In an August 28 special issue of the peer-reviewed online journal OBM Transplantation, stem cell biotechnology company Asymmetrex has now published a report describing how its technology for determining the specific dosage of therapeutic tissue stem cells works. The new technology is poised to revolutionize stem cell science and stem cell medicine by giving the long-needed means to quantity their essential focus, tissue stem cells.

BOSTON, Sept. 1, 2020 /PRNewswire-PRWeb/ --Stem cell biotechnology company, Asymmetrex, has been counting tissue stem cells like those used for bone marrow and cord blood transplantation therapies for a few years now. Recently, the company announced the issue of patents for its first-in-kind technology both in the U.S. and the U.K. However, until last Friday, August 28, Asymmetrex had not reported in the peer-reviewed academic literature how it achieves this feat that had been pursued by many distinguished labs for more than six decades.

Now in a report published in a special issue of OBM Transplantation, a peer-review journal for transplantation medicine research, Asymmetrex completes its introduction of the new technology to the fields of stem cell science and stem cell medicine. The report is the second invited article published in a special issue focused on the "Isolation and Characterization of Adult Therapeutic Cells."

The new report describes Asymmetrex's discovery of mathematical formulas, call algorithms, that can be used to determine the number of stem cells in complex tissue cell preparations, like experimental samples or patient treatments. The stem cell counting algorithms are specific for different types of tissue stem cells. So, the algorithms defined for blood stem cells are distinct from the algorithms for liver stem cells, or lung stem cells. Once an algorithm is defined by the Asymmetrex technology, it can be used repeatedly as a simple, rapid, and inexpensive test to determine the quantity and dosage of its specific tissue stem cell type.

Asymmetrex's founder and director, James L. Sherley, M.D., Ph.D., anticipated the August publication of the new algorithms in a talk given earlier at the 6th Annual Perinatal Stem Cell Society Congress in March of this year. Then and now, he says that he believes, "Now that the tissue stem cell counting algorithms are available, everything will change" in stem cell science and medicine.

Prior to Asymmetrex's technology, there was no method for counting tissue stem cells in research, medicine, or for any other of their many uses. So, the impact of the stem cell counting algorithms in research and medicine is far-reaching. Such information is a game changer for accelerating progress in stem cell science and stem cell medicine, including improving treatments like gene therapy whose success depends on targeting tissue stem cells. There will also be tremendous gains in cell biomanufacturing, drug development, and environmental toxicology, all whose capabilities are currently limited by the lack of a facile means to quantify tissue stem cells.

To make the new counting technology readily accessible for evaluation by the greater academic, medical, and industrial stem cell communities, Asymmetrex provides free tissue stem cell counting on its company website.

About Asymmetrex

Asymmetrex, LLC is a Massachusetts life sciences company with a focus on developing technologies to advance stem cell medicine. The company's U.S. and U.K. patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of effective use of human adult tissue stem cells for regenerative medicine and drug development. Asymmetrex markets the first technology for determination of the dose and quality of tissue stem cell preparations (the "AlphaSTEM Test") for use in stem cell transplantation therapies and pre-clinical drug evaluations. Asymmetrex is a member company of the Advanced Regenerative Manufacturing Institute BioFabUSA and the Massachusetts Biotechnology Council.

SOURCE Asymmetrex, LLC

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New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem...

Bone Marrow Stem Cells Comments Off on New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem… | September 1st, 2020

Evie Hodgson, 8, was due to undergo a bone marrow transplant this month, but her only match in the world pulled out at the last minute (Picture: North News)

A little girl battling a rare blood disorder has issued a desperate plea for donors after her only match in the world pulled out at the last minute.

Evie Hodgson, 8, was due to undergo a bone marrow transplant this month but her donor cancelled just as she was getting prepared.

Mum Tina, of Whitby, North Yorkshire, said the chances of finding another donor are so slim doctors are planning a different course of treatment.

