Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression

Translation of metabolic correction to clinical outcomes in first two patients continues to support potential of hematopoietic stem cell gene therapy in a second neurometabolic disorder

Data support planned initiation of registrational trial in 2021

BOSTON and LONDON, Sept. 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at theSan Raffaele Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today's results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up, saidMaria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. "These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigationalex vivoautologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele Telethon Institute for Gene TherapyinMilan, 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 onTwitterandLinkedIn.

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. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. 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 severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be successfully developed, approved 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 or that long-term adverse safety findings may be discovered; 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; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. 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 endedJune 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

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - BioSpace

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I – BioSpace | September 1st, 2020

Bone marrowaspiration 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 2016, 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 2016, 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.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 Business Analysis, New Innovation | Share, Revenue, And Sales Till 2025 - The Scarlet

Bone Marrow Stem Cells Comments Off on Bone Marrow Processing Systems Market Business Analysis, New Innovation | Share, Revenue, And Sales Till 2025 – The Scarlet | August 31st, 2020

If you think light therapyis a high-tech way to heal your skin, wait until you hear about this trending skin care ingredient thats going to sound incredibly brilliant: stem cells.

Dermatologists have turned to stem cells to fight wrinkles and improve skin turnover and overall appearance. Yep, you heard right. Stem cells, the same ones used in innovative medical research to treat Alzheimers and certain types of cancer, are now being sold over the counter in the form of creams, serums and other skin care products. Except theres one major difference here: These stems cells are usually derived from plants (or occasionally animals). However, they work similarly to human stem cells and may offer anti-aging benefits for your skin.

Human stem cells are unique because of their ability to divide. In certain organs, they can even become specialized to repair and replace damaged tissues. Stemcellsare rapidly dividingcellsin the body that have the ability to give rise to morestemscellsor become other types ofcellswith more specialized function, explains Dr. Sejal Shah, board-certified dermatologist and RealSelf contributor in New York City. Plant stem cells serve similar functions, she says.

Both plant and human stem cells contain proteins and amino acids, adds Dr. Michele Green, board-certified dermatologist and RealSelf contributor in New York City. These signal the bodys cells to rejuvenate and may result in younger-looking skin, she says.

As mentioned above,stemcellscontain amino acids and peptides, which are skin care powerhouse ingredients for skin rejuvenation. These are the building blocks forcellrejuvenation, so over the past few years, there have been a variety of both animal- and plant-basedstemcellsin skin care products, explains Green. Stemcellsnaturally have antioxidant properties and they nourish skincellswhich promotescell turnover and increases collagen production.

This could result in fewer lines and wrinkles, improved skin texture and tone, and younger, better-looking skin, she says.

But keep in mind, its not actually living stem cells that youre seeing in your face cream, Shah notes. Most cell skin care products contain plant stem cells, and more specifically, stem cell extracts. Thats not necessarily a bad thing, though. She says these extracts are often rich in antioxidants and may provide growth factors to help renew and repair the skin. The extracts themselves can benefit the skin, but its not accurate to think that part of this type of product will then become a new skincell. Remember, plantcellscannot become humancells,and they are no longer living once they have been processed and added into skin care.

Dr. Eve Lupenko, board-certified dermatologist at Greenberg Cosmetic Surgery in New York City, is starting to use treatments containing plant-derived stem cells in her practice. The reason why we prefer to use plant stem cells is that you dont have to worry about transmitting human and animal diseases, she says. We are seeing plant-based stem cells in skin care products these days because they repair the skin on a cellular level (a much deeper level). Most regular skin care products dont penetrate into those areas of the actual skin cells.

The efficacy of stem cells in skin care depends on who you ask. Some dermatologists like Lupenko swear by them. Stem cells have the potential to repair skin cells, and they also protect your skin from external factors and create a more youthful look, she says. They go into the skins cellular level, and they are able to deliver moisture and reparative agents to where they need to go.

Others arent convinced about why exactly stem cells are suddenly buzzing in the skin care world. They can be rich in antioxidants and often contain hydrators and moisturizers, so they can be good for the skin, Shah says. But do I think they are more effective than non-stem cell products? Not necessarily.

Green sees the potential in using stem cells in conjunction with other treatments. The products work, however, you can improve the benefits dramatically with other procedures such as the Fraxel laser, thermage, injectable fillers, botox and PRP, a mix of micro-needling with platelet-rich plasma.

If youre interested in trying out stem cells, work with your dermatologist to determine which products would work best for your skin, recommends Lupenko. Its important to use them regularly if youd like to see results, she says.

And even though you might find some stem cell products derived from animals, Green recommends sticking with plant-based stem cells. Theres more research in their efficacy, she says.

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Stem Cells in Skin Care: What They Do and How They Work ...

Skin Stem Cells Comments Off on Stem Cells in Skin Care: What They Do and How They Work … | August 31st, 2020

Using a new type of stem cells made from ordinary skin cells, U.S. researchers said on Thursday they treated mice with sickle cell anemia, proving in principle that such cells could be used as a therapy.

U.S. and Japanese researchers last month reported they had reprogrammed human skin cells into behaving like embryonic stem cells, the bodys master cells. They call the cells induced pluripotent stem cells, or iPS cells for short.

Hanna and colleagues working in Rudolf Jaenischs lab at Whitehead Institute took skin cells from diseased mice and inserted four genes that reprogram the cells into becoming iPS cells.

Pluripotent or multipurpose cells, such as embryonic stem cells and the new cells, can morph into any type of cell in the human body.

The researchers then coaxed these mouse master cells into becoming blood-forming stem cells and substituted the faulty gene that causes sickle cell anemia with a working one.

When they transplanted these cells into the diseased mice, tests showed normal blood and kidney function, they report in Fridays issue of the journal Science.

The four genes needed to turn skin cells into master cells are delivered using a type of virus called a retrovirus.

Once they enter the genome, there is the danger that they can silence some genes that are important or they can activate some dangerous genes that shouldnt be activated, Hanna said.

Another obstacle is that one of the four genes used is c-Myc, which is known to cause cancer.

Hanna and colleagues got around that by removing the c-Myc gene after it had done its job of converting the skin cells into iPS cells. It is far from solving the problem, he said.

Scientists hope to use stem cells to treat a host of diseases like diabetes, Parkinsons disease and spinal injuries. And the new technique for making stem cells will make them easier to study.

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Stem cells made from skin cells treats sickle cell anemia ...

Skin Stem Cells Comments Off on Stem cells made from skin cells treats sickle cell anemia … | August 31st, 2020

The global war against the coronavirus pandemic continues to wage on while researchers and medical experts seek to find a cure for COVID-19 symptoms. While many believe this is here to stay for an indefinite period, others feel that this too, shall pass.

The number of confirmed cases and death rates only seem to darken our world with over 400,000 confirmed cases in New York, the death toll is now over 31,000, with over 233,000 confirmed cases in Florida, the death toll is now over 4,000, and with over 250,000 confirmed cases in Texas, the death toll is now over 3,000.

And here we are in July 2020 where you can simply add those statistics and drive to find a cure for COVID-19 to our to-do list in the war on cancer and other diseases that still have not seen a cure.

With Florida continuing to make headlines by the day, most recently with one family facing federal charges after allegedly marketing a toxic bleach solution as a cure for multiple ailments, including COVID-19, the timing for our society to come together to help find a cure is essential.

And no, were not kidding. The Florida family (Mark Grennon and his three sons) were charged Wednesday with conspiracy to defraud the United States, conspiracy to violate the Federal Food, Drug and Cosmetic Act, and criminal contempt, according to the Department of Justice.

As for the Florida community, one Tampa business has been conducting the first-in-human clinical study for cutaneous melanoma. Morphogenesis, a clinical-stage company developing novel cell and gene therapies has been making headlines after receiving FDA approval to expand its human clinical trials into two more types of cancer: Merkel cell carcinoma (MCC) and cutaneous squamous cell carcinoma (cSCC).

Using its ImmuneFx (IFx) cancer vaccine technology that initiates the power of the immune system on the destruction of tumor cells, Morphogenesis of Tampa will focus its newly FDA approved trials on understanding these two new cancers, which follows in the footsteps of successful human trials on cutaneous melanoma, conducted in cooperation with Moffitt Cancer Center in 2019.

Other MCC and cSCC clinical trial sites across the country include the University of Southern California, the University of Utah, the University of Colorado, and the Dana Farber / Harvard Cancer Center. So why so many trial sites?

Well, according to Morphogenesis CEO Dr. Patricia Lawman, clinical trials need patients, which to qualify, requires an individual having been diagnosed with advanced Merkel or cutaneous squamous cell carcinoma, and having failed or refused other therapies.

As a cell and gene company, the mission from the beginning was to learn from the body and use the bodys building blocks and communication systems to treat chronic disease. Morphogenesis, according to Lawman, was built to identify, isolate and proliferate stem cells and progenitor cells to treat diseases such as diabetes.

With the world united to change the way in which chronic diseases are treated by engaging the innate intelligence of the body, how do companies (on a local level) push for national change?

