Im not saying my skin has aged significantly this year but my six-year-old recently asked me why I had asix-pack on my forehead. After six months of stressful days, sleepless nights and home-school nightmares, its become apparent that matters need taking in hand. And theres something about the early autumn, with its nip in the air, and its new-found appreciation for proper, non-negotiable routines that feels right for a skincare overhaul.

Fortunately, the seasons big skincare launches abound with new ways to reset your skin, from serious, sleeves-rolled-up jump-starting regimens, which last up to a month and deliver a rapid burst of intense reconditioning, to new strategies that claim todetoxify your daily regime without your having to somuch as cut down on caffeine.

Sorting out most modern-day skincare complaints from sluggish cell turnover caused by tiredness and stress to overstimulated skin (a result of using products not suited to one another), to undeserved lacklustre complexions caused by outdated products requires a bit of areboot. It could be a facial; it could be a peel. But inthedays when weve all become beauty hobbyists, performing DIY facials like pros, it could also be a pleasurable at-home experience for the price of a couple ofdecent salon treatments.

Dr Anita Sturnham, a London-based GP specialising in dermatology who launched her own excellent skincare line,Decree, last year, became so aware of how many of herpatients especially those suffering with breakouts, pigmentation and dehydration needed a thorough overhaul that she recently launched her own two-week Skin Reset Kit. Sturnham believes 90 per cent of the skin issues she sees are self-inflicted simply by using the wrong products and that stripping your skincare right back is an essential step for getting the best from your skin.

Another recent reset kit is Budapest brand Omoroviczas The Cure programme, which in nine days cycles through anacid phase (to resurface), a remineralise phase (tostimulate microcirculation) and a reconstruct phase (forrenewed elasticity). You can repeat it every three months, ideally to coincide with the change of seasons.

One of the best known brands for an intensive treatment is that of anthropologist-turned-dermatologist Dr Phillip Levy. A Geneva-based wound-healing specialist,he believes that only via resetting can you achieve some of the most visible anti-ageing results andhis Ultimate Stem Cell Spring Homecure (the springmeans spring clean but it can bestarted any time)is legendary. Manyofthe cures we have studied over the years seem to be everyday products nicely repackaged, he says.But to have something truly transformational, theyneed go deep enough to stimulate your own collagen, elastin and hyaluronic acid production, and last four weeks or even eight or more.

Its true that these regimes work best when they feel elevated from the everyday. And when it comes to products with a built-in sense of occasion, no one does it better than Sisley. Even before you get to the science and the scents, and the textures it has a particular French earnestness that makes every product feel like an event. Which must make LIntgral Anti-Age La Cure, its new skin-resetting regimen, at 775 for a four-week supply, a veritable tapis rouge.

For each of the four week-long phases Impulse, Reset, Consolidate, Renaissance theres a phial of creamy serum, about the size of an eye cream. You use each one for seven days, applying eight pumps of product morning and night (this feels a lot, and it takes a few minutes to properly sink in). You can follow with eye cream or moisturiser if you want to, but I didnt feel the need. The bottles have been slightly overfilled so as to ensure you dont run out, but when you get to the end of the seventh day, you must start the next one nonetheless. (This feels wasteful, but I was assured by Sisleys training manager Lorna Green that I could save up these last drops and use them a couple of weeks after the course, as a further boost).

The formulation works on the skins mitochondria the batteries where cellular energy is stored. Theylose the ability to restore themselves over time, particularly during intense periods of stress and hormonal changes, so following either one of those would be an ideal time to try it. The breakthrough wasthe discovery of the mechanisms of a process called autophagy (for which Japanese biologist Yoshinori Ohsumi won the Nobel Prize for medicine in2016), whereby damaged cell components such as mitochondria destroy themselves to protect the rest ofthe cell. La Cure boosts the elimination of these wasteelements, allowing the healthy cells left behind tosoak up energy and regenerate promoting the appearanceof healthier, more youthful skin. In skincare terms, this is no mean feat.

Where the real technology is happening, it wont be long before they eclipse the big jars of moisturiser completely

It sounds intense and its certainly super-active: by the end of the first week I had a small, yet determined, spot on my chin (which I cannot believe was a coincidence) and a little more redness than usual, too. The following week, cell detoxification week, my skin was starting to feel unusually smooth. By the end of the fourth week, my skin was smoother and clearer than I can ever remember. Its also, though, a real example of skincare as self-care: as much as the thought of a radically rejuvenated complexion, the daily reminder that youve sidelined your usual clutter of products in favour of something exceptional is almost enough to bring on a glow.

With any reset complete, the focus should then be on keeping your skin detoxified and renewed. One update worth looking at is a serum. Whereas the luxurious facecream at the end of your regime used to be the jewel in any skincare crown, these dayslightweight serums are where the real technology ishappening, and it wont be long before they eclipse thebig jars of moisturiser completely.

While serums used to be a targeted addition to your face cream specifically for age spots, say, or wrinkles the best new ones are genuinely impressive all-rounders. Este Lauder has just revamped Advanced Night Repair, one of the first ever mainstream skin serums and a product so ubiquitous that among beauty editors it has acronym status. (See also: Cliniques DDML, aka Dramatically Different Moisturizing Lotion). And in October, Suqqu, which hails from Japan where serums have been the mainstay of skincare much longer than here will launch Vialume, its most advanced line yet, containing glucosamine and amino-acid derivatives designed to targetall five key characteristics of great skin: moisture, firmness, smoothness, translucency and brightness.

Another product gaining increasingly scientific status is face oil, which should no longer be dismissed as the preserve of the militantly natural beauty brigade. Augustinus Bader, the world-leading wound-healing specialist whose Rich Cream was the runaway skincare success of 2018, has just launched The Face Oil, which contains a slew of delicious-sounding oils argan, babassu, hazelnut, karanja as wellas a healthy dose of TFC8, the complex of vitamins, amino acids and synthesised molecules that has made Baders products famous. Meanwhile, RVive Glow Elixir Hydrating Radiance Oil is bronze in colour and slightly shimmering although unusually, it leaves no evidence of glittery particles. Alongside a cocktail of seed oils, it contains the brands signature Bio-Renewal Protein, rendering it a real skincare/make-up hybrid and a great transitional product for this time of year.

Another need-to-know and a great option particularly for younger skin is Rihannas new Fenty Skin line. Theres Total Cleansr, which would work especially well as the first step of a double-cleanse, and Fat Water, which Ri-Ri calls a toner-serum hybrid but its the Hydra Vizor daily moisturiser that triumphs. This so-called Invisible Moisturizer has an SPF30 that leaves no white cast to the skin whatsoever, primarily because the product has a gorgeous pinkish hue and a blurring effect. The ghostly pallor left behind by so many SPF products is a particular challenge to people of colour and this range was designed to work seamlessly with make-up on all skin tones. It also smells great juicy with just the slightest medicinal tinge and comes in a refillable tube.

The recently launched skincare brand U Beauty wants to reset not just your skin, but the way you think about your whole regime. Were all doing too much, says founder Tina Craig, who until two years ago was working as an influencer/ambassador for the worlds biggest skincare brands but admits being as confused as anyone about what to use; she had ended up with a 13-step skincare routine. I started noticing that everyone Iknew had skin that looked translucent, which is not how it should look, she says. Then I looked at my grandma and relatives in Korea, and their skin was not like that. It was thick. Dense. Firm.

U Beauty is her answer to what she calls the cosmeticconfusion. Its first product, the Resurfacing Compound (which sold out three times on UK stockist Net-aPorter), was designed to replace toner, vitamin C, hyaluronicacid, AHAs, physical exfoliants, antioxidant serums and retinol products. From this month, theres alsoSuper Smart Hydrator, a moisturising serum that seeks out damaged cells and only treats the skin where itneeds it. Bookend these two with cleanser and SPF, saysCraig, and youre good to go.

Finally, could we reset the way we use products altogether? New US brand Noble Panacea is overseenby ascientific heavyweight: Sir Fraser Stoddart, who was awarded the 2016 Nobel Prize in chemistry. A microscopic delivery system releases its active ingredients into the skinin a programmed sequence, and it comes in individualdoses packed in mini sachets to ensure the optimal amount of these ingredients stays potent until theminute it reaches your skin.

On the one hand, they feel counter to the idea of luxury face creams more like a free sample from a beauty hall but on the other, the boxes made from renewable materials and ultra-hygienic 0.5ml doses feel modern and Covid-safe. (You can send them for recyling in a complimentary envelope to TerraCycle, with which the brand has partnered). And if nothing else, as its global ambassador ithas snapped up the actress Jodie Comer, who must havebeen pursued by every beauty company under the sun and as far as I can tell, theres no sign of a six-pack on her forehead.

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The great beauty reset: how to reboot your skin - Financial Times

DUBLIN, Sept. 2, 2020 /PRNewswire/ -- The "Global Cord Blood Banking Industry Report 2020" report has been added to ResearchAndMarkets.com's offering.

This report presents the number of cord blood units stored in inventory by the largest cord blood banks worldwide and the number of cord blood units (CBUs) released by registries across the world for hematopoietic stem cell (HSC) transplantation. Although cord blood is now used to treat more than 80 different diseases, this number could substantially expand if applications related to regenerative medicine start receiving approvals in major healthcare markets worldwide.

From the early 1900s through the mid-2000s, the global cord blood banking industry expanded rapidly, with companies opening for business in all major markets worldwide. From 2005 to 2010, the market reached saturation and stabilized.

Then, from 2010 to 2020, the market began to aggressively consolidate. This has created both serious threats and unique opportunities within the industry.

Serious threats to the industry include low rates of utilization for stored cord blood, expensive cord blood transplantation procedures, difficulty educating obstetricians about cellular therapies, and an increasing trend toward industry consolidation. There are also emerging opportunities for the industry, such as accelerated regulatory pathways for cell therapies in leading healthcare markets worldwide and expanding applications for cell-based therapies. In particular, MSCs from cord tissue (and other sources) are showing intriguing promise in the treatment and management of COVID-19.

Cord Blood Industry Trends

Within recent years, new themes have been impacting the industry, including the pairing of stem cell storage services with genetic and genomic testing services, as well as reproductive health services. Cord blood banks are diversifying into new types of stem cell storage, including umbilical cord tissue storage, placental blood and tissue, amniotic fluid and tissue, and dental pulp. Cord blood banks are also investigating means of becoming integrated therapeutic companies. With hundreds of companies offering cord blood banking services worldwide, maturation of the market means that each company is fighting harder for market share.

Growing numbers of investors are also entering the marketplace, with M&A activity accelerating in the U.S. and abroad. Holding companies are emerging as a global theme, allowing for increased operational efficiency and economy of scale. Cryoholdco has established itself as the market leader within Latin America. Created in 2015, Cryoholdco is a holding company that will control nearly 270,000 stem cell units by the end of 2020. It now owns a half dozen cord blood banks, as well as a dental stem cell storage company.

Globally, networks of cord blood banks have become commonplace, with Sanpower Group establishing its dominance in Asia. Although Sanpower has been quiet about its operations, it holds 4 licenses out of only 7 issued provincial-level cord blood bank licenses in China. It has reserved over 900,000 cord blood samples in China, and its reserves amount to over 1.2 million units when Cordlife' reserves within Southeast Asian countries are included. This positions Sanpower Group and it's subsidiary Nanjing Cenbest as the world's largest cord blood banking operator not only in China and Southeast Asia but in the world.

The number of cord blood banks in Europe has dropped by more than one-third over the past ten years, from approximately 150 to under 100. The industry leaders in this market segment include FamiCord Group, who has executed a dozen M&A transactions, and Vita34, who has executed approximately a half dozen. Stemlab, the largest cord blood bank in Portugal, also executed three acquisition deals prior to being acquired by FamiCord. FamiCord is now the leading stem cell bank in Europe and one of the largest worldwide.

Cord Blood Expansion Technologies

Because cord blood utilization is largely limited to use in pediatric patients, growing investment is flowing into ex vivo cord blood expansion technologies. If successful, this technology could greatly expand the market potential for cord blood, encouraging its use within new markets, such as regenerative medicine, aging, and augmented immunity.

Key strategies being explored for this purpose include:

Currently, Gamida Cell, Nohla Therapeutics, Excellthera, and Magenta Therapeutics have ex vivo cord blood expansion products proceeding through clinical trials. Growing numbers of investors have also entered the cord blood banking marketplace, led by groups such as GI Partners, ABS Capital Partners & HLM Management, KKR & Company, Bay City Capital, GTCR, LLC, and Excalibur.

Cord Blood Banking by Region

Within the United States, most of the market share is controlled by three major players: Cord Blood Registry (CBR), Cryo-Cell, and ViaCord. CBR has been traded twice, once in 2015 to AMAG Pharmaceuticals for $700 million and again in 2018 to GI Partners for $530 million. CBR also bought Natera's Evercord Cord Blood Banking business in September 2019. In total, CBR controls over 900,000 cord blood and tissue samples, making it one of the largest cord blood banks worldwide.

In China, the government controls the industry by authorizing only one cord blood bank to operate within each province, and official government support, authorization, and permits are required. Importantly, the Chinese government announced in late 2019 that it will be issuing new licenses for the first time, expanding from the current 7 licensed regions for cord blood banking to up to 19 regions, including Beijing.

In Italy and France, it is illegal to privately store one's cord blood, which has fully eliminated the potential for a private market to exist within the region. In Ecuador, the government created the first public cord blood bank and instituted laws such that private cord blood banks cannot approach women about private cord blood banking options during the first six months of pregnancy. This created a crisis for private banks, forcing most out of business.

Recently, India's Central Drugs Standard Control Organization (CDSCO) restricted commercial banking of stem cells from most biological materials, including cord tissue, placenta, and dental pulp stem cells - leaving only umbilical cord blood banking as permitted and licensed within the country.

While market factors vary by geography, it is crucial to have a global understanding of the industry, because research advances, clinical trial findings, and technology advances do not know international boundaries. The cord blood market is global in nature and understanding dynamics within your region is not sufficient for making strategic, informed, and profitable decisions.

Overall, the report provides the reader with the following details and answers the following questions:

1. Number of cord blood units cryopreserved in public and private cord blood banks globally2. Number of hematopoietic stem cell transplants (HSCTs) globally using cord blood cells3. Utilization of cord blood cells in clinical trials for developing regenerative medicines4. The decline of the utilization of cord blood cells in HSC transplantations since 20055. Emerging technologies to influence the financial sustainability of public cord blood banks6. The future scope for companion products from cord blood7. The changing landscape of cord blood cell banking market8. Extension of services by cord blood banks9. Types of cord blood banks10. The economic model of public cord blood banks11. Cost analysis for public cord blood banks12. The economic model of private cord blood banks13. Cost analysis for private cord blood banks14. Profit margins for private cord blood banks15. Pricing for processing and storage in private banks16. Rate per cord blood unit in the U.S. and Europe17. Indications for the use of cord blood-derived HSCs for transplantations18. Diseases targeted by cord blood-derived MSCs in regenerative medicine19. Cord blood processing technologies20. Number of clinical trials, number of published scientific papers and NIH funding for cord blood research21. Transplantation data from different cord blood registries

Key questions answered in this report are:

1. What are the strategies being considered for improving the financial stability of public cord blood banks?2. What are the companion products proposed to be developed from cord blood?3. How much is being spent on processing and storing a unit of cord blood?4. How much does a unit of cryopreserved cord blood unit fetch on release?5. Why do most public cord blood banks incur a loss?6. What is the net profit margin for a private cord blood bank?7. What are the prices for processing and storage of cord blood in private cord blood banks?8. What are the rates per cord blood units in the U.S. and Europe?9. What are the revenues from cord blood sales for major cord blood banks?10. Which are the different accreditation systems for cord blood banks?11. What are the comparative merits of the various cord blood processing technologies?12. What is to be done to increase the rate of utilization of cord blood cells in transplantations?13. Which TNC counts are preferred for transplantation?14. What is the number of registered clinical trials using cord blood and cord tissue?15. How many clinical trials are involved in studying the expansion of cord blood cells in the laboratory?16. How many matching and mismatching transplantations using cord blood units are performed on an annual basis?17. What is the share of cord blood cells used for transplantation from 2000 to 2020?18. What is the likelihood of finding a matching allogeneic cord blood unit by ethnicity?19. Which are the top ten countries for donating cord blood?20. What are the diseases targeted by cord blood-derived MSCs within clinical trials?

Key Topics Covered

1. REPORT OVERVIEW1.1 Statement of the Report1.2 Executive Summary1.3 Introduction1.3.1 Cord Blood: An Alternative Source for HPSCs1.3.2 Utilization of Cord Blood Cells in Clinical Trials1.3.3 The Struggle of Cord Blood Banks1.3.4 Emerging Technologies to Influence Financial Sustainability of Banks1.3.4.1 Other Opportunities to Improve Financial Stability1.3.4.2 Scope for Companion Products1.3.5 Changing Landscape of Cord Blood Cell Banking Market1.3.6 Extension of Services by Cord Blood Banks

2. CORD BLOOD & CORD BLOOD BANKING: AN OVERVIEW2.1 Cord Blood Banking (Stem Cell Banking)2.1.1 Public Cord Blood Banks2.1.1.1 Economic Model of Public Cord Blood Banks2.1.1.2 Cost Analysis for Public Banks2.1.1.3 Relationship between Costs and Release Rates2.1.2 Private Cord Blood Banks2.1.2.1 Cost Analysis for Private Cord Blood Banks2.1.2.2 Economic Model of Private Banks2.1.3 Hybrid Cord Blood Banks2.2 Globally Known Cord Blood Banks2.2.1 Comparing Cord Blood Banks2.2.2 Cord Blood Banks in the U.S.2.2.3 Proportion of Public, Private and Hybrid Banks2.3 Percent Share of Parents of Newborns Storing Cord Blood by Country/Region2.4 Pricing for Processing and Storage in Commercial Banks2.4.1 Rate per Cord Blood Unit in the U.S. and Europe2.5 Cord Blood Revenues for Major Cord Blood Banks

3. CORD BLOOD BANK ACCREDITATIONS3.1 American Association of Blood Banks (AABB)3.2 Foundation for the Accreditation of Cellular Therapy (FACT)3.3 FDA Registration3.4 FDA Biologics License Application (BLA) License3.5 Investigational New Drug (IND) for Cord Blood3.6 Human Tissue Authority (HTA)3.7 Therapeutic Goods Act (TGA) in Australia3.8 International NetCord Foundation3.9 AABB Accredited Cord Blood Facilities3.10 FACT Accreditation for Cord Blood Banks

4. APPLICATIONS OF CORD BLOOD CELLS4.1 Hematopoietic Stem Cell Transplantations with Cord Blood Cells4.2 Cord Cells in Regenerative Medicine

5. CORD BLOOD PROCESSING TECHNOLOGIES5.1 The Process of Separation5.1.1 PrepaCyte-CB5.1.2 Advantages of PrepaCyte-CB5.1.3 Treatment Outcomes with PrepaCyte-CB5.1.4 Hetastarch (HES)5.1.5 AutoXpress (AXP)5.1.6 SEPAX5.1.7 Plasma Depletion Method (MaxCell Process)5.1.8 Density Gradient Method5.2 Comparative Merits of Different Processing Methods5.2.1 Early Stage HSC Recovery by Technologies5.2.2 Mid Stage HSC (CD34+/CD133+) Recovery from Cord Blood5.2.3 Late Stage Recovery of HSCs from Cord Blood5.3 HSC (CD45+) Recovery5.4 Days to Neutrophil Engraftment by Technology5.5 Anticoagulants used in Cord Blood Processing5.5.1 Type of Anticoagulant and Cell Recovery Volume5.5.2 Percent Cell Recovery by Sample Size5.5.3 TNC Viability by Time Taken for Transport and Type of Anticoagulant5.6 Cryopreservation of Cord Blood Cells5.7 Bioprocessing of Umbilical Cord Tissue (UCT)5.8 A Proposal to Improve the Utilization Rate of Banked Cord Blood

6. CORD BLOOD CLINICAL TRIALS, SCIENTIFIC PUBLICATIONS & NIH FUNDING6.1 Cord Blood Cells for Research6.2 Cord Blood Cells for Clinical Trials6.2.1 Number of Clinical Trials involving Cord Blood Cells6.2.2 Number of Clinical Trials using Cord Blood Cells by Geography6.2.3 Number of Clinical Trials by Study Type6.2.4 Number of Clinical Trials by Study Phase6.2.5 Number of Clinical Trials by Funder Type6.2.6 Clinical Trials Addressing Indications in Children6.2.7 Select Three Clinical Trials Involving Children6.2.7.1 Sensorineural Hearing Loss (NCT02038972)6.2.7.2 Autism Spectrum (NCT02847182)6.2.7.3 Cerebral Palsy (NCT01147653)6.2.8 Clinical Trials for Neurological Diseases using Cord Blood and Cord Tissue6.2.9 UCB for Diabetes6.2.10 UCB in Cardiovascular Clinical Trials6.2.11 Cord Blood Cells for Auto-Immune Diseases in Clinical Trials6.2.12 Cord Tissue Cells for Orthopedic Disorders in Clinical Trials6.2.13 Cord Blood Cells for Other Indications in Clinical Trials6.3 Major Diseases Addressed by Cord Blood Cells in Clinical Trials6.4 Clinical Trials using Cord Tissue-Derived MSCs6.5 Ongoing Clinical Trials using Cord Tissue6.5.1 Cord Tissue-Based Clinical Trials by Geography6.5.2 Cord Tissue-Based Clinical Trials by Phase6.5.3 Cord Tissue-Based Clinical Trials by Sponsor Types6.5.4 Companies Sponsoring in Trials using Cord Tissue-Derived MSCs6.6 Wharton's Jelly-Derived MSCs in Clinical Trials6.6.1 Wharton's Jelly-Based Clinical Trials by Phase6.6.2 Companies Sponsoring Wharton's Jelly-Based Clinical Trials6.7 Clinical Trials Involving Cord Blood Expansion Studies6.7.1 Safe and Feasible Expansion Protocols6.7.2 List of Clinical Trials involved in the Expansion of Cord Blood HSCs6.7.3 Expansion Technologies6.8 Scientific Publications on Cord Blood6.9 Scientific Publications on Cord Tissue6.10 Scientific Publications on Wharton's Jelly-Derived MSCs6.11 Published Scientific Papers on Cord Blood Cell Expansion6.12 NIH Funding for Cord Blood Research

7. PARENT'S AWARENESS AND ATTITUDE TOWARDS CORD BLOOD BANKING7.1 Undecided Expectant Parents7.2 The Familiar Cord Blood Banks Known by the Expectant Parents7.3 Factors Influencing the Choice of a Cord Blood Bank