But the best hope Evie has of being cured is to find a stem cell transplant from a compatible donor.

The schoolgirl was diagnosed with deadly aplastic anaemia in May after she developed a pin prick rash on her back which didnt fade.

Tests revealed she had low blood platelet levels, which medics initially thought might be leukaemia but a biopsy found no cancerous cells.

They were then given the devastating diagnosis and Evie was told she would need a bone marrow transplant.

Tina, 37, dad Andy, 49, and five-year-old brother William, were sadly not a match so a worldwide search was launched to find a donor.

To the familys delight, a 10/10 match was found, with the anonymous donor agreeing to go ahead.

The family began to prepare for the transplant, including dental work and the removal of one of her ovaries, but to their horror, on August 14, they were told the donor had pulled out.

Tina, who works at RAF Flying Dales, said: We were devastated, it was a huge blow. We have no idea why the donor changed their mind. Everything is confidential.

Evie has already been through so much. She thought she had a donor and now she doesnt.

The donor pulling out is quite hard hitting but from our point of view we just want to raise awareness of the stem cell register.

Its so easy to be a donor. Its just like giving blood, but you could save a childs life. Some people dont even know they could be a match.

Its so easy to join the register but only about 1% of the UK population is registered.

Evie is set to undergo an immunosuppressant course of treatment while the search for a donor continues.

Her family have set up a Facebook group to raise awareness and to update progress on Evies journey.

Tina added: The condition Evie has is life-threatening. She wont survive without a transplant.

Thats why we are desperately appealing for any many people as possible to register as a stem cell donor.

Evie said: I need this transplant to save my life. Please sign the register to help save my life.

Get in touch with our news team by emailing us atwebnews@metro.co.uk.

For more stories like this,check our news page.

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Girl's only bone marrow donor in the world pulls out at last moment - Metro.co.uk

Bone Marrow Stem Cells Comments Off on Girl’s only bone marrow donor in the world pulls out at last moment – Metro.co.uk | September 1st, 2020

The parents of a girl battling a deadly blood disorder are begging people to join the stem cell donor register to save her life after her only match in the world pulled out at the last minute.

Evie Hodgson, eight, who suffers from aplastic anaemia, was due to have a bone marrow transplant this month but her donor backed out at the last possible moment.

Her mum, Tina, says the chances of finding another donor are so slim that doctors are now planning a different course of treatment. But, in future, a stem call transplant is Evies best hope of being cured.

The schoolgirl, from Whitby, North Yorks, was first taken to hospital with a rash and was diagnosed with aplastic anaemia in May.

After a global donor search was launched, a 10/10 match was found and the anonymous donor agreed to the procedure. In preparation, Evie had to have dental work and one of her ovaries was removed. But on August 14 the donor pulled out.

Tina, 37, who works at RAF Flyingdales, in Pickering, North Yorks, said: We were devastated, it was a huge blow. We have no idea why the donor changed their mind. Evie has already been through so much. She thought she had a donor and now she doesnt.

The donor pulling out is quite hard-hitting, but we want to raise awareness of the stem cell register. Its so easy to be a donor. Its just like giving blood, but you could save a childs life. Its so easy to join but only 1% of the UK population is registered.

Evie said: I need this transplant to save my life. Please sign the register to help.

Tina added: The condition Evie has is life-threatening. She wont survive without a transplant. We are desperately appealing for people to sign the stem cell register.

Evie was diagnosed with the condition after she developed a pin-prick rash on her back, which didnt fade. Tests revealed she had low blood platelet levels and she was told she needed a bone marrow transplant.

Aplastic anaemia is a rare life-threatening condition where the bone marrow fails to produce enough blood cells. Around 100-150 people are diagnosed in the UK each year.

Treatment can include immunosuppressants, chemotherapy, blood transfusions, or blood and bone marrow transplants.

Neither Tina, dad Andy, 49, or brother William, five, were a match and so an international search was launched.