The ability to genetically modify stem cells to enhance functionality is one aspect of this, but in order to perform a biological function, the stem cells must differentiate into mature cells, e.g. hematopoietic stem cells differentiate into macrophages that perform phagocytic and antigen presentation functions and T cells that kill cancer cells or virally infected cells. Morphogenesis means the evolution of form, which connotes the change for stem cells to these functional cells capable of mitigating chronic disease.

And what this means for our bodies, according to Lawman, is that regardless of the species, our bodies have developed systems that maintain structure and function over a long period of time. When we need to control blood sugar, beta islet cells produce just enough insulin as needed.

Indeed with modern technology, you would think that this is a relatively easy process to control.

There is an exquisite feedback system that regulates this. When things go awry, our best solution has been to provide insulin through pumps that are controlled in part by constant glucose monitors. This one example of where modern technology has tried to solve a problem mimicking how the body works.

However, even providing insulin through a pump cant do what a pancreas can. When it comes to dealing with foreign invaders, the immune system is unequaled. No drug, small molecule or compound can eliminate an invader as well as a fully functional immune system. We can kill cancer cells (while not foreign, they are still invaders) with chemo and radiation, but given the proper assurance, the immune system can eliminate the invader and do it with fewer adverse effects. Almost always, the body performs healing functions better than a synthetic drug or compound.

The companys recent FDA approval to move forward with stage 2 of its clinical trials is a gigantic win for the companys mission. The ability to expand our proof of concept studies from a single skin cancer into other, quite different skin cancers under the same Investigational New Drug (IND) is the next step in the execution of our clinical development plan.

And that starts with Morphogenesis focus on easily accessible tumors.

Since our therapy can be used to treat virtually any type of cancer, we wanted to start out with easily accessible tumors that could be directly injected with our plasmid DNA. The safety data collected from the cutaneous melanoma, Merkel cell carcinoma and cutaneous squamous cell carcinoma Phase 1 trials is a Segway into a Phase 2 skin cancer basket trial testing IFx-Hu2.0 as a monotherapy and in combination with a checkpoint inhibitor.

But anytime there is discussion over stem cell research or breakthroughs in the war on cancer, of course comes naysayers and disbelievers.

One thing that Dr. Lawman has noticed is the bias within scientific circles.

In scientific circles, there has been a bias against simple solutions, including the assumption that to get efficient transfer of genetic material you need viral vectors for all applications. These vectors are complicated to manufacture and use and pose a certain amount of risk to the patients. Plasmid DNA or mRNA, on the other hand, are much safer and are a viable alternative to viruses.

As an example, we inject our plasmid DNA directly into a patients tumor. We get sufficient uptake and expression of our protein to initiate an immune cascade with the effect spreading to multiple tumor antigens. The use of a viral vector in this case would be an unnecessary complication and risk.

Now in todays landscape with COVID-19 putting more pressure on experts to find a cure, Morphogenesis, like any company, is similarly faced with logistical challenges and supply issues.

COVID-19 has certainly affected patient recruitment to our trials. Hopefully, ways will be developed for patients to receive treatment for their terminal diseases even if restrictions continue. Otherwise, the death rate for these patients will be much higher than COVIDs. The biggest challenge for us is the continual process of raising funds that all small biotechs face.

As for ImmuneFx, the companys newest vaccine, we got the exclusive.

Beyond the Phase 2 skin cancer trial, we will be opening trials for head and neck cancer, gastric cancers, cervical cancer and colorectal cancer. The value add here is that successful trial results in multiple types of cancer will substantiate the efficacy and expand our label claims.

But with new products and solutions, come criticism. Lawman added that the one thing that is not usually discussed in such conversations is the importance of the safety profile of a new product.

Some of the new cellular immunotherapies not only come with a hefty price tag, the cost of treating the adverse side effects caused by the therapies can be as much as double the cost of the therapy itself (up to $1.5M total). Some of the newer gene therapies can have a price tag of up to $2M per treatment.

Not only can our plasmid DNA be cost effectively manufactured, it is causing minimal side effects, i.e. what we saw in hundreds of companion animals with naturally occurring cancers is being born out in human patients. Both of these cost saving factors means that ultimately, millions of people will have access to cancer treatment who otherwise would have none.

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Why The FDA's Recent Approval Of New Vaccine Is A Gigantic Win In The War On Cancer - Innovation & Tech Today

Skin Stem Cells Comments Off on Why The FDA’s Recent Approval Of New Vaccine Is A Gigantic Win In The War On Cancer – Innovation & Tech Today | August 31st, 2020

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Scope and Opportunities Analysis 2017 2025 - StartupNG

Cardiac Stem Cells Comments Off on Stem Cell Therapy Market Scope and Opportunities Analysis 2017 2025 – StartupNG | August 31st, 2020

The global Stem Cell-Derived Cells market study presents an all in all compilation of the historical, current and future outlook of the market as well as the factors responsible for such a growth. With SWOT analysis, the business study highlights the strengths, weaknesses, opportunities and threats of each Stem Cell-Derived Cells market player in a comprehensive way. Further, the Stem Cell-Derived Cells market report emphasizes the adoption pattern of the Stem Cell-Derived Cells across various industries.

The Stem Cell-Derived Cells market report examines the operating pattern of each player new product launches, partnerships, and acquisitions has been examined in detail.

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key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

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The Stem Cell-Derived Cells market report offers a plethora of insights which include:

The Stem Cell-Derived Cells market report answers important questions which include:

The Stem Cell-Derived Cells market report considers the following years to predict the market growth:

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Why Choose Stem Cell-Derived Cells Market Report?

Stem Cell-Derived Cells Market Reportfollows a multi- disciplinary approach to extract information about various industries. Our analysts perform thorough primary and secondary research to gather data associated with the market. With modern industrial and digitalization tools, we provide avant-garde business ideas to our clients. We address clients living in across parts of the world with our 24/7 service availability.

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Stem Cell-Derived Cells Market Forecast to 2025: Global Industry Analysis by Top Players, Types, Key Regions and Applications - The Scarlet

IPS Cell Therapy Comments Off on Stem Cell-Derived Cells Market Forecast to 2025: Global Industry Analysis by Top Players, Types, Key Regions and Applications – The Scarlet | August 30th, 2020

PHILADELPHIA Autism is a neurodevelopmental disorder which creates difficulties with social interactions, communication, and repetitive behaviors. According to the Centers for Disease Control and Prevention, autism affects one in 54 children in the United States. Although autism typically isnt diagnosed until children are at least 18 months-old, a new study finds that abnormal brain cell development likely occurs much earlier.

Researchers say this even occurs while babies are still in the womb.

To study developing brain cells, a team from Kings College London and Cambridge University use a type of cell known as an induced pluripotent stem cell (or iPSC). Essentially, iPSCs are adult cells that scientists force to become immature embryonic-like stem cells. Scientists can program iPSCs to become one of many different cell types, including neurons. Since iPSCs are forced to restart their cellular development, they mimic the processes occurring in the womb. This allows them to serve as a useful means of studying early brain development.

Using iPSCs from hair samples is the most ethical way to study early brain development in autistic people, explains study author Dwaipayan Adhya in a media release. It bypasses the need for animal research, it is non-invasive, and it simply requires a single hair or skin sample from a person.

Adhya is a molecular biologist at the Autism Research Centre in Cambridge and Department of Basic and Clinical Neuroscience at Kings College London.

To create the iPSCs, the study analyzes hair samples from nine adults with autism and six neurotypical adults. The hair cells were then treated with growth factors (naturally occurring bodily substances that regulate cell division and survival). The authors looked at the cells appearance and genetic makeup at different phases of development.

The scientists results reveal iPSCs from neurotypical people look different from those participants with autism. At day nine, neurons from neurotypical people develop a characteristic pattern of neural rosettes, which have a dandelion-like shape. In contrast, cells from people with autism have smaller rosettes or dont form them at all. Cells from autistic individuals also express lower levels of important developmental genes.

The use of iPSCs allows us to examine more precisely the differences in cell fates and gene pathways that occur in neural cells from autistic and typical individuals, co-author Deepak Srivastava explains. These findings will hopefully contribute to our understanding of why there is such diversity in brain development.

The brain has been the ultimate black box. Here, the authors have used nerve cells derived from peripheral stem cells to peek inside this box. This important study suggests that this is possible and is deepening our understanding of autism, says John Krystal, Editor-in-Chief of the journal Biological Psychiatry.

The study is published in the June 22 edition of Biological Psychiatry.

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Study: Brain cells of people with autism differ even before theyre born - Study Finds

Skin Stem Cells Comments Off on Study: Brain cells of people with autism differ even before theyre born – Study Finds | August 30th, 2020

Read: The plan that could give us our lives back

The science is paying off. Novavax, a Maryland-based company working on this type of vaccine, recently reported the results of its Phase 1 trial. The levels of antibodies generated were stunning, about four times higher than those in individuals who are recovering from a COVID-19 infection.

Scientists are also using different strains of another virus, adenovirus, as a vector or a missile to deliver genes that code for these same spike proteins and that also provoke an immune response. The vector has been engineered in the lab to be replication-defective; that is, the vector is able to deliver the spike gene into humans but once its done its job, the vector cannot replicate any further. At least three groups are testing these vectors. A University of Oxford group, in partnership with AstraZeneca, has employed an adenovirus from chimpanzees and has already entered Phase 3 trials in humans. The Beth Israel Deaconess Medical Center group, in partnership with Janssen Pharmaceutica, is using Ad26, a human adenovirus, and the Chinese-based CanSino Biologics has begun Phase 3 trials with yet another human adenovirus, Ad5.