8. CORD BLOOD: AS A TRANSPLANTATION MEDICINE8.1 Comparisons of Cord Blood to other Allograft Sources8.1.1 Major Indications for HCTs in the U.S.8.1.2 Trend in Allogeneic HCT in the U.S. by Recipient Age8.1.3 Trends in Autologous HCT in the U.S. by Recipient Age8.2 HCTs by Cell Source in Adult Patients8.2.1 Transplants by Cell Source in Pediatric Patients8.3 Allogeneic HCTs by Cell Source8.3.1 Unrelated Donor Allogeneic HCTs in Patients &lessThan;18 Years8.4 Likelihood of Finding an Unrelated Cord Blood Unit by Ethnicity8.4.1 Likelihood of Finding an Unrelated Cord Blood Unit for Patients &lessThan;20 Years8.5 Odds of using a Baby's Cord Blood8.6 Cord Blood Utilization Trends8.7 Number of Cord Blood Donors Worldwide8.7.1 Number of CBUs Stored Worldwide8.7.2 Cord Blood Donors by Geography8.7.2.1 Cord Blood Units Stored in Different Geographies8.7.2.2 Number of Donors by HLA Typing8.7.3 Searches Made by Transplant Patients for Donors/CBUs8.7.4 Types of CBU Shipments (Single/Double/Multi)8.7.5 TNC Count of CBUs Shipped for Children and Adult Patients8.7.6 Shipment of Multiple CBUs8.7.7 Percent Supply of CBUs for National and International Patients8.7.8 Decreasing Number of CBU Utilization8.8 Top Ten Countries in Cord Blood Donation8.8.1 HLA Typed CBUs by Continent8.8.2 Percentage TNC of Banked CBUs8.8.3 Total Number of CBUs, HLA-Typed Units by Country8.9 Cord Blood Export/Import by the E.U. Member States8.9.1 Number of Donors and CBUs in Europe8.9.2 Number of Exports/Imports of CBUs in E.U.8.10 Global Exchange of Cord Blood Units

9. CORD BLOOD CELLS AS THERAPEUTIC CELL PRODUCTS IN CELL THERAPY9.1 MSCs from Cord Blood and Cord Tissue9.1.1 Potential Neurological Applications of Cord Blood-Derived Cells9.1.2 Cord Tissue-Derived MSCs for Therapeutic use9.1.2.1 Indications Targeted by UCT-MSCs in Clinical Trials9.2 Current Consumption of Cord Blood Units by Clinical Trials9.3 Select Cord Blood Stem Cell Treatments in Clinical Trials9.3.1 Acquired Hearing Loss (NCT02038972)9.3.2 Autism (NCT02847182)9.3.3 Cerebral Palsy (NCT03087110)9.3.4 Hypoplastic Left Heart Syndrome (NCT01856049)9.3.5 Type 1 Diabetes (NCT00989547)9.3.6 Psoriasis (NCT03765957)9.3.7 Parkinson's Disease (NCT03550183)9.3.8 Signs of Aging (NCT04174898)9.3.9 Stroke (NCT02433509)9.3.10 Traumatic Brain Injury (NCT01451528)

10. MARKET ANALYSIS10.1 Public vs. Private Cord Blood Banking Market10.2 Cord Blood Banking Market by Indication

11. PROFILES OF SELECT CORD BLOOD BANKS11.1 AllCells11.1.1 Whole Blood11.1.2 Leukopak11.1.3 Mobilized Leukopak11.1.4 Bone Marrow11.1.5 Cord Blood11.2 AlphaCord LLC11.2.1 NextGen Collection System11.3 Americord Registry, Inc.11.3.1 Cord Blood 2.011.3.2 Cord Tissue11.3.3 Placental Tissue 2.011.4 Be The Match11.4.1 Hub of Transplant Network11.4.2 Partners of Be The Match11.4.3 Allogeneic Cell Sources in Be The Match Registry11.4.4 Likelihood of a Matched Donor on Be The Match by Ethnic Background11.5 Biocell Center Corporation11.5.1 Chorionic villi after Delivery11.5.2 Amniotic Fluid and Chorionic Villi during Pregnancy11.6 BioEden Group, Inc.11.6.1 Differences between Tooth Cells and Umbilical Cord Cells11.7 Biovault Family11.7.1 Personalized Cord Blood Processing11.8 Cell Care11.9 Cells4Life Group, LLP11.9.1 Cells4Life's pricing11.9.2 TotiCyte Technology11.9.3 Cord Blood Releases11.10 Cell-Save11.11 Center for International Blood and Marrow Transplant Research (CIBMTR)11.11.1 Global Collaboration11.11.2 Scientific Working Committees11.11.3 Medicare Clinical Trials and Studies11.11.4 Cellular Therapy11.12 Crio-Cell International, Inc.11.12.1 Advanced Collection Kit11.12.2 Prepacyte-CB11.12.3 Crio-Cell International's Pricing11.12.4 Revenue for Crio-Cell International11.13 Cord Blood Center Group11.13.1 Cord Blood Units Released11.14 Cordlife Group, Ltd.11.14.1 Cordlife's Cord Blood Release Track Record11.15 Core23 Biobank11.16 Cord Blood Registry (CBR)11.17 CordVida11.18 Crioestaminal11.18.1 Cord Blood Transplantation in Portugal11.19 Cryo-Cell International, Inc.11.19.1 Processing Method11.19.2 Financial Results of the Company11.20 CryoHoldco11.21 Cryoviva Biotech Pvt. Ltd11.22 European Society for Blood and Bone Marrow Transplantation (EBMT)11.22.1 EBMT Transplant Activity11.23 FamiCord Group11.24 GeneCell International11.25 Global Cord Blood Corporation11.25.1 The Company's Business11.26 HealthBaby Hong Kong11.26.1 BioArchive System Service Plan11.26.2 MVE Liquid Nitrogen System11.27 HEMAFUND11.28 Insception Lifebank11.29 LifebankUSA11.29.1 Placental Banking11.30 LifeCell International Pvt. Ltd.11.31 MiracleCord, Inc.11.32 Maze Cord Blood Laboratories11.33 New England Cord Blood Bank, Inc.11.34 New York Cord Blood Center (NYBC)11.34.1 Products11.34.2 Laboratory Services11.35 PacifiCord11.35.1 FDA-Approved Sterile Collection Bags11.35.2 AXP Processing System11.35.3 BioArchive System11.36 ReeLabs Pvt. Ltd.11.37 Smart Cells International, Ltd.11.38 Stem Cell Cryobank11.39 StemCyte, Inc.11.39.1 StemCyte Sponsored Clinical Trials11.39.1.1 Spinal Cord Injury Phase II11.39.1.2 Other Trials11.40 Transcell Biolife11.40.1 ScellCare11.40.2 ToothScell11.41 ViaCord11.42 Vita 34 AG11.43 World Marrow Donor Association (WMDA)11.43.1 Search & Match Service11.44 Worldwide Network for Blood & Marrow Transplantation (WBMT)

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Global Cord Blood Banking Market 2020 with Analysis of 44 Industry Players - PRNewswire

On April 15, 2002, the FDA approved a temporary treatment for wrinkles that would revolutionize aging. All of a sudden, you could waltz into a derms office and get your frown lines ironed out faster than it would take to iron an actual shirt. It was called botulinum toxin, Botox for short.

Eighteen years later, a few units of Botox every three months has become the norm for millions around the world (more than seven million yearly in the U.S. alone). Now, if someone had told your grandparents, or even your parents, 20 years ago that people would be getting their foreheads frozen to look younger, they likely would have scoffed at the idea. So just imagine what other wild fixes could be coming to a medi-spa near you.

Its exciting to think about how the next 10 years will look, says Dr. Rohan Bissoondath, medical director of Calgarys Preventous Cosmetic Medicine clinic. With lifespan increasing, people are routinely going to be living into their hundreds, so we want to look great as well. From magic pills to creams that mimic injections, we take a look at the incredible innovations on the horizon.

You wont need surgery to lift your face

The way science is progressing, facelifts are set to become obsolete, says Dr. Lisa Kellett of Torontos DLK on Avenue. I think that the gold standard will eventually be finding ways to regenerate and kick-start our own collagen instead of doing a facelift. Kellett is already trying out cutting-edge technology to accomplish this, such as a laser that delivers growth factors right in the dermis to regenerate tissue. Its pretty snazzy stuff, but she anticipates even greater advances in coming years. I think well be able to use stem cells in conjunction with technology to regenerate collagen I think thats what well be doing one day.

Youll (hopefully) be able to nix wrinkles without needles

Botox in a cream? This has been in the pipeline for a while, says Bissoondath. The challenge is getting the molecules to penetrate the skin so that they can act on the muscle. Maybe on crows feet because its a thinner area, thinner muscles; that may be an area where we see some utility for it, but its still out there. Topical Botox had some success in trials, but scientists still have kinks to work out. In the meantime, a Botox cream might be beneficial even if it doesnt reach muscles, says Bissoondath. I see the potential for having it in a cream and applying it to the whole face, not necessarily affecting facial expressions, but giving an improved glow and better skin quality.

Therell be more all-in-one solutions

If you want to smooth, you get Botox. If you want to brighten, you get IPL. If you want to tighten, you get Thermage. But what if there was a treatment that did it all? I think thats the future of aging, says Kellett, who is just about to launch such a treatment at her clinic. Marketed as the next generation of laser and light-based platform technology, Etherea MX is a multiple modality device that can tackle everything from dark spots and skin laxity to textural issues and wrinkles. It means that when patients come in, theyre not just doing one thing, says the doc. Instead, in the same appointment, shes able to address a variety of concerns with a single machine.

Youll be able to take a pill instead of hitting the gym

OK, this is very cool. Something I think is possible is a pill to replace exercise, says Bissoondath, who adds that this could be developed in the not so distant future. With the advances were making in understanding the functions of our body down to the cellular level and intracellular level, and understanding how our mitochondria actually ages, were looking at ways now where we can manipulate that from a pill perspective. The pill wouldnt deliver all the benefits of physical activity, such as the positive impact on our mood, but it would replicate its effects on our body. It wont take the place of walking around outside and soaking up nature it cant do the mental part of it. But as far as the physiologic, biochemical part of it, were really understanding that better and making big strides. Its exciting.

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Exciting new technologies could change the way we age - TheSpec.com

KENILWORTH, N.J.--(BUSINESS WIRE)--Sep 2, 2020--

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that new data from its broad and diverse oncology development program will be presented at the European Society for Medical Oncology (ESMO) Virtual Congress 2020 from Sept. 1921. Data spanning more than 15 types of cancer will be presented at the congress, with new findings from Mercks portfolio of established medicines including KEYTRUDA, Mercks anti-PD-1 therapy; LENVIMA (lenvatinib, in collaboration with Eisai); and LYNPARZA (in collaboration with AstraZeneca). Pivotal Phase 3 data evaluating KEYTRUDA in combination with chemotherapy for the first-line treatment of patients with locally advanced or metastatic esophageal cancer from the KEYNOTE-590 trial (Abstract #LBA8) and LYNPARZA in patients with metastatic castration-resistant prostate cancer (mCRPC) from the PROfound trial (Abstract #610O) were selected for inclusion in ESMO Presidential Symposium sessions. Additionally, new findings will be shared for three of Mercks novel investigational candidates: vibostolimab (MK-7684), an anti-TIGIT antibody; MK-4830, an antibody targeting ILT4; and MK-6482, an oral HIF-2 inhibitor.

At Merck, we are focused on further improving long-term outcomes for more patients living with cancer, and this commitment is reflected in the breadth and diversity of our oncology research program, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. At the ESMO Virtual Congress 2020, we look forward to sharing important new results including survival data for KEYTRUDA in esophageal cancer and long-term findings in lung cancer, melanoma, and head and neck cancer, as well as research from our expansive pipeline.

Key data from Mercks portfolio and pipeline to be presented at ESMO include:

KEYTRUDA

KEYTRUDA Plus LENVIMA

LYNPARZA

Pipeline

Merck Investor Event

Merck will hold a virtual investor event in conjunction with the ESMO Virtual Congress 2020 on Tuesday, Sept. 22 from 89 a.m. E.T. Details will be provided at a date closer to the event at https://www.merck.com/investor-relations.

Details on Abstracts Listed Above and Additional Key Abstracts for Merck

KEYTRUDA

Classical Hodgkin Lymphoma

Colorectal Cancer

Diffuse Large B-Cell Lymphoma

Esophageal Cancer

Head and Neck Cancer

Lung Cancer

Melanoma

Sarcoma

Solid Tumors

KEYTRUDA Plus LENVIMA (in collaboration with Eisai)

Lung Cancer

Melanoma

Renal Cell Carcinoma

Solid Tumors

LYNPARZA (in collaboration with AstraZeneca)

Ovarian Cancer

Prostate Cancer

Vibostolimab

Lung Cancer

MK-4830

Solid Tumors

MK-6482

Von-Hippel Lindau Disease

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Endometrial Carcinoma

KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

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New Scientific Data at the ESMO Virtual Congress 2020 Reflect Merck's Commitment to Advancing Cancer Research and Care - The Baytown Sun

Efforts to use regenerative medicinewhich seeks to address ailments as diverse as birth defects, traumatic injury, aging, degenerative disease, and the disorganized growth of cancerwould be greatly aided by solving one fundamental puzzle: How do cellular collectives orchestrate the building of complex, three-dimensional structures?

While genomes predictably encode the proteins present in cells, a simple molecular parts list does not tell us enough about the anatomical layout or regenerative potential of the body that the cells will work to construct. Genomes are not a blueprint for anatomy, and genome editing is fundamentally limited by the fact that its very hard to infer which genes to tweak, and how, to achieve desired complex anatomical outcomes. Similarly, stem cells generate the building blocks of organs, but the ability to organize specific cell types into a working human hand or eye has been and will be beyond the grasp of direct manipulation for a very long time.

But researchers working in the fields of synthetic morphology and regenerative biophysics are beginning to understand the rules governing the plasticity of organ growth and repair. Rather than micromanaging tasks that are too complex to implement directly at the cellular or molecular level, what if we solved the mystery of how groups of cells cooperate to construct specific multicellular bodies during embryogenesis and regeneration? Perhaps then we could figure out how to motivate cell collectives to build whatever anatomical features we want.

New approaches now allow us to target the processes that implement anatomical decision-making without genetic engineering. In January, using such tools, crafted in my lab at Tufts Universitys Allen Discovery Center and by computer scientists in Josh Bongards lab at the University of Vermont, we were able to create novel living machines, artificial bodies with morphologies and behaviors completely different from the default anatomy of the frog species (Xenopus laevis) whose cells we used. These cells rebooted their multicellularity into a new form, without genomic changes. This represents an extremely exciting sandbox in which bioengineers can play, with the aim of decoding the logic of anatomical and behavioral control, as well as understanding the plasticity of cells and the relationship of genomes to anatomies.

Deciphering how an organism puts itself together is truly an interdisciplinary undertaking.

Deciphering how an organism puts itself together is truly an interdisciplinary undertaking. Resolving the whole picture will involve understanding not only the mechanisms by which cells operate, but also elucidating the computations that cells and groups of cells carry out to orchestrate tissue and organ construction on a whole-body scale. The next generation of advances in this area of research will emerge from the flow of ideas between computer scientists and biologists. Unlocking the full potential of regenerative medicine will require biology to take the journey computer science has already taken, from focusing on the hardwarethe proteins and biochemical pathways that carry out cellular operationsto the physiological software that enables networks of cells to acquire, store, and act on information about organ and indeed whole-body geometry.

In the computer world, this transition from rewiring hardware to reprogramming the information flow by changing the inputs gave rise to the information technology revolution. This shift of perspective could transform biology, allowing scientists to achieve the still-futuristic visions of regenerative medicine. An understanding of how independent, competent agents such as cells cooperate and compete toward robust outcomes, despite noise and changing environmental conditions, would also inform engineering. Swarm robotics, Internet of Things, and even the development of general artificial intelligence will all be enriched by the ability to read out and set the anatomical states toward which cell collectives build, because they share a fundamental underlying problem: how to control the emergent outcomes of systems composed of many interacting units or individuals.

Many types of embryos can regenerate entirely if cut in half, and some species are proficient regenerators as adults. Axolotls (Ambystoma mexicanum) regenerate their limbs, eyes, spinal cords, jaws, and portions of the brain throughout life. Planarian flatworms (class Turbellaria), meanwhile, can regrow absolutely any part of their body; when the animal is cut into pieces, each piece knows exactly whats missing and regenerates to be a perfect, tiny worm.

The remarkable thing is not simply that growth begins after wounding and that various cell types are generated, but that these bodies will grow and remodel until a correct anatomy is complete, and then they stop. How does the system identify the correct target morphology, orchestrate individual cell behaviors to get there, and determine when the job is done? How does it communicate this information to control underlying cell activities?

Several years ago, my lab found that Xenopus tadpoles with their facial organs experimentally mixed up into incorrect positions still have largely normal faces once theyve matured, as the organs move and remodel through unnatural paths. Last year, a colleague at Tufts came to a similar conclusion: the Xenopus genome does not encode a hardwired set of instructions for the movements of different organs during metamorphosis from tadpole to frog, but rather encodes molecular hardware that executes a kind of error minimization loop, comparing the current anatomy to the target frog morphology and working to progressively reduce the difference between them. Once a rough spatial specification of the layout is achieved, that triggers the cessation of further remodeling.

The deep puzzle of how competent agents such as cells work together to pursue goals such as building, remodeling, or repairing a complex organ to a predetermined spec is well illustrated by planaria. Despite having a mechanistic understanding of stem cell specification pathways and axial chemical gradients, scientists really dont know what determines the intricate shape and structure of the flatworms head. It is also unknown how planaria perfectly regenerate the same anatomy, even as their genomes have accrued mutations over eons of somatic inheritance. Because some species of planaria reproduce by fission and regeneration, any mutation that doesnt kill the neoblastthe adult stem cell that gives rise to cells that regenerate new tissueis propagated to the next generation. The worms incredibly messy genome shows evidence of this process, and cells in an individual planarian can have different numbers of chromosomes. Still, fragmented planaria regenerate their body shape with nearly 100 percent anatomical fidelity.

Permanent editingof the encoded target morphology without genomic editing reveals a new kind of epigenetics.

So how do cell groups encode the patterns they build, and how do they know to stop once a target anatomy is achieved? What would happen, for example, if neoblasts from a planarian species with a flat head were transplanted into a worm of a species with a round or triangular head that had the head amputated? Which shape would result from this heterogeneous mixture? To date, none of the high-resolution molecular genetic studies of planaria give any prediction for the results of this experiment, because so far they have all focused on the cellular hardware, not on the logic of the softwareimplemented by chemical, mechanical, and electrical signaling among cellsthat controls large-scale outcomes and enables remodeling to stop when a specific morphology has been achieved.

Understanding how cells and tissues make real-time anatomical decisions is central not only to achieving regenerative outcomes too complex for us to manage directly, but also to solving problems such as cancer. While the view of cancer as a genetic disorder still largely drives clinical approaches, recent literature supports a view of cancer as cells simply not being able to receive the physiological signals that maintain the normally tight controls of anatomical homeostasis. Cut off from these patterning cues, individual cells revert to their ancient unicellular lifestyle and treat the rest of the body as external environment, often to ruinous effect. If we understand the mechanisms that scale single-cell homeostatic setpoints into tissue- and organ-level anatomical goal states and the conditions under which the anatomical error reduction control loop breaks down, we may be able to provide stimuli to gain control of rogue cancer cells without either gene therapy or chemotherapy.

During morphogenesis, cells cooperate to reliably build anatomical structures. Many living systems remodel and regenerate tissues or organs despite considerable damagethat is, they progressively reduce deviations from specific target morphologies, and halt growth and remodeling when those morphologies are achieved. Evolution exploits three modalities to achieve such anatomical homeostasis: biochemical gradients, bioelectric circuits, and biophysical forces. These interact to enable the same large-scale form to arise despite significant perturbations.

N.R. FULLER, SAYO-ART, LLC

BIOCHEMICAL GRADIENTS

The best-known modality concerns diffusible intracellular and extracellular signaling molecules. Gene-regulatory circuits and gradients of biochemicals control cell proliferation, differentiation, and migration.

BIOELECTRIC CIRCUITS

The movement of ions across cell membranes, especially via voltage-gated ion channels and gap junctions, can establish bioelectric circuits that control large-scale resting potential patterns within and among groups of cells. These bioelectric patterns implement long-range coordination, feedback, and memory dynamics across cell fields. They underlie modular morphogenetic decision-making about organ shape and spatial layout by regulating the dynamic redistribution of morphogens and the expression of genes.

BIOMECHANICAL FORCES

Cytoskeletal, adhesion, and motor proteins inside and between cells generate physical forces that in turn control cell behavior. These forces result in large-scale strain fields, which enable cell sheets to move and deform as a coherent unit, and thus execute the folds and bends that shape complex organs.

The software of life, which exploits the laws of physics and computation, is enabled by chemical, mechanical, and electrical signaling across cellular networks. While the chemical and mechanical mechanisms of morphogenesis have long been appreciated by molecular and cell biologists, the role of electrical signaling has largely been overlooked. But the same reprogrammability of neural circuits in the brain that supports learning, memory, and behavioral plasticity applies to all cells, not just neurons. Indeed, bacterial colonies can communicate via ionic currents, with recent research revealing brain-like dynamics in which information is propagated across and stored in a kind of proto-body formed by bacterial biofilms. So it should really come as no surprise that bioelectric signaling is a highly tractable component of morphological outcomes in multicellular organisms.

A few years ago, we studied the electrical dynamics that normally set the size and borders of the nascent Xenopus brain, and built a computer model of this process to shed light on how a range of various brain defects arise from disruptions to this bioelectric signaling. Our model suggested that specific modifications with mRNA or small molecules could restore the endogenous bioelectric patterns back to their correct layout. By using our computational platform to select drugs to open existing ion channels in nascent neural tissue or even a remote body tissue, we were able to prevent and even reverse brain defects caused not only by chemical teratogenscompounds that disrupt embryonic developmentbut by mutations in key neurogenesis genes.

Similarly, we used optogenetics to stimulate electrical activity in various somatic cell types totrigger regeneration of an entire tadpole tailan appendage with spinal cord, muscle, and peripheral innervationand to normalize the behavior of cancer cells in tadpoles strongly expressing human oncogenes such as KRAS mutations. We used a similar approach to trigger posterior regions, such as the gut, to build an entire frog eye. In both the eye and tail cases, the information on how exactly to build these complex structures, and where all the cells should go, did not have to be specified by the experimenter; rather, they arose from the cells themselves. Such findings reveal how ion channel mutations result in numerous human developmental channelopathies, and provide a roadmap for how they may be treated by altering the bioelectric map that tells cells what to build.