Tina said: Our world crumbled when Evie was diagnosed. Evie knew shed need chemotherapy. She donated her hair to The Little Princess Trust, after making friends with poorly children who have lost all their hair.

Evie will be treated with immunosuppressants while the search for a donor continues.

Blood cancer charity Anthony Nolan is looking for stem cell donors between the ages of 16-30.

Research shows that younger donors result in better outcomes for patients.

To find out how to donate click here.

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Parents plea for stem cell help to save life of daughter with rare blood disorder - Mirror Online

Bone Marrow Stem Cells Comments Off on Parents plea for stem cell help to save life of daughter with rare blood disorder – Mirror Online | September 1st, 2020

THE family of a girl battling a deadly blood disorder are pleading for help after her only donor match in the world pulled out at the last minute.

Evie Hodgson, eight, who suffers from aplastic anaemia - also known as bone marrow failure - was due to have a transplant this month but her donor backed out.

4

Her mum, Tina Hodgson, says the chances of finding another donor are so slim that doctors are now planning a different course of treatment.

The youngster from Sleights, Whitby, was rushed to hospital with a rash during the coronavirus lockdown.

She was diagnosed with aplastic anaemia in May.

Amazingly, a match was found and preparations were underway for surgery.

But just weeks before the transplant, the donor pulled out and the family's "world fell apart, again".

However, the courageous pupil at Fyling Hall School, in Robin Hoods Bay, is determined to battle the disease.

Evie's mum told how the family received the diagnosis: "In the middle of the Covid lockdown, Evie woke up one morning with a red pin rash all over her body.

"We first thought it was meningitis because it wasn't fading under a tumbler but she had no other symptoms."

The family contacted the NHS 111 service and were advised to take Evie to Whitby A&E.

"The doctor took one look at her and thought it was sepsis," Tina said.

"She had IV antibiotics there and we were rushed to James Cook University Hospital by ambulance."

4

Evie then had blood tests at the Middlesbrough hospital which revealed her platelets were low - but her red and white blood cells were fine.

She was diagnosed with a platelet condition - known as ITP - and told she would need to return to hospital once per week for tests.

But when the family returned to Teesside one week later, tests revealed all Evie's cell counts were low.

The youngster was then immediately transferred to Great North Childrens Hospital in Newcastle.

Tina, who works at RAF Fylingdales, said: "Deep down we thought this is quite serious and I think everyone though it was leukaemia."

In May, further tests revealed that Evie didn't have cancerous cells - and not many cells at all - as doctors broke the devastating news that Evie was suffering from aplastic anaemia.

4

As the family received the diagnosis, Tina said: "As parents we said thank god it isn't cancer but then the nurse said this is just as bad, it is a lot harder to treat.

"Our world fell apart at that point."

Aplastic anemia is a condition that occurs when your body stops producing enough new blood cells.

Around 100-150 people are diagnosed in the UK each year.

Treatment can include immunosuppressants, chemotherapy, blood transfusions, or blood and bone marrow transplants.

Evie's first option was a bone marrow transplant as a cure, but mum Tina, dad Andy and Evies five-year-old brother William were not a match.

The next step was to search the international database for a bone marrow transplant.

And luckily, Evie's perfect donor - the only one in the world - was found and preparations for surgery were scheduled.

Evie had one of her ovaries removed, dental work carried out and even chopped off her Rapunzel-like locks to donate to The Little Princess Trust.

Speaking through tears about her brave little girl, Tina, 37, said: "Evie was always that girl at the hairdressers that wanted her hair like Rapunzel.

"When we've been in the hospital she has saw her friends with no hair.

"One day she said to me 'will I lose my hair?'. So I said probably, yes."

Evie then made the courageous decision to chop off her hair and is now sporting a beautiful curly bob despite fighting her own battle.

The brave youngster, who is also a keen singer, has also been entertaining other patients and staff in the hospital with her talents.