These examples are not just beautiful science (although they are beautiful science). By harnessing the increased power of the biological sciences, researchers are developing entirely new ways of rapidly developing vaccines.

My optimism doesnt stop with these early results, although they are key. Im also encouraged because at least five very different approaches (Ive walked through only three above) are being explored to make a vaccine. As we say in Canada, if you want to win, you have to take many shots on goal.

Equally important is the unprecedented global collaboration among scientists around the world, as well as the high degree of cooperation between scientists and clinicians, biopharmaceutical companies, government, philanthropic funders, and regulators. They are all working together toward the common goal of developing as quickly as possible a safe and effective vaccine against COVID-19.

I dont know which of the vaccine candidates undergoing clinical testing in humans will ultimately be shown to be safe and effective. They might all prove effective, albeit in different age groups or in people with different preexisting conditions. But the encouraging news is that all of the vaccine candidates that have entered trials in humans so far are safe and have elicited high levels of antibodies against COVID-19. Some have also been shown to activate the cellular arm of our immune system, another crucial component of our defenses against foreign pathogens.

The public-health imperative to obtain a safe and effective vaccine as quickly as possible goes hand in hand with the mandate that the approval process be above any political considerations and solely based on data from the clinical trials. Anything else risks losing the publics confidence in a vaccine or, in a worst-case scenario, might result in a vaccine that is less effective than those that might be approved later, or the widespread administration of a vaccine that turns out to have serious adverse side effects. That would be a public-health tragedy.

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Im Optimistic That We Will Have a COVID-19 Vaccine Soon - The Atlantic

Skin Stem Cells Comments Off on Im Optimistic That We Will Have a COVID-19 Vaccine Soon – The Atlantic | August 30th, 2020

There isnt a third person in our marriage but there is a rival. My husband is passionate about a family-run fasting clinic in Uberlingen, overlooking Lake Constance, Germany. He goes alone twice a year and was there at the beginning of lockdown. He happily remained there for six weeks unable to get home. Buchinger Wilhelmi was founded seventy years ago by Otto Buchinger, a medical officer in the navy who cured himself of paralysis caused by rheumatic fever by fasting for 19 days in 1918. Now one of the worlds leading therapeutic fasting centres, Buchinger is run by Ottos great-grandson, Leonard Wilhelmi.

The German government demanded that the clinic remain open during lockdown, concerned that hospital over-crowding would necessitate patients being moved there. This proved unnecessary. But after Easter, having taken the requisite health and hygiene precautions, they encouraged guests. They are currently full with a completely different clientele, according to owner, Raimund Wilhelmi (Ottos grandson.) Normally, two-thirds of our guests are repeat guests. Now, fifty percent have come for the first time and they are much younger. There is a significant difference in the demographic because people are waking up to a new awareness about health as Covid affects everybody. His son, Leonard, adds: The medical community agree that the main causes in violent reactions to Covid are diabetes, high blood pressure and being over-weight. We have been treating these conditions for decades and our goal is now to equip people with a better immune system to fight Covid.

The minute my husband heard about their new immune-boosting programme, he signed us up for atwo weeks' holiday in August. This was an extravagance at a cost of over two thousand pounds a person. Health is our most valuable commodity, he reassured me. After an hour in this sleek minimalist medical centre, my sixteen-year-old daughter burst into tears. All her boarding school issues were ignited. My inner rebel similarly baulked at the first 24 hours that we had to spend eating in our rooms, until the results of our Covid tests, taken on arrival, came through. (Thankfully they were negative or we would have been quarantined in our rooms for our entire stay.)

There was something convict-like about dining on trays in our monastically simple rooms. Thank goodness the clinic do not advise couples share rooms 80 per centof guests go alone so we each had our own room. As Andrew was doing the full ten-day fast of 250 liquid calories a day, which I couldnt stomach(I was on 800 solid calories a day and Daisy, 1800,) a shared room would have destroyed our marriage. Not because Andrew had an enema on the bed every other day but because his preternatural joy freaked us out. His pious enthusiasm for the regime initially made us hate it. Until we were fully institutionalised, (or as Daisy said indoctrinated) the stricture wore us out.

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What I learnt from checking in to the 'Immunity Hotel' - Telegraph.co.uk

Skin Stem Cells Comments Off on What I learnt from checking in to the ‘Immunity Hotel’ – Telegraph.co.uk | August 30th, 2020

A California woman may be the first person to be cured of HIV without a bone marrow transplant, according to a recent report in Nature. More than 60 other so-called elite controllers, who have unusually potent immune responses to HIV, were found to have their virus sequestered in parts of their genome where it is unable to replicate.

The unusual case involves Loreen Willenberg, who acquired HIV in 1992. Her immune system has maintained control of the virus for decades without the use of antiretroviral treatment, and researchers have been unable to find any intact virus in more than 1.5 billion of her cells. Elite controllers are thought to make up less than half a percent of all people living with HIV.

I believe Loreen might indeed meet anyones definition of a cure, study coauthor Steven Deeks, MD, of the University of California at San Francisco, told POZ. Despite heroic efforts, we just could not find any virus that is able to replicate. Her immune system seems completely normal. Even her HIV antibodies levels are low, which is unprecedented in an untreated person.

Although antiretroviral therapy can keep HIV replication suppressed, the virus inserts its genetic material into the chromosomes of human cells, making it very difficult to eradicate. HIV can lie dormant in a reservoir of resting immune cells indefinitely, but when antiretrovirals are stopped and the cells become activated, they can start churning out new virus.

Previously, only two people were known to have been cured of HIV: Timothy Ray Brown, formerly know as the Berlin Patient, and a man in London. Both received bone marrow stem cell transplants from a donor with a rare genetic mutation that makes cells resistant to HIV entry. But this procedure is far too dangerous for people who dont need it to treat advanced cancer.

The new research suggests that Willenberg and some five dozen other people with long-term untreated HIV have their virus hidden away in their cells genomes in such as way that the viral genetic blueprint (known as a provirus) cant be used to produce new viral particles that can go on to infect other cells.

Xu Yu, MD, of the Ragon Institute of Massachusetts General Hospital, MIT and Harvard analyzed integrated HIV in millions of cells from 64 elite controllers and 41 typical HIV-positive people on antiretroviral therapy recruited at Mass General and San Francisco General Hospital.

In both groups, about 20% were women, the average age was approximately 56, they had been living with HIV for an average of 17 years and had undetectable virus according to standard tests for nine years. Overall, the elite controllers had a higher average CD4 count (about 900 versus 70, respectively).

The researchers used next-generation gene sequencing to characterize the participants viral blueprints, including where they were inserted into human chromosomes. They found that the elite controllers had fewer integrated proviruses, but a larger proportion of them were intact, or potentially capable of replicating. The virus in these individuals was highly consistent, without the wide variety of mutations seen in most people with HIV.

Whats more, their proviruses were integrated at distinct sites in the human genome, farther away from elements that enable viral replication. Specifically, the integrated DNA was not located near sites that switch on transcription or close to accessible chromatin, which contains histone proteins that package long DNA strands into a more compact form. The DNA must then be unwound from these proteins before it can be used to produce new virus.

These data suggest that a distinct configuration of the proviral reservoir represents a structural correlate of natural viral control, and that the quality, rather than the quantity, of viral reservoirs can be an important distinguishing feature for a functional cure of HIV-1 infection, Yu and colleagues wrote.

In a commentary accompanying the report, Nicolas Chomont, PhD, of the University of Montreal, characterized the proviruses in these elite controllers as being in a state of deep sleep compared with latent virus in typical people with HIV. This has only become apparent now because researchers have more sophisticated tools to pinpoint the location of proviruses within the genome.

It is unclear why this block and lock phenomenon happens in only a small proportion of people with HIV. Its possible that the virus ends up sequestered in these locations by chance. But the researchers think its more likely that the integrated proviruses at these sites are evolutionarily selected over time as the ones in locations more conducive to viral replication are eliminated by the immune system.

In Willenbergs case, the research team analyzed more than 1.5billion of her peripheral blood immune cells, including samples from gut tissue, where the virus often hides. Theycould not find any intact proviruses that could be used to produce new HIV. Given her absence of intact proviruses, the researcherswere unable to determine whether she ever fit the pattern of having latent HIV locked away in inaccessible locations.

Another 11 people, dubbed exceptional controllers, only had detectableprovirusesatremote sites in the genome where it could not replicate.Since this study, the researchers have discovered a couple more elite controllers who may qualify as additional cures, according to the New York Times.

This raises the possibility that a sterilizing cure of HIVmeaning complete eradicationmay be feasible in rare instances, the study authors suggested. A similar but less complete process may be at play in the subset of about 10% of people with HIV who maintain viral suppression after stopping antiretroviral therapy but who still have detectable proviruses (known as post-treatment controllers).