We also recently found a striking example of such reprogrammable bioelectrical software in control of regeneration in planaria. In 2011, we discovered that an endogenous electric circuit establishes a pattern of depolarization and hyperpolarization in planarian fragments that regulate the orientation of the anterior-posterior axis to be rebuilt. Last year, we discovered that this circuit controls the gene expressionneeded to build a head or tail within six hours of amputation, and by using molecules that make cell membranes permeable to certain ions to depolarize or hyperpolarize cells, we induced fragments of such worms to give rise to a symmetrical two-headed form, despite their wildtype genomes. Even more shockingly, the worms continued to generate two-headed progeny in additional rounds of cutting with no further manipulation. In further experiments, we demonstrated that briefly reducing gap junction-mediated connectivity between adjacent cells in the bioelectric network that guides regeneration led worms to regenerate head and brain shapes appropriate to other worm species whose lineages split more than 100 million years ago.

My group has developed the use of voltage-sensitive dyes to visualize the bioelectric pattern memory that guides gene expression and cell behavior toward morphogenetic outcomes. Meanwhile, my Allen Center colleagues are using synthetic artificial electric tissues made of human cells and computer models of ion channel activity to understand how electrical dynamics across groups of non-neural cells can set up the voltage patterns that control downstream gene expression, distribution of morphogen molecules, and cell behaviors to orchestrate morphogenesis.

The emerging picture in this field is that anatomical software is highly modulara key property that computer scientists exploit as subroutines and that most likely contributes in large part to biological evolvability and evolutionary plasticity. A simple bioelectric state, whether produced endogenously during development or induced by an experimenter, triggers very complex redistributions of morphogens and gene expression cascades that are needed to build various anatomies. The information stored in the bodys bioelectric circuitscan be permanently rewritten once we understand the dynamics of the biophysical circuits that make the critical morphological decisions. This permanent editing of the encoded target morphology without genomic editing reveals a new kind of epigenetics, information that is stored in a medium other than DNA sequences and chromatin.

Recent work from our group and others has demonstrated that anatomical pattern memories can be rewritten by physiological stimuli and maintained indefinitely without genomic editing. For example, the bioelectric circuit that normally determines head number and location in regenerating planaria can be triggered by brief alterations of ion channel or gap junction activity to alter the animals body plan. Due to the circuits pattern memory, the animals remain in this altered state indefinitely without further stimulation, despite their wildtype genomes. In other words, the pattern to which the cells build after damage can be changed, leading to a target morphology distinct from the genetic default.

N.R. FULLER, SAYO-ART, LLC

First, we soaked a planarian in voltage-sensitive fluorescent dye to observe the bioelectrical pattern across the entire tissue. We then cut the animal to see how this pattern changes in each fragment as it begins to regenerate.

We then applied drugs or used RNA interference to target ion channels or gap junctions in individual cells and thus change the pattern of depolarization/hyperpolarization and cellular connectivity across the whole fragment.

As a result of the disruption of the bodys bioelectric circuits, the planarian regrows with two heads instead of one, or none at all.

When we re-cut the two-headed planarian in plain water, long after the initial drug has left the tissue, the new anatomy persists in subsequent rounds of regeneration.

Cells can clearly build structures that are different from their genomic-default anatomical outcomes. But are cells universal constructors? Could they make anything if only we knew how to motivate them to do it?

The most recent advances in the new field at the intersection of developmental biology and computer science are driven by synthetic living machines known as biobots. Built from multiple interacting cell populations, these engineered machines have applications in disease modeling and drug development, and as sensors that detect and respond to biological signals. We recently tested the plasticity of cells by evolving in silico designs with specific movement and behavior capabilities and used this information to sculpt self-organized growth of aggregated Xenopus skin and muscle cells. In a novel environmentin vitro, as opposed to inside a frog embryoswarms of genetically normal cells were able to reimagine their multicellular form. With minimal sculpting post self-assembly, these cells form Xenobots with structures, movements, and other behaviors quite different from what might be expected if one simply sequenced their genome and identified them as wildtype X. laevis.

These living creations are a powerful platform to assess and model the computations that these cell swarms use to determine what to build. Such insights will help us to understand evolvability of body forms, robustness, and the true relationship between genomes and anatomy, greatly potentiating the impact of genome editing tools and making genomics more predictive for large-scale phenotypes. Moreover, testing regimes of biochemical, biomechanical, and bioelectrical stimuli in these biobots will enable the discovery of optimal stimuli for use in regenerative therapies and bioengineered organ construction. Finally, learning to program highly competent individual builders (cells) toward group-level, goal-driven behaviors (complex anatomies) will significantly advance swarm robotics and help avoid catastrophes of unintended consequences during the inevitable deployment of large numbers of artificial agents with complex behaviors.

Understanding how cells and tissues make real-time anatomical decisions is central to achieving regenerative outcomes too complex for us to manage directly.

The emerging field ofsynthetic morphology emphasizes a conceptual point that has been embraced by computer scientists but thus far resisted by biologists: the hardware-software distinction. In the 1940s, to change a computers behavior, the operator had to literally move wires aroundin other words, she had to directly alter the hardware. The information technology revolution resulted from the realization that certain kinds of hardware are reprogrammable: drastic changes in function could be made at the software level, by changing inputs, not the hardware itself.

In molecular biomedicine, we are still focused largely on manipulating the cellular hardwarethe proteins that each cell can exploit. But evolution has ensured that cellular collectives use this versatile machinery to process information flexibly and implement a wide range of large-scale body shape outcomes. This is biologys software: the memory, plasticity, and reprogrammability of morphogenetic control networks.

The coming decades will be an extremely exciting time for multidisciplinary efforts in developmental physiology, robotics, and basal cognition to understand how individual cells merge together into a collective with global goals not belonging to any individual cell. This will drive the creation of new artificial intelligence platforms based not on copying brain architectures, but on the multiscale problem-solving capacities of cells and tissues. Conversely, the insights of cognitive neurobiology and computer science will give us a completely new window on the information processing and decision-making dynamics in cellular collectives that can very effectively be targeted for transformative regenerative therapies of complex organs.

Michael Levinis the director of the Allen Discovery Center at Tufts University and Associate Faculty at Harvard Universitys Wyss Institute. Email him atmichael.levin@tufts.edu. M.L. thanks Allen Center Deputy DirectorJoshua Finkelsteinfor suggestions on the drafts of this story.

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How Groups of Cells Cooperate to Build Organs and Organisms - The Scientist

Cell Therapy Market Highlights

Acknowledging the increasing traction that the market is garnering currently, Market Research Future (MRFR) in its recently published analysis asserts that the global cell therapy market is expected to witness significant accruals, growing at a 10.6% CAGR during the forecast period (2017-2023).

Cell therapy has evolved as a recent phase of the biotechnological revolution in the medical sector. The key aim of cell therapy is to target various diseases at the cellular level by restoring a specific cell population as carriers of therapeutic cargo. Besides, cell therapy is used in combination with gene therapy for the treatment of several diseases.

Potential applications of this therapy include treatment of urinary problems, cancers, autoimmune disease, neurological disorders, and infectious disease. In the future, cell therapy will also be used for rebuilding damaged cartilage in joints, repairing spinal cord injuries, and improving the immune system.

Globalcell therapy marketis proliferating rapidly. Factors predominantly driving the growth of the market include the rising prevalence of chronic diseases and disorders, increasing geriatric population, increasing government assistance, and replacement of animal testing models. Besides, technological advancements transpired in the field of biotechnology are escalating the market on the global platform.

Additional factors pushing up the growth of the market include the growing number of neurological disorders and the improvement in the regulatory framework. Other dominant driving forces behind the growth of the global cell therapy market are the regulation of tissue engineering and the exciting possibilities that this therapy is offering in the field of therapeutics.

Conversely, factors such as the challenges that occurred during research and development activities impede the growth of the market. Also, the high cost associated with the development and reconstruction of cells is hampering the market growth especially in the developing and under-developed countries.

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Global Cell Therapy Market Segmentation

For enhanced understanding, the market has been segmented into six key dynamics:

By Type:Autologous and Allogeneic

By Technology:Somatic Cell Technology, Cell Immortalization Technology, Viral Vector Technology, Genome Editing Technology, Cell Plasticity Technology, and Three-Dimensional Technology among others.

By Source:Induced Pluripotent Stem Cells (iPSCs), Bone Marrow, Umbilical Cord Blood-Derived Cells, Adipose Tissue, and Neural Stem Cell among others.

By Application:Musculoskeletal, Cardiovascular, Gastrointestinal, Neurological, Oncology, Dermatology, Wounds & Injuries, and Ocular among others.

By End-users:Hospital & Clinics, Regenerative Medicine Centers, Diagnostic Centers, and Research Institutes among others.

By Regions:North America, Asia Pacific, Europe, and the Rest-of-the-World.

Major Players

Key players leading the global cell therapy market include GlaxoSmithKline plc, Novartis AG, MEDIPOST, PHARMICELL, Osiris,NuVasive, Inc.,Anterogen.Co., Ltd., JCR Pharmaceuticals Co., Ltd, CELLECTIS,Cynata,BioNTechIMFS, Cognate, EUFETS GmbH,Pluristem, Genzyme Corporation, Grupo Praxis, and Advanced Tissue among others.

Global Cell Therapy Market Regional Analysis

The North American region, heading with the successful advancements in therapies dominates the global cell therapy market with a significant share. The market is further expected to grow phenomenally, continuing its dominance from 2017 to 2023. Moreover, the growing number of patients suffering from chronic diseases such as cancer and cardiovascular disorders and well-defined per capita healthcare expenditure are acting as major tailwinds, driving the growth of the regional market.

The US, backed by its huge technological advancements, accounts for the major contributor to the cell therapy market in North America. Furthermore, an increasing number of care facilities offering cell therapies alongside the advanced devices contribute to the growth of the regional market. Also, factors such as the presence of the well-established players, availability of funding for the development of new therapeutics, and treatment positively impact the growth of the market.

The cell therapy market in the European region accounts for the second largest market, globally, expanding at a phenomenal CAGR. The resurging economy in Europe is undoubtedly playing a key role in fostering the growth of the regional market. Additionally, factors such as the availability of technologically advanced devices and the proliferation of quality healthcare along with the increasing healthcare cost contribute to the market growth in the region. Besides, the accessibility to the advanced technology and increasing government support for the R&D activities, propel the market growth in the region.

The Asia Pacific cell therapy market is rapidly emerging as a profitable market, globally. Factors such as the support provided by the government and private entities for research & development will drive the market in the region. Moreover, factors such as the vast advancements in biotechnology and cell reconstructive methods are fostering the growth in the regional market. Furthermore, the rapidly growing healthcare sector led by improving economic conditions positively impacts the regional market. Also, developing healthcare technology and the large unmet needs will foster the growth of the market in the region.

GlobalCell TherapyMarket Competitive Analysis

Highly competitive, the cell therapy market appears to be widely expanded and fragmented characterized by several small and large-scale players. To gain a competitive edge and to sustain their position in the market, these players incorporate various strategic initiatives such as partnership, acquisition, collaboration, expansion, and product launch.

The structure of the market is changing due to the acquisition of local players by multinational companies. Because of the increasing competition in the market, multinational companies are using the strategy of acquisition, which increases the profit of the company while significantly reducing the competition.

Industry, Innovation & Related News

March 12, 2019 -Cell Medica Ltd. (the UK), a leading global company engaging in the development, manufacture, and commercialization of cellular immunotherapy products for the treatment of cancer and viral infections announced the receiving of a grant of USD 8.7 MN from the Cancer Prevention and Research Institute of Texas (CPRIT the US) to accelerate off-the-shelf CAR-NKT cell therapy.

In addition to being available off-the-shelf, the new cell-based therapy CMD-502 uses donor-derived natural killer T-cells to fight cancer and is expected to have a better safety profile than current chimeric antigen receptor (CAR) T-cell therapies. The therapy is being developed and refined in collaboration with the Baylor College of Medicine (BCM Texas, the US).

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About Market Research Future:

AtMarket Research Future (MRFR), we enable our customers to unravel the complexity of various industries through our Cooked Research Report (CRR), Half-Cooked Research Reports (HCRR), Raw Research Reports (3R), Continuous-Feed Research (CFR), and Market Research & Consulting Services.

MRFR team have supreme objective to provide the optimum quality market research and intelligence services to our clients. Our market research studies by Components, Application, Logistics and market players for global, regional, and country level market segments, enable our clients to see more, know more, and do more, which help to answer all their most important questions.

In order to stay updated with technology and work process of the industry, MRFR often plans & conducts meet with the industry experts and industrial visits for its research analyst members.

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Knowing the Global Cell Therapy Market; MRFR Reveals Insights for 2017 2023 - The Daily Chronicle

When Ralph Fisher,a Texas cattle rancher, set eyes on one of the worlds first cloned calves in August 1999, he didnt care what the scientists said: He knew it was his old Brahman bull, Chance, born again. About a year earlier, veterinarians at Texas A&M extracted DNA from one of Chances moles and used the sample to create a genetic double. Chance didnt live to meet his second self, but when the calf was born, Fisher christened him Second Chance, convinced he was the same animal.

Scientists cautioned Fisher that clones are more like twins than carbon copies: The two may act or even look different from one another. But as far as Fisher was concerned, Second Chance was Chance. Not only did they look identical from a certain distance, they behaved the same way as well. They ate with the same odd mannerisms; laid in the same spot in the yard. But in 2003, Second Chance attacked Fisher and tried to gore him with his horns. About 18 months later, the bull tossed Fisher into the air like an inconvenience and rammed him into the fence. Despite 80 stitches and a torn scrotum, Fisher resisted the idea that Second Chance was unlike his tame namesake,telling the radio program This American Life that I forgive him, you know?

In the two decades since Second Chance marked a genetic engineering milestone, cattle have secured a place on the front lines of biotechnology research. Today, scientists around the world are using cutting-edge technologies, fromsubcutaneous biosensorstospecialized food supplements, in an effort to improve safety and efficiency within the$385 billion global cattle meat industry. Beyond boosting profits, their efforts are driven by an imminent climate crisis, in which cattle play a significant role, and growing concern for livestock welfare among consumers.

Gene editing stands out as the most revolutionary of these technologies. Although gene-edited cattle have yet to be granted approval for human consumption, researchers say tools like Crispr-Cas9 could let them improve on conventional breeding practices and create cows that are healthier, meatier, and less detrimental to the environment. Cows are also beinggiven genesfrom the human immune system to create antibodies in the fight against Covid-19. (The genes of non-bovine livestock such as pigs and goats, meanwhile, have been hacked togrow transplantable human organsandproduce cancer drugs in their milk.)

But some experts worry biotech cattle may never make it out of the barn. For one thing, theres the optics issue: Gene editing tends to grab headlines for its role in controversial research and biotech blunders. Crispr-Cas9 is often celebrated for its potential to alter the blueprint of life, but that enormous promise can become a liability in the hands of rogue and unscrupulous researchers, tempting regulatory agencies to toughen restrictions on the technologys use. And its unclear how eager the public will be to buy beef from gene-edited animals. So the question isnt just if the technology will work in developing supercharged cattle, but whether consumers and regulators will support it.

Cattle are catalysts for climate change. Livestockaccount for an estimated 14.5 percent of greenhouse gas emissions from human activities, of which cattle are responsible for about two thirds, according to the United Nations Food and Agriculture Organization (FAO). One simple way to address the issue is to eat less meat. But meat consumption is expected to increasealong with global population and average income. A 2012reportby the FAO projected that meat production will increase by 76 percent by 2050, as beef consumption increases by 1.2 percent annually. And the United States isprojected to set a recordfor beef production in 2021, according to the Department of Agriculture.

For Alison Van Eenennaam, an animal geneticist at the University of California, Davis, part of the answer is creating more efficient cattle that rely on fewer resources. According to Van Eenennaam, the number of dairy cows in the United Statesdecreasedfrom around 25 million in the 1940s to around 9 million in 2007, while milk production has increased by nearly 60 percent. Van Eenennaam credits this boost in productivity to conventional selective breeding.

You dont need to be a rocket scientist or even a mathematician to figure out that the environmental footprint or the greenhouse gases associated with a glass of milk today is about one-third of that associated with a glass of milk in the 1940s, she says. Anything you can do to accelerate the rate of conventional breeding is going to reduce the environmental footprint of a glass of milk or a pound of meat.

Modern gene-editing tools may fuel that acceleration. By making precise cuts to DNA, geneticists insert or remove naturally occurring genes associated with specific traits. Some experts insist that gene editing has the potential to spark a new food revolution.

Jon Oatley, a reproductive biologist at Washington State University, wants to use Crispr-Cas9 to fine tune the genetic code of rugged, disease-resistant, and heat-tolerant bulls that have been bred to thrive on the open range. By disabling a gene called NANOS2, he says he aims to eliminate the capacity for a bull to make his own sperm, turning the recipient into a surrogate for sperm-producing stem cells from more productive prized stock. These surrogate sires, equipped with sperm from prize bulls, would then be released into range herds that are often genetically isolated and difficult to access, and the premium genes would then be transmitted to their offspring.

Furthermore, surrogate sires would enable ranchers to introduce desired traits without having to wrangle their herd into one place for artificial insemination, says Oatley. He envisions the gene-edited bulls serving herds in tropical regions like Brazil, the worldslargestbeef exporter and home to around 200 million of the approximately 1.5 billion head of cattle on Earth.

Brazils herds are dominated by Nelore, a hardy breed that lacks the carcass and meat quality of breeds like Angus but can withstand high heat and humidity. Put an Angus bull on a tropical pasture and hes probably going to last maybe a month before he succumbs to the environment, says Oatley, while a Nelore bull carrying Angus sperm would have no problem with the climate.

The goal, according to Oatley, is to introduce genes from beefier bulls into these less efficient herds, increasing their productivity and decreasing their overall impact on the environment. We have shrinking resources, he says, and need new, innovative strategies for making those limited resources last.

Oatley has demonstrated his technique in mice but faces challenges with livestock. For starters, disabling NANOS2 does not definitively prevent the surrogate bull from producing some of its own sperm. And while Oatley has shown he can transplant sperm-producing cells into surrogate livestock, researchers have not yet published evidence showing that the surrogatesproduceenough quality sperm to support natural fertilization. How many cells will you need to make this bull actually fertile? asks Ina Dobrinski, a reproductive biologist at the University of Calgary who helped pioneer germ cell transplantation in large animals.

But Oatleys greatest challenge may be one shared with others in the bioengineered cattle industry: overcoming regulatory restrictions and societal suspicion. Surrogate sires would be classified as gene-edited animals by the Food and Drug Administration, meaning theyd face a rigorous approval process before their offspring could be sold for human consumption. But Oatley maintains that if his method is successful, the sperm itself would not be gene-edited, nor would the resulting offspring. The only gene-edited specimens would be the surrogate sires, which act like vessels in which the elite sperm travel.

Even so, says Dobrinski, Thats a very detailed difference and Im not sure how that will work with regulatory and consumer acceptance.

In fact, American attitudes towards gene editing have been generally positive when the modification is in the interest of animal welfare. Many dairy farmers prefer hornless cows horns can inflict damage when wielded by 1,500-pound animals so they often burn them off in apainful processusing corrosive chemicals and scalding irons. Ina study published last yearin the journal PLOS One, researchers found that most Americans are willing to consume food products from cows genetically modified to be hornless.

Still, experts say several high-profile gene-editing failures in livestock andhumansin recent years may lead consumers to consider new biotechnologies to be dangerous and unwieldy.

In 2014, a Minnesota startup called Recombinetics, a company with which Van Eenennaams lab has collaborated, created a pair of cross-bred Holstein bulls using the gene-editing tool TALENs, a precursor to Crispr-Cas9, making cuts to the bovine DNA and altering the genes to prevent the bulls from growing horns. Holstein cattle, which almost always carry horned genes, are highly productive dairy cows, so using conventional breeding to introduce hornless genes from less productive breeds can compromise the Holsteins productivity. Gene editing offered a chance to introduce only the genes Recombinetics wanted. Their hope was to use this experiment to prove that milk from the bulls female progeny was nutritionally equivalent to milk from non-edited stock. Such results could inform future efforts to make Holsteins hornless but no less productive.

The experiment seemed to work. In 2015, Buri and Spotigy were born. Over the next few years, the breakthrough received widespread media coverage, and when Buris hornless descendant graced thecover of Wired magazine in April 2019, it did so as the ostensible face of the livestock industrys future.

But early last year, a bioinformatician at the FDA ran a test on Buris genome and discovered an unexpected sliver of genetic code that didnt belong. Traces of bacterial DNA called a plasmid, which Recombinetics used to edit the bulls genome, had stayed behind in the editing process, carrying genes linked to antibiotic resistance in bacteria. After the agency publishedits findings, the media reaction was swift and fierce: FDA finds a surprise in gene-edited cattle: antibiotic-resistant, non-bovine DNA,readone headline. Part cow, part bacterium?readanother.

Recombinetics has since insisted that the leftover plasmid DNA was likely harmless and stressed that this sort of genetic slipup is not uncommon.

Is there any risk with the plasmid? I would say theres none, says Tad Sonstegard, president and CEO of Acceligen, a Recombinetics subsidiary. We eat plasmids all the time, and were filled with microorganisms in our body that have plasmids. In hindsight, Sonstegard says his teams only mistake was not properly screening for the plasmid to begin with.

While the presence of antibiotic-resistant plasmid genes in beef probably does not pose a direct threat to consumers, according to Jennifer Kuzma, a professor of science and technology policy and co-director of the Genetic Engineering and Society Center at North Carolina State University, it does raise the possible risk of introducing antibiotic-resistant genes into the microflora of peoples digestive systems. Although unlikely, organisms in the gut could integrate those genes into their own DNA and, as a result, proliferate antibiotic resistance, making it more difficult to fight off bacterial diseases.

The lesson that I think is learned there is that science is never 100 percent certain, and that when youre doing a risk assessment, having some humility in your technology product is important, because you never know what youre going to discover further down the road, she says. In the case of Recombinetics. I dont think there was any ill intent on the part of the researchers, but sometimes being very optimistic about your technology and enthusiastic about it causes you to have blinders on when it comes to risk assessment.

The FDA eventually clarified its results, insisting that the study was meant only to publicize the presence of the plasmid, not to suggest the bacterial DNA was necessarily dangerous. Nonetheless, the damage was done. As a result of the blunder,a plan was quashedforRecombinetics to raise an experimental herd in Brazil.

