Advance Market Analytics published a new research publication on Stem Cells Market Insights, to 2026 with 232 pages and enriched with self-explained Tables and charts in presentable format. In the Study you will find new evolving Trends, Drivers, Restraints, Opportunities generated by targeting market associated stakeholders. The growth of the Stem Cells Market was mainly driven by the increasing R&D spending across the world.

Some of the key players profiled in the study are:

Smith & Nephew (United Kingdom),Celgene Corporation (United States),BIOTIME, INC. (United States),Cynata (Australia),Applied Cell Technology (Hungary),STEMCELL Technologies Inc. (Canada),BioTime Inc. (United States),Cytori Therapeutics, Inc. (United States),Astellas Pharma Inc. (Japan),U.S. Stem Cell, Inc. (United States),Takara Holdings. (Japan)

Get Free Exclusive PDF Sample Copy of This Research @ https://www.advancemarketanalytics.com/sample-report/72815-global-stem-cells-market-1

Scope of the Report of Stem Cells

The stem cell is used for treating chronic diseases such as cardiovascular disorders, cancer, diabetes, and others. Growing research and development in stem cell isolation techniques propelling market growth. For instance, a surgeon from Turkey developed a method for obtaining stem cells from the human body without enzymes which are generally used for the isolation of stem cells. Further, growing healthcare infrastructure in the developing economies and government spending on the life science research and development expected to drive the demand for stem cell market over the forecasted period.

Market Trend:

Emphasizing On Development of Regenerative Medicine

Technological Advancement in Stem Cell Harvesting and Isolation Techniques

Market Drivers:

Rising Prevalence of Chronic Diseases such as Cardiovascular Disorders, Cancer, and others

Growing Healthcare Infrastructure in the Developing Economies

Challenges:

Lack of Awareness Regarding Stem Cell Therapy in the Low and Middle Income Group Countries

Opportunities:

Growing Demand for Cellular Therapies

Rising Application of Autologous Therapy

The titled segments and sub-section of the market are illuminated below:by Type (Adult Stem Cells (Neuronal, Hematopoietic, Mesenchymal, Umbilical Cord, Others), Human Embryonic Stem Cells (hESC), Induced Pluripotent Stem Cells, Very Small Embryonic-Like Stem Cells), Application (Regenerative Medicine (Neurology, Orthopedics, Oncology, Hematology, Cardiovascular and Myocardial Infraction, Injuries, Diabetes, Liver Disorder, Incontinence, Others), Drug Discovery and Development), Technology (Cell Acquisition (Bone Marrow Harvest, Umbilical Blood Cord, Apheresis), Cell Production (Therapeutic Cloning, In-vitro Fertilization, Cell Culture, Isolation), Cryopreservation, Expansion and Sub-Culture), Therapy (Autologous, Allogeneic)

Have Any Questions Regarding Global Financial Advisory Market Report, Ask Our [emailprotected] https://www.advancemarketanalytics.com/enquiry-before-buy/72815-global-stem-cells-market-1

Region Included are: North America, Europe, Asia Pacific, Oceania, South America, Middle East & Africa

Country Level Break-Up: United States, Canada, Mexico, Brazil, Argentina, Colombia, Chile, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Belgium, Austria, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, China, Japan, Taiwan, South Korea, Singapore, India, Australia and New Zealand etc.

Strategic Points Covered in Table of Content of Global Stem Cells Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the Stem Cells market

Chapter 2: Exclusive Summary the basic information of the Stem Cells Market.

Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges of the Stem Cells

Chapter 4: Presenting the Stem Cells Market Factor Analysis Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.

Chapter 5: Displaying market size by Type, End User and Region 2015-2020

Chapter 6: Evaluating the leading manufacturers of the Stem Cells market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile

Chapter 7: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries (2021-2026).

Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source

Finally, Stem Cells Market is a valuable source of guidance for individuals and companies in decision framework.

