Warm Up with Homemade Broths and Stocks – The Source Weekly
By daniellenierenberg
Abowl of hot soup or warm broth can take the chill out of a cold winter day. While grocery store shelves are usually stocked with an array of canned and boxed varieties, making a delicious broth for sipping or a stock as a base for other soups and recipes is something you can do while doing other things, such as working on your computer or doing the laundry. Once you get it going, you only have to check on it occasionally until it's done.
According to Food & Wine, the difference between stock and broth is minimal. A stock is to be made with bones in addition to a mirepoix, a mix of carrots, onions and celery. At its most basic, broth is simply any liquid that meat has been cooked in. A broth can also be made with just vegetables. While broth is something you sip, stock is typically used as a base in sauces and soups, providing body rather than flavor.
As author Sally Fallon Morell points out in her book, "Nourishing Broth: An Old-Fashioned Remedy for the Modern World," bone broth has rich dissolves of collagen, cartilage, bone and marrow which give the body the right stuff to rebuild and rejuvenatestuff such as vitamins, minerals, amino acids and healing sugars.
"Deep in the center of bones is marrow, a creamy substance valued by our ancestors for its life-giving, reproduction-enhancing, and brain-building fat and cholesterol. As the seed of blood and stem cells, it's prized as a sacred, energizing, and regenerative food in native cultures all over the world," Morell writes. Of course, most are aware of the benefits of eating plenty of plants in our diet, too.
Whether you're going for bones or carrots or both, the basic technique is the same. Simmer veggie scraps or bones in water for a long, slow time (in the case of straight-up vegetable broth, it can be finished in one hour or less).
Simple Vegetable BrothA swirl of your favorite oil1 onion, chopped2 stalks celery, chopped2 large carrots, choppedLeftover vegetable scraps you have on hand (onion skins, carrot ends, celery leaves, herbs, potato peels, greens, etc.)Several cloves of garlic, smashedFresh parsley/thymePinch of saltTwo bay leavesWater to cover
Saut chopped veggies in a bit of oil or water to soften. Add salt, herbs, bay leaves and water to cover. Bring to almost boiling, then turn heat down to simmer for 45-60 minutes, longer if desired. Strain. Cool.
Basic Bone Broth
Roast bones on baking sheet in hot oven (400 degrees) for 30 minutes. Place bones and vegetables in big pot. Cover with water. Bring to an easy roll then immediately turn heat down. Simmer uncovered, skimming scum as it rises. Cook for 24-72 hours. Turn off overnight, turn back on to simmer next morning. During last 10 minutes of cooking, toss in fresh parsley for added flavor. Let broth cool before straining. Store in fridge up to one week or freezer up to six months.
Pro Tips:
The number one goal for bone broth/stock is to get it gelatinous, meaning it sets up in a solid gel if you put it in the fridge. Bones, such as knucklebones and chicken/pig feet with lots of cartilage help make the broth gelatinous. Include meaty bones, such as short ribs, to add flavor.
Water should just cover the bones.
Never overheat the broth/stock. A roiling boil will break down collagen fibers that won't coagulate when cooled. So heat over medium heat only until the liquid starts to "roll," then turn the heat down until it barely simmers.
Simmer with the lid off to prevent boiling and allow the gradual reduction of the stock and concentration of gelatin.
To avoid cloudiness, skim the scum that rises to the top as the liquid starts to cook and occasionally throughout cooking.
To freeze stock, only fill the container full.
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Warm Up with Homemade Broths and Stocks - The Source Weekly
Gamida Cell to Present Full Data from Phase 3 Study of Omidubicel at TCT, the Combined Transplantation and Cellular Therapy Meetings of ASTCT and…
By daniellenierenberg
CARLSBAD, Calif., Jan. 12, 2021 /PRNewswire/ Today Callaway Golf Company (NYSE: ELY), an industry leader in golf equipment and innovation, announced its new family of Apex Irons and Apex Hybrids. These irons provide exceptional forged performance and A.I.-designed ball speed technologies for a wide range of players. And the hybrids feature new Jailbreak A.I. Velocity Blades for fast ball speeds in versatile, high performance offerings.
Apex 21, Apex Pro 21, and Apex DCB Irons
Apex is widely recognized for establishing the forged distance category, and the Apex 21 Irons deliver an exceptional level of performance and craftsmanship. They're the first forged Apex irons with an A.I-designed Flash Face Cup, for high ball speeds and increased spin robustness across the face. Increased forgiveness comes from a massive Tungsten Energy Core, while the 100% forged body and proprietary urethane microspheres deliver remarkable sound and feel at impact. Callaway has also enhanced the shaping for even better turf interaction.
In the players category, new Apex Pro 21 Irons promote Tour distance and performance for scratch or single-digit handicap golfers. They also utilize an A.I.-designed Flash Face in each iron for high COR's and fast ball speeds, along with extremely soft feel from an all-new forged 1025 hollow body construction and urethane microspheres. There are up to 90 grams of tungsten in the longer irons, the most ever for any Apex model, to improve launch characteristics while simultaneously improving forgiveness.
In the game-improvement category, new Apex DCB Irons extend the forged Apex offering to a wider group of golfers than ever before. They combine the look, feel and performance of a forged players club with the forgiveness of a deep cavity back. The deep cavity back design and enhanced sole width promote easy launch and solid turf interaction out of a variety of lies. The irons are engineered with an A.I.-designed Flash Face Cup, up to 50 grams of tungsten per iron for outstanding launch and forgiveness on off-center hits, and forged feel.
All of these Apex Irons will be available for online pre-order on January 26, and at retail on February 11, at a price of $1,480 for a standard 8-piece steel set ($185 per individual steel iron), and $1,600 for a standard 8-piece graphite set ($200 per individual graphite iron). Combo sets are also available.
Apex 21 and Apex Pro 21 Hybrids
The Apex 21 Hybrids are suited to help a wide range of players. They feature new Jailbreak A.I. Velocity Blades designed to increase vertical stiffness near the sole of the club, promoting more speed low on the face where players often mishit their hybrids. The blades allow the Face Cup to flex on the crown to create better spin rate consistency, and the bars are spread to enhance torsional stiffness, to provide more forgiveness across the face.
Every model and every face in the Apex Hybrids are uniquely designed using advanced A.I. This proven ball speed technology puts an even greater emphasis on center and off-center ball speeds. To create high launch and forgiveness. Callaway has implemented a massive amount of tungsten, and the adjustable hosel helps to optimize loft, trajectory and control.
Apex Pro Hybrids also incorporate Jailbreak A.I. Velocity Blades and an A.I-designed Flash Face. The Forged 455 steel provides strength and flexibility, while the iron-like design and fixed hosel create a look at address that highly skilled players prefer.
These new hybrids will all be available for online pre-order on January 26, and at retail on February 11, at a price of $269.99 each.
The Apex Family
Apex is synonymous with legendary performance and we've created a truly special offering with our new 2021 lineup, said Callaway Sr. VP of R&D, Dr. Alan Hocknell. Now we've implemented A.I.-designed ball speeds, enhanced launch characteristics, and reliable forgiveness to advance this iconic name. There's nothing like our best, and that's Apex.
About Callaway Golf Company Callaway Golf Company (NYSE: ELY) is a premium golf equipment and active lifestyle company with a portfolio of global brands, including Callaway Golf, Odyssey, OGIO, TravisMathew and Jack Wolfskin. Through an unwavering commitment to innovation, Callaway manufactures and sells premium golf clubs, golf balls, golf and lifestyle bags, golf and lifestyle apparel and other accessories. For more information please visitwww.callawaygolf.com, http://www.odysseygolf.com,www.ogio.com, http://www.travismathew.com,andwww.jack-wolfskin.com.
MEDIA CONTACTS: Jeff NewtonCallaway Golf Company[emailprotected]
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Gamida Cell to Present Full Data from Phase 3 Study of Omidubicel at TCT, the Combined Transplantation and Cellular Therapy Meetings of ASTCT and...
Bone Marrow Processing Systems Market Recent developments in the competitive landscape forecast 2018 2025 – SoccerNurds
By daniellenierenberg
Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.
The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.
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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.
In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.
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Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.
Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.
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Bone Marrow Processing Systems Market Recent developments in the competitive landscape forecast 2018 2025 - SoccerNurds
Four promising COVID-19 therapies being tested at nearby UVA – Rappahannock News
By daniellenierenberg
Over the past nine months, clinical trials conducted at the University of Virginia have led to new treatments for patients fighting COVID-19 and new tools for health care workers saving lives around the commonwealth and world.
We have been able to learn very quickly, and try new things that have changed the way we approach treatment for this virus, said Dr. Kyle Enfield, a professor and physician in pulmonary and critical care medicine who has helped to coordinate clinical trials at UVA Health.
We are seeing clinical research happen at a speed that has never been seen before, both for drug therapies and vaccine development, Dr. Linda Duska, associate dean for clinical research in the School of Medicine, added. Weve also seen funding and the regulatory apparatus really adapt to this pandemic, while maintaining rigorous standards.
Four clinical trials of COVID-19 drug therapies either underway or completed at UVA, and their implications for patients and for the ongoing pandemic, are summarized below.