Backlash to the FDA study exposed a fundamental disagreement between the agency and livestock biotechnologists. Scientists like Van Eenennaam, who in 2017 received a $500,000 grant from the Department of Agriculture to study Buris progeny, disagree with the FDAs strict regulatory approach to gene-edited animals. Typical GMOs aretransgenic, meaning they have genes from multiple different species, but modern gene-editing techniques allow scientists to stay roughly within the confines of conventional breeding, adding and removing traits that naturally occur within the species.

That said, gene editing is not yet free from errors and sometimes intended changes result in unintended alterations, notes Heather Lombardi, division director of animal bioengineering and cellular therapies at the FDAs Center for Veterinary Medicine. For that reason, the FDA remains cautious.

Theres a lot out there that I think is still unknown in terms of unintended consequences associated with using genome-editing technology, says Lombardi. Were just trying to get an understanding of what the potential impact is, if any, on safety.

Bhanu Telugu, an animal scientist at the University of Maryland and president and chief science officer at the agriculture technology startup RenOVAte Biosciences, worries that biotech companies willmigrate their experimentsto countries with looser regulatory environments. Perhaps more pressingly, he says strict regulation requiring long and expensive approval processes may incentivize these companies to work only on traits that are most profitable, rather than those that may have the greatest benefit for livestock and society, such as animal well-being and the environment.

What company would be willing to spend $20 million on potentially alleviating heat stress at this point? he asks.

On a windywinter afternoon, Raluca Mateescu leaned against a fence post at the University of Floridas Beef Teaching Unit while a Brahman heifer sniffed inquisitively at the air and reached out its tongue in search of unseen food. Since 2017, Mateescu, an animal geneticist at the university, has been part of a team studying heat and humidity tolerance in breeds like Brahman and Brangus (a mix between Brahman and Angus cattle). Her aim is to identify the genetic markers that contribute to a breeds climate resilience, markers that might lead to more precise breeding and gene-editing practices.

In the South, Mateescu says, heat and humidity are a major problem. That poses a stress to the animals because theyre selected for intense production to produce milk or grow fast and produce a lot of muscle and fat.

Like Nelore cattle in South America, Brahman are well-suited for tropical and subtropical climates, but their high tolerance for heat and humidity comes at the cost of lower meat quality than other breeds. Mateescu and her team have examined skin biopsies and found that relatively large sweat glands allow Brahman to better regulate their internal body temperature. With funding from the USDAs National Institute of Food and Agriculture, the researchers now plan to identify specific genetic markers that correlate with tolerance to tropical conditions.

If were selecting for animals that produce more without having a way to cool off, were going to run into trouble, she says.

There are other avenues in biotechnology beyond gene editing that may help reduce the cattle industrys footprint. Although still early in their development,lab-cultured meatsmay someday undermine todays beef producers by offering consumers an affordable alternative to the conventionally grown product, without the animal welfare and environmental concerns that arise from eating beef harvested from a carcass.

Other biotech techniques hope to improve the beef industry without displacing it. In Switzerland, scientists at a startup called Mootral areexperimenting with a garlic-based food supplementdesigned to alter the bovine digestive makeup to reduce the amount of methane they emit. Studies have shown the product to reduce methane emissions by about 20 percent in meat cattle, according to The New York Times.

In order to adhere to the Paris climate agreement, Mootrals owner, Thomas Hafner, believes demand will grow as governments require methane reductions from their livestock producers. We are working from the assumption that down the line every cow will be regulated to be on a methane reducer, he told The New York Times.

Meanwhile, a farm science research institute in New Zealand, AgResearch, hopes to target methane production at its source by eliminating methanogens, the microbes thought to be responsible for producing the greenhouse gas in ruminants. The AgResearch team isattempting to developa vaccine to alter the cattle guts microbial composition, according to the BBC.

Genomic testing may also allow cattle producers to see what genes calves carry before theyre born, according to Mateescu, enabling producers to make smarter breeding decisions and select for the most desirable traits, whether it be heat tolerance, disease resistance, or carcass weight.

Despite all these efforts, questions remain as to whether biotech can ever dramatically reduce the industrys emissions or afford humane treatment to captive animals in resource-intensive operations. To many of the industrys critics, including environmental and animal rights activists, the very nature of the practice of rearing livestock for human consumption erodes the noble goal of sustainable food production. Rather than revamp the industry, these critics suggest alternatives such as meat-free diets to fulfill our need for protein. Indeed,data suggestsmany young consumers are already incorporating plant-based meats into their meals.

Ultimately, though, climate change may be the most pressing issue facing the cattle industry, according to Telugu of the University of Maryland, which received a grant from the Bill and Melinda Gates Foundation to improve productivity and adaptability in African cattle. We cannot breed our way out of this, he says.

Dyllan Furness is a Florida-based science and technology journalist. His work has appeared in Quartz, OneZero, and PBS, among other outlets. Follow him on Twitter @dyllonline

This article was originally published at Undark and has been republished here with permission. Follow Undark on Twitter @undarkmag

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CRISPR cows could boost sustainable meat production, but regulations, wary consumers stand in the way - Genetic Literacy Project

On April 15, 2002, the FDA approved a temporary treatment for wrinkles that would revolutionize aging. All of a sudden, you could waltz into a derms office and get your frown lines ironed out faster than it would take to iron an actual shirt. It was called botulinum toxin,Botox for short.

Eighteen years later, a few units of Botox every three months has become the norm for millions around the world (more than seven million yearly in the U.S. alone). Now, if someone had told your grandparents, or even your parents, 20 years ago that people would be getting their foreheads frozen to look younger, they likely would have scoffed at the idea. So just imagine what other wild fixes could be coming to a medi-spa near you.

Its exciting to think about how the next 10 years will look, says Dr. Rohan Bissoondath, medical director of Calgarys Preventous Cosmetic Medicine clinic. With lifespan increasing, people are routinely going to be living into their hundreds, so we want to look great as well. From magic pills to creams that mimic injections, we take a look at the incredible innovations on the horizon.

The best products worth your time, money and energy get beauty news and more delivered to your inbox with our daily newsletter.

The way science is progressing, facelifts are set to become obsolete, says Dr. Lisa Kellett of Torontos DLK on Avenue. I think that the gold standard will eventually be finding ways to regenerate and kick-start our own collagen instead of doing a facelift. Kellett is already trying out cutting-edge technology to accomplish this, such as a laser that delivers growth factors right in the dermis to regenerate tissue. Its pretty snazzy stuff, but she anticipates even greater advances in coming years. I think well be able to use stem cells in conjunction with technology to regenerate collagenI think thats what well be doing one day.

Botox in a cream? This has been in the pipeline for a while, says Bissoondath. The challenge is getting the molecules to penetrate the skin so that they can act on the muscle. Maybe on crows feet because its a thinner area, thinner muscles; that may be an area where we see some utility for it, but its still out there. Topical Botox had some success in trials, but scientists still have kinks to work out. In the meantime, a Botox cream might be beneficial even if it doesnt reach muscles, says Bissoondath. I see the potential for having it in a cream and applying it to the whole face, not necessarily affecting facial expressions, but giving an improved glow and better skin quality.

If you want to smooth, you get Botox. If you want to brighten, you get IPL. If you want to tighten, you get Thermage. But what if there was a treatment that did it all? I think thats the future of aging, says Kellett, who is just about to launch such a treatment at her clinic. Marketed as the next generation of laser and light-based platform technology, Ethera is a multiple modality device that can tackle everything from dark spots and skin laxity to textural issues and wrinkles. It means that when patients come in, theyre not just doing one thing, says the doc. Instead, in the same appointment, shes able to address a variety of concerns with a single machine.

Okay, this is very cool. Something I think is possible is a pill to replace exercise, says Bissoondath, who adds that this could be developed in the not so distant future. With the advances were making in understanding the functions of our body down to the cellular level and intracellular level, and understanding how our mitochondria actually ages, were looking at ways now where we can manipulate that from a pill perspective. The pill wouldnt deliver all the benefits of physical activity, such as the positive impact on our mood, but it would replicate its effects on our body. It wont take the place of walking around outside and soaking up natureit cant do the mental part of it. But as far as the physiologic, biochemical part of it, were really understanding that better and making big strides. Its exciting.

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Best Anti Aging Advances to ComeHow We'll Soon Be Looking Younger - The Kit

NEW YORK, Aug. 14, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in cellular medicines for inflammatory diseases, today announced that the Oncologic Drugs Advisory Committee (ODAC) of the United States Food and Drug Administration (FDA) voted overwhelmingly in favor that the available data support the efficacy of remestemcel-L (RYONCIL) in pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD).

Mesoblast Chief Medical Officer Dr Fred Grossman said: Steroid-refractory acute graft versus host disease is an area of extreme need, especially in vulnerable children under 12 years old where there is no approved therapy. We are very encouraged by todays outcome and are committed to working closely with the FDA as they complete their review of our submission regarding approval of RYONCIL for this life-threatening complication of an allogeneic bone marrow transplant.

The ODAC is an independent panel of experts that evaluates efficacy and safety of data and makes appropriate recommendations to the FDA.Although the FDA will consider the recommendation of the panel, the final decision regarding the approval of the product is made solely by the FDA, and the recommendations by the panel are non-binding. RYONCIL has been accepted for Priority Review by the FDA with an action date of September 30, 2020, under the Prescription Drug User Fee Act (PDUFA). If approved by the PDUFA date, Mesoblast plans to launch RYONCIL in the United States in 2020.

Pediatric transplant physician Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatrics and Professor of Pathology, and Director, Pediatric Blood and Marrow Transplant Program at Duke University Medical Center, said: This devastating condition has an extremely poor prognosis and there are no FDA-approved options for children under the age of 12. The clinical studies I have directed have demonstrated the potential for this treatment to fill a significant unmet medical need.

Conference CallAn audio webcast can be accessed via https://webcast.boardroom.media/mesoblast-limited/20200813/NaN5f3237e85300840019de909d

The archived webcast is also available on the Investor page of the Companys website http://www.mesoblast.com

About Acute Graft Versus Host Disease Acute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.1 In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.2,3 There are currently no FDA-approved treatments in the United States for children under 12 with SR-aGVHD, a potentially life-threatening complication of an allogeneic bone marrow transplant for blood cancer.

About RYONCILTM Mesoblasts lead product candidate, RYONCIL (remestemcel-L), is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in steroid-refractory acute graft versus host disease by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

1. Niederwieser D, Baldomero H, Szer J. Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. Bone Marrow Transplant 2016; 51(6):778-85. 2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology 2011;2011:601953. 3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation 2019;54(11):1805-1814

About MesoblastMesoblast Limited (Nasdaq:MESO; ASX:MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblast has a strong and extensive global intellectual property (IP) portfolio with protection extending through to at least 2040 in all major markets. The Companys proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblasts Biologics License Application to seek approval of its product candidate RYONCIL (remestemcel-L) for pediatric steroid-refractory acute graft versus host disease (acute GVHD) has been accepted for priority review by the United States Food and Drug Administration (FDA), and if approved, product launch in the United States is expected in 2020. Remestemcel-L is also being developed for other inflammatory diseases in children and adults including moderate to severe acute respiratory distress syndrome. Mesoblast is completing Phase 3 trials for its product candidates for advanced heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of Mesoblasts preclinical and clinical studies, and Mesoblasts research and development programs; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblasts ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals (including any decision that the FDA may make based upon the recommendation of the ODAC in relation to the efficacy of remestemcel-L), manufacturing activities and product marketing activities, if any; the commercialization of Mesoblasts product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblasts product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblasts ability to enter into and maintain established strategic collaborations; Mesoblasts ability to establish and maintain intellectual property on its product candidates and Mesoblasts ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

For further information, please contact:

Excerpt from:
U.S. FDA Advisory Committee Votes Nine to One in Favor of Remestemcel-L (Ryoncil) for Efficacy in Children With Steroid-Refractory Acute Graft Versus...

Patient enrollment to commence immediately

VANCOUVER, Washington, Aug. 20, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today the Clinical Trials Unit of the Medicines & Healthcare product Regulatory Agency (MHRA) of the U.K. government authorized the Company to enroll for its ongoing Phase 3 COVID-19 trial for severe-to-critical patients in the United Kingdom. The MHRAs decision follows several months of its review of CytoDyns manufacturing processes and leronlimabs safety profile.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, stated, We are very pleased with the MHRAs confidence in leronlimab to initiate enrollment of patients in the U.K. for our current CD12 protocol. CytoDyn recently requested fast track approval from the MHRA for its completed Phase 2 COVID-19 trial for the mild-to-moderate population, with strong efficacy and safety data. We believe leronlimab has multiple opportunities for several clinical indications and we are very optimistic about our future based upon how far we have advanced this drug in about 5 years. In addition, we plan to file a BLA for HIV in the U.K. within the next 4 weeks.

About Coronavirus Disease 2019CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a randomized clinical trial for mild-to-moderate patients in the U.S. Enrollment continues in its Phase 3 randomized clinical trial for the severe-to-critically ill COVID-19 population in several hospitals throughout the country.

SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 is believed to typically transmit person-to-person through respiratory droplets. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140)The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA has agreed to provide written responses to the Companys questions concerning its recent Biologics License Application by September 4, 2020, in lieu of a Type A teleconference meeting for this HIV combination therapy.

CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years.

CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Michael MulhollandOffice: 360.980.8524, ext. 102Mobile: 503.341.3514mmulholland@cytodyn.com

Continued here:
After Several Months of Providing Requested Information About Manufacturing and Safety of Leronlimab, U.K.'s MHRA Accepts CytoDyn's Request to Enroll...

New York, Aug. 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Hydrogels Industry" - https://www.reportlinker.com/p05896103/?utm_source=GNW Strong R&D interest is already underway for hydrogel biomaterials. New developments in hydrogel design and hydrogel synthesis are resulting in the development of hydrogels with mechanical properties. Superporous comb-type grafted hydrogels with fast response times; hybrid graft copolymers based self-assembling hydrogels; protein based hydrogels and hybrid hydrogels are the emerging new future of smart hydrogel based biomaterials. Stimuli-sensitive hydrogels, especially polypeptide based responsive hydrogels hold promising potential. Protein hydrogel are more biocompatible than synthetic hydrogel as they do not require the use of oxic chemical crosslinkers. This represents a key growth opportunity in the market given that traditional hydrogels have been largely limited by their poor mechanical properties and slow response times to stimuli. Temperature-sensitive hydrogels especially will find attractive opportunities in biomedicine.

Wound dressings currently remain a popular application area with hydrogel being effective for treating dry necrotic wounds and rapid healing of burn wounds. Hydrogel enables painless debridement of infected tissue and provides a moist wound environment for faster healing. Chitosan-based hydrogels, in this regard, are growing in popularity for their biocompatible, antimicrobial, and hemostatic effects. Acellular Hydrogel is especially valuable in accelerated healing of third-degree burn wounds and is a welcome substitute for complicated and infection prone skin grafts. Encouraging progress is being made in the use of hydrogels for targeted & controlled drug delivery. Hydrogels can prolong drug release kinetics. Their porosity and aqueous features make them perfect biocompatible drug delivery vehicles. Chitosan-based hydrogels can be loaded with active drug compounds like growth factors or stem cells that are important in providing scaffold for cell growth. The growing focus on controlled and targeted drug delivery systems in the field of cardiology, oncology, immunology, and pain management bodes well for future growth in the market. Some of the physical properties of hydrogel that can be manipulated and tuned to suit drug delivery needs include porosity, swelling and elasticity in response to stimuli such as temperature, solvent quality, pH, electric field; resistance to dissolution; free diffusion of solute molecules in water; among others. These properties help in controlled drug release and protect from drug degradation, thereby making them highly effective vehicles for drug delivery systems. Some of the types of hydrogels development for drug delivery include DNA-hydrogels; supramolecular hydrogels; bio-inspired hydrogels; and multi-functional and stimuli-responsive hydrogels. New emerging uses in contact lenses and tissue engineering will also benefit growth in the market in the coming years. The United States and Europe represent large markets worldwide with a combined share of 52.4% of the market. China ranks as the fastest growing market with a CAGR of 7.3% over the analysis period supported by the governments focus on revolutionizing biomedical engineering in the country. The country today ranks as the top country for biomedical research encouraged by a permissive regulatory climate.

Read the full report: https://www.reportlinker.com/p05896103/?utm_source=GNW

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW Expanding Applications and Product Innovations Spur Growth in the Global Hydrogel Market Emerging Economies to Post Strong Growth Industry Witnesses Rise in Demand for Synthetic Hydrogels Synthetic Hydrogels by Polymer Type: A Snapshot Global Competitor Market Shares Hydrogel Competitor Market Share Scenario Worldwide (in %): 2019 Hydrogel Competitor Market Share Scenario Worldwide (in %): 2019

2. FOCUS ON SELECT PLAYERS 3M Company (USA) ACELITY L.P, Inc. (USA) Ashland, Inc. (USA) Braun Melsungen AG (Germany) Cardinal Health, Inc. (USA) Coloplast A/S (Denmark) ConvaTec, Inc. (USA) Integra LifeSciences Holdings Corporation (USA) Evonik Industries AG (Germany) Gentell, Inc. (USA) Hollister, Inc. (USA) Mlnlycke Health Care AB (Sweden) Ocular Therapeutix, Inc. (USA) Sekisui Plastics Co. Ltd. (Japan) Smith & Nephew, Plc (UK)

3. MARKET TRENDS & DRIVERS Innovations Expand Addressable Market for Hydrogels Rise in Incidence of Chronic Diseases and High Treatment Costs Drive Demand for Hydrogel Dressings for Wound Healing Global Prevalence of Wounds Global Wound Care Market: Percentage Breakdown of Spending by Wound Type Personal Care Product: An Evolving Niche Market Global Skin Care Market Size in US$ Billion for the Years 2019, 2021, 2023 and 2025 Consumer Adoption of Hydrogel Contact Lenses Augurs Well for Market Growth Global Contact Lens Fits by Category (In %): 2019 Hydrogels Evolve as Emerging Alternative for Food Packaging Agriculture Sector Depicts Strong Growth Potential Global Water Utilization: Percentage Share Breakdown for Agricultural Practices, Industrial Processes, and Domestic Usage Rising Concerns over Polluting Water Resources: An Opportunity for Hydrogels Market Need for Wastewater Treatment Presents Opportunity for Hydrogels: Percentage of Wastewater Treated in Europe, Asia, Latin America, and Africa Growing Emphasis on Sustainability and Positive Impact on Hydrogels Growth in Biomedical Applications of Hydrogels Hydrogels for Cartilage Regeneration Growing Need for Targeted Controlled Drug Delivery (TCDD) Drives Importance of Hydrogels Hydrogel Nanoparticles: The New Hydrogels for Drug Delivery Evaporative Cooling Hydrogel Packaging: Increasing Storage Stability of Pharmaceuticals Growing Focus on Baby Hygiene Products Spells Steady Growth Opportunities for Hydrogels Annual Usage of Baby Disposable Diapers Per Infant by Region: ( Age upto 2.5 years) Global New Births (in Millions) per Annum by Geographic Region World Fertility Rate in % by Region (2013 & 2050F) Increased Demand for Feminine Hygiene Products Global Female Population by Geographic Region: Percentage Breakdown by Region for 2018 Number of Menstruating Women Worldwide by Country: 15-49 Years Female Population (in Millions) for 2013 & 2025P Aging Population and the Associated Complications Drive the Demand for Hydrogel Global Population Statistics for the 65+ Age Group in Million by Geographic Region for the Years 2019, 2025, 2035 and 2050 Rise in Demand for Novel Hydrogel Dressings for Wound Healing Propels Innovations PRODUCT OVERVIEW Hydrogel Types of Hydrogel Natural Hydrogels Select Natural Hydrogels: Advantages and Disadvantages Synthetic Hydrogels Select Synthetic Hydrogels: Advantages and Disadvantages Hybrid Hydrogels

4. GLOBAL MARKET PERSPECTIVE Table 1: Hydrogels Global Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020-2027

Table 2: Hydrogels Global Retrospective Market Scenario in US$ Thousand by Region/Country: 2012-2019

Table 3: Hydrogels Market Share Shift across Key Geographies Worldwide: 2012 VS 2020 VS 2027

Table 4: Natural (Raw Material) World Market by Region/Country in US$ Thousand: 2020 to 2027

Table 5: Natural (Raw Material) Historic Market Analysis by Region/Country in US$ Thousand: 2012 to 2019

Table 6: Natural (Raw Material) Market Share Breakdown of Worldwide Sales by Region/Country: 2012 VS 2020 VS 2027

Table 7: Synthetic (Raw Material) Potential Growth Markets Worldwide in US$ Thousand: 2020 to 2027

Table 8: Synthetic (Raw Material) Historic Market Perspective by Region/Country in US$ Thousand: 2012 to 2019

Table 9: Synthetic (Raw Material) Market Sales Breakdown by Region/Country in Percentage: 2012 VS 2020 VS 2027

Table 10: Hybrid (Raw Material) Geographic Market Spread Worldwide in US$ Thousand: 2020 to 2027

Table 11: Hybrid (Raw Material) Region Wise Breakdown of Global Historic Demand in US$ Thousand: 2012 to 2019

Table 12: Hybrid (Raw Material) Market Share Distribution in Percentage by Region/Country: 2012 VS 2020 VS 2027

Table 13: Polyacrylate (Composition) World Market Estimates and Forecasts by Region/Country in US$ Thousand: 2020 to 2027

Table 14: Polyacrylate (Composition) Market Historic Review by Region/Country in US$ Thousand: 2012 to 2019

Table 15: Polyacrylate (Composition) Market Share Breakdown by Region/Country: 2012 VS 2020 VS 2027

Table 16: Polyacrylamide (Composition) World Market by Region/Country in US$ Thousand: 2020 to 2027

Table 17: Polyacrylamide (Composition) Historic Market Analysis by Region/Country in US$ Thousand: 2012 to 2019

Table 18: Polyacrylamide (Composition) Market Share Distribution in Percentage by Region/Country: 2012 VS 2020 VS 2027

Table 19: Silicon (Composition) World Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020 to 2027

Table 20: Silicon (Composition) Market Worldwide Historic Review by Region/Country in US$ Thousand: 2012 to 2019

Table 21: Silicon (Composition) Market Percentage Share Distribution by Region/Country: 2012 VS 2020 VS 2027

Table 22: Other Compositions (Composition) Market Opportunity Analysis Worldwide in US$ Thousand by Region/Country: 2020 to 2027

Table 23: Other Compositions (Composition) Global Historic Demand in US$ Thousand by Region/Country: 2012 to 2019

Table 24: Other Compositions (Composition) Market Share Distribution in Percentage by Region/Country: 2012 VS 2020 VS 2027

Table 25: Agriculture (Application) Worldwide Sales in US$ Thousand by Region/Country: 2020-2027

Table 26: Agriculture (Application) Historic Demand Patterns in US$ Thousand by Region/Country: 2012-2019