This research was first presented at the International AIDS Society Conference on HIV Science last summer, where Willenberg was referred to as the San Francisco Patient. Willenberg later went public with her status, and she and Yu discussed the study findings during a webinar with HIV cure advocates last November.

I broke out in tears when saw Dr. Yus final slide, said Willenberg, who over the years has participated in more than a dozen studies. I can only hope and pray that with continued dedication we can figure out how I have dumped the virus into the DNA junkyard.

The question now is whether its possible to develop treatments that could enable the millions of typical people with progressive HIV to become elite controllers like Willenberg. Chomont suggested that immune-based therapiesincluding CAR-T cellsmight be able to shrink the viral reservoir until it consists only of deeply latent proviruses that are unable to replicate.

The key question is how did her immune system achieve this remarkable state, Deeks said. We do not know. We need to find more people who are exceptional controllers like Loreen and get to work on figuring out the mechanism.

In this video fromamfAR, the Foundation for AIDS Research,Loreen Willenbergtalks about living as an elite controller of HIV.

Click here to read the Nature article.Click here for more news about HIV cure research.

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First Woman May Be Cured of HIV Without a Bone Marrow Transplant - POZ

Bone Marrow Stem Cells Comments Off on First Woman May Be Cured of HIV Without a Bone Marrow Transplant – POZ | August 30th, 2020

Autologous stem cell and non-stem cell based therapiesinvolve an individuals cell to be cultured and then re-introduced to the donors body. These therapies do not use foreign organism cells and are therefore free from HLA incompatibility, disease transmission, and immune reactions.Increasing demand for the new therapies in the field of regenerative medicine is directly facilitating the growth of autologous stem cell and non-stem cell based therapies market. Furthermore, since the risk to transplantation surgeries is significantly reduced in these therapies, they are increasingly being preferred for treatment of bone marrow diseases, aplastic anemia, multiple myeloma, non-Hodgkins lymphoma, Hodgkins lymphoma, Parkinsons disease, thalassemia, and diabetes.

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Moreover, rising incidents of cancer, diabetes and cardiovascular diseases along with growing geriatric population is another factor attributed for its high growth. However, side-effects of autologous stem cell and non-stem cell based therapies such as nausea, infection, hair loss, vomiting, diarrhea, etc. are expected to affect the market to an extent. High cost is another factor that can act as challenge to autologous stem cell and non-stem cell based therapies market. In spite of this, less risk post transplantation surgeries and favorable tax reimbursement policies are anticipated to reduce the impact of these limitation during the forecast period.Autologous stem cell and non-stem cell based therapies market can be segmented on the basis of application, end-user, and region.

In terms of application, the autologous stem cell and non-stem cell based therapies market can be segmented into blood pressure (BP) monitoring devices, intracranial pressure (ICP) monitoring devices, and pulmonary pressure monitoring devices. In terms of end-user, the market can be segmented into ambulatory surgical center and hospitals. By region, the market can be segmented into North America, Europe, Asia Pacific, Middle East and Africa and South America. Amongst all, Asia Pacific is anticipated to be the most attractive market owing to favorable reimbursement policies in the region.The players operating in autologous stem cell and non-stem cell based therapies market are limited. They are consistently involved in research and development activities for product development to keep up with the growing competition, thereby aiding the growth of autologous stem cell and non-stem cell based therapies market across the world.

The major players operating in autologous stem cell and non-stem cell based therapies market are Regennex, Antria(Cro), Bioheart, Orgenesis Inc., Virxys corporation , Dendreon Corporation, Tigenix, Georgia Health Sciences University, Neostem Inc, Genesis Biopharma, Brainstorm Cell Therapeutics, Tengion Inc., Fibrocell Science Inc., Opexa Therapeutics Inc, Regeneus Ltd, and Cytori Inc., among others.

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Autologous Stem Cell And Non Stem Cell Based Therapies Market to Witness Widespread Expansion During 2018-2026 - Scientect

Bone Marrow Stem Cells Comments Off on Autologous Stem Cell And Non Stem Cell Based Therapies Market to Witness Widespread Expansion During 2018-2026 – Scientect | August 30th, 2020

DetailsCategory: DNA RNA and CellsPublished on Sunday, 30 August 2020 13:01Hits: 250

Long-term results from Phase 2/3 Starbeam study (ALD-102/LTF-304) suggest durability of response post eli-cel with all 20 patients who were free of major functional disabilities (MFDs) at two years (out of 23 evaluable patients) remaining MFD-free through last available follow-up, including all 10 patients who reached at least Year 5 follow-up visit

31 out of 32 patients in ALD-102 had stable Neurologic Function Scores following treatment with eli-cel, including 24 patients with a score of zero as of the last available visit

In clinical studies of eli-cel to date, there have been no reports of graft failure, graft rejection, graft-versus-host disease (GVHD), replication competent lentivirus, or insertional oncogenesis

Company on track to submit Marketing Authorization Application in EU by year-end 2020, and Biologics License Application in U.S. in mid-2021

CAMBRIDGE, MA, USA I August 29, 2020 I bluebird bio, Inc. (Nasdaq: BLUE) announced updated results from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including long-term results from the Phase 2/3 Starbeam study (ALD-102/LTF-304) and data from the Phase 3 ALD-104 study. These data were presented today at the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2020), taking place virtually from August 29 - September 1, 2020.

CALD is a fatal neurodegenerative disease primarily affecting young boys. Currently, the only treatment available is allogeneic hematopoietic stem cell transplantation (allo-HSCT), which comes with associated, significant risks, including transplant-related mortality, graft failure or rejection, and graft-versus-host disease (GVHD), said David Davidson, M.D., chief medical officer, bluebird bio. Eighty-seven percent of patients in our Phase 2/3 Starbeam study of eli-cel are alive and free of major functional disabilities (MFDs) at 24 months or more of follow-up. Importantly, there were no reports of graft failure, graft rejection, or GVHD. It is gratifying to see the consistent outcomes with eli-cel and the durability of the treatment effect demonstrated in the children participating in our long-term follow-up study including 10 boys who have now reached at least their Year 5 follow-up visit.

Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide. ALD is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP) and subsequently cause toxic accumulation of very long-chain fatty acids (VLCFAs) primarily in the adrenal cortex and white matter of the brain and spinal cord.

Approximately 40% of boys with adrenoleukodystrophy will develop CALD, the most severe form of ALD. CALD is a progressive neurodegenerative disease that involves breakdown of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms of CALD usually occur in early childhood and progress rapidly, if untreated, leading to severe loss of neurologic function, and eventual death, in most patients. CALD is associated with six MFDs, which severely compromise a patients ability to function independently: loss of communication, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement. Nearly half of boys with CALD who do not receive treatment will die within five years of symptom onset.

Patients with CALD experience a rapid decrease in neurologic function after the initial onset of clinical symptoms, so early diagnosis and treatment is critical in order to stop the disease progression and preserve their neurological function. In the Phase 2/3 Starbeam study, 31 of 32 patients had a stable neurologic function score, suggesting that disease progression had stabilized and minimal neurological function was lost, following eli-cel infusion, said Dr. Jrn-Sven Khl, Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women's and Children's Medicine, University Hospital Leipzig. These results presented at EBMT 2020 are very encouraging and suggest treatment with eli-cel may prevent neurological decline in boys with CALD.

Eli-cel is a one-time investigational gene therapy designed to address the underlying genetic cause of CALD by adding functional copies of the ABCD1 gene into a patients own hematopoietic (blood) stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of a functional gene allows patients to produce the ALDP, which is thought to break down the toxic accumulation of VLCFAs in the brain. There is no need for donor HSCs from another person, as is required for allo-HSCT.

Starbeam Study (ALD-102)/Long-Term Follow-Up Study (LTF-304)

The ALD-102 study has completed enrollment. All reported data below are as of January 2020 and reflect a total population of 32 patients with a median follow-up time of 30.0 months (9.1 70.7 months).

Of the 32 patients who have received eli-cel as of January 2020, 20 have completed ALD-102 and enrolled in a long-term follow-up study (LTF-304). Nine additional patients continue to be followed in ALD-102 and have not reached 24 months post-treatment. As previously reported, two patients withdrew from the study at investigator discretion, and one experienced rapid disease progression early on-study resulting in MFDs and death. To date, 104.3 patient-years of follow-up have been reported for ALD-102 and LTF-304.

The primary efficacy endpoint in the study is the proportion of patients who are alive and free of MFDs at Month 24. Of those patients who have or would have reached Month 24, 87% have met the primary endpoint and continue to be alive and MFD-free at more than two years of follow-up (N=20/23). Fourteen patients have at least four years of follow-up, including 10 patients who have reached at least their Year 5 follow-up visit. The nine patients from ALD-102 that have not reached Month 24 have shown no evidence of MFDs.

Data on several secondary and exploratory efficacy outcomes are reported, including changes in neurologic function score (NFS), a 25-point score used to evaluate the severity of gross neurologic dysfunction across 15 symptoms in six categories; resolution of gadolinium enhancement (GdE), an indicator of active inflammation in the brain; and change in Loes score, an MRI measurement of white matter changes in CALD. Of the 32 patients treated, 31 had stable NFS following treatment with eli-cel, defined as NFS <4, without a change of >3 from baseline, and 24 patients maintained an NFS of 0. An NFS of 0 indicates that there are no concerns with the neurologic functions that are assessed on the 25-point scale. Loes scores generally stabilized within 12-24 months and GdE was no longer seen in most patients following eli-cel treatment.