A single-site trial based at UVA, the study examines the use of convalescent plasma derived from blood donated by recovered COVID-19 patients to treat patients hospitalized with the virus, but not yet in intensive care. In theory, the antibodies in the plasma will bind to virus cells, blocking them from harming healthy cells.
Convalescent plasma therapy has been around for more than 100 years, and there has been a lot of interest in it since COVID-19 appeared, said Dr. Jeff Sturek, who specializes in pulmonary and critical care medicine and is the principal investigator for the trial. We wanted to bring this therapy to UVA, to contribute to the development of the field and to offer our patients as many options as possible.
The trial was approved in April and patients were enrolled at UVA from May to August. Researchers are now in the process of analyzing results, which look promising.
UVA is part of a multisite Adaptive COVID-19 Treatment Trial, or ACTT, testing the antiviral drug remdesivir in adults hospitalized with COVID-19.
Results from the first part of the trial found that the drug sped recovery time in patients with advanced cases of COVID-19, prompting the U.S. Food and Drug Administration to issue an emergency use authorization for remdesivir. It was the first drug authorized to treat COVID-19.
The trial is now in its third phase; it began with comparing remdesivir to a placebo drug, and then progressed to pairing different drugs with remdesivir, to see which combination was most effective.
The trial was designed to be iterative, to allow us to continue to adapt the study as we learn more about the drug, Duska said. That lets us continually improve treatment without having to go through a complete restart.
Another multisite trial that includes UVA is investigating if infusion of the mesenchymal stromal cell remestemcel-L, a type of stem cell derived from bone marrow, can increase survival rates among COVID-19 patients experiencing acute respiratory distress syndrome.
The cells have been shown to migrate to the lungs when inflammation occurs and release anti-inflammatory factors that can reduce cytokines secreted by the immune systems. High levels of cytokine production have been associated with severe illness and death among COVID-19 patients.
These adult bone marrow stem cells have been used to treat a variety of inflammatory diseases, which means they have already been through early safety trials and we could move more quickly into a larger trial, said Sturek, also the principal investigator for this trial. We hope that the cells can turn down inflammation in the lungs and help the lungs repair themselves, especially for critical ill patients on ventilators.
The trial is halfway through its enrollment process, with a target of enrolling 300 patients. It has already passed initial safety checks with the National Institutes of Healths Data Safety Monitoring Board.
In this multisite trial, researchers are working to determine if monoclonal antibodies made by the drug company Regeneron Pharmaceuticals can prevent COVID-19 infection among people who have been exposed by someone in their household, but have not yet developed the disease. The trial is testing the same antibody cocktail given to President Trump when he was hospitalized with COVID-19, though with a different use.
In this case, the antibodies are intended to prevent people from getting sick if they have a household member with COVID, Enfield said. So far, UVA has done a good job with recruitment, which is particularly tricky in this case as you have to find people who have been exposed to COVID in their household, but who do not yet have COVID.
UVA is recruiting 40 participants for the study, each of whom will receive four injections of either the antibodies or a placebo. Participants must have been exposed to COVID-19 by someone in their household within the previous 96 hours and continue to live with that person for a month.
Its been a rapid process, and a testament to the multidisciplinary team involved, from infectious disease clinicians and researchers to cell therapy, pulmonary critical care and several other departments, Sturek said. Its been all-hands-on-deck.
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Four promising COVID-19 therapies being tested at nearby UVA - Rappahannock News
Brave West Lothian women discovers back pain is actually deadly blood cancer – Daily Record
By daniellenierenberg
A brave West Lothian mum was floored after doctors found her sciatica pain was actually a symptom of a deadly blood cancer which had hollowed out her bones.
Judith Green had suffered from back pain on several occasions over the last 10 years but was repeatedly told it was likely due to a trapped nerve and would resolve itself.
The 42-year-olds pain became too much in June 2019 when she woke screaming in the middle of the night before repeatedly vomiting blood over the next two days.
She took herself to St Johns Hospital in Livingston where doctors soon made the shock diagnosis of myeloma cancer which had left her kidneys functioning at only 15 per cent.
The mum-of-two was told that the condition - which normally affects men over the age of 60 - was incurable but doctors hoped to extend her life through various treatments.
She underwent a stem cell transplant with her own cells in January 2019 but was heartbroken when medics revealed the cancer had returned just seven months later.
The former waitress has vowed to keep fighting so she can meet her future grandchildren and is urging people to register as stem cell donors in a bid to save more lives.
She explained: I remember thinking but its just a sore back. I had never heard of myeloma before I got diagnosed with it.
I 100 per cent thought I was going to hospital that day because I had sciatica. With myeloma, it eats away at your bone marrow.
My ribs were sore but I brushed it off thinking it was my new bra digging in. When my back hurt, I thought it was the new car seat causing it.
But in reality, I had almost no bone marrow. It was 90 per cent cancerous cells. I just made excuse after excuse but looking back I now realise that it was all part of it.
My kidneys were only working at 15 per cent, which explained why I was so thirsty.
Doctors immediately started Judith on a course of chemotherapy and steroids before attempting to harvest some of her remaining bone marrow.
The first attempt was unsuccessful but the next managed to gather enough cells to provide at least three more transplants.
The cells were then deep frozen before being transplanted back into the mum-of-two in January this year - a move which they hoped would buy her at least 18 more months.
But a blood test in August revealed that the myeloma had returned a lot quicker than expected meaning she now has to undergo a second transplant from a mystery donor.
They then discovered Judith had sepsis and MRSA and having no immune system and blood cancer, Judith said she was the sickest she had ever been.
She continued: They were hoping I would make it 18 months post transplant but they discovered in August that the cancer had returned and it had only worked for seven months.
Thats when we found out that they wouldnt be able to use my own cells again because it wasnt worth putting me through all that again.
So now Ill be going back on chemo in January and getting a transplant from a worldwide donor. Thankfully the transplant team has already found a match for me on the system.
Judith continued: Im really lucky that theres a match out there for me. But there are so many others, who are a lot sicker than I am, that dont have theirs yet.
The reason I wanted to speak out is to raise awareness of myeloma and stem cell donation.
You really could be giving someone a second chance at life by spitting into a tube. Back in the day it was a bone marrow transplant but now its stem cells.
Its no different from giving blood. I would just ask everyone to go have a look into it and see if they want to or are able to register.
Judith, who lives with her two sons and partner Steven (46), added: I may not be able to do some of the things I did before like go to the cinema with the boys but Im still here.
And I hope to be here long enough to see my grandkids. I know Ill keep fighting after that to see them grow up then. But for now, its just taking each day as it comes.
To find out more about stem cell donation for those aged under 30 visit https://www.anthonynolan.org/.
Those over 30 can visit https://www.dkms.org.uk/en.
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Brave West Lothian women discovers back pain is actually deadly blood cancer - Daily Record
Orchard Therapeutics Announces 2021 Corporate Priorities Supporting the Build-out of its Commercial Business in Hematopoietic Stem Cell (HSC) Gene…
By daniellenierenberg
Preparations on Track for First Half 2021 Commercial Launch of Libmeldy (OTL-200), the First Approved Product for Metachromatic Leukodystrophy (MLD) in the EU
Filing Strategy for OTL-200 Biologics License Application (BLA) in MLD in the U.S. to be Communicated by Mid-2021 Following Additional Regulatory Interactions
Marketing Authorization Application (MAA) Filing for OTL-103 in Wiskott-Aldrich Syndrome (WAS) on Track for Year End 2021 in the EU; Followed by BLA Filing in 2022 in the U.S.
New Clinical Data for OTL-203 (for MPS-I) and OTL-201 (for MPS-IIIA) Accepted for Oral Presentation at February 2021 WORLD Symposium; Preclinical Data from Research Programs in Larger Indications Expected in 2021
$192M in Cash and Investments to Support Strategic Execution into the First Half of 2022
BOSTONandLONDON, Jan. 11, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today outlined the companys 2021 strategic priorities in advance of its attendance at the virtual 39thAnnual J.P. Morgan Healthcare Conference. These priorities support the companys plan of building a successful commercial business in HSC gene therapy and advancing its portfolio of investigational medicines for high-value, high-need indications.
In a year that challenged how we live and work, Im extremely proud of Orchards achievements in 2020, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. Our accomplishments were a direct result of the drive and innovation that fuels our commitment to bring our potentially life-saving HSC therapies to patients, including Libmeldy, which is the first product approved for the treatment of eligible patients with early-onset MLD in the EU. With the HSC approach to gene therapy as our scientific foundation, we are focused on the capabilities that can deliver our therapies on a global commercial scale and support our ability to also treat larger indications over time. It has been a privilege to be a pioneer in changing the way medicine is practiced in these conditions, and we look forward to another year of continued execution and scientific progress.
2021 Corporate PrioritiesOrchard has outlined the following key corporate objectives and expected milestones for 2021:
In preparation for a European launch, Orchard has put in place the commercial infrastructure to support Libmeldy as well as future product launches. The company is qualifying five treatment centers in the UK, Germany, Italy, France and the Netherlands with specialized expertise in transplant and disease area knowledge. In addition, the company expects to leverage cross-border and treatment abroad reimbursement pathways in both Europe and markets such as the Middle East and Turkey. Activities are also underway to drive timely MLD patient identification and access, including disease awareness, genetic testing and newborn screening studies, which have started or are on track to initiate in five countries in 2021.