Read Detailed Index of full Research Study at @ https://www.advancemarketanalytics.com/reports/72815-global-stem-cells-market-1

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Stem Cells Market to Witness Gigantic Growth by 2026 LSMedia - LSMedia

Bone Marrow Stem Cells Comments Off on Stem Cells Market to Witness Gigantic Growth by 2026 LSMedia – LSMedia | December 23rd, 2021

Mysuru

An 88-year-old gentleman presented to Manipal Hospital Mysore with blackish discoloration of the heel of left foot. He was diabetic & was on regular treatment for the same. For the current problem, he had already received several medications including intra venous antibiotics with little improvement. Upon examination he was detected to have Critical Limb Ischemia (CLI) with gangrene of heel of left foot. Large number of such patients end up with amputation of leg. Our aim in such situation is to first try to save the limb. Amputation should be the last resort when everything else fails. Said Dr. Upendra Shenoy Cardiothoracic and Vascular Surgeon Manipal Hospital Mysore, while giving details about the patient. While addressing the media Dr. C B Keshavamurthy Consultant Interventional Cardiology, Manipal Hospital Mysore said, Patients angiogram showed diffuse disease in all blood vessels of the leg with critical blocks in many areas.

We performed angioplasty on the limb. The procedure improved the blood supply to the limb, but additional treatment was required to restore blood circulation to the foot and toes. Dr. Shenoy and team decided to implement stem cell therapy, hybrid procedure of peripheral angioplasty with stem cell injection. First of its kind procedure in Mysore. Stem cell therapy involves the injection of stem cells obtained from the bone marrow of healthy individuals.

These stem cells can transform themselves into different tissues according to the requirement. In this case, the stem cells stimulate the formation of new blood vessels, said Dr Upendra Shenoy while explaining about the therapy. Dr Shenoy further added, On the day after angioplasty, we injected the stem cell into the calf muscles of the patient.

The dose depends upon the weight of the patient. If the weight is below 60 kg, the doctor injects about 150 million stem cells. In patients with more than 60 kg, the dose is about 200 million.

Continue reading here:
Stem cell therapy holds immense promise for the treatment of patients with non-healing ischemic leg wounds - Ibcworldnews

Bone Marrow Stem Cells Comments Off on Stem cell therapy holds immense promise for the treatment of patients with non-healing ischemic leg wounds – Ibcworldnews | December 23rd, 2021

PALM BEACH, Fla., Dec. 21, 2021 /PRNewswire/ -- FinancialNewsMedia.com News Commentary - Systemic mastocytosis is rare disease which affects very few people and it causes due to C-kit mutation which leads to higher number of mast cell production in the body resulting in accumulation of excessive mast cells in the internal body organs such as spleen, liver, bone marrow and small intestine etc. Recently, the World Health Organization (WHO) updated the prognosis, diagnosis and systemic mastocytosis treatment guidelines for the disease which in turn helped to bring uniformity in the approach by healthcare professionals. The manufacturers in the systemic mastocytosis treatment market are focusing on evaluating possible treatment options to cure the disease by investing heavily in the research & development. Various leading manufacturers are focusing on gaining FDA approval to respective drugs for the systemic mastocytosis treatment to enhance their revenue generation. A reportfrom Future Market Insights said:"Increasing awareness about the systemic mastocytosis treatment as well as symptoms of the disease due to extended effort by non-profit organizations, governmental associations and through other platforms expected to drive the growth of the systemic mastocytosis treatment market Increasing approvals and launches of drugs for the systemic mastocytosis treatment expected to drive the growth of the market Increasing spending on research & development by various pharmaceutical companies to develop novel systemic mastocytosis treatment expected to further fuel the growth of market. Increasing early diagnosis rate subsequently followed by increasing treatment seeking rate further expected to drive the growth of the systemic mastocytosis treatment market." Active companies in the markets today include: Hoth Therapeutics, Inc. (NASDAQ:HOTH), Longeveron Inc. (NASDAQ: LGVN), Bristol Myers Squibb (NYSE: BMY), Takeda Pharmaceutical Company Limited (NYSE: TAK), Amgen (NASDAQ: AMGN).