The primary safety endpoint is the proportion of patients who experience acute (Grade 2) or chronic GvHD by Month 24. GvHD is a condition that may occur after an allo-HSCT, where the donated cells view the recipients body as foreign and attack the body. No events of acute or chronic GvHD have been reported post-eli-cel treatment. There have been no reports of graft failure or graft rejection.

In addition, there have been no cases of replication competent lentivirus or insertional oncogenesis to date. Integration site analysis (ISA) was conducted to determine the pattern of integration post-eli-cel infusion and assess whether dominant or expanding clones were present. In one patient, now enrolled in LTF-304 for long-term follow up, a case of benign clonal expansion was observed with three separate integrations in the DNA of the cell at ACER3, RFX3, and MECOM. As of the patients Month 62 visit in March 2020, the patient remained clinically stable. Bone marrow analyses showed no dysplasia (abnormal cell growth) or molecular abnormalities.

The treatment regimen, comprising mobilization/apheresis, conditioning, and eli-cel infusion, had a safety and tolerability profile primarily reflective of the known effects of mobilization/apheresis and conditioning. In ALD-102, as previously reported, three adverse events (AE) were considered possibly related to drug product and include one serious AE (SAE), BK viral cystitis (N=1, SAE, Grade 3), and two non-serious AEs, vomiting (N=2, Grade 1). All three AEs resolved using standard measures.

ALD-104 Study

bluebird bio is currently enrolling patients for ALD-104, a Phase 3 study designed to assess the efficacy and safety of eli-cel in patients with CALD after myeloablative conditioning using busulfan and fludarabine, a different chemotherapy conditioning regimen than what is used in ALD-102 (busulfan and cyclophosphamide). The primary efficacy endpoint is the proportion of patients who are alive and free of MFDs at Month 24, and the primary safety endpoint is the proportion of patients with neutrophil engraftment after eli-cel infusion. All reported data below are as of February 2020.

In ALD-104, the 13 patients currently on study have a median of 6.1 months of follow-up to date (min-max: 2.2 10.3 months). All 13 patients achieved neutrophil engraftment and 12/13 evaluable patients had platelet engraftment (platelet engraftment pending in one patient as of data cut date). Due to the limited duration of follow-up, only safety data are being presented.

No events of acute or chronic GvHD have been reported and there have been no reports of graft failure, graft rejection, cases of insertional oncogenesis, or replication competent lentivirus.

The treatment regimen, comprising mobilization/apheresis, conditioning, and eli-cel infusion had a safety and tolerability profile primarily reflective of the known effects of mobilization/apheresis and conditioning. In ALD-104, two AEs of pancytopenia were considered possibly related to eli-cel. These two ongoing AEs were deemed as suspected unexpected serious adverse reactions (SUSARs) by the principal investigator and were diagnosed approximately two months post-eli-cel infusion in two patients (one Grade 2 and one Grade 3). An additional AE was ongoing as of February 2020, a Grade 3 SAE of transverse myelitis that was diagnosed in the presence of viral infection (adenovirus and rhinovirus/enterovirus positivity) approximately six months after eli-cel infusion and deemed unrelated to eli-cel.

eli-cel Presentation at EBMT

Lenti-D hematopoietic stem cell gene therapy stabilizes neurologic function in boys with cerebral adrenoleukodystrophy

Presenting Author: Dr. Jrn-Sven Khl, Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women's and Children's Medicine, University Hospital Leipzig Poster Session & Number: Gene Therapy; ePoster O077

Presentations will be available for virtual viewing throughout the duration of the live meeting on the EBMT 2020 website and content will be accessible online following the close of the meeting until November 1, 2020.

About elivaldogene autotemcel (eli-cel, formerly Lenti-D)

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

bluebird bio is currently enrolling patients for a Phase 3 study (ALD-104) designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Contact This email address is being protected from spambots. You need JavaScript enabled to view it. for more information and a list of study sites.

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

The Phase 2/3 Starbeam study (ALD-102) has completed enrollment.

For more information about bluebird bio-sponsored studies visit: http://www.bluebirdbio.com/our-science/clinical-trialsor clinicaltrials.gov.

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

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

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

About CALD Early Diagnosis

Early diagnosis of CALD is important, as the outcome of available treatment varies with the clinical stage of the disease. Newborn screening for ALD is a critical enabler of early diagnosis and thus of successful treatment of ALD. Once a patient has been diagnosed with ALD, regular MRI scans are critical to detect white matter changes indicative of progression to CALD.

In the U.S., newborn screening for ALD was added to the Recommended Universal Screening Panel in February 2016 and is currently active in 17 states, accounting for > 58 percent of U.S. newborns. Outside the U.S., the Minister of Health in the Netherlands has approved the addition of ALD to their newborn screening program. Even though ALD newborn screening has not been implemented in most EU countries, efforts to begin pilot programs are slowly progressing.

About bluebird bio, Inc.

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

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

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

SOURCE: bluebird bio

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bluebird bio Presents New Results from Clinical Development Program of elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for Cerebral...

Bone Marrow Stem Cells Comments Off on bluebird bio Presents New Results from Clinical Development Program of elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for Cerebral… | August 30th, 2020

This report studies the Stem Cells market size (value and volume) by players, regions, product types and end industries, history data 2013-2017 and forecast data 2018-2025; This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

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Stem cells are a class of undifferentiated cells that are able to differentiate into specialized cell types. Commonly, stem cells come from two main sources: Embryos formed during the blastocyst phase of embryological development (embryonic stem cells) and Adult tissue (adult stem cells).

Both types are generally characterized by their potency, or potential to differentiate into different cell types (such as skin, muscle, bone, etc.).

Stem Cells market, by technology, is Cell Acquisition, Cell Production, Cryopreservation, Expansion, and Sub-Culture. Stem Cell Therapy in China is not mature, so in this report we mainly cover Stem Cell Banking market.

Stem Cells market, by technology, is Cell Acquisition, Cell Production, Cryopreservation, Expansion, and Sub-Culture. Stem Cell Therapy in China is not mature, so in this report we mainly cover Stem Cell Banking market.

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Geographically, this report is segmented into several key regions, with sales, revenue, market share and growth Rate of Stem Cells in these regions, from 2013 to 2025, covering

North America (United States, Canada and Mexico)

Europe (Germany, UK, France, Italy, Russia and Turkey etc.)

Asia-Pacific (China, Japan, Korea, India, Australia, Indonesia, Thailand, Philippines, Malaysia and Vietnam)

South America (Brazil etc.)

Middle East and Africa (Egypt and GCC Countries)

The various contributors involved in the value chain of the product include manufacturers, suppliers, distributors, intermediaries, and customers. The key manufacturers in this market include

CCBC

Vcanbio

Boyalife

Beikebiotech

By the product type, the market is primarily split into

Umbilical Cord Blood Stem Cell

Embryonic Stem Cell

Adult Stem Cell

Other

By the end users/application, this report covers the following segments

Diseases Therapy

Healthcare

We can also provide the customized separate regional or country-level reports, for the following regions:

North America

United States

Canada

Mexico

Asia-Pacific

China

India

Japan

South Korea

Australia

Indonesia

Singapore

Malaysia

Philippines

Thailand

Vietnam

Rest of Asia-Pacific

Europe

Germany

France

UK

Italy

Spain

Russia

Rest of Europe

Central & South America

Brazil

Rest of Central & South America

Middle East & Africa

GCC Countries

Turkey

Egypt

South Africa

Rest of Middle East & Africa

The study objectives of this report are:

To study and analyze the global Stem Cells market size (value & volume) by company, key regions/countries, products and application, history data from 2013 to 2017, and forecast to 2025.

To understand the structure of Stem Cells market by identifying its various subsegments.

To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).

Focuses on the key global Stem Cells manufacturers, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.

To analyze the Stem Cells with respect to individual growth trends, future prospects, and their contribution to the total market.

To project the value and volume of Stem Cells submarkets, with respect to key regions (along with their respective key countries).

To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.

To strategically profile the key players and comprehensively analyze their growth strategies.

About Us:

Precision Business Insights is one of the leading market research and management consulting firm, run by a group of seasoned and highly dynamic market research professionals with a strong zeal to offer high-quality insights. We at Precision Business Insights are passionate about market research and love to do the things in an innovative way. Our team is a big asset for us and great differentiating factor. Our company motto is to address client requirements in the best possible way and want to be a part of our client success. We have a large pool of industry experts and consultants served a wide array of clients across different verticals. Relentless quest and continuous endeavor enable us to make new strides in market research and business consulting arena.

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Stem Cells Market is Expected to Thrive at Impressive CAGR by 2025 - Scientect

Skin Stem Cells Comments Off on Stem Cells Market is Expected to Thrive at Impressive CAGR by 2025 – Scientect | August 28th, 2020

Jean Chen Smith, Correspondent for Memphis Commercial Appeal Published 6:01 a.m. CT Aug. 28, 2020 | Updated 11:26 a.m. CT Aug. 28, 2020

Lily Lolo's Lip Gloss Set offers two colors perfect for day or night.(Photo: Lily Lolo)

You dont have to spend a ton of money on beauty products that are safe, effective and easy to use.With so many options in the marketplace today, it's easy to get lost among the offerings.