The company also provided an update concerning the impact of the COVID-19 pandemic on certain development activities. These include restrictions to laboratory access at Orchard and third-party service providers, which is impacting the timeline to develop a specific functional potency assay for OTL-103 in WAS, as requested by the FDA. As a result, the company now expects to file a BLA for OTL-103 in the U.S. in 2022. Orchard is utilizing the benefits provided under OTL-103s RMAT designation and plans to continue interacting with the FDA in 2021 to confirm the data package for the BLA filing. In addition, with several of the follow-up visits associated with the companys active clinical trials impacted by COVID-19 travel restrictions and other trial site limitations, Orchard is using alternative data collection approaches to capture the necessary data to support future regulatory filings.
Frank Thomas, president and chief operating officer continued, Starting 2021 with a clear set of strategic priorities is crucial to our ability to effectively manage the business while fueling Orchards continued growth. Our launch preparations for Libmeldy not only mark our evolution towards a fully integrated company but establish a common manufacturing, commercial and operational infrastructure to support multiple future potential products. This work is complemented by our exciting proof-of-concept and research pipeline that we look forward to advancing internally or in partnership.
Key 2020 AchievementsOrchards key 2020 achievements are highlighted below.
Cash GuidanceThe company ended 2020 with approximately $192 million of cash and investments. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2020 will enable the funding of its currently anticipated operating expenses and capital expenditure requirements into the first half of 2022. This excludes the $50 million expected to be available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers.
About Libmeldy / OTL-200
Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.
The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.
For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.
Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.
Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
About Orchard
Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Ourex vivoautologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy inMilan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.
Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on TwitterandLinkedIn.
Availability of Other Information About Orchard
Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.
Forward-Looking Statements
This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2022. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.
Contacts
InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com
MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com
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Orchard Therapeutics Announces 2021 Corporate Priorities Supporting the Build-out of its Commercial Business in Hematopoietic Stem Cell (HSC) Gene...
Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2027 – LionLowdown
By daniellenierenberg
Stem Cell Therapy Market is expected to reach 202.77 billion by 2027 from XX billion in 2019 at CAGR of XX %.
The report includes the analysis of impact of COVID-19 lock-down on the revenue of market leaders, followers, and disrupters. Since lock down was implemented differently in different regions and countries, impact of same is also different by regions and segments. The report has covered the current short term and long term impact on the market, same will help decision makers to prepare the outline for short term and long term strategies for companies by region.
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Stands for use of stem cells to treat or prevent disease or condition.Bone marrow transplant and some therapies derived from umbilical cord blood are mainly used in stem cell therapy. Advancement, in order to establish new sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions such as diabetes, heart disease, and other conditions, are increased in recent years.
Stem Cell Therapy Market Researchers are making efforts to discover novel methods to create human stem cells. This will increase the demand as well as supply for stem cell production and potential investigation in disease management. Increasing investment & research grants for developing safe and effective stem cell therapy products, the growing patient base for target diseases, concentrated product pipelines, increasing approval of the new clinical trials, rapid technological advancement in genomics, and the rising awareness about the stem cell are expected to drive the growth of the Stem Cell Therapy solutions market during the forecast period.
However, improper infrastructure, insufficient storage systems, nascent technology in underdeveloped economies, Ethical issues related to an embryonic stem cell, low patient acceptance rate, Difficulty in the preservation of stem cell are expected to restrain the market growth. North America is expected to be the largest growing region by 2026; the reason behind that is extensive funding by Government. However, Emerging countries like India, china, Korea have low growth rate as compared to Developed regions in 2017 but increase in awareness about stem cell therapy will lead the Asia Pacific to generate a significant level of revenue by 2026.
Key Highlights of Stem Cell Therapy Market report
Detailed quantitative analysis of the current and future trends from 2017 to 2026, which helps to identify the prevailing market opportunities.Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, core competencies in terms of market development, growth strategies and identification of key companies that can influence this market on a global and regional scale.Assessment of Market definition along with the identification of key drivers, restraints opportunities and challenges for this market during the forecast period.Complete analysis of micro-markets with respect to individual growth trends, prospects, and contributions to the overall Stem Cell Therapy Solutions market.Stem Cell Therapy market analysis and comprehensive segmentation with respect to the Application, End users, Treatment, and geography to assist in strategic business planning.Stem Cell Therapy market analysis and forecast for five major geographies-North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key regions.For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.
Research Methodology:
The market is estimated by triangulation of data points obtained from various sources and feeding them into a simulation model created individually for each market. The data points are obtained from paid and unpaid sources along with paid primary interviews with key opinion leaders (KOLs) in the market. KOLs from both, demand and supply side were considered while conducting interviews to get an unbiased idea of the market. This exercise was done at a country level to get a fair idea of the market in countries considered for this study. Later this country-specific data was accumulated to come up with regional numbers and then arrive at a global market value for the stem cell therapy market.Key Players in the Stem Cell Therapy Market are:
Chiesi Farmaceutici S.P.A Are:Gamida CellReNeuron Group, plcOsiris Therapeutics, Inc.Stem Cells, Inc.Vericel Corporation.Mesoblast, Ltd.
Key Target Audience:
Stem Cell Associations and OrganizationsGovernment Research Boards and OrganizationsResearch and consulting firmsStem Cell Therapy Market InvestorsHealthcare Service Providers (including Hospitals and Diagnostic Centers)Stem Cell Therapeutic Product Manufacturing OrganizationsResearch LabsClinical research organizations (CROs)Stem Cell Therapy Marketing PlayersPharmaceutical Product Manufacturing Companies
DO INQUIRY BEFORE PURCHASING REPORT HERE: https://www.maximizemarketresearch.com/inquiry-before-buying/522
Scope of the Stem Cell Therapy Market Report:
Stem Cell Therapy market research report categorizes the Stem Cell Therapy market based on Application, End users, Treatment, and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Stem Cell Therapy market with key developments in companies and market trends.Stem Cell Therapy Market, By Treatments:
Allogeneic Stem Cell TherapyAutologous Stem Cell Therapy
Stem Cell Therapy Market, By End Users:
HospitalsAmbulatory Surgical Centers
Stem Cell Therapy Market, By Application:
OncologyCentral Nervous System DiseasesEye DiseasesMusculoskeletal DiseasesWound & InjuriesMetabolic DisordersCardiovascular DisordersImmune System DisordersStem Cell Therapy Market, By Geography:
North AmericaEuropeAsia PacificMiddle East & AfricaLatin America
Available Customization:
With the given market data, Maximize Market Research offers customization of report and scope of the report as per the requirement
Regional Analysis:
Breakdown of the North America stem cell therapy marketBreakdown of the Europe stem cell therapy marketBreakdown of the Asia Pacific stem cell therapy marketBreakdown of the Middle East & Africa stem cell therapy marketBreakdown of the Latin America stem cell therapy market
MAJOR TOC OF THE REPORT
Chapter One: Stem Cell Therapy Market Overview
Chapter Two: Manufacturers Profiles
Chapter Three: Global Stem Cell Therapy Market Competition, by Players
Chapter Four: Global Stem Cell Therapy Market Size by Regions
Chapter Five: North America Stem Cell Therapy Revenue by Countries
Chapter Six: Europe Stem Cell Therapy Revenue by Countries
Chapter Seven: Asia-Pacific Stem Cell Therapy Revenue by Countries
Chapter Eight: South America Stem Cell Therapy Revenue by Countries
Chapter Nine: Middle East and Africa Revenue Stem Cell Therapy by Countries
Chapter Ten: Global Stem Cell Therapy Market Segment by Type
Chapter Eleven: Global Stem Cell Therapy Market Segment by Application
Chapter Twelve: Global Stem Cell Therapy Market Size Forecast (2019-2026)
Browse Full Report with Facts and Figures of Stem Cell Therapy Market Report at: https://www.maximizemarketresearch.com/market-report/stem-cell-therapy-market/522/
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Maximize Market Research provides B2B and B2C market research on 20,000 high growth emerging technologies & opportunities in Chemical, Healthcare, Pharmaceuticals, Electronics & Communications, Internet of Things, Food and Beverages, Aerospace and Defense and other manufacturing sectors.
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Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2027 - LionLowdown
AGC Biologics Confirms Cell and Gene Therapy Commercial Expertise as Manufacturer of Orchard Therapeutics’ Newly Approved Libmeldy(TM) – Daily…
By daniellenierenberg
SEATTLE, Jan. 4, 2021 /PRNewswire/ -- AGC Biologics, a leading global Biopharmaceutical Contract Development and Manufacturing Organization (CDMO), is the first manufacturer of Orchard Therapeutics' Libmeldy, which was recently approved by the European Commission (EC) as a one-time therapy for eligible patients with early-onset Metachromatic Leukodystrophy (MLD).
The EC has granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy manufactured at AGC Biologics' Milan facility. Orchard Therapeutics is already underway with EU launch preparations to support commercial-scale drug manufacturing at the AGC Biologics Milan facility.
"AGC Biologics is delighted to have successfully partnered with Orchard Therapeutics on this great milestone," says AGC Biologics Chief Business Officer, Mark Womack. "We're very proud to be one of few Cell and Gene Therapy CDMOs with commercial experience Libmeldy being the third commercial product we've manufactured and we look forward to continuing our partnership with Orchard to provide this life-changing therapy to those affected by MLD."