The family's second devastating blow came when Evie was in hospital and doctors broke the news the donor had pulled out.

4

"It was like the diagnosis all over again. We will never know why they pulled out," Tina added.

Evie is due to start a three-month immunosuppressive therapy treatment which aims for her body to generate bone marrow.

As the treatment has only a 60 per cent success rate, Evie's family are now raising awareness about joining the stem cell donation register.

Speaking about the importance of signing up, Tina said: "I was shocked to learn that only 2 per cent of the UK population is registered on the blood stem cell register.

"It is such an easy procedure, similar to giving blood.

"It is so easy to get on the register and donate if you are a match.

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"If people are made aware they could save someone's life. It isn't just Evie, there are so many families waiting for a donor."

You can join on the DKMS register, here, or through Anthony Nolan register, here.

A dedicated Facebook page has been set up to follow Evie's journey with aplastic anaemia - you can follow her progress here.

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Familys plea to help save girl, 8, after her only donor match in the world pulls out at the last minute - The Sun

Bone Marrow Stem Cells Comments Off on Familys plea to help save girl, 8, after her only donor match in the world pulls out at the last minute – The Sun | September 1st, 2020

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and Metagenomi, a gene editing company discovering breakthrough systems for curing genetic disease, today announced that Vor will evaluate the potential use of Metagenomis gene editing technology to develop engineered hematopoietic stem cell-based therapies for the treatment of blood cancers, such as acute myeloid leukemia.

Cancer patients deserve therapies with strong effects on cancer cells and minimal effects on all other cells, said Tirtha Chakraborty, Ph.D., Vors VP and Head of Research. Our new partnership with Metagenomi will help us achieve this goal by engineering hematopoietic stem cells using precise yet flexible gene editing thereby ensuring that targeted therapies can live up to their name."

The collaboration is non-exclusive and applies to pre-clinical research only. Further terms of the agreement are not being disclosed.

This partnership unites two transformative technologies our proprietary gene editing enzymes, and Vors platform for engineering hematopoietic stem cells such that they are inherently treatment-resistant, said Brian C. Thomas, Metagenomis CEO and co-founder. We are excited to be working together to bring both of these cutting-edge approaches into the clinic.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

About Metagenomi

Metagenomi is harnessing the vast information found in life on Earth to develop cures for genetic disease. Using proprietary data collected from around the world, Metagenomi has developed novel gene editing tools that enable next-generation gene and cell therapies.

Metagenomi is based out of Emeryville, California, and was founded by pioneers in the field of metagenomics, Jill Banfield and Brian C. Thomas. Metagenomi generates massive quantities of data from natural environments, producing complete genomes from organisms that are otherwise unknown. Metagenomi then unlocks the information captured in these genomes to develop game-changing in vivo and ex vivo therapeutics.

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Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies - Business Wire

Bone Marrow Stem Cells Comments Off on Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies – Business Wire | September 1st, 2020

The Hematopoietic Stem Cells Transplantation (HSCT) market research report study recently presented by AMR provides comprehensive knowledge on the development activities by Global industry players, growth possibilities or opportunities and market sizing for Hematopoietic Stem Cells Transplantation (HSCT) along with analysis by key segments, leading and emerging players, and their presence geographies. This is the latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions

This research study has 125 pages, it covers the complete market overview of various profiled players and their development history, on-going development strategies along with the current situation.

Hematopoietic stem cells transplantation is the treatment of patients with blood dysfunction such as leukemia or aplastic anemia by intravenous injection of normal bone marrow cells.

The research benefits in recognizing and following arising players in the market and their portfolios, to enhance decision-making abilities and helps to create effective counter-strategies to gain a competing advantage. Some of the players profiled/ part of study coverage are ViaCord, Vita34, Omeros Corporation, Cesca Therapeutics, Smart Cells, Cryo-Cell, Kiadis Pharma, Cryo-Save AG.