Table 27: Agriculture (Application) Market Share Shift across Key Geographies: 2012 VS 2020 VS 2027

Table 28: Healthcare & Hygiene (Application) Global Market Estimates & Forecasts in US$ Thousand by Region/Country: 2020-2027

Table 29: Healthcare & Hygiene (Application) Retrospective Demand Analysis in US$ Thousand by Region/Country: 2012-2019

Table 30: Healthcare & Hygiene (Application) Market Share Breakdown by Region/Country: 2012 VS 2020 VS 2027

Table 31: Contact Lenses (Application) Demand Potential Worldwide in US$ Thousand by Region/Country: 2020-2027

Table 32: Contact Lenses (Application) Historic Sales Analysis in US$ Thousand by Region/Country: 2012-2019

Table 33: Contact Lenses (Application) Share Breakdown Review by Region/Country: 2012 VS 2020 VS 2027

Table 34: Drug Delivery (Application) Worldwide Latent Demand Forecasts in US$ Thousand by Region/Country: 2020-2027

Table 35: Drug Delivery (Application) Global Historic Analysis in US$ Thousand by Region/Country: 2012-2019

Table 36: Drug Delivery (Application) Distribution of Global Sales by Region/Country: 2012 VS 2020 VS 2027

Table 37: Tissue Engineering (Application) Sales Estimates and Forecasts in US$ Thousand by Region/Country for the Years 2020 through 2027

Table 38: Tissue Engineering (Application) Analysis of Historic Sales in US$ Thousand by Region/Country for the Years 2012 to 2019

Table 39: Tissue Engineering (Application) Global Market Share Distribution by Region/Country for 2012, 2020, and 2027

Table 40: Other Applications (Application) Global Opportunity Assessment in US$ Thousand by Region/Country: 2020-2027

Table 41: Other Applications (Application) Historic Sales Analysis in US$ Thousand by Region/Country: 2012-2019

Table 42: Other Applications (Application) Percentage Share Breakdown of Global Sales by Region/Country: 2012 VS 2020 VS 2027

III. MARKET ANALYSIS

GEOGRAPHIC MARKET ANALYSIS

UNITED STATES Table 43: United States Hydrogels Market Estimates and Projections in US$ Thousand by Raw Material: 2020 to 2027

Table 44: Hydrogels Market in the United States by Raw Material: A Historic Review in US$ Thousand for 2012-2019

Table 45: United States Hydrogels Market Share Breakdown by Raw Material: 2012 VS 2020 VS 2027

Material: 2012 VS 2020 VS 202

Table 47: Hydrogels Market in the United States by Composition: A Historic Review in US$ Thousand for 2012-2019

Table 48: United States Hydrogels Market Share Breakdown by Composition: 2012 VS 2020 VS 2027

Table 49: United States Hydrogels Latent Demand Forecasts in US$ Thousand by Application: 2020 to 2027

Table 50: Hydrogels Historic Demand Patterns in the United States by Application in US$ Thousand for 2012-2019

Table 51: Hydrogels Market Share Breakdown in the United States by Application: 2012 VS 2020 VS 2027

CANADA Table 52: Canadian Hydrogels Market Estimates and Forecasts in US$ Thousand by Raw Material: 2020 to 2027

Table 53: Canadian Hydrogels Historic Market Review by Raw Material in US$ Thousand: 2012-2019

Table 54: Hydrogels Market in Canada: Percentage Share Breakdown of Sales by Raw Material for 2012, 2020, and 2027

Table 55: Canadian Hydrogels Market Estimates and Forecasts in US$ Thousand by Composition: 2020 to 2027

Table 56: Canadian Hydrogels Historic Market Review by Composition in US$ Thousand: 2012-2019

Table 57: Hydrogels Market in Canada: Percentage Share Breakdown of Sales by Composition for 2012, 2020, and 2027

Table 58: Canadian Hydrogels Market Quantitative Demand Analysis in US$ Thousand by Application: 2020 to 2027

Table 59: Hydrogels Market in Canada: Summarization of Historic Demand Patterns in US$ Thousand by Application for 2012-2019

Table 60: Canadian Hydrogels Market Share Analysis by Application: 2012 VS 2020 VS 2027

JAPAN Table 61: Japanese Market for Hydrogels: Annual Sales Estimates and Projections in US$ Thousand by Raw Material for the Period 2020-2027

Table 62: Hydrogels Market in Japan: Historic Sales Analysis in US$ Thousand by Raw Material for the Period 2012-2019

Table 63: Japanese Hydrogels Market Share Analysis by Raw Material: 2012 VS 2020 VS 2027

Table 64: Japanese Market for Hydrogels: Annual Sales Estimates and Projections in US$ Thousand by Composition for the Period 2020-2027

Table 65: Hydrogels Market in Japan: Historic Sales Analysis in US$ Thousand by Composition for the Period 2012-2019

Table 66: Japanese Hydrogels Market Share Analysis by Composition: 2012 VS 2020 VS 2027

Table 67: Japanese Demand Estimates and Forecasts for Hydrogels in US$ Thousand by Application: 2020 to 2027

Table 68: Japanese Hydrogels Market in US$ Thousand by Application: 2012-2019

Table 69: Hydrogels Market Share Shift in Japan by Application: 2012 VS 2020 VS 2027

CHINA Table 70: Chinese Hydrogels Market Growth Prospects in US$ Thousand by Raw Material for the Period 2020-2027

Table 71: Hydrogels Historic Market Analysis in China in US$ Thousand by Raw Material: 2012-2019

Table 72: Chinese Hydrogels Market by Raw Material: Percentage Breakdown of Sales for 2012, 2020, and 2027

Table 73: Chinese Hydrogels Market Growth Prospects in US$ Thousand by Composition for the Period 2020-2027

Table 74: Hydrogels Historic Market Analysis in China in US$ Thousand by Composition: 2012-2019

Table 75: Chinese Hydrogels Market by Composition: Percentage Breakdown of Sales for 2012, 2020, and 2027

Table 76: Chinese Demand for Hydrogels in US$ Thousand by Application: 2020 to 2027

Table 77: Hydrogels Market Review in China in US$ Thousand by Application: 2012-2019

Table 78: Chinese Hydrogels Market Share Breakdown by Application: 2012 VS 2020 VS 2027

EUROPE Table 79: European Hydrogels Market Demand Scenario in US$ Thousand by Region/Country: 2020-2027

Table 80: Hydrogels Market in Europe: A Historic Market Perspective in US$ Thousand by Region/Country for the Period 2012-2019

Table 81: European Hydrogels Market Share Shift by Region/Country: 2012 VS 2020 VS 2027

Table 82: European Hydrogels Market Estimates and Forecasts in US$ Thousand by Raw Material: 2020-2027

Table 83: Hydrogels Market in Europe in US$ Thousand by Raw Material: A Historic Review for the Period 2012-2019

Table 84: European Hydrogels Market Share Breakdown by Raw Material: 2012 VS 2020 VS 2027

Table 85: European Hydrogels Market Estimates and Forecasts in US$ Thousand by Composition: 2020-2027

Table 86: Hydrogels Market in Europe in US$ Thousand by Composition: A Historic Review for the Period 2012-2019

Table 87: European Hydrogels Market Share Breakdown by Composition: 2012 VS 2020 VS 2027

Table 88: European Hydrogels Addressable Market Opportunity in US$ Thousand by Application: 2020-2027

Table 89: Hydrogels Market in Europe: Summarization of Historic Demand in US$ Thousand by Application for the Period 2012-2019

Table 90: European Hydrogels Market Share Analysis by Application: 2012 VS 2020 VS 2027

See the rest here:
The global market for Hydrogel is projected to reach US$15.3 billion by 2025 - GlobeNewswire

The global market for cell harvesting should grow from $885 million in 2018 to reach $1.5 billion by 2023 at a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023.

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Report Scope:

The scope of the report encompasses the major types of cell harvesting that have been used and the cell harvesting technologies that are being developed by industry, government agencies and nonprofits. It analyzes current market status, examines drivers on future markets and presents forecasts of growth over the next five years.

The report provides a summary of the market, including a market snapshot and profiles of key players in the cell harvesting market. It provides an exhaustive segmentation analysis of the market with in-depth information about each segment. The overview section of the report provides a description of market trends and market dynamics, including drivers, restraints and opportunities. it provides information about market developments and future trends that can be useful for organizations, including wholesalers and exporters. It provides market positionings of key players using yardsticks of revenue, product portfolio, and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Readers will also find historical and current market sizes and a discussion of the markets future potential. The report will help market players and new entrants make informed decisions about the production and exports of goods and services.

Report Includes:

41 data tables and 22 additional tables Description of segments and dynamics of the cell harvesting market Analyses of global market trends with data from 2017, 2018, and projections of compound annual growth rates (CAGRs) through 2023 Characterization and quantification of market potential for cell harvesting by type of harvesting, procedure, end user, component/equipment and region A brief study and intact information about the market development, and future trends that can be useful for the organizations involved in Elaboration on the influence of government regulations, current technology, and the economic factors that will shape the future marketplace Key patents analysis and new product developments in cell harvesting market Detailed profiles of major companies of the industry, including Becton, Dickinson and Co., Corning, Inc., Fluidigm Corp., General Electric Co., Perkinelmer, Inc., and Thermo Fisher Scientific, Inc.

Summary

Stem cells are unspecialized cells that have the ability to divide indefinitely and produce specialized cells. The appropriate physiological and experimental conditions provided to the unspecialized cells give rise to certain specialized cells, including nerve cells, heart muscle cells and blood cells. Stem cells can divide and renew themselves over long periods of time. These cells are extensively found in multicellular organisms, wherein mammals, there are two types of stem cells embryonic stem cells and adult stemcells. Embryonic stem cells are derived from a human embryo four or five days old that is in the blastocyst phase of development. Adult stem cells grow after the development of the embryo and are found in tissues such as bone marrow, brain, blood vessels, blood, skin, skeletal muscles and liver. Stemcell culture is the process of harvesting the exosomes and molecules released by the stem cells for the development of therapeuticsfor chronic diseases such as cancer and diabetes.

The process is widely used in biomedical applications such as therapy, diagnosis and biological drug production. The global cell harvesting market is likely to witness a growth rate of REDACTED during the forecast period of 2018-2023.The value of global cell harvesting market was REDACTED in 2017 and is projected to reach REDACTED by 2023. Market growth is attributed to factors such as increasing R&D spending in cell-based research,the introduction of 3D cell culture technology, increasing government funding, and the growing prevalence of chronic diseases such as cancer and diabetes.

The growing incidence and prevalence of cancer is seen as one of the major factors contributing to the growth of the global cell harvesting market. According to the World Health Organization (WHO), cancer is the second-leading cause of mortality globally and was responsible for an estimated 9.6 million deaths in 2018. Therefore, there is an increasing need for effective cancer treatment solutions globally. Cell harvesting is the preferred method used in cancer cell-related studies including cancer cell databases (cancer cell lines), and other analyses and drug discovery in a microenvironment.

The rising prevalence of such chronic diseases has led governments to provide R&D funding to research institutes and biotechnology companies to develop advanced therapeutics. Various 3D cell culture technologies have been developed by researchers and biotechnology companies such as Lonza Group and Thermo Fischer Scientific for research applications such as cancer drug discovery. The application of cell culture in cancer research is leading to more predictive models for research, drug discovery and regenerative medicine applications.

Platelet-rich plasma (PRP) therapy, a new biotechnology solution that has a heightened interest among researchers in tissue engineering and cell-based therapies, has various applications in the treatment of tissue healing in tendinopathy, osteoarthritis and muscle injury. It has been conventionally employed in orthopedics, maxillofacial surgery, periodontal therapy and sports medicines. PRP therapy can be used in the treatment of fat grafting, acne scars, and hair regrowth.

Major factors driving market growth include increasing healthcare costs and the high rate of adoption for modern medicines in emerging economies such as China and India. It has been estimated that India will witness a CAGR of REDACTED in the cell harvesting market during the forecast period. The active participation of foreign pharmaceutical companies has tapped the Indian healthcare sector with a series of partnerships and mergers and acquisitions, which in turn is positively impacting the growth of the market in this region.

Consistent development and clinical trials for stem cell therapies, plus contribution from the government and private sectors through investments and cohesive reimbursement policies in the development of cancer biomarkers, is further fueling market growth. InSweden, a research team at Lund University has developed a device to collect fluid and harvest stem mesenchymal stem cells (MSCs). The device is developed with 3D-printed bio-inert plastics which, when used by doctors, can result in the safe extraction of fluids (medical waste) from the patients body. The liquid is then passed through a gauze filter for purifying thoroughly and MSCs are separated from the fluid by centrifugation and are grown in culture.

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Cell Harvesting Market Market Will Grow At CAGR During 2018-2023 Global Evaluation By Trends, Proportions, Share, Swot, And Key Developments -...

In a surgical procedure last month, neurosurgeons from Kyoto University implanted 2.4 million cells into the brain of a patient with Parkinsons disease. The cellsderived from peripheral blood cells of an anonymous donorhad been reprogrammed into induced pluripotent stem cells (iPSCs) and then into dopaminergic precursor cells, which researchers hope will boost dopamine levels and ameliorate the patients symptoms.

The procedure is the most recent attempt by clinicians to test whether iPSCs can treat disease. In recent years, Japanese scientists have launched several clinical studies to examine their efficacy in heart disease and macular degeneration of the eye. And other researchers around the globe are exploring ways to turn the cells into treatments for everything from endometriosis to spinal cord injury. The initial foray into clinical trials raises hopes that the technology will bear fruit 12 years after its Nobel Prizewinning discovery.

Im excited that theyre trying to move it to the clinical level, because the iPS field does at some point need to start demonstrating that [these cells] have regenerative potential, says Jalees Rehman of the University of Illinois at Chicago. But the move towards clinical work is also revealing the difficulties of developing therapies. Its a learning curve, he adds.

So far, only a handful of patients have undergone iPSC-based treatments. In 2014, a woman with macular degeneration of the eye received a transplant of iPSC-based retinal cells derived from her own cells. The woman treated showed no apparent improvement in her vision, but the safety of the iPSC-derived cells was confirmed, writes Jun Takahashi, a stem cell biologist at Kyoto University who helped derive the dopaminergic precursor cells implanted into the Parkinsons disease patient. It was his wife, Masayo Takahashi of the RIKEN Center for Developmental Biology, who created the retinal cells used in that trial.

Last year, five patients were treated for the same eye condition with iPSC-derived retinal cells, which were taken from different donors. One of them patients developed a serious, but non-lifethreatening, reaction to the transplant, forcing doctors to remove it, according to the Japan Times.

More clinical studies are underway: Next year, heart surgeons plan to implant sheets of iPSC-derived cardiomyocytes into the hearts of three patients with heart disease, and Takahashi hopes to treat six more patients with Parkinsons disease by 2022. These are all in the earliest phases of testing. It is too early to say something [about the cells efficacy] in our trial, he adds.

While some researchers are waiting for the results of clinical studies to determine whether iPSCs have regenerative potential, others are racing ahead with preclinical studies presenting ever more ways on how to use them therapeutically. For instance, April Pyle, a stem cell biologist at the University of California, Los Angeles, recently developed an approach she believes is promising in treating Duchenne muscular dystrophy, a devastating disease caused by a mutation in the gene encoding the muscle-strengthening protein dystrophin. She and her colleagues used CRISPR-Cas9 to repair the gene in human iPSCs, turned them into skeletal muscle cells, and injected them into the muscle of dystrophin-deficient mice. We [could] actually see that weve restored dystrophin in pockets of the muscle, she explains.

I think its really just the beginning, she says. I think that were finally seeing the payoff for all of the hard work . . . and there will be many more trials to follow from these initial studies.

By now, researchers have figured out how to coax iPSCs to grow into most known cell types, Rehman says. But to get these cells to take on the roles of mature cells in a new tissue environment is another issue. In the heart, for instance, researchers have found that new stem cells have to be electrically aligned with the other cells. Experiments on human iPSC-derived heart muscle cells in culture show that by subjecting them to electrically induced contractions as they develop, the cells mature faster, suggesting that they become more able to handle the adult workload in vivo. How to integrate the new cells so they will survive in injured or diseased tissue is another question. Do you need a special matrix, a gel, a patch, an organoid, to ensure the success of these cells long term? Rehman asks. These challenges are faced in all the organs.

Researchers have been relying on monkey models to evaluate the efficacy of engraftment procedures before testing them in human patients, explains Takahashi. Last year, his team demonstrated on monkeys that human iPSCderived dopaminergic neurons stably integrated into existing brain tissue, where they produced dopamine and ultimately improved Parkinsonian symptoms.

The closer we get to [clinical] applications, the more we obviously realize the challenges that lie ahead.

Jalees Rehman, University of Illinois at Chicago

Another challenge with the implantation of iPSC-derived tissue is the ever-present risk that the cells might trigger cancer, because they stem from a cell type that is by nature highly proliferative. To avoid this, Takahashi and his colleagues filter the implanted cells to eliminate undifferentiated ones that are most prone to overgrowth, and also test the cell lines for tumorgenicity by implanting a sample into mice.

Still, we cannot completely eliminate the possibility of tumor formation, notes Tetsuo Maruyama, an associate professor of obstetrics and gynecology from Keio University School of Medicine. He thinks that such procedures should focus on non-essential organs, such as the eye or the uterus, for instance. He recently succeeded in deriving healthy uterine cells from iPSCs and plans to use these to study how endometriosis occurs, and also to generate human endometrium that could eventually be used clinically.

Another concern researchers have frequently raised are the immunosuppressive drugs that patients require if the iPSCs are derived from cells other than the patients own. Takahashis patient with Parkinsons, for instance, will be on immunosuppressants for a year, possibly making the patient less able to fight off infections and cancer. But despite the risks, many researchers have opted to use allogeneic stem cellsthose from a donorforemost because the approach will save time, cost, and labor when the time comes to scale up such treatments for commercialization. It is important when you think about industrialization, Takahashi writes in an email.

The possibility to create off the shelf iPSC therapies has also attracted industry, not just academics. For instance, Australia-based biotech company Cynata Therapeutics recently concluded a Phase I trial using iPSC-derived mesenchymal stem cells to treat graft-versus-host disease (GVHD). The condition occurs after bone marrow transplants when immune cells of the donor recognize cells in the recipients body as foreign and attack them, often resulting in death. But mesenchymal stem cells, which can mature into a variety of cell types, suppress the proliferation and activation of the donors T cells, explains Kilian Kelly, the companys vice president for product development. The company produced these cells by starting from iPSCs, reprogramming them in to an intermediary cell called a mesenchymoangioblast, and then directing them to become mesenchymal cells.

The trial, which the company claims is the worlds first to use iPSCs, administered the cells intravenously to 15 patients with GVHD who had previously failed to respond to steroid treatment and as such faced a grim prognosis. Although its too soon to evaluate efficacy, Kelly says, he sees it as a positive sign that 14 of them showed a notable improvement in their condition. And conveniently, immune rejection isnt an issue with mesenchymal stem cells because they dont express the donor-specific antigens that trigger rejection. So that means that we can use cells from a single iPS [cell] bank to treat essentially anybody, says Kelly.

Developing off-the-shelf treatments is also vastly more cost effective than maturing iPSC-derived cells for individual patients, adds Ross McDonald, the companys CEO. He points to personalized T-cell immunotherapiestwo of which have been recently FDA-approvedwhich can nearly$500,000 per patient. Its too soon to predict how much his product might cost, he adds.

This is one reason why several groups are developing banks of iPSCs that can be used to develop regenerative therapies at scale. For instance, the Japanese government decided to put around $250 million towards developing an iPSC stock for biomedical research. The donors from whom these cells are derived were carefully selected with immune compatibility in mind: the bank is designed to encompass a diverse set of commonly present human leukocyte antigen (HLA) types, so that they are broadly representative of the majority of the population. Then, implantation will require only a minimum amount of immune suppression. This is kind of a middle ground between using patient-specific cells and cells chosen at random, explains Amanda Mack, director of iPSC reprogramming at Fujifilm Cellular Dynamics, a Wisconsin-based company that grows human cells for biomedical research.

Together, the cells will be immunocompatible with almost 70 percent of the Japanese population, says Maruyama. This might be more difficult for countries such as the US, where the genetic makeup is more diverse, but similar efforts are also underway there. For instance, Macks company aims to develop a bank of iPSCs that are matched to a majority of the US population.

While efforts like these continue, researchers around the world are still figuring out the nuts and bolts of applying these cells therapeutically. The closer we get to [clinical] applications, the more we obviously realize the challenges that lie ahead, says Rehman. I think thats a very normal process for scientific discovery.

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Increasing Number of iPS Cell Therapies Tested in Clinical ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced that new data from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including data from the Phase 2/3 Starbeam study (ALD-102) and available data from the Phase 3 ALD-104 study, will be presented at the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2020), taking place virtually from August 29 - September 1, 2020.

New Cerebral Adrenoleukodystrophy (CALD) Data at EBMT 2020

Lenti-D hematopoietic stem cell gene therapy stabilizes neurologic function in boys with cerebral adrenoleukodystrophy (ALD-102 and ALD-104)Presenting Author: Dr. Jrn-Sven Khl, Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Womens and Childrens Medicine, University Hospital LeipzigPoster Session & Number: Gene Therapy; ePoster O077

Additional bluebird bio data at EBMT 2020 includes encore presentations from the companys CALD, sickle cell disease (SCD), transfusion-dependent -thalassemia (TDT) and multiple myeloma programs.