"We are very honored to have been a part of the entire clinical journey of this product, from the very first treated patient, all the way to market approval. As manufacturer of both lentiviral vector and drug product, we are proud to support Orchard in treating this devastating disease," says AGC Biologics General Manager of Milan, Luca Alberici.
"The EC approval of Libmeldy opens up tremendous possibilities for eligible MLD children faced with this devastating disease," saysBobby Gaspar, M.D., Ph.D., Chief Executive Officer of Orchard. "We are humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU, and confident in the capabilities of our partners at AGC Biologics to help us achieve this goal."
MLD is a rare neurodegenerative disorder caused by mutations in the ARSA gene. While there are several forms of MLD, the disorder primarily affects young children and disease progression and life expectancy varies based on age of symptom onset. Libmeldy is designed to correct the genetic cause of MLD by inserting functional copies of the ARSA gene into the genome of a patient's own hematopoietic stem cells (HSCs) using a self-inactivating (SIN) lentiviral vector. It is approved in the EU for children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.
About AGC Biologics: AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to deliver the highest standard of service as we work side-by-side with our clients and partners, every step of the way. We provide world-class development and manufacture of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), viral vectors and genetically engineered cells. Our global network spans the U.S., Europe and Asia, with cGMP-compliant facilities in Seattle, Washington; Boulder, Colorado; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba, Japan and we currently employ more than 1,600 employees worldwide. Our commitment to continuous innovation fosters the technical creativity to solve our clients' most complex challenges, including specialization in fast-track projects and rare diseases. AGC Biologics is the partner of choice. To learn more, visit http://www.agcbio.com.
About Orchard Therapeutics:Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK's rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.
Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us onTwitterandLinkedIn.
About OTL-200/Libmeldy:Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union (as appropriate may add: "UK, Iceland, Liechtenstein and Norway" as an alternative can rewrite as "was approved by the European Commission" or something similar) for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.
The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA). In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.
For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC).
Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy, which has not been approved by the U.S. Food and Drug Administration or any other health authority.
OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
MorphoSys and Incyte Announce the Acceptance of the Swissmedic Marketing Authorization Application f – PharmiWeb.com
By daniellenierenberg
DGAP-News: MorphoSys AG / Key word(s): Miscellaneous05.01.2021 / 08:00 The issuer is solely responsible for the content of this announcement.
Media Release
MorphoSys and Incyte Announce the Acceptance of the Swissmedic Marketing Authorization Application for Tafasitamab
- The Swissmedic MAA seeks approval for tafasitamab in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma
PLANEGG/MUNICH, Germany and MORGES, Switzerland - January 5, 2021 - MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ:MOR) and Incyte (NASDAQ:INCY) today announced that the Swiss Agency for Therapeutic Products (Swissmedic) has accepted the marketing authorization application (MAA) for tafasitamab, a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. The MAA seeks approval for tafasitamab, in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from low grade lymphoma, who are not candidates for autologous stem cell transplantation (ASCT). The MAA will now enter the formal review process by Swissmedic.
The Swissmedic MAA for tafasitamab will be reviewed as part of the U.S. Food and Drug Administration's (FDA) modified Project Orbis, which provides a framework for concurrent submission and review of oncology drug applications among the FDA's international collaborators. Collaboration among international regulators may allow patients with cancer to receive earlier access to products in other countries.
"Currently about 40% of DLBCL patients do not respond to initial therapy or relapse thereafter leading to a high medical need for new, effective therapies," said Peter Langmuir, M.D., Group Vice President, Targeted Therapeutics, Incyte. "The acceptance of the MAA for tafasitamab for review by Swissmedic is a pivotal step towards bringing tafasitamab in combination with lenalidomide to eligible patients in Switzerland."
"Tafasitamab in combination with lenalidomide may represent an important new targeted treatment option for patients with relapsed or refractory DLBCL," said Mike Akimov, M.D., Ph.D., Head of Global Clinical Development, MorphoSys. "We look forward to continuing to work with the regulatory authorities alongside our partners at Incyte to bring this novel therapeutic option to eligible patients with a high unmet medical need."
The Swissmedic application, submitted by Incyte in collaboration with MorphoSys, is supported by data from the L-MIND study evaluating tafasitamab in combination with lenalidomide as a treatment for patients with relapsed or refractory DLBCL and data from the RE-MIND study, an observational retrospective study in relapsed or refractory DLBCL. If approved, Incyte will hold the marketing authorization, and have exclusive commercialization rights for tafasitamab in Switzerland.
Incyte has exclusive commercialization rights for tafasitamab outside the United States.
About Diffuse Large B-cell Lymphoma (DLBCL)DLBCL is the most common type of non-Hodgkin lymphoma in adults worldwide[1], characterized by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs. It is an aggressive disease with about 40% of patients not responding to initial therapy or relapsing thereafter[2]. In Europe, each year approximately 16,000 patients are diagnosed with relapsed or refractory DLBCL[3],[4],[5].
About L-MINDThe L-MIND trial is a single arm, open-label, multicenter Phase 2 study (NCT02399085) investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who have had at least one, but no more than three prior lines of therapy, including an anti-CD20 targeting therapy (e.g. rituximab), who are not eligible for high-dose chemotherapy or refuse subsequent autologous stem cell transplant. The study's primary endpoint is Overall Response Rate (ORR). Secondary outcome measures include Duration of Response (DoR), Progression-Free Survival (PFS) and Overall Survival (OS). In May 2019, the study reached its primary completion.
For more information about L-MIND, visit https://clinicaltrials.gov/ct2/show/NCT02399085
About RE-MINDRE-MIND, an observational retrospective study (NCT04150328), was designed to isolate the contribution of tafasitamab in combination with lenalidomide and to prove the combinatorial effect. The study compares real-world response data of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who received lenalidomide monotherapy with the efficacy outcomes of the tafasitamab-lenalidomide combination, as investigated in MorphoSys' L-MIND trial. RE-MIND collected the efficacy data from 490 relapsed or refractory DLBCL patients in the U.S. and the EU. Qualification criteria for matching patients of both studies were pre-specified. As a result, 76 eligible RE-MIND patients were identified and matched 1:1 to 76 of 80 L-MIND patients based on important baseline characteristics. Objective Response Rates (ORR) were validated based on this subset of 76 patients in RE-MIND and L-MIND, respectively. The primary endpoint of RE-MIND was met and shows a statistically significant superior best ORR of the tafasitamab-lenalidomide combination compared to lenalidomide monotherapy.
For more information about RE-MIND, visit https://clinicaltrials.gov/ct2/show/NCT04150328.
About TafasitamabTafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).
Monjuvi(R) (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration (FDA) in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.
Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.
Monjuvi(R) is a registered trademark of MorphoSys AG.
XmAb(R) is a registered trademark of Xencor, Inc.
About MorphoSysMorphoSys (FSE & NASDAQ: MOR) is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of exceptional, innovative therapies for patients suffering from serious diseases. The focus is on cancer. Based on its leading expertise in antibody, protein and peptide technologies, MorphoSys, together with its partners, has developed and contributed to the development of more than 100 product candidates, of which 27 are currently in clinical development. In 2017, Tremfya(R), developed by Janssen Research & Development, LLC and marketed by Janssen Biotech, Inc., for the treatment of plaque psoriasis, became the first drug based on MorphoSys' antibody technology to receive regulatory approval. In July 2020, the U.S. Food and Drug Administration (FDA) granted accelerated approval of MorphoSys' proprietary product Monjuvi(R) (tafasitamab-cxix) in combination with lenalidomide in patients with a certain type of lymphoma.
Headquartered near Munich, Germany, the MorphoSys group, including the fully owned U.S. subsidiary MorphoSys US Inc., has ~500 employees. More information at http://www.morphosys.com or http://www.morphosys-us.com.
Monjuvi(R) is a registered trademark of MorphoSys AG.
Tremfya(R) is a registered trademark of Janssen Biotech, Inc.
About Incyte Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.
MorphoSys Forward-looking Statements This communication contains certain forward-looking statements concerning the MorphoSys group of companies, including the expectations regarding Monjuvi's ability to treat patients with relapsed or refractory diffuse large B-cell lymphoma, the further clinical development of tafasitamab-cxix, including ongoing confirmatory trials, additional interactions with regulatory authorities and expectations regarding future regulatory filings and possible additional approvals for tafasitamab-cxix as well as the commercial performance of Monjuvi. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "would," "could," "potential," "possible," "hope" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The forward-looking statements contained herein represent the judgment of MorphoSys as of the date of this release and involve known and unknown risks and uncertainties, which might cause the actual results, financial condition and liquidity, performance or achievements of MorphoSys, or industry results, to be materially different from any historic or future results, financial conditions and liquidity, performance or achievements expressed or implied by such forward-looking statements. In addition, even if MorphoSys' results, performance, financial condition and liquidity, and the development of the industry in which it operates are consistent with such forward-looking statements, they may not be predictive of results or developments in future periods. Among the factors that may result in differences are MorphoSys' expectations regarding risks and uncertainties related to the impact of the COVID-19 pandemic to MorphoSys' business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products, the global collaboration and license agreement for tafasitamab, the further clinical development of tafasitamab, including ongoing confirmatory trials, and MorphoSys' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials, additional interactions with regulatory authorities and expectations regarding future regulatory filings and possible additional approvals for tafasitamab-cxix as well as the commercial performance of Monjuvi, MorphoSys' reliance on collaborations with third parties, estimating the commercial potential of its development programs and other risks indicated in the risk factors included in MorphoSys' Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. MorphoSys expressly disclaims any obligation to update any such forward-looking statements in this document to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements, unless specifically required by law or regulation.