Download the Sample ToC and understand the COVID19 impact and be smart in redefining business strategies. https://www.amplemarketreports.com/sample-request/covid-19-outbreak-global-hematopoietic-stem-cells-transplantation-industry-1910831.html

AMRs research team has examined complete data across the globe comprising 20+ countries with a comprehensive data plan spread from 2013 to 2026 and approximately 12+ regional indicators complemented with 20+ company level coverage.

The study is organized utilizing data and knowledge sourced of various primary and secondary sources, proprietary databases, company/university websites, regulators, conferences, SEC filings, investor presentations and featured press releases from company sites and industry-specific third party sources.

Know more about focused companies, countries before buy at: https://www.amplemarketreports.com/enquiry-before-buy/covid-19-outbreak-global-hematopoietic-stem-cells-transplantation-industry-1910831.html

Characteristics of the Table of Content:

The comprehensive study presented by considering all the important aspects and sections. Some of these were

Hematopoietic Stem Cells Transplantation (HSCT) MARKET RESEARCH SCOPE OBJECTIVES, TARGET AND KEY FINDINGS

Preferably, that approaching major uptrend failed to arrive on schedule, but the Hematopoietic Stem Cells Transplantation (HSCT) market raised without posting any drops and surely witnesses zeniths in years to come.

Buy this research report at: https://www.amplemarketreports.com/buy-report.html?report=1910831&format=1

Leukemia, Lymphoproliferative Disorders, Solid Tumors, Non-Malignant Disorders, Others segment interpreted and sized in this research report by application/end-users reveals the inherent growth and several shifts for the period 2014 to 2026.

The changing dynamics supporting the growth perform it perilous for manufacturers in this extent to keep up-to-date with the changing pace of the market. Find out which segment is doing great and will return in strong earnings adding the significant drive to overall growth.

Furthermore, the research contributes an in-depth overview of regional level break-up categorized as likely leading growth rate territory, countries with the highest market share in past and current scenario. Some of the geographical break-up incorporated in the study are North America (Covered in Chapter 7 and 14), United States, Canada, Mexico, Europe (Covered in Chapter 8 and 14), Germany, UK, France, Italy, Spain, Russia.

In the Type segment Autologous Transplant, Allogenic Transplant included for segmenting Hematopoietic Stem Cells Transplantation (HSCT) market by type.

The industry is performing well and few emerging business institutions are in their peak as per growth rate and their existence with major players of Hematopoietic Stem Cells Transplantation (HSCT) market whereas conflict between 2 Global economies continues in 2020.

ViaCord, Vita34, Omeros Corporation, Cesca Therapeutics, Smart Cells, Cryo-Cell, Kiadis Pharma, Cryo-Save AG major key players included in this research along with their sales and revenue data show how they are performing well?

Find out more about this report at: https://www.amplemarketreports.com/report/covid-19-outbreak-global-hematopoietic-stem-cells-transplantation-industry-1910831.html

Thanks for reading this article, you can also get individual chapter wise section or region wise report versions like North America, Western / Eastern Europe or Southeast Asia.

With the given market data, Research on Global Markets offers customization according to specific needs.

About Author

Ample Market Research provides comprehensive market research services and solutions across various industry verticals and helps businesses perform exceptionally well. Our end goal is to provide quality market research and consulting services to customers and add maximum value to businesses worldwide. We desire to delivery reports that have the perfect concoction of useful data. Our mission is to capture every aspect of the market and offer businesses a document that makes solid grounds for crucial decision making.

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Comprehensive Report on Hematopoietic Stem Cells Transplantation Market Set to Witness Huge Growth by 2026 | ViaCord, Vita34, Omeros Corporation,...

Bone Marrow Stem Cells Comments Off on Comprehensive Report on Hematopoietic Stem Cells Transplantation Market Set to Witness Huge Growth by 2026 | ViaCord, Vita34, Omeros Corporation,… | September 1st, 2020

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

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

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.