Here are someof our favoritebeauty finds under $25.

Beauty Society, an American skincare and cosmetics company that specializes in affordable products, was named one of 2019s top eco-friendly skincare companies because of itsrefilling and biodegradable packaging practices. Its matte liquid lipsticks ($22) are smudge free and long lasting. The unique formula prevents color from bleeding, which makes for fewer applications.

Details: beautysociety.com

Actsyl-3 ($25)is a fast-absorbing, non-greasy, formulated serum designed to improve follicle thickness and stimulate hair stem cells. The Actsyl-3 serum contains Redensyl (3%) and Capixyl (2%) two ingredients proven to help regrow hair. Key peptides also help to strengthen hair roots and improve scalp health all without affecting hormones.

Details: actsyl.com

Trinny Londons Eye2Eye / Eyeshade ($24) is a collection of flattering shades that function as both eyeshadows and eyeliners. Rich in pigment, moisturizing and easy to use, these glide on smoothly, allowing you to smudge, smoke or line your eyes. The company also carries a full line of accessories such as The Elizabeth ($20), which is a bright yellow bag for storing your makeup essentials.Fifteen percent of the proceeds for every The Elizabeth sold go to The Princes Trust, which supports the Change A Girl's Life Campaign, an initiative of Women Supporting Women.

Details: trinnylondon.com

Wonderskin Wonder Blading Lip Gloss ($22.50) delivers draw-dropping shine that makes lips look plump without a sticky or tacky feel. The high-lacquer finish can last for hours and works great over any of the companys coordinating Peel & Reveal Lip Colors.

Details: wonderskin.com

Crabtree & Evelyn's Petal Power Lip Scrub ($18) helps to cleanse and hydrate your lips using shea butter and coconut oil.It is gentle enough to use daily and great for exfoliating the dead skin from dry, chapped lips.

Details: crabtree-evelyn.com

Facial Works Sea Mist Calming Cucumber Toning Mist ($24) is the perfect travel companion.This is a soothing and calming toner providing antioxidant benefits from CleanSea Complex and is packed with Sea Whip, an anti-inflammatory ingredient from the Caribbean Sea. This 1-ounce bottle is travel-sized, so you can bring it with you anywhere. A full-size bottle is also available.

Details: thefacialworks.com

Beautycounter Countersun Mineral Sunscreen Stick will keep you protected.(Photo: Beautycounter)

Refresh and renew with Beautycounters Citrus Mimosa Body Wash ($25), a gentle and moisturizing cleanser that energizes with a citrus scent. Certified vegan and cruelty-free, this will leave your skin feeling soft, clean and hydrated.Dont forget the sunscreen!The Countersun Mineral Sunscreen Stick SPF 30C ($21) comes in a convenient stick form with plenty of protection. Water-resistant and formulated with non-nano zinc and California poppy, the SPF 30 formula provides an effective shield from UVA and UVB rays, while moisturizing with cocoa butter, without leaving a white residue on your skin. Best of all, it is reef-friendly and water-resistant for up to 80 minutes.

Details: beautycounter.com

These Purifying Facial Towelettes From Rooted Beauty will keep you refreshed.(Photo: Rooted Beauty)

On the Grove Collaborative website, you can find affordable beauty products that adhere to the highest standards.The site prioritizes products made with sustainable, plant-based ingredients that arent tested on animals. Each brand must meet strict guidelines regarding safety, ethics, environmental impact and animal welfare. Purifying Facial Towelettes ($6.95) from Rooted Beauty will keep you refreshed throughout the day. They remove dirt, oiland makeup with free-radical-fighting white tea, calming calendula and the R7 complex, which contains antioxidant-rich roots.Throw Lily Lolos Lip Gloss Set ($25) into your bag the colors are perfect for both day and night.

Details: grove.co/roven

Sjal Skincare's Balans is a gentle pore cleanser.(Photo: Sjal Skincare)

Sjal Skincare Balans ($75) is a lightweight deep pore cleanser that hydrates, detoxifies and protects the skin. This powerhouse cleaner contains potent ingredients such as pearl extract for brightening, African whitewood extract and fig leaf extract, a natural barrier to protect the skin against water loss.

Details: sjalskincare.com/products/balans

Westmore Beauty-Skin Conditioning Exfoliator ($39) is a creamy exfoliator packed with moisturizing ingredients like shea butter and squalene. The oil-free formula contains gentle exfoliating beads to instantly smooth away dirt and dry skin, making your skin feel soft and silky. Fragrance-free and made without the use of parabens, sulfates or phthalates, this is something you will want to put on your skin.

Details:westmorebeauty.com

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Here are some must-try beauty products for $25 or less (and a couple splurges) - Commercial Appeal

Skin Stem Cells Comments Off on Here are some must-try beauty products for $25 or less (and a couple splurges) – Commercial Appeal | August 28th, 2020

Photo: Cichlid fish, by Russell D. Fernald and Sabrina S. Burmeister / CC BY (https://creativecommons.org/licenses/by/2.5).

Anarticle yesterdayforEvolution Newsabout allostery showed how an individual protein or RNA can send information to its distant domains. Information sharing can also occur between chains of molecules arranged in a signaling cascade, where each one triggers action in the next. This is a bit more like the Rube Goldberg technique, except that in cells, it is much more logical and reliable. Here are new examples ofmechanosensing(the ability to sense a touch) andmechanotransduction(the ability to pass on touch information). A paper onbioRxivexplains, Cells sense the physical properties of their environment, translate them into biochemical signals and adapt their behaviour accordingly.

One such system is the MAPK/ERK pathway that all eukaryotic cells use to get information from the cell surface into the nucleus. A diagram onWikipedias page makes it clear that many individual factors take part. Once the EGFR receptor triggers ERK on the cells exterior membrane, a signaling cascade begins with at least 16 cofactors and proteins transporting the information to the cell nucleus, which responds by transcribing code proteins or enzymes. ERK signals can also spread throughout the cytoplasm, leading to a variety of responses depending on the nature of the triggering molecule.

Now, Japanese scientists have noticed a further response in neighboring cells. When one cells ERK pathway is triggered, that cell shrinks. Neighboring cells sense the change and respond by shrinking themselves, causing a chain reaction.Researchers at Kyoto Universitylikened this to how crowds do The Wave at sporting arenas, passing collective motion throughout the stadium.

Cells are tightly connected and packed together, so when one starts contracting from ERK activation, it pulls in its neighbors, elaborates [Tsuyoshi] Hirashima. This then caused surrounding cells to extend, activating their ERK, resulting in contractions thatlead to a kind of tug-of-war propagating into colony movement.[Emphasis added.]

The response involves both chemical and mechanical factors. Our work clearly shows that the ERK-mediatedmechano-chemical feedback systemgenerates complicated multicellular patterns, the lead author comments.

Another touch-sensitive mechanism is the so-called Hedgehog (Hh) pathway, so named because defects in its function cause fruit fly embryos to look like the spiny animals. Hedgehog pathways are often associated with the primary cilium, an organelle that sticks out like an antenna from the cell membrane and senses its environment. When triggered, it also causes a cascade of reactions inside the cell.

Craig Albertson, a researcher at theUniversity of Massachusetts, Amherstwas curious why cichlid fish can evolve so quickly to environmental changes, including changing the shapes and densities of bone in their jaws. This capacity for phenotypic plasticity is not evolution of a Darwinian kind, but rather a programmed response to environmental cues.

Albertson works with a system cichlid fishes known throughout the scientific world as champions of phenotypic plasticity thatcan alter, in a single season, jawbone hardness or shape to match feeding conditions.They are also well known for their rapid evolution and diversity in jaw shapes, which hasenabled cichlids to adapt to many different food sources, including algae, plankton, fish, snails and even the scales of other fishes.

Albertson speculated that this capacity for rapid response to environmental cues might be associated with the Hedgehog signaling pathway. By tuning the amount of Hh signal, his research team discovered that more bone was deposited, or vice versa.

Albertson, explains, Bone cells in these fish are innatelysensitive to differentmechanical environments. But we were able to play with this system using a single molecular switch you turn up the Hh signal and the cells become more sensitive to the environment, or you turn the molecular sensor down and the cells become almost deaf to the environment.

Like ERK, the Hedgehog signaling pathway involves numerous factors that interact in chain reactions. And it is triggered by a mechano-sensor on the cell, the primary cilium.

An important clue came as Albertson learned more about how this molecular pathway works. He explains, There isa well-known mechano-sensor on most cells, including those that make the skeleton, called the primary cilium. Cells that lack this organelle are unable to sense or respond to environmental input, includingmechanical load.It turns out that several key protein components of the Hedgehog pathway are physically associated with this structure, making it an obvious candidate for an environmentally sensitive signal.

The team believes this kind of response to environmental cues could be responsible for other kinds of rapid evolution in other animals. The Hh signal has also been shown to regulate plasticity inbeetle horns, so there may be something special that positions it to be anenvironmental sensor across tissues and animals, Albertson says. This is not Darwinism; it is pre-programmed response using molecular machines capable of sensing touch.