Incyte Forward-looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether or when tafasitamab might be approved in Switzerland for the treatment of, and whether or when tafasitamab might provide a successful treatment option for, in combination with lenalidomide, certain patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), and the L-MIND and RE-MIND clinical trial programs. These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by European regulatory authorities or other regulatory authorities, including the U.S. FDA; the Company's dependence on its relationships with its collaboration partners; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in the Company's reports filed with the Securities and Exchange Commission, including its Form 10-Q for the quarter ending September 30, 2020. The Company disclaims any intent or obligation to update these forward-looking statements. # # #
Contacts:
References[1] Sarkozy C, et al. Management of relapsed/refractory DLBCL. Best Practice Research & Clinical Haematology. 2018 31:209-16. doi.org/10.1016/j.beha.2018.07.014.[2] Skrabek P, et al. Emerging therapies for the treatment of relapsed or refractory diffuse large B cell lymphoma. Current Oncology. 2019 26(4): 253-265. doi.org/10.3747/co.26.5421.[3] DRG Epidemiology data.[4] Kantar Market Research (TPP testing 2018).[5] Friedberg, Jonathan W. Relapsed/Refractory Diffuse Large B-Cell Lymphoma. Hematology Am Soc Hematol Educ Program 2011; 2011:498-505. doi: 10.1182/asheducation-2011.1.498.
05.01.2021 Dissemination of a Corporate News, transmitted by DGAP - a service of EQS Group AG.The issuer is solely responsible for the content of this announcement.
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MorphoSys and Incyte Announce the Acceptance of the Swissmedic Marketing Authorization Application f - PharmiWeb.com
Direct Biologics Announces First Patient Treated Under Phase II Expanded Access Protocol Using ExoFlo(TM) to Treat COVID-19 – Tyler Morning Telegraph
By daniellenierenberg
AUSTIN, Texas, Dec. 30, 2020 /PRNewswire/ --Direct Biologics, LLC, announced today that the first patient has been treated under its expanded access protocol for ExoFlo Infusion Treatment for COVID-19 Associated ARDS (EXIT COVID-19).
"The Phase II trial is actively enrolling patients at several sites nationally. It has met several key milestones and is nearing completion," states Co-Founder and Chief Executive Officer, Mark Adams. "We look forward to sharing our results and hopefully proceeding to Phase III very soon."
The expanded access protocol is an open label study to treat patients who often have more advanced disease under a "compassionate use" application.
"The expanded access gives us an opportunity to treat very sick patients who do not have other meaningful options," notes Joe Schmidt, Co-Founder and President. "It also enhances our ability to treat patients at additional hospitals and therefore communities in need across the country."
Chief Medical Officer, Vik Sengupta, MD, adds, "We at Direct Biologics are grateful for every opportunity to help these additional patients in critical need of treatment."
This study is under the same IND application utilizing bone marrow-derived extracellular vesicles to treat COVID-19-associated ARDS. Extracellular vesicles secreted by bone marrow-derived mesenchymal stem cells (bmMSCs) have been studied extensively in preclinical studies of lung disease and inflammation and are notable for their ability to downregulate inflammation and upregulate tissue repair.
Physicians can learn more at clinicaltrials.gov and may request access for a patient by emailing msl@directbiologics.com. Requests for expanded access to ExoFlo must be made by a licensed U.S. treating physician.
About ExoFlo
ExoFlo is an investigational new drug that has not been approved or licensed by the FDA. It is an extracellular vesicle product isolated from human bone marrow mesenchymal stem or stromal cells (MSCs). ExoFlo provides natural bioactive signals that have been shown to modulate inflammation and direct cellular communication.
About Direct Biologics
Direct Biologics, LLC, is headquartered in Austin, Texas, with a recently expanded R&D facility located at the University of California, and an Operations and Order Fulfillment Center located in St. Louis, Missouri. Direct Biologics is a market-leading innovator and cGMP manufacturer of regenerative medical products, including a robust line of extracellular vesicle-based biological products. The Company was created to expand the science of regenerative healing by delivering cutting-edge biologic technologies. Direct Biologics' management team holds extensive collective experience in biologics research, development, and commercialization, making the Company a leader in the evolving, next-generation segment of the biotherapeutics industry. Direct Biologics is dedicated to pursuing additional clinical applications of its extracellular vesicle biologic products through the FDA's investigational new drug application process. For more information, visithttp://www.directbiologics.com.
Phone:1-800-791-1021Email:info@directbiologics.com
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Direct Biologics Announces First Patient Treated Under Phase II Expanded Access Protocol Using ExoFlo(TM) to Treat COVID-19 - Tyler Morning Telegraph
Lonza’s Cocoon will soon make dozens of stem cell therapies all at once – Innovation Origins
By daniellenierenberg
The name Lonza comes from the river in Switzerland near where the company was originally founded more than a hundred years ago, As such, it has nothing to do with the biomedical products and services that Lonza provides.
Lonza is active around the world and has around 15,000 employees with branches in the Netherlands as well. Such as the one on the BrightlandsChemelot Campus in Geleen. where approximately 250 people work there. This company division started as a start-up in Maastricht in 2005 and developed a production facility for stem cell and gene therapy to combat diseases. It became part of Lonza in 2018.
The reason that Lonza is on the Chemelot campus is due to its strategic location. This is very convenient for having access to all kinds of industry-related services that are already available there, says Willem Dullaers. He is the senior manager of quality control at Lonza in Geleen. Security is well organized, and we use a number of other facilities so that as a company you dont need to arrange these yourself.
The interesting thing about the Lonza production facility in Geleen is that the company isolates living cells and is even able to manipulate them on behalf of pharmaceutical groups. These companies then supply them to hospitals for the treatment of patients, primarily those suffering from forms of cancer. So what does Lonza do exactly and what does it ultimately deliver to those hospitals? is the question for Dullaers. This is not so easy to explain.
We work in cleanrooms at Lonzas premises in Geleen with the aim of selecting body cells from a sample of the patient taken in the hospital via a blood transfusion or bone marrow puncture. For example, body cells that are selected have the ability to fight cancer cells by virtue of their specific properties. If these cells are selected and reproduce in number after being cultured, it can be useful to add DNA to them so that they are able to attack the cancer cells even more effectively. Modification is done using a piece of deactivated virus that is used as a vector to introduce DNA into the selected cells.
Once that process is completed, the number of cells, which is usually very small, is cultivated to a larger quantity so that after various quality checks and the preparation for transport (cooled or frozen) are carried out, the cells are introduced into the patient. It is very common that patients are successfully treated afterward, says Dullaers. He refers to a report that made the world news last year. An Italian two-year-old boy with a rare immune disease (HLH) who was initially given up by doctors, Alex Montresor, was cured after stem cell therapy.
About 30 people are currently working on the production process for cell and gene therapy to treat people, Dullaers adds. The tasks that the biomedical doctors have to perform take time and require careful attention. They have to enter the cleanroom themselves to put the cells through the process to be transformed into stem cell therapies. They have to wear protective, hermetically sealed suits under the strictest safety conditions. That is to safeguard their own safety but also to prevent any potential contamination of the cells. Patients for whom this therapy is intended are often severely debilitated. They are not allowed to get sick as a result of a bacterium or particulate matter that has entered the cultured cells. A check always takes place to make sure that the product is completely clean. If this is not the case, it must be remade as a last resort.
At the moment, Lonza is working on a method to fully automate the culture process of the cells in the cleanroom. A pilot is currently underway at the Sheba Medical Center in Israel. It has a test setup with a so-called cocoon. The cocoon looks like an egg-shaped module of white plastic that is about one meter long. Inside the egg there is a small factory that automates all operations, from cell selection to DNA insertion and preparation for transport and administration.
Over the coming years, this innovative culture method for cell and gene therapy must be approved through clinical trials and by medicinal regulatory agencies such as the U.S. FDA and the European EMA. Only then can this robotized method be applied on a large scale.
I hope it will be achievable within five to ten years, says Dullaers. That will change a lot in terms of affordability and supply options for cell and gene therapy. Because it is such a cumbersome treatment, the costs are high right now. The production also takes a lot of time. Depending on the complexity of the process, the duration varies from a few days to sometimes more than two months, Dullaers notes.
If the entire process can be robotized, fewer people will be needed to do the work. I think that whereas we now work with 150 people, you will be able to do it with 15. However, you will need a different set of employees: People with a software background and an understanding of the machinery.
You can simultaneously fill a room with dozens of cocoons where cell therapies are made. That means that productivity is bound to skyrocket. Consequently, it will also be possible to make more medication based on the cells of individual patients, which will also be cheaper since less staff is needed. The chance of making mistakes is smaller than with work that involves human hands, Dullaers points out.