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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."

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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

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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.

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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

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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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.

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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

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.

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

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Researchers have found a way, in mice and human tissue, to regenerate the cartilage that eases movement between bones.

Loss of this slippery and shock-absorbing tissue layer, called articular cartilage, is responsible for many cases of joint pain and arthritis, which afflicts more than 55 million Americans.

The researchers can envision a time when people are able to avoid getting arthritis in the first place by rejuvenating their cartilage before it is badly degraded.

Nearly 1 in 4 adult Americans suffer from arthritis, and far more are burdened by joint pain and inflammation generally.

The researchers figured out how to regrow articular cartilage by first causing slight injury to the joint tissue, then using chemical signals to steer the growth of skeletal stem cells as the injuries heal.

Cartilage has practically zero regenerative potential in adulthood, so once its injured or gone, what we can do for patients has been very limited, says co-senior author Charles K.F. Chan, assistant professor of surgery at Stanford Universitys School of Medicine.

Its extremely gratifying to find a way to help the body regrow this important tissue, Chan says.

The work builds on previous research that resulted in isolation of the skeletal stem cell, a self-renewing cell that is also responsible for the production of bone, cartilage and a special type of cell that helps blood cells develop in bone marrow.

Articular cartilage is a complex and specialized tissue that provides a slick and bouncy cushion between bones at the joints. When this cartilage is damaged by trauma, disease, or simply thins with age, bones can rub directly against each other, causing pain and inflammation, which can eventually result in arthritis.

Damaged cartilage can be treated through a technique called microfracture, in which tiny holes are drilled in the surface of a joint. The microfracture technique prompts the body to create new tissue in the joint, but the new tissue is not much like cartilage.

I realized the only way to understand the process was to look at what stem cells are doing after microfracture.

Microfracture results in what is called fibrocartilage, which is really more like scar tissue than natural cartilage, says Chan. It covers the bone and is better than nothing, but it doesnt have the bounce and elasticity of natural cartilage, and it tends to degrade relatively quickly.

The most recent research arose, in part, through the work of surgeon and lead author Matthew Murphy, a visiting researcher at Stanford who is now at the University of Manchester.

I never felt anyone really understood how microfracture really worked, Murphy says. I realized the only way to understand the process was to look at what stem cells are doing after microfracture.

For a long time, Chan says, people assumed that adult cartilage did not regenerate after injury because the tissue did not have many skeletal stem cells that could be activated. Working in a mouse model, the team documented that microfracture did activate skeletal stem cells. Left to their own devices, however, those activated skeletal stem cells regenerated fibrocartilage in the joint.

But what if the healing process after microfracture could be steered toward development of cartilage and away from fibrocartilage?

The researchers knew that as bone develops, cells must first go through a cartilage stage before turning into bone. They had the idea that they might encourage the skeletal stem cells in the joint to start along a path toward becoming bone, but stop the process at the cartilage stage.

The researchers used a powerful molecule called bone morphogenetic protein 2 (BMP2) to initiate bone formation after microfracture, but then stopped the process midway with a molecule that blocked another signaling molecule important in bone formation, called vascular endothelial growth factor (VEGF).

What we ended up with was cartilage that is made of the same sort of cells as natural cartilage with comparable mechanical properties, unlike the fibrocartilage that we usually get, Chan says. It also restored mobility to osteoarthritic mice and significantly reduced their pain.

As a proof of principle that this might also work in humans, the researchers transferred human tissue into mice that were bred to not reject the tissue, and were able to show that human skeletal stem cells could be steered toward bone development but stopped at the cartilage stage.

The next stage of research is to conduct similar experiments in larger animals before starting human clinical trials. Murphy points out that because of the difficulty in working with very small mouse joints, there might be some improvements to the system they could make as they move into relatively larger joints.

