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.

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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

Covid 19 Impact[emailprotected]https://www.trendsmarketresearch.com/report/covid-19-analysis/3184

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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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

And that starts with Morphogenesis focus on easily accessible tumors.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Global Stem Cell Therapy Market: Overview

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

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

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Global Stem Cell Therapy Market: Key Trends

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

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

Global Stem Cell Therapy Market: Market Potential

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

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

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

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

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

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

Global Stem Cell Therapy Market: Competitive Analysis

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

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

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

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

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

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

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

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

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

Segmentation by Product Type

Segmentation by End User

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

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

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

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

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

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

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Why Choose Stem Cell-Derived Cells Market Report?

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Read: The plan that could give us our lives back

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

ALD-104 Study

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

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

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

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

eli-cel Presentation at EBMT

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

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

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

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

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

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

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

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

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

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

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

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

About CALD Early Diagnosis

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

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

About bluebird bio, Inc.

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

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

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

SOURCE: bluebird bio

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

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

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

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

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

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

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

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

North America (United States, Canada and Mexico)

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

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

South America (Brazil etc.)

Middle East and Africa (Egypt and GCC Countries)

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

CCBC

Vcanbio

Boyalife

Beikebiotech

By the product type, the market is primarily split into

Umbilical Cord Blood Stem Cell

Embryonic Stem Cell

Adult Stem Cell

Other

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

Diseases Therapy

Healthcare

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

North America

United States

Canada

Mexico

Asia-Pacific

China

India

Japan

South Korea

Australia

Indonesia

Singapore

Malaysia

Philippines

Thailand

Vietnam

Rest of Asia-Pacific

Europe

Germany

France

UK

Italy

Spain

Russia

Rest of Europe

Central & South America

Brazil

Rest of Central & South America

Middle East & Africa

GCC Countries

Turkey

Egypt

South Africa

Rest of Middle East & Africa

The study objectives of this report are:

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

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

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

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

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

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

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

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

About Us:

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

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

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

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

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

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

Here are someof our favoritebeauty finds under $25.

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

Details: beautysociety.com

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

Details: actsyl.com

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

Details: trinnylondon.com

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

Details: wonderskin.com

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

Details: crabtree-evelyn.com

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

Details: thefacialworks.com

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

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

Details: beautycounter.com

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

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

Details: grove.co/roven

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

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

Details: sjalskincare.com/products/balans

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

Details:westmorebeauty.com

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

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