Another alternative is for hospital laboratories to make the gene and cell therapies themselves. It is conceivable that they would like to have a cocoon in their own hospital that they can use to treat patients.
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Lonza's Cocoon will soon make dozens of stem cell therapies all at once - Innovation Origins
Bone Marrow Processing Systems Market to Make Great Impact in near Future by -2025 – The Monitor
By daniellenierenberg
Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.
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The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.
Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.
In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.
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Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.
Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.
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Bone Marrow Processing Systems Market to Make Great Impact in near Future by -2025 - The Monitor
The Myeloproliferative Disorders Drugs Market to grow on an exceptional note in the next 10 years – LionLowdown
By daniellenierenberg
Myeloproliferative disorders are disease of blood and bone marrow which have unknown cause and there are wide range of symptoms. The treatment of myeloproliferative disorders generally depends on the type and presence of symptoms. Myeloproliferative disorders is generally considered as clonal disorder which begins with one or more change in the DNA of a single stem cells in the bone marrow. The changes to the hematopoietic stem cell cause the cell to reproduce repeatedly, creating more abnormal stem cells and these abnormal cells become one or more types of blood cells. Myeloproliferative disorders gets worst with time as the number of extra blood cells build up in the bone marrow and bloodstream.
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Emergence of new treatment for the myeloproliferative disorders and availability of novel drug drive the market for myeloproliferative disorders drugs market in the near future. Rising incidence of myeloproliferative disorders and presence of strong product pipeline spur the myeloproliferative disorders drugs market. Growing geriatric population, change in lifestyle and growing awareness among general population is expected to drive the market of myeloproliferative disorders in the forecast period. Advancement in the treatment for oncology further expand the treatment option for myeloproliferative disorders. Various clinical trial undergoing for the treatment of myeloproliferative disorders which further drive the growth of the myeloproliferative disorders drugs market. However, high cost of drug and treatment along with the lack of awareness among the population in developing and under developed nations hinder the growth of myeloproliferative disorders drugs market.
The global myeloproliferative disorders drugs market is segmented on basis of Type, Drug Type, Distribution Channel, End User and Geography.
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Improvement in the symptoms and reduction of in splenomegaly among patients receiving available therapy is expected to boost the market of myeloproliferative disorders. Development in new therapeutic drug and target therapy further drive the market growth of myeloproliferative disorders. Increased research and development and increased funding by the government towards the development of novel therapy spur the market growth. With the discovery of specific gene mutations in myeloproliferative disorders the market is expected to grow in the forecast period owing to increased adoption of new drugs and increased awareness along with the favorable reimbursement scenarios for the treatment of myeloproliferative disorders.
The North America market holds the largest revenue share for myeloproliferative disorders drugs, due to presence of major pharmaceutical players undergoing various clinical innovation, government initiative and increase research and development funding for the Myeloproliferative disorders. Europe is expected to contribute for the second largest revenue share after North America in the global myeloproliferative disorders drugs market, owing to merging treatment option and development of oncology drug discovery and rising prevalence of myeloproliferative disorders. Asia Pacific is expected to show rapid growth, due to increasing number of vascular surgeons and low cost of peripheral interventions. China is expected to register fast growth, due to significant increase in the number of innovative firm and research organization and increasing importance of pharmaceutical research & development activities and investments in research for developing new drugs. Latin America and Middle East & Africa are projected to exhibit sluggish growth in myeloproliferative disorders Drugs market, due to proper healthcare systems and adoption of new drug and therapy.
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Examples of some of the key manufacturer present in the global myeloproliferative disorders drugs market are
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The Myeloproliferative Disorders Drugs Market to grow on an exceptional note in the next 10 years - LionLowdown
2020: The year science took centre-stage – The Hindu
By daniellenierenberg
Apart from new findings on coronavirus every single day, the year was also filled with stories from outer space, archeology and anatomy
The year 2020 also termed as the year of the pandemic, social distancing, work from home, was also the year of research at breakneck speed. Virologists, immunologists, computational biologists, epidemiologists, and medical professionals across the globe turned into superheroes without capes.
Quick sequencing of the whole genome of the novel coronavirus (SARS-CoV-2) helped develop various test kits. We now have not one or two, but multiple COVID-19 vaccines that have succeeded in human clinical trials. Moderna's and Pfizer-BioNTechs vaccines that use messenger RNA have reported efficacy of about 95%, and the United Kingdom, the United States and the United Arab Emirates have already launched mass vaccinations.
Apart from new findings on coronavirus every single day, the year was also filled with stories from outer space, archeology and anatomy. Here is a list of a few of them in random order
In October, NASA confirmed, for the first time, water on the sunlit side of the Moon indicating that water may be distributed across the moons surface, and not limited to the cold and shadowed side.
Researchers from the Netherlands Cancer Institute announced in October that they have discovered a new pair of salivary glands hidden between the nasal cavity and throat. The team proposed the name tubarial glands and noted that this identification could help to explain and avoid radiation-induced side-effects such as trouble during eating, swallowing, and speaking.
In September, an international scientific team announced that they have spotted phosphine gas on Venus. On Earth, microorganisms that live in anaerobic (with no oxygen) environments produce phosphine. Massachusetts Institute of Technology molecular astrophysicist and study co-author Clara Sousa-Silva said in a release, This is important because, if it is phosphine, and if it is life, it means that we are not alone. It also means that life itself must be very common, and there must be many other inhabited planets throughout our galaxy.
Read our detailed explainer here.
In March, a person suffering from Leber congenital amaurosis, a rare inherited disease that leads to blindness, became the first to have CRISPR/Cas-9-based therapy directly injected into the body.
In June, two patients with beta-thalassemia and one with sickle cell disease had their bone marrow stem cells edited using CRISPR techniques.
Click here to read our explainer on the genome-editing tool that won this years Nobel Prize for Chemistry.
The year 2020 marks 100 years of discovery of Indus Valley Civilisation, and a new study showed that dairy products were being produced by the Harappans as far back as 2500 BCE.
Another study found the presence of animal products, including cattle and buffalo meat, in ceramic vessels dating back about 4,600 years.
Chinas Change-5 probe brought back about 1,731 grams of samples from the moon becoming the third country to bring moon samples after the U.S and Soviet Union.
Also, Japans Hayabusa 2 brought back the first extensive samples from an asteroid. The spacecraft, launched from Japan's Tanegashima space centre in 2014, took four years to reach the asteroid Ryugu before taking a sample and heading back to Earth in November 2019.
Mars rover Perseverance blasted off for the red planet on July 30 to bring the first Martian rock samples back to Earth. If all goes well, the rover will descend to the Martian surface on February 18, 2021.
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2020: The year science took centre-stage - The Hindu
Stem cells from cord blood can now be used across many conditions: Mayur Abhaya, MD & CEO, LifeCell Internat.. – ETHealthworld.com
By daniellenierenberg
Shahid Akhter, editor, ETHealthworld, spoke to Mayur Abhaya, MD & CEO, LifeCell International, to know more about the latest advancements in Stem cell industry and how it has recovered from the Covid challenges.Impact and challenges of Covid-19 on the Stem cell industry ?One of the biggest issues faced by the stem cell industry during the pandemic was the transport of the cells after collection at birth. It needs to reach the lab within 72 hours and in the case of bone marrow stem cell from donation all the way until it reaches the patient that also has to be completed within 72 hours. The bone marrow cells cannot be handed over in courier. It has to be manually hand carried and that created a huge logistics hurdle were transplants significantly reduced in numbers because of the availability of donors and the transport issues around it.
What are the current global trends in the Stem Cell Industry at large?The recent clinical progress that has been made in the medical space of stem cell transplantation is that now cord blood is considered as a better source than bone marrow cells. This was found in a research study based in US, published in 2020. They have also shown that stem cells from the cord blood can now be used across many conditions with the same treatment protocol. Besides that, the preparation of the patient is different in different conditions but now they have simplified that and reduced the risk of death to a very, very low number. So the cord blood is preferred, as the outcomes are improving.How has Life Cell managed the scenario during the pandemic? Do let us know your challenges and the way forward plan?One of the biggest issues during the pandemic was transport, especially the flight operations because we heavily depend on them for moving the samples across the country. We had to revert to an alternate plan where we had to transport these samples through a relay network from one city to another, through a road network. Luckily LifeCell has operations across the country covering more than 250 cities. So still we had the ability to ensure that our commitment of getting the samples to the lab within those 72 hours was very much possible.
Another major milestone during this pandemic that we were able to help was to support a transplant were a child having Aplastic Anaemia needed not one but two cord blood units for the transplant and within the family they couldnt find a match. Luckily because of the LifeCell network and the inventory size of 50,000 units we were able to meet the requirements of the transplant and happy to share the outcome was very successful. So LifeCell ensures that we have appropriate training for its paramedical staff and they are also provided with the appropriate personal protective gears. There are restrictions on the entry of the team inside during the collection we work with the medical staff in the collection rooms, in the operation theatres to ensure a smooth and a well organised collection and even at the lab we have protocols that ensures hygiene and safety within the team and, the operating rooms we have for processing are also well managed.