Cerebral Adrenoleukodystrophy (CALD) Encore Data at EBMT 2020

Outcomes of allogeneic hematopoietic stem cell transplant in patients with cerebral adrenoleukodystrophy vary by donor cell source, conditioning regimen, and stage of cerebral disease status (ALD-103)Presenting Author: Dr. Jaap Jan Boelens, Chief, Pediatric Stem Cell Transplantation and Cellular Therapies Service, Memorial Sloan Kettering Cancer CenterPoster Session & Number: Haemoglobinopathy and inborn errors; ePoster O106

Multiple Myeloma Correlative Encore Data at EBMT 2020

Markers of initial and long-term responses to idecabtagene vicleucel (ide-cel; bb2121) in the CRB-401 study in relapsed/refractory multiple myelomaPresenting Author: Dr. Ethan G. Thompson, Bristol Myers SquibbPoster Session & Number: CAR-based Cellular Therapy clinical; ePoster A089

Sickle Cell Disease (SCD) Encore Data at EBMT 2020

LentiGlobin for sickle cell disease (SCD) gene therapy (GT): updated results in Group C patients from the Phase 1/2 HGB-206 studyPresenting Author: Dr. Markus Y. Mapara, Director, Adult Blood and Marrow Transplantation Program, Columbia University Medical CenterOral Session & Number: Inborn Errors; O080Date & Time: September 1, 2020; 4:35 4:42 PM CET/10:35 10:42 AM ET

Transfusion-Dependent -Thalassemia (TDT) Encore Data at EBMT 2020

Clinical outcomes following autologous hematopoietic stem cell transplantation with LentiGlobin gene therapy in the Phase 3 Northstar-2 and Northstar-3 studies for transfusion-dependent -thalassemiaPresenting Author: Professor Franco Locatelli, Director, Department of Pediatric Hematology and Oncology, Ospedale Pediatrico Bambino GesPoster Session & Number: Gene Therapy; ePoster O074

LentiGlobin gene therapy treatment of two patients with transfusion-dependent -thalassemia (case report)Presenting Author: Dr. Mattia Algeri, Department of Pediatric Oncohematology - Transplantation Unit and Cell Therapies, Ospedale Pediatrico Bambino GesPoster Session & Number: Haemoglobinopathy and inborn errors; ePoster A328

Cross Indication Encore Data at EBMT 2020

Safety of autologous hematopoietic stem cell transplantation with gene addition therapy for transfusion-dependent -thalassemia, sickle cell disease, and cerebral adrenoleukodystrophyPresenting Author: Dr. Evangelia Yannaki, Director, Gene and Cell Therapy Center, Hematology Department, George Papanicolaou HospitalPoster Session & Number: Gene Therapy; ePoster O078

Abstracts outlining bluebird bios accepted data at EBMT 2020 are available on the Annual Meeting website. On August 29, 2020, at 12:30 PM CET/6:30 AM ET, the embargo will lift for ePosters and oral presentations accepted for EBMT 2020. Presentations will be available for virtual viewing throughout the duration of the live meeting and content will be accessible online following the close of the meeting until November 1, 2020.

About elivaldogene autotemcel (eli-cel, Lenti-D gene therapy)In July 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted an accelerated assessment to eli-cel gene therapy for cerebral adrenoleukodystrophy (CALD). bluebird bio is currently on track to submit the Marketing Authorization Application (MAA) in the EU for eli-cel for CALD by year-end 2020, and the Biologics License Application (BLA) in the U.S. in mid-2021.

bluebird bio is currently enrolling patients for a Phase 3 study (ALD-104) designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Contact clinicaltrials@bluebirdbio.com for more information and a list of study sites.

Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.

The Phase 2/3 Starbeam study (ALD-102) has completed enrollment. For more information about the ALD-102 study visit: http://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT01896102.

Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide. Approximately 40 percent of boys with ALD will develop CALD, the most severe form of ALD. CALD is a progressive neurogenerative disease that involves breakdown of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms of CALD usually occur in early childhood and progress rapidly, if untreated, leading to severe loss of neurologic function, and eventual death, in most patients.

The European Medicines Agency (EMA) accepted eli-cel gene therapy for the treatment of CALD into its Priorities Medicines scheme (PRIME) in July 2018, and previously granted Orphan Medicinal Product designation to eli-cel.

The U.S. Food and Drug Administration (FDA) granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation for the treatment of CALD.

Eli-cel is not approved for any indication in any geography.

About idecabtagene vicleucel (ide-cel; bb2121)Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

In addition to the pivotal KarMMa trial evaluating ide-cel in patients with relapsed and refractory multiple myeloma, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

In July 2020, Bristol Myers Squibb (BMS) and bluebird bio submitted the Biologics License Application for ide-cel to the U.S. Food and Drug Administration for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. Ide-cel is the first CAR T cell therapy submitted for regulatory review to target BCMA and for multiple myeloma.

Ide-cel was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) and PRIority Medicines (PRIME) designation, as well as Accelerated Assessment status, by the European Medicines Agency for relapsed and refractory multiple myeloma.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between BMS and bluebird bio.

Ide-cel is not approved for any indication in any geography.

About LentiGlobin for Sickle Cell DiseaseLentiGlobin for sickle cell disease (SCD) is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel and LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS). HbS causes red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive crises (VOCs). For adults and children living with SCD, this means painful crises and other life-altering or life-threatening acute complicationssuch as acute chest syndrome (ACS), stroke and infections. If patients survive the acute complications, vasculopathy and end-organ damage, resulting complications can lead to pulmonary hypertension, renal failure and early death; in the U.S. the median age of death for someone with sickle cell disease is 43 - 46 years.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration (FDA) granted Orphan Drug status and Regenerative Medicine Advanced Therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for the treatment of SCD.

bluebird bio reached general agreement with the U.S. Food and Drug Administration (FDA) that the clinical data package required to support a Biologics Licensing Application (BLA) submission for LentiGlobin for SCD will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. The planned submission will be based on an analysis using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint with at least 18 months of follow-up post-treatment with LentiGlobin for SCD. Globin response will be used as a key secondary endpoint.

bluebird bio anticipates additional guidance from the FDA regarding the commercial manufacturing process, including suspension lentiviral vector. bluebird bio announced in a May 11, 2020 press release it plans to seek an accelerated approval and expects to submit the U.S. BLA for SCD in the second half of 2021.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About betibeglogene autotemcel (beti-cel; formerly LentiGlobin gene therapy for -thalassemia)The European Commission granted conditional marketing authorization (CMA) for betibeglogene autotemcel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for ZYNTEGLO, supported by data from 32 patients treated with ZYNTEGLO, including three patients with up to five years of follow-up.

In the HGB-207 clinical study supporting the conditional marketing approval of ZYNTEGLO, the primary endpoint was transfusion independence (TI) by Month 24, defined as a weighted average Hb 9 g/Dl without any RBC transfusions for a continuous period of 12 months at any time during the study after infusion of ZYNTEGLO. Ten patients were evaluable for assessment of TI. Of these, 9/10 (90.0%, 95% CI 55.5-99.7%) achieved TI at last follow-up. Among these nine patients, the median (min, max) weighted average Hb during TI was 12.22 (11.4, 12.8) g/dLl.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Beti-cel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during the clinical studies that were attributed to betibeglogene autotemcel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, and non-cardiac chest pain. Two serious adverse events (SAE) of thrombocytopenia were considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the United States.

Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

About bluebird bio, Inc.bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

Lenti-D and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking StatementsThis release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the plans for regulatory submissions for beti-cel (marketed as ZYTENGLO in the European Union), eli-cel, ide-cel, and LentiGlobin for SCD, including anticipated endpoints to support regulatory submissions and timing expectations; the companys expectations regarding the potential for the suspension manufacturing process for lentiviral vector; its expectations for commercialization efforts for ZYNTEGLO in Europe; as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the COVID-19 pandemic and resulting economic conditions will have a greater impact on the companys operations and plans than anticipated; that our amended collaboration with BMS will not continue or be successful; that preliminary positive efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or future clinical trials; the risk that our plans for submitting a BLA for LentiGlobin for SCD may be delayed if the FDA does not accept our comparability plans for the use of the suspension manufacturing process for lentiviral vector; the risk that the submission of BLA for ide-cel is not accepted for filing by the FDA or approved in the timeline we expect, or at all; the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, including due to delays from the COVID-19 pandemics impact on healthcare systems; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product, in the adoption of value-based payment models, or in obtaining sufficient coverage or reimbursement for our products; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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bluebird bio to Present New Data from Clinical Studies of elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for Cerebral Adrenoleukodystrophy...

Mystified by the need for defibrillation to save a 10-year-old from drowning, Michael Ackerman, M.D., Ph.D., vowed to dig for answers. That pivotal case during a Mayo Clinic pediatric cardiology residency was the catalyst for Dr. Ackermans career in genetic sleuthing of inherited sudden cardiac death syndromes. With help from the Center for Regenerative Medicine Biotrust, Dr. Ackermans team reprograms cell lines to zero in on precise causes and possible treatments for genetic heart disorders that increase the risk of sudden cardiac death. His research and practice focus on inherited conditions like long QT syndrome (LQTS), catecholaminergic polymorphic ventricular tachycardia (CPVT) and Brugada syndrome (BrS) along with heart muscle diseases such as hypertrophic cardiomyopathy (HCM).

Working with the Center for Regenerative Medicine has opened up a whole new investigative arm to our lab. It is bench to bedside research. We take cells from a blood sample from my patients and then reprogram those cells to become cardiac cells. This research effort has been a powerful tool in gene discovery to prove beyond a shadow of a doubt when a monogenetic variant is indeed the cause of a sudden cardiac death syndrome, says Dr. Ackerman.

Reprogramming cells to identify disease-causing mutations

Reprogramming a patients cells is like a step back in time to when the cells were initially forming in the mothers womb. At that time, cells were dividing and could become any type of cell or tissue in the body. Reprogrammed cells, known as induced pluripotent stem cells, can be redirected to become new heart cells. Dr. Ackermans team uses these patient-specific cell lines to create a disease in a dish model and investigate whether genetic mutations are causing the patients genetic heart disease such as long QT syndrome.

Once we think weve found the root cause of disease, we then go to the patients cell line. We ask, does it show in the dish, in that patients re-engineered heart cells, a prolonged QT cellular phenotype? If it does, then we edit out and correct that variant of interest and at the cellular level test whether the abnormality disappears, says Dr. Ackerman.

Dr. Ackermans team then introduces that genetic variant into normal, healthy cells. If those cells produce a long QT phenotype, they have proof that exact genetic variant is the cause.

Using this disease in a dish model and other genetic sleuthing strategies, Dr. Ackermans team has discovered six of the 17 known genes that cause long QT syndrome. And, they have recently described two entirely new syndromes. One is triadin knockout syndrome, a heart arrhythmia that could lead to cardiac arrest in children during exercise. The second is an autosomal recessive genetic mechanism for calcium release channel deficiency syndrome, prevalent within Amish communities. That key discovery solved the mystery of why so many Amish children were dying suddenly during ordinary childhood play. The disease in a dish model is also useful for discovering new therapies. After creating the patients disease in a dish, Dr. Ackermans team tests potential new drug compounds to see if they could be effective.

We are developing a new gene therapy for the most common genetic subtype of long QT syndrome.With this model, the gene therapy vector is essentially curing the diseased long QT phenotype in the dish, says Dr. Ackerman.

Almost quit research

Dr. Ackerman began medical and graduate school at Mayo Clinic in 1988, where he worked in a research lab next to then fellow trainee, Andre Terzic, M.D., Ph.D., who now is director of Mayo Clinic Center for Regenerative Medicine. Initially not seeing the relevance to patient care, Dr. Ackerman finished his Ph.D. and left research vowing to never, ever return. True to his mentors predictions that youll be back, Mike, Dr. Ackerman felt the pull back to research to address unmet medical needs of his patients.He joined Mayo Clinics faculty in 2000 as one of the first genetic cardiologists with a goal of establishing a practice for patients at risk of sudden cardiac death from genetic heart diseases. Dr. Ackerman now directs the Mayo Clinic Windland Smith Rice Genetic Heart Rhythm Clinic and the Mayo Clinic Windland Smith Rice Sudden Death Genomics Laboratory.

Dr. Ackermans return to research has provided many answers for patients, with over 600 peer-reviewed publications that have occurred since that time 23 years ago when Dr. Ackerman and his team first solved that 10-year-old boys near fatal drowning. It was a mutation in the gene causing type 1 long QT syndrome.

Dr. Ackerman is one of the innovators the Center for Regenerative Medicine collaborates with as it seeks to be a global leader and trusted destination for regenerative care driven by research and education.

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Tags: Brugada syndrome, Center for Regenerative Medicine Biotrust, hypertrophic cardiomyopathy, long Q T syndrome, Mayo Clinic Center for Regenerative Medicine, Michael Ackerman, People, Research, Stem cell research, sudden cardiac death

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Using stem cells to find causes and treatments to prevent ...

Unlike other CAR T-cell therapies, clinical success was not associated with significant complications from therapy, said Dr. Jonathan Serody. This means this treatment should be available to patients in a clinic setting and would not require patients to be hospitalized, which is critical in our current environment.

Results from the parallel phase 1 and phase 2 studies also demonstrated that the CAR T-cell therapy was safe and did not produce any serious or severe side effects.

Researchers from the UNC Lineberger Comprehensive Cancer Center and Baylor College of Medicine administered anti-CD30 CAR T cells to 41 patients with relapsed or refractory Hodgkin lymphoma. All patients underwent lymphodepletion with bendamustine alone, bendamustine and fludarabine, or cyclophosphamide and fludarabine prior to the anti-CD30 CAR T-cell therapy.

Measuring safety was the primary goal of the two parallel studies.

The overall response rate, or the percentage of partial or complete responses to therapy, among 37 evaluable patients was 62%. Thirty-four of the patients received fludarabine-based lymphodepletion 17 of which received it with bendamustine, and the other half received it with cyclophosphamide. Two of these patients were considered to be complete response at infusion and maintained the response, so they were not included in final analysis.

The overall response rate among the remaining patients was 72%, with 59% of patients achieving a complete response. After a median follow-up of 533 days, researchers identified the one-year progression free survival rate to be 36% and the one-year overall survival rate to be 94%.

This is particularly exciting because the majority of these patients had lymphomas that had not responded well to other powerful new therapies, said senior study author Dr. Barbara Savoldo, professor in the Department of Microbiology and Immunology at the UNC School of Medicine, in a press release.Patients within the study had received a median of seven previous lines of therapy that included checkpoint inhibitors and autologous or allogeneic stem cell therapies, therapies known to be powerful but also tend to come with a host of side effects.

However, treatment with the anti-CD30 CART cells demonstrated a favorable safety profile. Although 10 patients developed cytokine release syndrome, all cases were considered minor.

Patients who received fludarabine-containing lymphodepletion were the only participants in the study to have a response to the anti-CD30 CAR T-cell therapy.

Although CD30 CAR T (cells) showed modest activity in (Hodgkin lymphoma) when infused without lymphodepletion, robust clinical responses were achieved when these cells were infused in hosts lymphodepleted with fludarabine-containing regimens, the authors wrote.

The activity of this new therapy is quite remarkable and while we need to confirm these findings in a larger study, this treatment potentially offers a new approach for patients who currently have very limited options to treat their cancer, said Dr. Jonathan Serody, director of the bone marrow transplant and cellular therapy program at UNC Lineberger Comprehensive Cancer Center, in the release. Additionally, unlike other CAR T-cell therapies, clinical success was not associated with significant complications from therapy. This means this treatment should be available to patients in a clinic setting and would not require patients to be hospitalized, which is critical in our current environment.

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DUBLIN, Aug. 11, 2020 /PRNewswire/ -- The "Global Induced Pluripotent Stem Cell (iPS Cell) Industry Report" report has been added to ResearchAndMarkets.com's offering.

Since the discovery of induced pluripotent stem cells (iPSCs) a large and thriving research product market has grown into existence, largely because the cells are non-controversial and can be generated directly from adult cells. It is clear that iPSCs represent a lucrative market segment because methods for commercializing this cell type are expanding every year and clinical studies investigating iPSCs are swelling in number.

Therapeutic applications of iPSCs have surged in recent years. 2013 was a landmark year in Japan because it saw the first cellular therapy involving the transplant of iPSCs into humans initiated at the RIKEN Center in Kobe, Japan. Led by Masayo Takahashi of the RIKEN Center for Developmental Biology (CDB), it investigated the safety of iPSC-derived cell sheets in patients with macular degeneration. In another world-first, Cynata Therapeutics received approval in 2016 to launch the world's first formal clinical trial of an allogeneic iPSC-derived cell product (CYP-001) for the treatment of GvHD. Riding the momentum within the CAR-T field, Fate Therapeutics is developing FT819, its off-the-shelf iPSC-derived CAR-T cell product candidate. Numerous physician-led studies using iPSCs are also underway in Japan, a leading country for basic and applied iPSC applications.

iPS Cell Commercialization

Methods of commercializing induced pluripotent stem cells (iPSCs) are diverse and continue to expand. iPSC cell applications include, but are not limited to:

Since the discovery of iPSC technology in 2006, significant progress has been made in stem cell biology and regenerative medicine. New pathological mechanisms have been identified and explained, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated. The main objectives of this report are to describe the current status of iPSC research, patents, funding events, industry partnerships, biomedical applications, technologies, and clinical trials for the development of iPSC-based therapeutics.

Key Topics Covered:

1. Report Overview

2. Introduction

3. History of Induced Pluripotent Stem Cells (IPSCS)

4. Research Publications on IPSCS

5. IPSCS: Patent Landscape

6. Clinical Trials Involving IPSCS

7. Funding for IPSC

8. Generation of Induced Pluripotent Stem Cells: An Overview

9. Human IPSC Banking

10. Biomedical Applications of IPSCS

11. Other Novel Applications of IPSCS

12. Deals in the IPSCS Sector

13. Market Overview

14. Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/kpc95y

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Opportunities in the Global Induced Pluripotent Stem Cell (iPS Cell) Industry - PRNewswire

Overview

We are a clinical stage biopharmaceutical company focused on the discovery,development and commercialization of drugs for the treatment of cancer. We aredeveloping proprietary drugs independently and through research and developmentcollaborations. Our core objective is to leverage our proprietary phospholipiddrug conjugate (PDC) delivery platform to develop PDCs that are designed tospecifically target cancer cells and deliver improved efficacy and better safetyas a result of fewer off-target effects. Our PDC platform possesses thepotential for the discovery and development of the next generation ofcancer-targeting treatments, and we plan to develop PDCs both independently andthrough research and development collaborations. The COVID-19 pandemic hascreated uncertainties in the expected timelines for clinical stagebiopharmaceutical companies such as us, and because of such uncertainties, it isdifficult for us to accurately predict expected outcomes at this time. We havenot yet experienced any significant impacts as a result of the pandemic and havecontinued to enroll patients in our clinical trials. However, COVID-19 mayimpact our future ability to recruit patients for clinical trials, obtainadequate supply of CLR 131 and obtain additional financing.

Our lead PDC therapeutic, CLR 131 is a small-molecule PDC designed to providetargeted delivery of iodine-131 directly to cancer cells, while limitingexposure to healthy cells. We believe this profile differentiates CLR 131 frommany traditional on-market treatment options. CLR 131 is the company's leadproduct candidate and is currently being evaluated in a Phase 2 study inrelapsed/refractory (r/r) B-cell malignancies, including multiple myeloma (MM),chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),lymphoplasmacytic lymphoma/Waldenstrom's macroglobulinemia (LPL/WM), marginalzone lymphoma (MZL), mantle cell lymphoma (MCL), and diffuse large B-celllymphoma (DLBCL).CLR 131 is also being evaluated in a Phase 1 dose escalationstudy in pediatric solid tumors and lymphoma. The U.S. Food and DrugAdministration ("FDA") granted CLR 131 Fast Track Designation for both r/r MMand r/r DLBCL and Orphan Drug Designation (ODD) of MM, LPL/WM, neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. CLR 131 was also grantedRare Pediatric Disease Designation (RPDD) for the treatment of neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. Most recently, the EuropeanCommission granted an ODD for r/r MM.

Our product pipeline also includes one preclinical PDC chemotherapeutic program(CLR 1900) and several partnered PDC assets. The CLR 1900 Series is beingtargeted for solid tumors with a payload that inhibits mitosis (cell division) avalidated pathway for treating cancers.

We have leveraged our PDC platform to establish four collaborations featuringfive unique payloads and mechanisms of action. Through research and developmentcollaborations, our strategy is to generate near-term capital, supplementinternal resources, gain access to novel molecules or payloads, accelerateproduct candidate development and broaden our proprietary and partnered productpipelines.

Our PDC platform provides selective delivery of a diverse range of oncologicpayloads to cancerous cells, whether a hematologic cancer or solid tumor, aprimary tumor, or a metastatic tumor and cancer stem cells. The PDC platform'smechanism of entry does not rely upon specific cell surface epitopes or antigensas are required by other targeted delivery platforms. Our PDC platform takesadvantage of a metabolic pathway utilized by all tumor cell types in all stagesof the tumor cycle. Tumor cells modify specific regions on the cell surface as aresult of the utilization of this metabolic pathway. Our PDCs bind to theseregions and directly enter the intracellular compartment. This mechanism allowsthe PDC molecules to accumulate over time, which enhances drug efficacy, and toavoid the specialized highly acidic cellular compartment known as lysosomes,which allows a PDC to deliver molecules that previously could not be delivered.Additionally, molecules targeting specific cell surface epitopes face challengesin completely eliminating a tumor because the targeted antigens are limited inthe total number on the cell surface, have longer cycling time frominternalization to being present on the cell surface again and available forbinding and are not present on all of the tumor cells in any cancer. This meansa subpopulation of tumor cells always exist that cannot be targeted by therapiestargeting specific surface epitopes. In addition to the benefits provided by themechanism of entry, PDCs offer the ability to conjugate payload molecules innumerous ways, thereby increasing the types of molecules selectively deliveredvia the PDC.

The PDC platform features include the capacity to link with almost any molecule,provide a significant increase in targeted oncologic payload delivery and theability to target all types of tumor cells. As a result, we believe that we cangenerate PDCs to treat a broad range of cancers with the potential to improvethe therapeutic index of oncologic drug payloads, enhance or maintain efficacywhile also reducing adverse events by minimizing drug delivery to healthy cells,and increasing delivery to cancerous cells and cancer stem cells.

We employ a drug discovery and development approach that allows us toefficiently design, research and advance drug candidates. Our iterative processallows us to rapidly and systematically produce multiple generations ofincrementally improved targeted drug candidates.

In June 2020, the European Medicines Agency (EMA) granted us Small andMedium-Sized Enterprise status by the EMA's Micro, Small and Medium-sizedEnterprise office. SME status allows us to participate in significant financialincentives that include a 90% to 100% EMA fee reduction for scientific advice,clinical study protocol design, endpoints and statistical considerations,quality inspections of facilities and fee waivers for selective EMA pre andpost-authorization regulatory filings, including orphan drug and PRIMEdesignations. We are also eligible to obtain EMA certification of quality andmanufacturing data prior to review of clinical data. Other financial incentivesinclude EMA-provided translational services of all regulatory documents requiredfor market authorization, further reducing the financial burden of the marketauthorization process.