The first human clinical trials might be for people who have arthritis in their fingers and toes. We might start with small joints, and if that works we would move up to larger joints like knees, Murphy says.

Right now, one of the most common surgeries for arthritis in the fingers is to have the bone at the base of the thumb taken out. In such cases we might try this to save the joint, and if it doesnt work we just take out the bone as we would have anyway. Theres a big potential for improvement, and the downside is that we would be back to where we were before.

One advantage of their discovery is that the main components of a potential therapy are approved as safe and effective by the FDA, says co-senior author Michael Longaker, professor of surgery.

BMP2 has already been approved for helping bone heal, and VEGF inhibitors are already used as anti-cancer therapies, he says. This would help speed the approval of any therapy we develop.

Joint replacement surgery has revolutionized how doctors treat arthritis and is very common: By age 80, 1 in 10 people will have a hip replacement and 1 in 20 will have a knee replaced. But such joint replacement is extremely invasive, has a limited lifespan and is performed only after arthritis hits and patients endure lasting pain.

The researchers say they can envision a time when people are able to avoid getting arthritis in the first place by rejuvenating their cartilage in their joints before it is badly degraded.

One idea is to follow a Jiffy Lube model of cartilage replenishment, Longaker says. You dont wait for damage to accumulateyou go in periodically and use this technique to boost your articular cartilage before you have a problem.

The work appears in the journal Nature Medicine.

Support for the research came from the National Institutes of Health, the California Institute for Regenerative Medicine, the Oak Foundation, the Pitch Johnson Fund, the Gunn/Olivier Research Fund, the Stinehart/Reed Foundation, The Siebel Foundation, the Howard Hughes Medical Institute, the German Research Foundation, the PSRF National Endowment, National Center for Research Resources, the Prostate Cancer Research Foundation, the American Federation of Aging Research, and the Arthritis National Research Foundation.

Source: Stanford University

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Method regrows cartilage to cushion bones - Futurity: Research News

Bone Marrow Stem Cells Comments Off on Method regrows cartilage to cushion bones – Futurity: Research News | August 27th, 2020

SAN DIEGO Cystinosis is a disease that slowly and aggressively attacks your organs, tissues, muscles, bones, eyes, even your brain.

It's a genetic disorder with no cure.

And right now, the only option for treatment is an army of pills to slow it down but missing even one dose can be devastating.

Now one man is "patient one", the first to try a new treatment that may save thousands of lives.

21-year old Jordan Janz is living with the rare, unrelenting disorder.

"I was diagnosed at eight months old and basically have been living with it my whole life."

In Jordan, cystine, an amino acid, gets trapped in his cells.

When cystine levels rise, crystals build up all over the body leaving a trail of damage... even causing him to vomit up to 13 times a day.

"It's not how strong you are physically," he said.

"I think it's how strong you are mentally when you come into this."

Traditional cystinosis treatments aim to slow the build up of cystine inside cells.

In order to do that Jordan takes 56 pills each day, but now he hopes to change that, Jordan is the first patient to test a unique gene therapy.

UC San Diego professor Stephanie Cherquie's took stem cells from Jordan's bone marrow, re-engineered the cells, introduced genes that will produce cystinosin, then reinfused Jordan with his own cystinosin-producing cells.

"So, then these cells become a source of healthy stem cells for the rest of the life of the patient," said Stephanie.

Jordan had to take chemo twice a day, but he hasn't let that scare him away.

"I'm doing this obviously for other cystinosis families, right?," said Jordan.

Hoping that many others after him will now get the chance at a better, longer life.

For those born with cystinosis who make it into adulthood, the average lifespan is around 28 years old.

We're told Jordan Janz is making a good recovery. though it is still too soon to tell his long-term prognosis.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens atjim.mertens@wqad.comor Marjorie Bekaert Thomas atmthomas@ivanhoe.com.