Your future plans to ensure the smooth collection of Cord Blood?To ensure business continuity we have our teams located very close to our lab itself, you know, so about 100+ member team are placed within a Kilometer of the operating facility. We have adequate stocks, lots of the testing and the processing, consumables that we use are imported. We, at least, maintain 3 month inventory. We also have onsite power back up systems which include a month of diesel supply, month of liquid nitrogen supply and the teams also have a plan that we have a back site also with arrangements done. If for any reason we have cut off of the Chennai centre we have arrangements with an alternate lab to ensure the continuity of the operations
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Stem cells from cord blood can now be used across many conditions: Mayur Abhaya, MD & CEO, LifeCell Internat.. - ETHealthworld.com
‘We had to take our health into our hands’ – Meet the siblings fighting for their lives with sickle cell disease – South West Londoner
By daniellenierenberg
Siblings Nabila Nakigozi and Fauzan Ssebaggala were diagnosed with sickle cell disease at birth, and with time running out they are taking action.
20-year old Nabila was also diagnosed with avascular necrosis the collapse of bones and as a result is losing her left hip.
She said: Sickle cell is very unpredictable. You can go from happy, to in pain and crying in a matter of minutes. Pain has stolen my childhood and now my adulthood.
Sickle cell disease (SCD) is a group of inherited red blood cell disorders. Those with SCD red blood cells are crescent or sickle shape. This can block blood flow through the body and can lead to many health problems including strokes and episodes of pain.
Fauzan, 24, is now disabled also due to avascular necrosis which has led to the collapse of his spine and hips.
He said: I spent last night crying in pain, every breath I take is painful.
There are two known cures for sickle cell, bone marrow and stem cell transplants.
After extensive research, the siblings have located a private doctor in India who specialises in sickle cell and are fundraising for the expensive but life- saving bone marrow transplant.
Nabila said: Discussions about this procedure is what should be happening within the NHS. We have waited for action but there is always an excuse.
On 11 November, The Parliaments Joint Committee released a report titled Black People, Racism and Human Rights.
The report found that more than 60% of Black people in the UK do not believe their health is as equally protected by the NHS compared to white people.
SCD commonly affects people of African and Caribbean descent.
Fauzan said: The NHS as a whole needs to re-evaluate its position on sickle cell because they can be very insensitive towards us.
There are assumptions that we are not really in pain and just addicted to morphine.
For Nabila, going through this with her brother makes every day easier.
She said: It might sound odd, but its probably the best thing that could have happened to both of us. We lean on each other for support.
But for Fauzan, seeing his sister suffer is difficult to watch.
He said: As an older brother, Im meant to be looking after her. Its heart-breaking knowing there are things I cant do for her.
Nabila is in hospital 80% of the time. I struggle looking into her eyes and confidently saying, tomorrow will be fine.
The east London duo are passionate about highlighting the pain those with SCD go through beyond the surface.
Nabila said: We were born in Uganda and within the African community it isnt always acknowledged that the disease affects us mentally too.
Fauzan added: Ive really struggled with my mental health, and I felt a lot of pressure as a result of not working.
Despite their struggles, the siblings hope to be a voice for those with SCD through their charitable fashion brand, Werent Born Rich.
They make streetwear clothing, with a portion of the profits going to sickle cell patients.
Nabila said: I want people to look at Werent Born Rich and be inspired. If I can be this sick and continue to fight, so can you.
Tracy Williams, project manager for blood donation at the Sickle Cell Society, has advocated through multiple projects for more Black and mixed heritage blood donors.
She manages two projects South London Gives and Give Blood, Spread Love.
Tracy said: Only 1% of people who give blood are of Black heritage. This number needs to increase to help those with sickle cell with Exchange Blood Transfusions.
We advocate for the patient voice, and of that of families affected by SCD. Our goal is to improve the overall wellbeing for people with sickle cell by accessing excellent treatment, mental health support and easily accessible ethnically matched blood.
Despite SCD being the fastest growing genetically inherited condition in the UK, research remains limited.
She said: We have seen positive changes however we would like to see more research for widely accessible treatments. We also continue to campaign for their exemption from prescription charges.
Williams message for the Black community is clear.
She said: Your blood is needed. Donating blood is safe and the process takes half an hour. Its your chance to literally save someones life.
If you are not from our target communities be an ally and share our message too.
New class of drugs to treat blood and bone marrow cancers: Research – Hindustan Times
By daniellenierenberg
A group of scientists in the US, including an Indian-American from the prestigious Cleveland Clinic, have identified a potential new class of drugs that may prove effective in treating certain common types of blood and bone marrow cancers.
First published in the latest edition of Blood Cancer Discovery, the decade long research which reports that a new pharmacological strategy to preferentially target and eliminate leukemia cells with TET2 mutations, was carried out by Jaroslaw Maciejewski and his collaborator Babal Kant Jha from the Cleveland Clinic Department of Translational Hematology & Oncology Research.
Myeloid leukemias are cancers derived from stem and progenitor cells in the bone marrow that give rise to all normal blood cells.
One of the most common mutations involved in driving myeloid leukemias are found in the TET2 gene, which has been investigated for the last decade by Maciejewski and Jha.
In preclinical models, we found that a synthetic molecule called TETi76 was able to target and kill the mutant cancer cells both in the early phases of disease--what we call clonal hematopoiesis of indeterminate potential, or CHIP--and in fully developed TET2 mutant myeloid leukemia, said Dr Maciejewski.
According to a media release, the research team designed TETi76 to replicate and amplify the effects of a natural molecule called 2HG (2-hydroxyglutarate), which inhibits the enzymatic activity of TET genes.
The TET DNA dioxygenase gene family codes for enzymes that remove chemical groups from DNA molecules, which ultimately changes what genes are expressed and can contribute to the development and spread of disease.
While all members of the TET family are dioxygenases, the most powerful enzymatic activity belongs to TET2, the press release said.
Even when TET2 is mutated, however, its related genes TET1 and TET3 provide residual enzymatic activity. While significantly less, this activity is still enough to facilitate the spread of mutated cancer cells.
Maciejewskis and Jhas new pharmacologic strategy to selectively eliminate TET2 mutant leukemia cells centers on targeting their reliance on this residual DNA dioxygenase activity, it said.
We took lessons from the natural biological capabilities of 2HG, explained Jha, a principal investigator.
We studied the molecule and rationally designed a novel small molecule, synthesized by our chemistry group headed by James Phillips, PhD. Together, we generated TETi76a similar, but more potent version capable of inhibiting not just TET2, but also the remaining disease-driving enzymatic activity of TET1 and TET3, Jha said.
Cleveland Clinic said that the researchers studied TETi76s effects in both preclinical disease and xenograft models (where human cancer cells are implanted into preclinical models). Additional studies will be critical to investigate the small molecules cancer-fighting capabilities in patients.
We are optimistic about our results, which show not just that TETi76 preferentially restricts the growth and spread of cells with TET2 mutations, but also gives survival advantage to normal stem and progenitor cells, Jha said.
(This story has been published from a wire agency feed without modifications to the text.)
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New class of drugs to treat blood and bone marrow cancers: Research - Hindustan Times
He got his cheek swabbed at 24. Nothing happened for 14 years. – Las Vegas Review-Journal
By daniellenierenberg
Cade Cridland thought a lot about fate as he sat tethered to a machine that drained blood from one arm and pumped it back into his body through the other arm.
After four hours, blood stem cells processed by the machine would be flown thousands of miles to a young child he has never met. A child whose name he does not know.
A child battling blood cancer.
This story unfolded in a Denver hospital in September. But it began 14 years earlier with a split-second decision in Las Vegas when Cridland was 24.
Before the child was born.
Im not a religious person by any means, Cridland, now 38, said. But I do believe that theres a lot of fate that takes place in our actions on a day-to-day basis.
The year is 2006. George W. Bush is president.
And Cridland, a recent UNLV graduate with a bachelors degree in journalism and media studies, had just left his part-time job with Vegas PBS for a full-time job with the local chapter of the Leukemia and Lymphoma Society.
Through his new job, Cridland soon found himself at a donation drive, hosted by a charity called Be the Match, for a family desperately searching for a bone marrow donor.
For patients diagnosed with leukemia or lymphoma, a bone marrow or cord blood transplant may be their only hope for a cure.
Cridland was there to work the event, but he drew inspiration from those around him getting swabbed. Whats the harm, he thought.
That day, his DNA was packaged and shipped off to be entered in the National Bone Marrow Registry.
He wasnt a match. Life went on, and eventually Cridland forgot about the swab.
In the meantime, he moved on to a job with the Clark County School District, got married, adopted a dog, had two children, bought a house, got another dog.
Fourteen years came and went, and now it was 2020.
The phone call
The call came at the best time, during arguably one of the worst years in modern history. The 2020 pandemic was in full swing. Protests over racism and police brutality had taken hold of a divided nation.
Cridland, a spokesman for the school district, needed something good to focus on.
But when that something good came calling earlier this year, he almost didnt answer. A toll-free number lit up the screen. A telemarketer maybe, or a scam?
Cridland surprised himself and took the call.
I dont know if you remember this, Cridland recalled a woman on the other end explaining. But you gave a cheek swab at one of our events, and theres a possibility that this cheek swab is a match for a child in need of stem cells or marrow to help them fight blood cancer.
The woman, an employee of Be the Match, ended the call with a question: Would you be willing to donate?