How does skin stretch when a body grows? The answers may rely on mechanosensitive factors.Nature News and Views said recently, Stretching the skin of mice reveals thatmechanical strain is communicated by a subpopulation of stem cellsthat proliferate and promote mechanical resistance,and so generate extra skin.

One of the most remarkable examples of touch communication was announced this month inNature. Researchers at theUniversity of Montrealconfirmed the existence of nanotubes that grow out of cell membranes and reach across comparatively large distances to touch other cells, affect their behaviors, and even share organelles with them. They found these nanotubes in the retinas of mice, and believe they are responsible for controlling blood flow in the capillaries.

For the first time, we have identifieda communication structure between cellsthat is required to coordinate blood supply in the living retina, said Dr. Adriana Di Polo, a neuroscience professor at Universit de Montral and holder of a Canada Research Chair in glaucoma and age-related neurodegeneration, who supervised the study.

We already knew that activated retinal areas receive more blood than non-activated ones, she said, butuntil now no one understood how this essential blood delivery was finely regulated.

These nanotubes tunnel through the mass of retinal cells to distant capillaries, where they contact pericytes, cells that have the ability to control the amount of blood passing through a single capillary simply by squeezing and releasing it. This touch communication allows a retinal cell to tell the capillary it needs more blood or less blood.

Using a microscopy technique to visualize vascular changes in living mice, we showed that pericytes project very thin tubes, calledinter-pericyte tunnelling nanotubes, tocommunicate with other pericytes located in distant capillaries, said Alarcon-Martinez. Through these nanotubes,the pericytes can talk to each other to deliver blood where it is most needed.

Video micrographs show that even mitochondria can be passed down these nanotubes. The paper inNaturesays:

Here we identify nanotube-like processes that connect two bona fide pericytes on separate capillary systems, forming a functional network in the mouse retina, which we named interpericyte tunnelling nanotubes (IP-TNTs). We provide evidence that these (i) have an open-ended proximal side and a closed-ended terminal (end-foot) that connects with distal pericyte processes via gap junctions, (ii)carry organelles including mitochondria, which can travelalong these processes, and (iii) serve as a conduit for intercellular Ca2+waves, thusmediating communication between pericytes.

The cells literally reach out and touch other pericytes bound to other capillaries, and hand off signals and organelles. This gives the retinal cells, dependent on a steady supply of oxygen and nutrients, a way to fine-tune their own blood supply. The gap junctions act like filters: Small particles, such as ions, can pass through this junction, but larger objects, such as organelles, cannot. Tunneling nanotubes had been noted between cells in a petri dish before, but a companion article onNature News and Viewscalls this the firstin vivoevidence for the existence of a type of TNT-like protrusion. Maybe it wont be the first for long. The research teams headline calls them, Nanotubes in the eye that help us see.

These are just some of the ways that cells respond to mechanical forces. The chains of reactions can be very elaborate and irreducibly complex. But first, they have to be triggered by well-designed mechanosensors that can feel a factor in the environment and then pass along that information to downstream processes that can do something about it. Undoubtedly many more examples of mechanosensing and mechanotransduction remain to be discovered. Its hard to conceive of any of these systems arising piecemeal by accumulated mistakes (mutations).

Instead, they appear as systems of coordinated parts that were planned to adapt to changes, providing robustness. It is exciting to ponder how such pre-programmed responses to environmental cues could trigger rapid adaptations, giving rise to some of the spectacular variations seen in birds, beetles, fish, and other organisms. Prematurely attributed to Darwinian processes, these examples of phenotypic plasticity actually show foresight and design.

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Cells Reach Out and Touch, Providing Evidence of Foresight and Design - Discovery Institute

Skin Stem Cells Comments Off on Cells Reach Out and Touch, Providing Evidence of Foresight and Design – Discovery Institute | August 28th, 2020

MIKE TYSON has revealed he was injected with nearly-translucent blood in his bid to make a comeback... and the former heavyweight champ said it made him feel "weird".

The 54-year-old - who initially retired from boxing in 2005 - will fight Roy Jones Jr in November in his eagerly-anticipated comeback fight.

2

His return to action has been aided by stem-cell research therapy, which has left him feeling like a "different person".

In May, Tyson claimed: "You know what I had done? I had stem-cell research therapy.

"I feel like a different person but I can't comprehend why I feel this way.

"It's really wild what scientists can do."

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition that usually takes the form of a bone marrow transplantation.

Tyson opened up on the effects the treatment has had on him in an recent interview with rapper LL Cool J on the Rock the Bells Radio show on SiriusXM, earlier in 2020.

Commenting on the mental aspect of training for a fight for the first time in 15 years, he said: "My mind wouldnt belong to me.

"My mind would belong to somebody that disliked me enough to break my soul, and I would give them my mind for that period of time.

"Six weeks of this and Id be in the best shape Ive ever dreamed of being in. As a matter of fact, Im going through that process right now. And you know what else I did, I did stem-cell research."

Tyson was then asked whether that meant if his white blood had been spun and then put back in, to which he replied: "Yes. As they took the blood it was red and when it came back it was almost transfluid (sic).

"I could almost see through the blood, and then they injected it in me.

"And Ive been weird ever since, Ive got to get balanced now."

2

Exclusive

BOIS POISEDDubois confident he can take over soon after shock KOs for Joshua and Whyte

HALl-MARKHow Eddie Hall is transforming into boxer for Thor fight with intense training

WEIGH TO GOConor Benn tips 'animal' Vergil Ortiz to surpass Spence and Crawford

NOT FINE WITH ITDelfine Persoon now claims she deserved to beat Katie Taylor

BROTHERLY GLOVETyson Fury tips Tommy to go 'all the way' as brothers share training vid

FUR SUREDubois tips Fury to beat Joshua having sparred with BOTH heavyweight champs

WHAT IS STEM CELL TREATMENT USED FOR?

Stem cell transplants are carried out when bone marrow is damaged or isnt able to produce healthy blood cells.

It can also be used to replace damaged blood cells as the result of intensive cancer treatment.

Here are conditions that stem cell transplants can be used to treat:

Iron Mike had been called out by former rival Evander Holyfield to complete their trilogy following their two meetings in 1990s.

But he has since looked elsewhere, taking on Jones Jr later this year - potentially in front of a packed house.

Tyson is looking in incredible condition, too as he continues this hard graft.

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Mike Tyson reveals all after doctors gave him blood injection that left him feeling weird during stem cell t - The Irish Sun

Bone Marrow Stem Cells Comments Off on Mike Tyson reveals all after doctors gave him blood injection that left him feeling weird during stem cell t – The Irish Sun | August 28th, 2020

IndustryGrowthInsights (IGI), a prominent market research firm in its own industry, has published a detailed report on Global Hematopoietic Stem Cell Transplantation (HSCT) Market. This market research report provides comprehensive and in-depth analysis on the market which can possibly help an enterprise to identify lucrative opportunities and assist them with fabricating creative business strategies. The market report provides information about the current market scenario regarding the global supply and demand, key market trends and opportunities in the market, and challenges and threats faced by the industry players.

The Hematopoietic Stem Cell Transplantation (HSCT) market report talks about the competitive scenario among the industry players and imparts aspiring and emerging industry players with the future market insights in a detailed manner. This market report includes crucial data and figures which are structured out in a concise yet understandable manner. The research report covers the updates on the government regulations and policies which illustrates key opportunities and challenges of the market. IndustryGrowthInsights (IGI) has been monitoring the market since few years and collaborated with eminent players of the industry to give better insights on the market. It has conducted vigorous research and implied robust methodology to provide accurate predictions about the market.

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Impacts of Advancements and COVID-19 on the market.

Amidst the COVID-19, few segments of the market have witnessed a disruption due to the gap in supply and demand which has impacted the growth of the Hematopoietic Stem Cell Transplantation (HSCT) market. Along with this, the latest advancements have changed the market dynamics of the market. This research report covers the wide-range analysis of the COVID-19 impact to the industry and gives out insights on the change in the market scenario due to the advancements.

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Market Segmentation

Some of the major companies that are covered in the report.

Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord Inc

Note: Additional companies

Based on the type, the market is segmented into

AllogeneicAutologous

Based on the application, the market is segregated into

Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)

Based on the geographical location, the market is segregated into

Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa

IndustryGrowthInsights (IGI) provides yearly updates on the Hematopoietic Stem Cell Transplantation (HSCT) market that assist the clients to stay ahead in the competitive space.

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The market research report provides all valuable constituents of the market such as revenue growth, product pricing & analysis, growth potential, and guidelines to tackle the challenges in the market. The report covers all the crucial mergers & acquisitions, partnerships, and collaborations that created further created opportunities or in some cases, challenges for the industry players.

This report includes latest product news, advancements, and updates from the prominent player of the industry that has leveraged their position in the market. It also provides business strategies implemented by the key players and yardstick to arrive on informed business decisions. Moreover, it gives insights on the consumer behavior patterns that can help the enterprise to curate the business strategies accordingly.