A description of our PDC product candidates follows:

Our lead PDC therapeutic, CLR 131 is a small-molecule, PDC designed to providetargeted delivery of iodine-131 directly to cancer cells, while limitingexposure to healthy cells. We believe this profile differentiates CLR 131 frommany traditional on-market treatments and treatments in development. CLR 131 iscurrently being evaluated in a Phase 2 study in r/r B-cell lymphomas, and twoPhase 1 dose-escalating clinical studies, one in r/r MM and one in r/r pediatricsolid tumors and lymphoma. The initial Investigational New Drug (IND)application was accepted by the FDA in March 2014 with multiple INDs submittedsince that time. Initiated in March 2017, the primary goal of the Phase 2 studyis to assess the compound's efficacy in a broad range of hematologic cancers.The Phase 1 study is designed to assess the compound's safety and tolerabilityin patients with r/r MM (to determine maximum tolerated dose) and was initiatedin April 2015. The FDA previously accepted our IND application for a Phase 1open-label, dose escalating study to evaluate the safety and tolerability of asingle intravenous administration of CLR 131 in up to 30 children andadolescents with cancers including neuroblastoma, sarcomas, lymphomas (includingHodgkin's lymphoma) and malignant brain tumors. This study was initiated duringthe first quarter of 2019. These cancer types were selected for clinical,regulatory and commercial rationales, including the radiosensitive nature andcontinued unmet medical need in the r/r setting, and the rare diseasedeterminations made by the FDA based upon the current definition within theOrphan Drug Act.

In December 2014, the FDA granted ODD for CLR 131 for the treatment of MM.Multiple myeloma is an incurable cancer of the plasma cells and is the secondmost common form of hematologic cancers. In 2018, the FDA granted ODD and RPDDfor CLR 131 for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing'ssarcoma and osteosarcoma. The FDA may award priority review vouchers to sponsorsof rare pediatric disease products that meet its specified criteria. The keycriteria to receiving a priority review voucher is that the disease beingtreated is life-threatening and that it primarily effects individuals under theage of 18. Under this program, a sponsor who receives an approval for a drug orbiologic for a rare pediatric disease can receive a priority review voucher thatcan be redeemed to receive a priority review of a subsequent marketingapplication for a different product. Additionally, these priority reviewvouchers can be exchanged or sold to other companies for them to use thevoucher. In May 2019, the FDA granted Fast Track designation for CLR 131 for thetreatment of multiple myeloma in July 2019 for the treatment of DLBCL, inSeptember, CLR 131 received Orphan Drug Designation from the European Union forMultiple Myeloma, and in January 2020, the FDA granted Orphan Drug Designationfor CLR 131 in lymphoplasmacytic lymphoma (LPL).

Phase 2 Study in Patients with r/r select B-cell Malignancies

In February 2020, we announced positive data from our Phase 2 CLOVER-1 study inpatients with relapsed/refractory B-cell lymphomas. Relapsed/Refractory MM andnon-Hodgkin lymphoma (NHL) patients were treated with three different doses(<50mCi, ~50mCi and ~75mCi total body dose (TBD). The <50mCi total body dose wasa deliberately planned sub-therapeutic dose. CLR 131 achieved the primaryendpoint for the study. Patients with r/r MM who received the highest dose ofCLR 131 showed a 42.8% overall response rate (ORR). Those who received ~50mCiTBD had a 26.3% ORR with a combined rate of 34.5% ORR (n=33) while maintaining awell-tolerated safety profile. Patients in the studies were elderly with amedian age of 70, and heavily pre-treated, with a median of five prior lines oftreatment (range: 3 to 17), which included immunomodulatory drugs, proteasomeinhibitors and CD38 antibodies for the majority of patients. Additionally, amajority of the patients (53%) were quad refractory or greater and 44% of alltreated multiple myeloma patients were triple class refractory. 100% of allevaluable patients (n=43) achieved clinical benefit (primary outcome measure) asdefined by having stable disease or better. 85.7% of multiple myeloma patientsreceiving the higher total body dose levels of CLR 131 experienced tumorreduction. The 75mCi TBD demonstrated positive activity in both high-riskpatients and triple class refractory patients with a 50% and 33% ORR,respectively.

Patients with r/r NHL who received ~50mCi TBD and the ~75mCi TBD had a 42% and43% ORR, respectively and a combined rate of 42%. These patients were alsoheavily pre-treated, having a median of three prior lines of treatment (range, 1to 9) with the majority of patients being refractory to rituximab and/oribrutinib. The patients had a median age of 70 with a range of 51 to 86. Allpatients had bone marrow involvement with an average of 23%. In addition tothese findings, subtype assessments were completed in the r/r B-cell NHLpatients. Patients with DLBCL demonstrated a 30% ORR with one patient achievinga complete response (CR), which continues at nearly 24 months post-treatment.The ORR for CLL/SLL/MZL patients was 33%. Current data from our Phase 2 CLOVER-1clinical study show that four LPL/WM patients demonstrated 100% ORR with onepatient achieving a CR which continues at nearly 27 months post-treatment. Thismay represent an important improvement in the treatment of relapsed/refractoryLPL/WM as we believe no approved or late-stage development treatments forsecond- and third-line patients have reported a CR. LPL/WM is a rare, indolentand incurable form of NHL that is composed of a patient population in need ofnew and better treatment options.

The most frequently reported adverse events in r/r MM patients were cytopenias,which followed a predictable course and timeline. The frequency of adverseevents have not increased as doses were increased and the profile of cytopeniasremains consistent. Importantly, these cytopenias have had a predictable patternto initiation, nadir and recovery and are treatable. The most common grade ?3events at the highest dose (75mCi TBD) were hematologic toxicities includingthrombocytopenia (65%), neutropenia (41%), leukopenia (30%), anemia (24%) andlymphopenia (35%). No patients experienced cardiotoxicities, neurologicaltoxicities, infusion site reactions, peripheral neuropathy, allergic reactions,cytokine release syndrome, keratopathy, renal toxicities, or changes in liverenzymes. The safety and tolerability profile in patients with r/r NHL wassimilar to r/r MM patients except for fewer cytopenias of any grade. Based uponCLR 131 being well tolerated across all dose groups and the observed responserate, especially in difficult to treat patients such as high risk and tripleclass refractory or penta-refractory, and corroborating data showing thepotential to further improve upon current ORRs and durability of thoseresponses, the study has been expanded to test a two-cycle dosing optimizationregimen of CLR 131.

In July 2016, we were awarded a $2,000,000National Cancer Institute (NCI)Fast-Track Small Business Innovation Research grant to further advance theclinical development of CLR 131. The funds are supporting the Phase 2 studyinitiated in March 2017 to define the clinical benefits of CLR 131 in r/r MM andother niche hematologic malignancies with unmet clinical need. These nichehematologic malignancies include Chronic Lymphocytic Leukemia, Small LymphocyticLymphoma, Marginal Zone Lymphoma, Lymphoplasmacytic Lymphoma and DLBCL. Thestudy is being conducted in approximately 10 U.S. cancer centers in patientswith orphan-designated relapse or refractory hematologic cancers. The study'sprimary endpoint is clinical benefit response (CBR), with additional endpointsof ORR, progression free survival (PFS,) median Overall Survival (mOS) and othermarkers of efficacy following a single 25.0 mCi/m2 dose of CLR 131, with theoption for a second 25.0 mCi/m2dose approximately 75-180 days later. Based onthe performance results from Cohort 5 of our Phase 1 study in patients with r/rMM, reviewed below, we have modified the dosing regimen of this study to afractionated dose of 15.625 mCi/m2 administered on day 1 and day 8.

In May 2020, we announced that the FDA granted Fast Track Designation for CLR131 in LPL/WM in patients having received two prior treatment regimens or more.

Phase 1 Study in Patients with r/r Multiple Myeloma

In February 2020, we announced the successful completion of our Phase 1 doseescalation study. Data from the study demonstrated that CLR 131 was safe andtolerated at total body dose of approximately 90mCi in r/r MM. The Phase 1multicenter, open-label, dose-escalation study was designed to evaluate thesafety and tolerability of CLR 131 administered as a 30-minute I.V. infusion,either as a single bolus dose or as two fractionated doses. The r/r multiplemyeloma patients in this study received single cycle doses ranging fromapproximately 20mCi to 90mCi total body dose. To date, an independent DataMonitoring Committee determined that all doses have been safe and well-toleratedby patients.

CLR 131 in combination with dexamethasone is currently under investigation inadult patients with r/r MM. Patients must have been refractory to or relapsedfrom at least one proteasome inhibitor and at least one immunomodulatory agent.The clinical study is a standard three-plus-three dose escalation safety studyto determine the maximum tolerable dose. Multiple myeloma is an incurable cancerof the plasma cells and is the second most common form of hematologic cancers.Secondary objectives include the evaluation of therapeutic activity by assessingsurrogate efficacy markers, which include M protein, free light chain (FLC), PFSand OS. All patients have been heavily pretreated with an average of five priorlines of therapy. CLR 131 was deemed by an Independent Data Monitoring Committee(IDMC) to be safe and tolerable up to its planned maximum single, bolus dose of31.25 mCi/m2. The four single dose cohorts examined were: 12.5 mCi/m2(~25mCiTBD), 18.75 mCi/m2 (~37.5mCi TBD), 25 mCi/m2(~50mCi TBD), and 31.25mCi/m2(~62.5mCi TBD), all in combination with low dose dexamethasone (40 mgweekly). Of the five patients in the first cohort, four achieved stable diseaseand one patient progressed at Day 15 after administration and was taken off thestudy. Of the five patients admitted to the second cohort, all five achievedstable disease however one patient progressed at Day 41 after administration andwas taken off the study. Four patients were enrolled to the third cohort and allachieved stable disease. In September 2017, we announced results for cohort 4,showing that a single infusion up to 30-minutes of 31.25mCi/m2 of CLR 131 wassafe and tolerated by the three patients in the cohort. Additionally, all threepatients experienced CBR with one patient achieving a partial response (PR). Weuse the International Myeloma Working Group (IMWG) definitions of response,which involve monitoring the surrogate markers of efficacy, M protein and FLC.The IMWG defines a PR as a greater than or equal to 50% decrease in FLC levels(for patients in whom M protein is unmeasurable) or 50% or greater decrease in Mprotein. The patient experiencing a PR had an 82% reduction in FLC. This patientdid not produce M protein, had received seven prior lines of treatment includingradiation, stem cell transplantation and multiple triple combination treatmentsincluding one with daratumumab that was not tolerated. One patient experiencingstable disease attained a 44% reduction in M protein. In January 2019, weannounced that the pooled mOS data from the first four cohorts was 22.0 months.In late 2018, we modified this study to evaluate a fractionated dosing strategyto potentially increase efficacy and decrease adverse events.

Following the determination that all prior dosing cohorts were safe andtolerated, we initiated a cohort 7 utilizing a 40mCi/m2 fractionated doseadministered 20mCi/m2 (~40mCi TBD) on days 1 and day 8. Cohort 7 was the highestpre-planned dose cohort and subjects have completed the evaluation period. Finalstudy report and study close-out will be completed later this year.

In May 2019, we announced that the FDA granted Fast Track Designation for CLR131 in fourth line or later r/r MM. CLR 131 is our small-moleculeradiotherapeutic PDC designed to deliver cytotoxic radiation directly andselectively to cancer cells and cancer stem cells. It is currently beingevaluated in our ongoing CLOVER-1 Phase 2 clinical study in patients withrelapsed or refractory multiple myeloma and other select B-cell lymphomas.

Phase 1 Study in r/r Pediatric Patients with select Solid tumors, Lymphomas andMalignant Brain Tumors

In December 2017 the Division of Oncology at the FDA accepted our IND and studydesign for the Phase 1 study of CLR 131 in children and adolescents with selectrare and orphan designated cancers. This study was initiated during the firstquarter of 2019. In December 2017, we filed an IND application for r/r pediatricpatients with select solid tumors, lymphomas and malignant brain tumors. ThePhase 1 clinical study of CLR 131 is an open-label, sequential-group,dose-escalation study evaluating the safety and tolerability of intravenousadministration of CLR 131 in up to 30 children and adolescents with cancersincluding neuroblastoma, sarcomas, lymphomas (including Hodgkin's lymphoma) andmalignant brain tumors. Secondary objectives of the study are to identify therecommended Phase 2 dose of CLR 131 and to determine preliminary antitumoractivity (treatment response) of CLR 131 in children and adolescents. In 2018,the FDA granted OD and RPDD for CLR 131 for the treatment of neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. Should any of theseindications reach approval, the RPDD would enable us to receive a priorityreview voucher. Priority review vouchers can be used by the sponsor to receivepriority review for a future New Drug Application ("NDA") or Biologic LicenseApplication ("BLA") submission, which would reduce the FDA review time from 12months to six months. Currently, these vouchers can also be transferred or soldto another entity.

Phase 1 Study in r/r Head and Neck Cancer

In August 2016, the University of Wisconsin Carbone Cancer Center ("UWCCC") wasawarded a five-year Specialized Programs of Research Excellence ("SPORE") grantof $12,000,000 from the National Cancer Institute and the National Institute ofDental and Craniofacial Research to improve treatments and outcomes for head andneck cancer, HNC, patients. HNC is the sixth most common cancer across the worldwith approximately 56,000 new patients diagnosed every year in the U.S. As a keycomponent of this grant, the UWCCC researchers completed testing of CLR 131 invarious animal HNC models and initiated the first human clinical trial enrollingup to 30 patients combining CLR 131 and external beam radiation with recurrentHNC in Q4 2019. This clinical trial was suspended due to the COVID-19 pandemicbut has now been reopened for enrolment.

We believe our PDC platform has potential to provide targeted delivery of adiverse range of oncologic payloads, as exemplified by the product candidateslisted below, that may result in improvements upon current standard of care("SOC") for the treatment of a broad range of human cancers:

Research and development expense. Research and development expense consist ofcosts incurred in identifying, developing and testing, and manufacturing productcandidates, which primarily include salaries and related expenses for personnel,cost of manufacturing materials and contract manufacturing fees paid to contractmanufacturers and contract research organizations, fees paid to medicalinstitutions for clinical trials, and costs to secure intellectual property. TheCompany analyzes its research and development expenses based on four categoriesas follows: clinical project costs, preclinical project costs, manufacturing andrelated costs, and general research and development costs that are not allocatedto the functional project costs, including personnel costs, facility costs,related overhead costs and patent costs.

General and administrative expense. General and administrative expense consistsprimarily of salaries and other related costs for personnel in executive,finance and administrative functions. Other costs include insurance, costs forpublic company activities, investor relations, directors' fees and professionalfees for legal and accounting services.

Three Months Ended June 30, 2020 and 2019

Research and Development. Research and development expense for the three monthsended June 30, 2020 was approximately $2,465,000 compared to approximately$1,810,000 for the three months ended June 30, 2019.

The following table is an approximate comparison summary of research anddevelopment costs for the three months ended June 30, 2020 and June 30, 2019:

General research and development costs 1,018,000 384,000 634,000

The overall increase in research and development expense of $655,000, or 36%,was primarily a result of increased general research and development costsresulting from increased personnel related costs and in clinical project costs.Manufacturing and related costs decreased due to a decrease in materialsproduction processes and related costs. Pre-clinical study costs were relativelyconsistent.

General and administrative. General and administrative expense for the threemonths ended June 30, 2020 was approximately $1,157,000, compared toapproximately $1,391,000 in the three months ended June 30, 2019. The decreaseof approximately $234,000, or 17%, was primarily a result of lower stock-basedcompensation expense.

Six Months Ended June 30, 2020 and 2019

Research and Development. Research and development expense for the six monthsended June 30, 2020 was approximately $5,082,000 compared to approximately$4,118,000 for the six months ended June 30, 2019.

The following table is a comparison summary of research and development costsfor the six months ended June 30, 2020 and June 30, 2019:

General research and development costs 1,779,000 914,000 865,000

The overall increase in research and development expense of approximately$964,000, or 23%, was primarily a result of increased general research anddevelopment costs resulting from increased personnel related costs and inclinical project costs. Manufacturing and related costs decreased due to adecrease in materials production processes and related costs. Pre-clinical studycosts were relatively consistent.

General and Administrative. General and administrative expense for the sixmonths ended June 30, 2020 was approximately $2,499,000, compared toapproximately $2,712,000 in the six months ended June 30, 2019. The decrease ofapproximately $213,000, or 8%, was primarily a result of lower stock-basedcompensation expense.

Liquidity and Capital Resources

As of June 30, 2020, we had cash and cash equivalents of approximately$22,450,000 compared to $10,615,000 as of December 31, 2019. This increase wasdue primarily to the approximately $18,300,000 of net proceeds received inconnection with the June 5, 2020 public offering. Net cash used in operatingactivities during the six months ended June 30, 2020 was approximately$6,562,000.

Our cash requirements have historically been for our research and developmentactivities, finance and administrative costs, capital expenditures and overallworking capital. We have experienced negative operating cash flows sinceinception and have funded our operations primarily from sales of common stockand other securities. As of June 30, 2020, we had an accumulated deficit ofapproximately $119,251,000.

We believe that the cash balance is adequate to fund our basic budgetedoperations for at least 12 months from the filing of these financial statements.However, our future results of operations involve significant risks anduncertainties. Our ability to execute our operating plan beyond that timedepends on our ability to obtain additional funding via the sale of equityand/or debt securities, a strategic transaction or otherwise. We plan toactively pursue all available financing alternatives; however, there can be noassurance that we will obtain the necessary funding. Other than theuncertainties regarding our ability to obtain additional funding, there arecurrently no known trends, demands, commitments, events or uncertainties thatare likely to materially affect our liquidity. Because we have had recurringlosses and negative cash flows from operating activities, and in light of ourexpected expenditures, the report of our independent auditors with respect tothe financial statements as of December 31, 2019 and for the year ended December31, 2019 contains an explanatory paragraph as to the potential inability tocontinue as a going concern. This opinion indicated at that time, thatsubstantial doubt existed regarding our ability to remain in business.

Edgar Online, source Glimpses

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CELLECTAR BIOSCIENCES : Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - marketscreener.com

CRISPR is a catchy acronym that originally described a naturally occurring gene editing tool, derived from a bacterial defense mechanism against viruses. Its the name on everybodys lips in the intersecting realms of science, medicine, ethics, and politics. From the moment of its discovery, CRISPR-Cas9 looked like a miraculous solution to all of the problems that gene editing efforts have experienced over decades of trial and error. This revolutionary new gene editing technique has opened the doors to both massive scientific progress and ethical controversy. Now more than ever, were seeing that CRISPR still has massive kinks to work out. Can we ever fully understand the social and scientific implications of gene editing, and should we use it in humans before we learn how to properly harness it?

What is gene editing?

The 20th century saw genetic scientists increasingly focus their pursuits on the sub-microscopic. As science delved deeper into the human body in an attempt to uncover the molecular minutiae of life, the possibility of reaching into the cell and manipulating its genetic material began to look more and more real. Even by the 1950s, evidence had been mounting for decades that deoxyribonucleic acid (DNA), an unassuming molecule residing in a central cellular compartment called the nucleus, was the physical genetic material that passed information from parent to child. Finally, in 1953, landmark work by Kings College biochemist Rosalind Franklin allowed Cambridge researchers to reveal the structure of DNA and confirm its role in heredity once and for all.

Starting from a hesitant foundation, molecular genetics exploded in both scope and popularity over subsequent decades. With the secrets of heredity increasingly out in the open, human ambition demanded that we try to bend DNA to our willand now we can. These days, targeted gene editing techniques revolve around artificially-engineered molecular tools known as nucleases, whose earliest use was in 1996not even 50 years after the discovery of DNAs structure. Engineered nucleases are often described as molecular scissors. Fundamentally, they have two main parts: one part that finds and grabs onto the target DNA within a cell, and one part that snips a piece out of that DNA.

How CRISPR works

CRISPR is similar to other directed nucleases, but its much better at its job. The CRISPR part is secondary to the systems gene editing applications; the truly important discovery, which Jennifer Doudna made in 2012, was a protein that she called CRISPR-associated protein 9, or Cas9. This protein is the nuclease tool, the pair of molecular scissors that finds, sticks to, and snips target DNAand its more accurate than anything weve ever seen before.

In bacteria, CRISPR is a section of the genome that acts as an immune memory, storing little snippets of different viruses genetic material as DNA after failed infections, like trophies. When a once-active virus attempts to invade a bacterium, the mobile helper Cas9 copies down the relevant snippet from CRISPR in the form of ribonucleic acid, or RNA. RNA is a molecule thats virtually identical to DNA, except for one extra oxygen atom. Because of this property, the RNA sequence that Cas9 holds can pair exactly, nucleotide by nucleotide, with the viral targets DNA, making it extremely efficient at finding that DNA. With a freshly transcribed RNA guide, the bacterium can deploy Cas9 to findand cut outthe corresponding section of viral genetic material, rendering the attacker harmless.

The existence of CRISPR in bacteria was old news by 2012, but Doudnas discovery of Cas9s function was revolutionary. With a little creativity and ingenuity, such a simple and accurate nuclease can be modified to be much more than just a pair of scissors. Using synthetic RNA guides and certain tweaks, Cas9 can be used to remove specific genes, cause new insertions to genomes, tag DNA sequences with fluorescent probes, and much more.

The possibilities seem endless.What if we could go into the body of a human affected by a hereditary disease and change that persons DNA to cure them? What if we could modify reproductive germ cells in human bodies (which give rise to sperm and eggs), or make targeted genetic edits in the very first cell of an embryo? Nine months of division and multiplication later, that cell would give rise to a human being whose very nature has been deliberately tweakedand their childrens nature, and their childrens. With the accuracy and accessibility of the CRISPR/Cas9 system, these ideas arent hypotheticals. In 2019, CRISPR edits in bone marrow stem cells were successfully used to cure sickle cell anemia in a Mississippi woman. Beta thalassaemia, another genetic disease of the blood, has also been treated this way. In 2018, Chinese scientist He Jiankui even claimed that he had conferred HIV immunity upon twin girls using embryonic editing.

CRISPRs complications

At first glance, CRISPR looks like a miraclebut it isnt perfect. What if some cells were affected by edits, but others werent, creating a strange genetic mosaic in a human body? What if, in trying to modify a specific gene, we accidentally hit a different section of DNA nearby? What if we got the right gene, but it also affected a different part of the body that we didnt know about?

These problems arent hypotheticals either. So-called mosaicism and off-target editing are huge concerns among CRISPR scientists. Mosaicism is of particular concern in embryonic editing. Though CRISPR injections are carried out when an embryo is single-celled, CRISPR doesnt always appear to work until after several rounds of cell divisionand it doesnt work in every cell. If not all the cells in the body are affected by gene editing that is intended to eliminate a genetic disease, the disease could remain in the body. It may be possible to combat mosaicism with faster gene editing (so that cells dont replicate before theyve had a chance to become CRISPR-modified), altering sperm and egg cells before they meet to form an embryo, and developing more precise CRISPR gene editing which is in itself a challenge, thanks to off-target editing.