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YOUR HEALTH: When the body turns to crystals - WQAD.com

Bone Marrow Stem Cells Comments Off on YOUR HEALTH: When the body turns to crystals – WQAD.com | August 27th, 2020

DataIntelo has published a latest market research report on Global Hematopoietic Stem Cell Transplantation (HSCT) Market. The global report is prepared in collaboration with the leading industry experts and dedicated research analyst team to provide an enterprise with in-depth market insights and help them to take crucial business decisions. This report covers current market trends, opportunities, challenges, and detailed competitive analysis of the industry players in the market.

The published report explains about the current supply and demand scenario and presents the future outlook of the market in a detailed manner. DataIntelo has applied a robust market research methodology to bestow the new entrants and emerging players with 360 wide-view analysis on the latest advancements and their impacts on the market. It has congregated massive amount of data on the key segments of the market in an easy to understand format. The research report has laid out the numbers and figures in a comprehensive manner with the help of graphical and pictorial representation which embodies more clarity on the market.

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Report Covers Impacts of COVID-19 to the market.

The on-going pandemic has overhauled various facets of the market. This research report provides the financial impacts and market disturbance on the Hematopoietic Stem Cell Transplantation (HSCT) market. It also includes analysis on the potential lucrative opportunities and challenges in the foreseeable future. DataIntelo has interviewed various delegates of the industry and got involved in the primary and secondary research to confer the clients with information and strategies to fight against the market challenges amidst and after COVID-19 pandemic.

Market Segmentation:

Few of the 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 can be included in the list upon the request.

By Product Type:

AllogeneicAutologous

By Applications:

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

By Geographical Location: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

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Hematopoietic Stem Cell Transplantation (HSCT) Supply Chain Analysis

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Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size, Analytical Overview, Growth Factors, Demand, Trends And Forecast To 2026 -...

Bone Marrow Stem Cells Comments Off on Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size, Analytical Overview, Growth Factors, Demand, Trends And Forecast To 2026 -… | August 27th, 2020

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

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The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

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As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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Demand for Myelofibrosis Treatment Market to Witness Rapid Surge During the Period 2016 2022 - Scientect

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Background:Microfracture is a surgical technique that involves creating multiple holes of 3-4 mm depth in the subchondral bone to recruit stem cells in the bone marrow to the lesion, inducing fibrocartilage repair and knee cartilage regeneration. Recently, it has been reported that increasing the exposed area of the lower cartilaginous bone (drilling a lot of holes) increases the outflow of stem cells, which is expected to affect the physical properties of the subchondral bone when the exposed area is large. The purpose of this study was to analyse the effect of the distance between the holes in the microfracture procedure on the structural stability of the osteochondral bone using a finite element method.

Methods:In this study, lateral aspects of the femoral knee, which were removed during total knee arthroplasty were photographed using microtomography. The model was implemented using a solitary walks program, which is a three-dimensional simplified geometric representation based on the basic microtomography data. A microfracture model was created by drilling 4 mm-deep holes at 1, 1.5, 2, 2.5, 3, 4, and 5 mm intervals in a simplified three-dimensional (3D) geometric femoral model. The structural stability of these models was analysed with the ABAQUS program. We compared the finite element model (FEM) based on the microtomography image and the simplified geometric finite element model.

Results:Von Mises stress of the subchondral bone plate barely increased, even when the distance between holes was set to 1 mm. Altering the distance between the holes had little impact on the structural stability of the subchondral bone plate. Safety factors were all below 1.

Conclusions:Although we did not confirm an optimal distance between holes, this study does provide reference data and an epidemiological basis for determining the optimal distance between the holes used in the microfracture procedure.

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The effect of distance between holes on the structural stability of subchondral bone in microfracture surgery: a finite element model study - DocWire...

Bone Marrow Stem Cells Comments Off on The effect of distance between holes on the structural stability of subchondral bone in microfracture surgery: a finite element model study – DocWire… | August 25th, 2020