She gave Cridland the weekend to think it over. But for Cridland and his wife, who have two young children, it was a no-brainer.
Not long after, a second cheek swab confirmed what the woman had told Cridland during their phone call.
He was a match for the patient. In this case, that meant at least eight specific genetic markers in Cridlands DNA, called human leukocyte antigens, matched the patients DNA.
Every one individual has their own unique genetic DNA code, said Erica Sevilla, a spokeswoman for Be the Match. What were looking to attach to that code is protein markers that tell your body what cells belong in the body and what cells do not. Essentially, youre looking to match immune systems between the donor and the patient.
From there, the patients doctor will decide the best course for treatment.
According to Be the Match, many believe that the only way to donate blood stem cells is through a surgical procedure, during which the donor receives anesthesia and a needle is used to extract liquid marrow from the pelvic bone. But 79 percent of donations are done through a nonsurgical procedure known as a peripheral blood stem cell donation.
The doctor in Cridlands donation case chose the nonsurgical route.
During the week leading up to the donation, Be the Match sent a nurse to Cridlands home in Henderson once a day for five consecutive days to administer injections of a medication that increased the number of blood-forming cells in Cridlands bloodstream.
Cridland and his wife were flown to Denver for the procedure.
At times throughout the donation process, which spanned about five months from the first phone call to the day of the procedure, Cridland would find himself moved to tears, overcome by his gratitude for the chance to help save a young child.
To me, the crazy thing about all this is that my actions from 14 years ago have had a dramatic effect on how somebody else is living in 2020, he said in an interview this month at his home. With all the negativity weve seen this year, this one family may look at 2020 as the best year of their lives because of this one specific moment in my life that took place 14 years ago.
Even now, three months removed from donation day, Cridland at random will break down in tears.
Sometimes he thinks about his blood pumping through the childs body. How a piece of him will always be with that child. How someone he has never met, and may never meet, could be such a close genetic match to him.
How were all more alike than we think.
Epilogue
I wish I could take this feeling, put it in a can and throw a lid on top, Cridland said of his experience donating stem cells.
If that were possible, Cridland said, he would pass it around like a party favor but he cant.
So instead, hes hoping his story will inspire others, especially people of color, to join the registry and give the gift of a cure to another patient in need.
Currently, there is a severe shortage of diverse donors in the national registry, which consists of 22 million donors. More than 13 percent of the American population is Black, for example, yet only about 4 percent of registered donors are Black.
And the disparity is costing lives.
Matching is based on genetic markers that can be traced back to your grandparents and your great-grandparents, said Sevilla, the spokeswoman for Be the Match. Theyre traced back to the very origins of your family, so thats why people who are of European descent have an easier time finding a match, whereas people who descended from slavery, for example, have a harder time.
To join the National Bone Marrow Registry and request a cheek swab kit, visit http://www.join.bethematch.org.
Meanwhile, as of mid-December, Cridland knew only that the patient had received his stem cells. He wasnt sure, even, if hed ever meet the child.
Both parties must consent to meeting, and different countries operate under varied cooling-off periods. For some countries, a patient and a donor must wait two years before they can meet or even speak.
As a donor, Cridland was told the patients age, specific diagnosis and city and country of residence. But he is not allowed to disclose that information to protect the patients privacy.
According to Be The Match, 50 percent of all marrow or stem cell donations are international.
We are either exporting cells or importing cells, said Sevilla, the charity spokeswoman.
Cridland gave his consent for the patients family to reach out to him in the future. For now, its a waiting game.
If the moment comes that they try to connect, Ill be there, he said.
Contact Rio Lacanlale at rlacanlale@reviewjournal.com or 702-383-0381. Follow @riolacanlale on Twitter.
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He got his cheek swabbed at 24. Nothing happened for 14 years. - Las Vegas Review-Journal
Study clarifies the impact of getting old on hematopoietic stem cells – Microbioz India
By daniellenierenberg
By shifting mouse elderly hematopoietic stem cells (aged HSCs) to the environment of young mice (bone marrow niche), it was shown that the pattern of stem cell gene expression was rejuvenated to that of young hematopoietic stem cells. On the other hand, the function of elderly HSCs failed to recover in the young bone marrow niche. The epigenome (DNA methylation) of aged HSCs didnt change significantly even in the young bone marrow niche, and DNA methylation profiles were found to be a better index than the gene expression pattern of aged HSCs.
A research group headed by Professor Atsushi Iwama in the Division of Stem Cell and Molecular Medicine, The Institute of Medical Science, The University of Tokyo (IMSUT) declared these world-first Outcomes and was published in the Journal of Experimental Medicine (online) on November 24th.
The results will contribute to the development of treatments for age-related blood diseases.
Professor Atsushi Iwama, Lead Scientist, IMSUT
The research group investigated whether rejuvenating aged HSCs in a young bone marrow market environment would rejuvenate.
Tens of thousands of elderly hematopoietic stem/progenitor cells gathered from 20-month-old mice were transplanted into 8-week-old young mice without pretreatment like irradiation. After two months of follow-up, they collected bone marrow cells and performed flow cytometric analysis.
The research team also transplanted 10-week-old young mouse HSCs for comparison. In addition, engrafted aged HSCs were fractionated and RNA sequence analysis and DNA methylation analysis were conducted.
They discovered that engrafted elderly HSCs were less capable of producing hematopoietic cells compared to younger HSCs. They also showed that differentiation of aged HSCs into multipotent progenitor cells was persistently impaired even in the young bone marrow market, and that the direction of differentiation was biased. It was found that the transfer of aged HSCs into the young bone marrow market does not enhance their stem cell function.
A more detailed analysis may reveal mechanisms that irreversibly affect aged HSC functionAging studies focusing on HSCs have been chased in mice with a bone marrow transfer model. However, the effect of aging on HSCs remains to be clarified.
Professor Iwama says as follows. This analysis has a substantial impact because it clarified the effect of aging on HSCs. Our results are expected to contribute to further elucidation of the mechanism of aging in HSCs and comprehension of the pathogenic mechanism of age-related blood disorders.
Source:
Journal reference:
Kuribayashi, W.,et al.(2020) Limited rejuvenation of aged hematopoietic stem cells in young bone marrow niche.Journal of Experimental Medicine.doi.org/10.1084/jem.20192283.
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Study clarifies the impact of getting old on hematopoietic stem cells - Microbioz India
Scientists find new class of drugs that may treat blood, bone marrow cancer – Business Standard
By daniellenierenberg
A group of scientists in the US, including an Indian-American from the prestigious Cleveland Clinic, have identified a potential new class of drugs that may prove effective in treating certain common types of blood and bone marrow cancers.
First published in the latest edition of Blood Cancer Discovery, the decade long research which reports that a new pharmacological strategy to preferentially target and eliminate leukemia cells with TET2 mutations, was carried out by Jaroslaw Maciejewski and his collaborator Babal Kant Jha from the Cleveland Clinic Department of Translational Hematology & Oncology Research.
Myeloid leukemias are cancers derived from stem and progenitor cells in the bone marrow that give rise to all normal blood cells.
One of the most common mutations involved in driving myeloid leukemias are found in the TET2 gene, which has been investigated for the last decade by Maciejewski and Jha.
In preclinical models, we found that a synthetic molecule called TETi76 was able to target and kill the mutant cancer cells both in the early phases of disease--what we call clonal hematopoiesis of indeterminate potential, or CHIP--and in fully developed TET2 mutant myeloid leukemia," said Dr Maciejewski.
According to a media release, the research team designed TETi76 to replicate and amplify the effects of a natural molecule called 2HG (2-hydroxyglutarate), which inhibits the enzymatic activity of TET genes.
The TET DNA dioxygenase gene family codes for enzymes that remove chemical groups from DNA molecules, which ultimately changes what genes are expressed and can contribute to the development and spread of disease.
While all members of the TET family are dioxygenases, the most powerful enzymatic activity belongs to TET2, the press release said.
Even when TET2 is mutated, however, its related genes TET1 and TET3 provide residual enzymatic activity.
While significantly less, this activity is still enough to facilitate the spread of mutated cancer cells.
Maciejewski's and Jha's new pharmacologic strategy to selectively eliminate TET2 mutant leukemia cells centers on targeting their reliance on this residual DNA dioxygenase activity, it said.
We took lessons from the natural biological capabilities of 2HG, explained Jha, a principal investigator.
We studied the molecule and rationally designed a novel small molecule, synthesized by our chemistry group headed by James Phillips, PhD. Together, we generated TETi76a similar, but more potent version capable of inhibiting not just TET2, but also the remaining disease-driving enzymatic activity of TET1 and TET3, Jha said.
Cleveland Clinic said that the researchers studied TETi76's effects in both preclinical disease and xenograft models (where human cancer cells are implanted into preclinical models). Additional studies will be critical to investigate the small molecule's cancer-fighting capabilities in patients.
We are optimistic about our results, which show not just that TETi76 preferentially restricts the growth and spread of cells with TET2 mutations, but also gives survival advantage to normal stem and progenitor cells, Jha said.
(Only the headline and picture of this report may have been reworked by the Business Standard staff; the rest of the content is auto-generated from a syndicated feed.)
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Scientists find new class of drugs that may treat blood, bone marrow cancer - Business Standard