In nature, a little bit of off-target editing could actually make the CRISPR-Cas9 defense system stronger with the principle of redundancy. Flexibility in the form of imprecision could allow a bacterium to neutralize viruses whose exact genetic sequences have not yet been encountered: viruses related to, but not identical to, previous attackers. In clinical and therapeutic applications, on the other hand, precision is everything. And unfortunately, as time passes, CRISPRs level of precision seems further and further off. Preprints released just this year reveal that the frequency and magnitude of CRISPRs off-target edits in human cells may be worse than we had previously known. Large proportions of cells with massive unwanted DNA deletions, losses of entire chromosomes in experimental embryos, and shuffling of genetic sequences were observed.

Of course, not only do scientists need to avoid off-target edits, but they also need to know when such undesired edits have occurred. Off-target effects can be detected by genome sequencing and computer prediction tools, but theres no perfect way to do it yetthere may still be editing misses that were, well, missing. Off-target edits themselves could be minimized by altering the RNA transcript that Cas9 carries to make it more accurate, altering Cas9 itself, or reducing the actual amount of Cas9 protein released into the cell (though this could also reduce on-target effects). Replacing Cas9 itself with other Cas variants, like smaller and more easily deliverable CasX and CasY proteins, is a promising possibility for more efficient editing, but these candidates still run into many of the same problems as Cas9. More strategies are constantly being discovered, proposed, and explored, but were still nowhere near perfect.

Perhaps most importantly, even barring any purely technical problems, is that humans remain in sheer ignorance of much of the extent and consequences of pleiotropy, a phenomenon where a genes presence or deletion has more than one effect in the human body. Even genes whose function we think we know well might have totally unexpected additional functions. On the other side of the coin, we dont have a comprehensive understanding of how many different genetic contributors there are to any given trait or disease, much less where they lie in the genome. We dont understand the way that thousands of variations across the entire genome contribute to appearance, personality, and health. Assuming that some genes are good and others are bad is morally dangerous, and scientifically reprehensible. In reality, we are not ready for genetic determinism, and may never be.

A great responsibility

Humanity has discovered a great power, but we all know what comes with great power. Questions of which edits are necessary for health (is mild Harlequin syndrome a disease or a cosmetic concern?), whether edits are ethical (should autism and homosexuality be considered curable conditions?), and the possibility of designer babies, among others, are pertinent and require thorough discussion. We also need to realize that making these types of changes isnt our decision until we can get CRISPR right, and understand the genome well enough to target particular phenotypes. Though most scientists are aware of the difficulties of CRISPR and its use is generally tightly regulated, some scientistsand laypeopleare less careful. He Jiankuis apparent miracle HIV cure led to his arrest and imprisonment for unapproved and unethical practice. Its no great surprise that his work likely fell prey to off-target effects and mosaicism; even if he got it right, his intended change could alter cognitive function, and who knows what else?

Non-scientists are getting involved too: in 2018, self-proclaimed biohacker Josiah Zayner publicly injected his own arm with what he claimed was muscle-enhancing CRISPR. Though Zayner is one of the most vocal, hes not the only one of his kind. Quieter biohackers, untrained people without a scientific background or a good understanding of how CRISPR can go wrong, are attempting to edit themselves and even their pets.

Laypeople have an unquestionable place in science: the scientific discipline needs fresh perspectives and creativity that stuffy academics cant offer. CRISPR is still in its infancy, though. Before we know much, much more about its capabilities and consequences, there can be no place for black market gene editing kits, rogue scientists altering human embryonic and germline DNA, or basement geneticists injecting Cas9 into their dogs. Who can say what effects these interventions might have, not just on edited individuals, but on the futures of entire species?

Some say that gene editing is an act of hubris, destined to backfire spectacularly and horrendously. Others believe that its our responsibility to use CRISPR to improve lives. Which of these opinions is true depends on how science walks a narrow tightrope, though Im inclined to agree with the latterand add that our responsibility is not just to master gene editing, but to make clear and public its many faults and failings. The truth, in all its complexity, needs to overcome pop sciences oversimplification and sensationalism. Promising new advances and techniques are on the horizon, but we have a long way to go. Gene editing is no joke; humanity is playing with fire. With an incredibly accurate and accessible nuclease making its way into labs and garages across the world (while its flaws continue to be uncovered year by year), it is more important than ever for the world to understand and discuss the long-reaching consequences and responsible use of gene editing technology. CRISPR is not a miracle, but gene editing may very well be the future of humanityand its on us to keep it under control.

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The Trouble With CRISPR The Strand - Strand

The Covid-19 pandemic is one of the greatest challenges modern medicine has ever faced. Doctors and scientists are scrambling to find treatments and drugs that can save the lives of infected people and perhaps even prevent them from getting sick in the first place.

Below is an updated list of 20 of the most-talked-about treatments for the coronavirus. While some are accumulating evidence that theyre effective, most are still at early stages of research. We also included a warning about a few that are just bunk.

We are following 20 coronavirus treatments for effectiveness and safety:

Tentative or

mixed evidence

We are following 20 coronavirus treatments

for effectiveness and safety:

Tentative or

mixed evidence

We are following 20 coronavirus treatments

for effectiveness and safety:

There is no cure yet for Covid-19. And even the most promising treatments to date only help certain groups of patients and await validation from further trials. The F.D.A. has not fully licensed any treatment specifically for the coronavirus. Although it has granted emergency use authorization to some treatments, their effectiveness against Covid-19 has yet to be demonstrated in large-scale, randomized clinical trials.

This list provides a snapshot of the latest research on the coronavirus, but does not constitute medical endorsements. Always consult your doctor about treatments for Covid-19.

New additions and recent updates:

Added ivermectin, a drug typically used against parasitic worms that is being increasingly prescribed in Latin America. Aug. 10

Updated descriptions for several treatments. Aug. 10

We will update and expand the list as new evidence emerges. For details on evaluating treatments, see the N.I.H. Covid-19 Treatment Guidelines. For the current status of vaccine development, see our Coronavirus Vaccine Tracker.

WIDELY USED: These treatments have been used widely by doctors and nurses to treat patients hospitalized for diseases that affect the respiratory system, including Covid-19.

PROMISING EVIDENCE: Early evidence from studies on patients suggests effectiveness, but more research is needed. This category includes treatments that have shown improvements in morbidity, mortality and recovery in at least one randomized controlled trial, in which some people get a treatment and others get a placebo.

TENTATIVE OR MIXED EVIDENCE: Some treatments show promising results in cells or animals, which need to be confirmed in people. Others have yielded encouraging results in retrospective studies in humans, which look at existing datasets rather than starting a new trial. Some treatments have produced different results in different experiments, raising the need for larger, more rigorously designed studies to clear up the confusion.

NOT PROMISING: Early evidence suggests that these treatments do not work.

PSEUDOSCIENCE OR FRAUD: These are not treatments that researchers have ever considered using for Covid-19. Experts have warned against trying them, because they do not help against the disease and can instead be dangerous. Some people have even been arrested for their false promises of a Covid-19 cure.

EVIDENCE IN CELLS, ANIMALS or HUMANS: These labels indicate where the evidence for a treatment comes from. Researchers often start out with experiments on cells and then move onto animals. Many of those animal experiments often fail; if they dont, researchers may consider moving on to research on humans, such as retrospective studies or randomized clinical trials. In some cases, scientists are testing out treatments that were developed for other diseases, allowing them to move directly to human trials for Covid-19.

All treatmentsWidely usedPromisingTentative or mixedNot promisingPseudoscience

Antivirals can stop viruses such as H.I.V. and hepatitis C from hijacking our cells. Scientists are searching for antivirals that work against the new coronavirus.

PROMISING EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSEMERGENCY USE AUTHORIZATIONRemdesivirRemdesivir, made by Gilead Sciences, was the first drug to get emergency authorization from the F.D.A. for use on Covid-19. It stops viruses from replicating by inserting itself into new viral genes. Remdesivir was originally tested as an antiviral against Ebola and Hepatitis C, only to deliver lackluster results. But preliminary data from trials that began this spring suggested the drug can reduce the recovery time of people hospitalized with Covid-19 from 15 to 11 days. (The study defined recovery as either discharge from the hospital or hospitalization for infection-control purposes only.) These early results did not show any effect on mortality, though retrospective data released in July hints that the drug might reduce death rates among those who are very ill.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSFavipiravirOriginally designed to beat back influenza, favipiravir blocks a viruss ability to copy its genetic material. A small study in March indicated the drug might help purge the coronavirus from the airway, but results from larger, well-designed clinical trials are still pending.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSMK-4482Another antiviral originally designed to fight the flu, MK-4482 (previously known as EIDD-2801) has had promising results against the new coronavirus in studies in cells and on animals. Merck, which has been running clinical trials on the drug this summer, has announced it will launch a large Phase III trial in September.Updated Aug. 6

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS Recombinant ACE-2To enter cells, the coronavirus must first unlock them a feat it accomplishes by latching onto a human protein called ACE-2. Scientists have created artificial ACE-2 proteins which might be able to act as decoys, luring the coronavirus away from vulnerable cells. Recombinant ACE-2 proteins have shown promising results in experiments on cells, but not yet in animals or people.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS AND HUMANS IvermectinFor decades, ivermectin has served as a potent drug to treat parasitic worms. Doctors use it against river blindness and other diseases, while veterinarians give dogs a different formulation to cure heartworm. Studies on cells have suggested ivermectin might also kill viruses. But scientists have yet to find evidence in animal studies or human trials that it can treat viral diseases. As a result, Ivermectin is not approved to use as an antiviral.

In April, Australian researchers reported that the drug blocked coronaviruses in cell cultures, but they used a dosage that was so high it might have dangerous side effects in people. The FDA immediately issued a warning against taking pet medications to treat or prevent Covid-19. These animal drugs can cause serious harm in people, the agency warned.

Since then a number of clinical trials have been launched to see if a safe dose of ivermectin can fight Covid-19. In Singapore, for example, the National University Hospital is running a 5,000-person trial to see if it can prevent people from getting infected. As of now, theres no firm evidence that it works. Nevertheless ivermectin is being prescribed increasingly often in Latin America, much to the distress of disease experts.Updated Aug. 10

NOT PROMISING EVIDENCE IN CELLS AND HUMANS Lopinavir and ritonavirTwenty years ago, the F.D.A. approved this combination of drugs to treat H.I.V. Recently, researchers tried them out on the new coronavirus and found that they stopped the virus from replicating. But clinical trials in patients proved disappointing. In early July, the World Health Organization suspended trials on patients hospitalized for Covid-19. They didnt rule out studies to see if the drugs could help patients not sick enough to be hospitalized, or to prevent people exposed to the new coronavirus from falling ill. The drug could also still have a role to play in certain combination treatments.

NOT PROMISING EVIDENCE IN CELLS, ANIMALS AND HUMANSHydroxychloroquine and chloroquineGerman chemists synthesized chloroquine in the 1930s as a drug against malaria. A less toxic version, called hydroxychloroquine, was invented in 1946, and later was approved for other diseases such as lupus and rheumatoid arthritis. At the start of the Covid-19 pandemic, researchers discovered that both drugs could stop the coronavirus from replicating in cells.

Since then, theyve had a tumultuous ride. A few small studies on patients offered some hope that hydroxychloroquine could treat Covid-19. The World Health Organization launched a randomized clinical trial in March to see if it was indeed safe and effective for Covid-19, as did Novartis and a number of universities. Meanwhile, President Trump repeatedly promoted hydroxychloroquine at press conferences, touting it as a game changer, and even took it himself. The F.D.A. temporarily granted hydroxychloroquine emergency authorization for use in Covid-19 patients which a whistleblower later claimed was the result of political pressure. In the wake of the drugs newfound publicity, demand spiked, resulting in shortages for people who rely on hydroxychloroquine as a treatment for other diseases.

But more detailed studies proved disappointing. A study on monkeys found that hydroxychloroquine didnt prevent the animals from getting infected and didnt clear the virus once they got sick. Randomized clinical trials found that hydroxychloroquine didnt help people with Covid-19 get better or prevent healthy people from contracting the coronavirus. Another randomized clinical trial found that giving hydroxychloroquine to people right after being diagnosed with Covid-19 didnt reduce the severity of their disease. (One large-scale study that concluded the drug was harmful as well was later retracted.) The World Health Organization, the National Institutes of Health and Novartis have since halted trials investigating hydroxychloroquine as a treatment for Covid-19, and the F.D.A. revoked its emergency approval. The F.D.A. now warns that the drug can cause a host of serious side effects to the heart and other organs when used to treat Covid-19.

In July, researchers at Henry Ford hospital in Detroit published a study finding that hydroxychloroquine was associated with a reduction in mortality in Covid-19 patients. President Trump praised the study on Twitter, but experts raised doubts about it. The study was not a randomized controlled trial, in which some people got a placebo instead of hydroxychloroquine. The studys results might not be due to the drug killing the virus. Instead, doctors may have given the drug to people who were less sick, and thus more likely to recover anyway.

Despite negative results, a number of hydroxychloroquine trials have continued, although most are small, testing a few dozen or a few hundred patients. A recent analysis by STAT and Applied XL found more than 180 ongoing clinical trials testing hydroxychloroquine or chloroquine, for treating or preventing Covid-19. Although its clear the drugs are no panacea, its theoretically possible they could provide some benefit in combination with other treatments, or when given in early stages of the disease. Only well-designed trials can determine if thats the case.Updated Aug. 10

Most people who get Covid-19 successfully fight off the virus with a strong immune response. Drugs might help people who cant mount an adequate defense.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS AND HUMANS Convalescent plasmaA century ago, doctors filtered plasma from the blood of recovered flu patients. So-called convalescent plasma, rich with antibodies, helped people sick with flu fight their illness. Now researchers are trying out this strategy on Covid-19. In May, the F.D.A. designated convalescent plasma an investigational product. That means that despite not yet being shown as safe and effective, plasma can be used in clinical trials and given to some patients who are seriously ill with Covid-19. Tens of thousands of patients in the U.S. have received plasma through a program launched by the Mayo Clinic and the federal government.

The Trump administration has praised convalescent plasma, despite the lack of evidence yet that it works. The first wave of trials have been small and the results have been mixed. Large randomized clinical trials are underway, but theyve struggled to enroll enough participants, some of whom worry they will receive a placebo instead of the treatment itself.

Experts say that its vital to complete these trials to determine if convalescent plasma is safe and effective. If these trials are successful, it could serve as an important stopgap measure until more potent therapies become widely available.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSMonoclonal antibodiesConvalescent plasma from people who recover from Covid-19 contains a mix of different antibodies. Some of the molecules can attack the coronavirus, but many are directed at other pathogens. Researchers have sifted through this slurry to find the most potent antibodies against Covid-19. They have then manufactured synthetic copies of these molecules, known as monoclonal antibodies. Researchers have begun investigating them as a treatment for Covid-19, either individually or in cocktails.

Monoclonal antibodies were first developed as a therapy in the 1970s, and since then the F.D.A. has approved them for 79 diseases, ranging from cancer to AIDS. Since the start of the pandemic, researchers have found dozens of monoclonal antibodies that show promise against Covid-19 in preclinical studies on cells and animals. Companies like Eli Lilly and Regeneron recently began clinical trials studying monoclonal antibodies. Several other firms, as well as teams at universities, are slated to enter the race soon as well.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSInterferonsInterferons are molecules our cells naturally produce in response to viruses. They have profound effects on the immune system, rousing it to attack the invaders, while also reining it in to avoid damaging the bodys own tissues. Injecting synthetic interferons is now a standard treatment for a number of immune disorders. Rebif, for example, is prescribed for multiple sclerosis.

As part of its strategy to attack our bodies, the coronavirus appears to tamp down interferon. That finding has encouraged researchers to see whether a boost of interferon might help people weather Covid-19, particularly early in infection. Early studies, including experiments in cells and mice, have yielded encouraging results that have led to clinical trials.

An open-label study in China suggested that the molecules could help prevent healthy people from getting infected. On July 20, the British pharmaceutical company Synairgen announced that an inhaled form of interferon called SNG001 lowered the risk of severe Covid-19 in infected patients in a small clinical trial. The full data have not yet been released to the public, or published in a scientific journal. On August 6, the National Institute of Allergy and Infectious Diseases launched a Phase III trial on a combination of Rebif and the antiviral remdesivir, with results expected by fall 2020.Updated Aug. 10

The most severe symptoms of Covid-19 are the result of the immune systems overreaction to the virus. Scientists are testing drugs that can rein in its attack.

PROMISING EVIDENCE EVIDENCE IN HUMANS DexamethasoneThis cheap and widely available steroid blunts many types of immune responses. Doctors have long used it to treat allergies, asthma and inflammation. In June, it became the first drug shown to reduce Covid-19 deaths. That study of more than 6,000 people, which in July was published in the New England Journal of Medicine, found that dexamethasone reduced deaths by one-third in patients on ventilators, and by one-fifth in patients on oxygen. It may be less likely to help and may even harm patients who are at an earlier stage of Covid-19 infections, however. In its Covid-19 treatment guidelines, the National Institutes of Health recommends only using dexamethasone in patients with COVID-19 who are on a ventilator or are receiving supplemental oxygen.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS Cytokine InhibitorsThe body produces signaling molecules called cytokines to fight off diseases. But manufactured in excess, cytokines can trigger the immune system to wildly overreact to infections, in a process sometimes called a cytokine storm. Researchers have created a number of drugs to halt cytokine storms, and they have proven effective against arthritis and other inflammatory disorders. Some turn off the supply of molecules that launch the production of the cytokines themselves. Others block the receptors on immune cells to which cytokines would normally bind. A few block the cellular messages they send. Depending on how the drugs are formulated, they can block one cytokine at a time, or muffle signals from several at once.

Against the coronavirus, several of these drugs have offered modest help in some trials, but faltered in others. Drug companies Regeneron and Roche drug both recently announced that two drugs called sarilumab and tocilizumab, which both target the cytokine IL-6, did not appear to benefit patients in Phase 3 clinical trials. Many other trials remain underway, several of which combine cytokine inhibitors with other treatments.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS EMERGENCY USE AUTHORIZATIONBlood filtration systemsThe F.D.A. has granted emergency use authorization to several devices that filter cytokines from the blood in an attempt to cool cytokine storms. One machine, called Cytosorb, can reportedly purify a patients entire blood supply about 70 times in a 24-hour period. A small study in March suggested that Cytosorb had helped dozens of severely ill Covid-19 patients in Europe and China, but it was not a randomized clinical trial that could conclusively demonstrate it was effective. A number of studies on blood filtration systems are underway, but experts caution that these devices carry some risks. For example, such filters could remove beneficial components of blood as well, such as vitamins or medications.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS Stem cellsCertain kinds of stem cells can secrete anti-inflammatory molecules. Over the years, researchers have tried to use them as a treatment for cytokine storms, and now dozens of clinical trials are under way to see if they can help patients with Covid-19. But these stem cell treatments havent worked well in the past, and its not clear yet if theyll work against the coronavirus.

Doctors and nurses often administer other supportive treatments to help patients with Covid-19.

WIDELY USEDProne positioningThe simple act of flipping Covid-19 patients onto their bellies opens up the lungs. The maneuver has become commonplace in hospitals around the world since the start of the pandemic. It might help some individuals avoid the need for ventilators entirely. The treatments benefits continue to be tested in a range of clinical trials.

WIDELY USEDEMERGENCY USE AUTHORIZATIONVentilators and other respiratory support devicesDevices that help people breathe are an essential tool in the fight against deadly respiratory illnesses. Some patients do well if they get an extra supply of oxygen through the nose or via a mask connected to an oxygen machine. Patients in severe respiratory distress may need to have a ventilator breathe for them until their lungs heal. Doctors are divided about how long to treat patients with noninvasive oxygen before deciding whether or not they need a ventilator. Not all Covid-19 patients who go on ventilators survive, but the devices are thought to be lifesaving in many cases.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS AnticoagulantsThe coronavirus can invade cells in the lining of blood vessels, leading to tiny clots that can cause strokes and other serious harm. Anticoagulants are commonly used for other conditions, such as heart disease, to slow the formation of clots, and doctors sometimes use them on patients with Covid-19 who have clots. Many clinical trials teasing out this relationship are now underway. Some of these trials are looking at whether giving anticoagulants before any sign of clotting is beneficial.

False claims about Covid-19 cures abound. The F.D.A. maintains a list of more than 80 fraudulent Covid-19 products, and the W.H.O. debunks many myths about the disease.

WARNING: DO NOT DO THISDrinking or injecting bleach and disinfectantsIn April, President Trump suggested that disinfectants such as alcohol or bleach might be effective against the coronavirus if directly injected into the body. His comments were immediately refuted by health professionals and researchers around the world as well as the makers of Lysol and Clorox. Ingesting disinfectant would not only be ineffective against the virus, but also hazardous possibly even deadly. In July, Federal prosecutors charged four Florida men with marketing bleach as a cure for COVID-19.

WARNING: NO EVIDENCEUV lightPresident Trump also speculated about hitting the body with ultraviolet or just very powerful light. Researchers have used UV light to sterilize surfaces, including killing viruses, in carefully managed laboratories. But UV light would not be able to purge the virus from within a sick persons body. This kind of radiation can also damage the skin. Most skin cancers are a result of exposure to the UV rays naturally present in sunlight.

WARNING: NO EVIDENCESilverThe F.D.A. has threatened legal action against a host of people claiming silver-based products are safe and effective against Covid-19 including televangelist Jim Bakker and InfoWars host Alex Jones. Several metals do have natural antimicrobial properties. But products made from them have not been shown to prevent or treat the coronavirus.

Note: After additional discussions with experts we have adjusted several labels on the tracker. The Strong evidence label has been removed until further research identifies treatments that consistently benefit groups of patients infected by the coronavirus. In its place, Promising evidence will be used for drugs such as remdesivir and dexamethasone that have shown promise in at least one randomized controlled trial, and Widely used for treatments such as proning and ventilators that are often used with severely ill patients, including those with Covid-19. And we may reintroduce the Ineffective label when ongoing clinical trials repeatedly end with disappointing results.

Sources: National Library of Medicine; National Institutes of Health; William Amarquaye, University of South Florida; Paul Bieniasz, Rockefeller University; Jeremy Faust, Brigham & Womens Hospital; Matt Frieman, University of Maryland School of Medicine; Noah Haber, Stanford University; Swapnil Hiremath, University of Ottawa; Akiko Iwaskai, Yale University; Paul Knoepfler, University of California, Davis; Elena Massarotti, Brigham and Womens Hospital; John Moore and Douglas Nixon, Weill Cornell Medical College; Erica Ollman Saphire, La Jolla Institute for Immunology; Regina Rabinovich, Harvard T.H. Chan School of Public Health; Ilan Schwartz, University of Alberta; Phyllis Tien, University of California, San Francisco.

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Coronavirus Drug and Treatment Tracker - The New York Times