IndustryGrowthInsights (IGI) bestows the clients with the specialized customized options related to the regional analysis, company analysis, and product analysis, among others.

Complete Table Content of the Market

Executive Summary

Assumptions and Acronyms Used

Research Methodology

Hematopoietic Stem Cell Transplantation (HSCT) Market Overview

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Type

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Application

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Sales Channel

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Region

North America Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast

Latin America Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast

Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast

Asia Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast

Asia Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Size and Volume Forecast by Application

Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast

Competition Landscape

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Global Hematopoietic Stem Cell Transplantation (HSCT) Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts...

Bone Marrow Stem Cells Comments Off on Global Hematopoietic Stem Cell Transplantation (HSCT) Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts… | August 28th, 2020

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Gene in fruit flies, worms, zebrafish, mice and people may help explain some fertility issues

Researchers at Washington University School of Medicine in St. Louis have identified a gene that plays an important role in fertility across multiple species. Pictured is a normal fruit fly ovary (left) and a fruit fly ovary with this gene dialed down (right). Male and female animals missing this gene had substantially defective reproductive organs. The study could have implications for understanding human infertility and early menopause.

A new study from Washington University School of Medicine in St. Louis identifies a specific genes previously unknown role in fertility. When the gene is missing in fruit flies, roundworms, zebrafish and mice, the animals are infertile or lose their fertility unusually early but appear otherwise healthy. Analyzing genetic data in people, the researchers found an association between mutations in this gene and early menopause.

The study appears Aug. 28 in the journal Science Advances.

The human gene called nuclear envelope membrane protein 1 (NEMP1) is not widely studied. In animals, mutations in the equivalent gene had been linked to impaired eye development in frogs.

The researchers who made the new discovery were not trying to study fertility at all. Rather, they were using genetic techniques to find genes involved with eye development in the early embryos of fruit flies.

We blocked some gene expression in fruit flies but found that their eyes were fine, said senior author Helen McNeill, PhD, the Larry J. Shapiro and Carol-Ann Uetake-Shapiro Professor and a BJC Investigator at the School of Medicine. So, we started trying to figure out what other problems these animals might have. They appeared healthy, but to our surprise, it turned out they were completely sterile. We found they had substantially defective reproductive organs.

Though it varied a bit by species, males and females both had fertility problems when missing this gene. And in females, the researchers found that the envelope that contains the eggs nucleus the vital compartment that holds half of an organisms chromosomes looked like a floppy balloon.

This gene is expressed throughout the body, but we didnt see this floppy balloon structure in the nuclei of any other cells, said McNeill, also a professor of developmental biology. That was a hint wed stumbled across a gene that has a specific role in fertility. We saw the impact first in flies, but we knew the proteins are shared across species. With a group of wonderful collaborators, we also knocked this gene out in worms, zebrafish and mice. Its so exciting to see that this protein that is present in many cells throughout the body has such a specific role in fertility. Its not a huge leap to suspect it has a role in people as well.

To study this floppy balloon-like nuclear envelope, the researchers used a technique called atomic force microscopy to poke a needle into the cells, first penetrating the outer membrane and then the nucleuss membrane. The amount of force required to penetrate the membranes gives scientists a measure of their stiffness. While the outer membrane was of normal stiffness, the nucleuss membrane was much softer.

Its interesting to ask whether stiffness of the nuclear envelope of the egg is also important for fertility in people, McNeill said. We know there are variants in this gene associated with early menopause. And when we studied this defect in mice, we see that their ovaries have lost the pool of egg cells that theyre born with, which determines fertility over the lifespan. So, this finding provides a potential explanation for why women with mutations in this gene might have early menopause. When you lose your stock of eggs, you go into menopause.

On the left is a normal fruit fly ovary with hundreds of developing eggs. On the right is a fruit fly ovary that is totally missing the NEMP gene. It is poorly developed and no eggs are visible.

McNeill and her colleagues suspect that the nuclear envelope has to find a balance between being pliant enough to allow the chromosomes to align as they should for reproductive purposes but stiff enough to protect them from the ovarys stressful environment. With age, ovaries develop strands of collagen with potential to create mechanical stress not present in embryonic ovaries.

If you have a softer nucleus, maybe it cant handle that environment, McNeill said. This could be the cue that triggers the death of eggs. We dont know yet, but were planning studies to address this question.

Over the course of these studies, McNeill said they found only one other problem with the mice missing this specific gene: They were anemic, meaning they lacked red blood cells.

Normal adult red blood cells lack a nucleus, McNeill said. Theres a stage when the nuclear envelope has to condense and get expelled from the young red blood cell as it develops in the bone marrow. The red blood cells in these mice arent doing this properly and die at this stage. With a floppy nuclear envelope, we think young red blood cells are not surviving in another mechanically stressful situation.

The researchers would like to investigate whether women with fertility problems have mutations in NEMP1. To help establish whether such a link is causal, they have developed human embryonic stem cells that, using CRISPR gene-editing technology, were given specific mutations in NEMP1 listed in genetic databases as associated with infertility.

We can direct these stem cells to become eggs and see what effect these mutations have on the nuclear envelope, McNeill said. Its possible there are perfectly healthy women walking around who lack the NEMP protein. If this proves to cause infertility, at the very least this knowledge could offer an explanation. If it turns out that women who lack NEMP are infertile, more research must be done before we could start asking if there are ways to fix these mutations restore NEMP, for example, or find some other way to support nuclear envelope stiffness.

This work was supported by the Canadian Institutes of Health, research grant numbers 143319, MOP-42462, PJT-148658, 153128, 156081, MOP-102546, MOP-130437, 143301, and 167279. This work also was supported, in part, by the Krembil Foundation; the Canada Research Chair program; the National Institutes of Health (NIH), grant number R01 GM100756; and NSERC Discovery grant; and the Medical Research Council, unit programme MC_UU_12015/2. Financial support also was provided by the Wellcome Senior Research Fellowship, number 095209; Core funding 092076 to the Wellcome Centre for Cell Biology; a Wellcome studentship; the Ontario Research FundsResearch Excellence Program. Proteomics work was performed at the Network Biology Collaborative Centre at the Lunenfeld-Tanenbaum Research Institute, a facility supported by Canada Foundation for Innovation funding, by the Ontarian Government, and by the Genome Canada and Ontario Genomics, grant numbers OGI-097 and OGI-139.

Tsatskis Y, et al. The NEMP family supports metazoan fertility and nuclear envelope stiffness. Science Advances. Aug. 28, 2020.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

Original post:
Genetic mutations may be linked to infertility, early menopause - Washington University School of Medicine in St. Louis

Bone Marrow Stem Cells Comments Off on Genetic mutations may be linked to infertility, early menopause – Washington University School of Medicine in St. Louis | August 28th, 2020

A familys wishes have come true after an inspirational search for a bone marrow donor for Darcy Keeley, four, uncovered a match.

The young Perth boy was diagnosed in June with acute myeloid leukaemia, needing a bone marrow transplant.

Finding a match proved difficult and sparked the campaign Do it for Darcy.

His family took to social media, relentless in their pursuit to help their little boy.

When the story of the boy dubbed the little lion started to spread, thousands of people came forward to try to lend a hand.

Recently one of our most loyal, passionate and enthusiastic supporters, Darcy Keeley, was diagnosed with Leukaemia, requiring 6 months of intensive chemotherapy followed by a bone marrow transplant, Hamersley Carine Footy Club posted on GoFundMe in July.

On their campaign Do it for Darcy, the family explained he was in isolation in Perth Childrens Hospital.

He needs a bone marrow donation and currently the national and international bone marrow registry is yet to find one, they wrote.

Thousands of people rallied around Darcy and his family, signing up to see if they were a potential match to be a donor.

On Thursday, his mum Casey announced in a heartfelt social media post they finally had a match.

IT HAPPENED... guys it actually happened!!! We got the match weve been waiting for! she said.

Darcys transplant doctor confirmed yesterday we have stem cells from some cord blood!

This is a huge relief and allows us to focus on the next step to recovery.

She said an employee at Lifeblood said literally thousands of people registered to try to help Darcy.

Casey also added that the campaign had subsequently led to many new donors.

There have been literally THOUSANDS of ABMDR [Australian Bone Marrow Donor Registry] registrations and new donors as a result of our Do it for Darcy campaign, she said.

The mum said this would help people all around the world, adding they were proud of the awareness the campaign had created.

It is impossible to express our gratitude for all of you who have rallied behind us and registered to be bone marrow donors, donated blood products and spread the word, she said.

As parents, there is nothing more heartwarming than people supporting your child, especially in a time of need.

And the love we have felt wrapped around us [has] helped us as a family immensely.

The loving mother conceded that while they are only at the beginning of fighting this illness, this win will help them move forward.

She also encouraged people to keep spreading awareness.

This is not the end of the fight, Casey said.

Its really just the beginning.

The transplant process will be challenging, but for right now lets focus on this win.

Continued here:
'We got the match': Happy news for family fighting four-year-old's Darcy Keeley's leukaemia - 7NEWS.com.au

Bone Marrow Stem Cells Comments Off on ‘We got the match’: Happy news for family fighting four-year-old’s Darcy Keeley’s leukaemia – 7NEWS.com.au | August 28th, 2020