Future Market Insights continued:"The global systemic mastocytosis treatment market is expected to experience significant growth due to increasing novel treatment options. By drug class, systemic mastocytosis treatment market is expected to be dominated by the mast cell stabilizers due to superior efficacy. By indication, systemic mastocytosis treatment market is expected to be dominated by indolent systemic mastocytosis (ISM) due to higher prevalence. By route o administration, systemic mastocytosis treatment market is expected to be dominated by injectables. By distribution channel, systemic mastocytosis treatment market is expected to be dominated by the retail pharmacies due to higher patient footfall. The global systemic mastocytosis treatment market is expected to be dominated by the North America due to comparatively higher prevalence of the disease. Europe systemic mastocytosis treatment market is expected to be second most lucrative market due to higher treatment seeking rate. Latin America expected to show gradual growth in the systemic mastocytosis treatment market due to steadily increasing diagnosis. Asia-Pacific is emerging systemic mastocytosis treatment market due to increasing diagnosis subsequently followed by treatment. Middle East & Africa is the least lucrative systemic mastocytosis treatment market due to least diagnostic rate and lower awareness about the symptoms."

Hoth Therapeutics, Inc. (NASDAQ:HOTH) BREAKING NEWS: Hoth Therapeutics Announces Submission of Orphan Designation Application for HT-KIT to Treat Mastocytosis Hoth Therapeutics, Inc., a patient-focusedclinical-stage biopharmaceutical company, announced it submitted an Orphan Drug Designation Application to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocyctosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting, resulting in apoptosis of neoplastic mast cells. The KIT signaling pathway is implicated in multiple diseases, including all types of mastocytosis (such as aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN)), acute myeloid leukemia, gastrointestinal stromal tumors, and anaphylaxis.

Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US)are eligible to apply for Orphan Drug Designation (ODD), which provides multiple benefits to the sponsor during development and after approval. Hoth intends to pursue these benefits as part of the drug development for HT-KIT for treatment of mastocytosis, pending designation of the ODD application.

Benefits of Orphan Drug Designation - Under the Orphan Drug Act, drug companies can apply for ODD, and if granted, the drug will have a status which gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug. A tax credit of 50% of the qualified clinical drug testing costs awarded upon drug approval is also possible. Regulatory streamlining and provide special assistance to companies that develop drugs for rare patient populations. In addition to exclusive rights and cost benefits, the FDA will provide protocol assistance, potential decreased wait-time for drug approval, discounts on registration fees, and eligibility for market exclusivity after approval.

Key benefits of ODD:

Hoth recently announcedthat its novelanti-cancer therapeuticexhibited highly positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma. CONTINUED Read the Hoth Therapeutics full press release by going to: https://ir.hoththerapeutics.com/news-releases

In other news and developments of note in the markets this week:

Amgen (NASDAQ: AMGN) recently announced that the U.S. Food and Drug Administration (FDA) has approved Amgen and AstraZeneca'sTezspire (tezepelumab-ekko) for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.

Tezspirewas approved following a Priority Review by the FDA and based on results from the PATHFINDER clinical trial program. The application included results from the pivotal NAVIGATOR Phase 3 trial in whichTezspiredemonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.

Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

Bristol Myers Squibb (NYSE: BMY) recently announced thatOrencia(abatacept) was approved by the U.S. Food and Drug Administration (FDA) for the prophylaxis, or prevention, of acute graft versus host disease (aGvHD), in combination with a calcineurin inhibitor (CNI) and methotrexate (MTX), in adults and pediatric patients 2 years of age and older undergoing hematopoietic stem cell transplantation (HSCT) from a matched or 1 allele-mismatched unrelated donor (URD).

"Orenciais the first FDA-approved therapy to prevent acute graft versus host disease following hematopoietic stem cell transplant, a potentially life-threatening complication that can pose a comparatively higher risk to racial and ethnic minority populations in the U.S. due to difficulty finding appropriately matched donors," said Tina Deignan, senior vice president, U.S. Immunology, Bristol Myers Squibb. "With this fourth indication forOrencia,Bristol Myers Squibb draws on its legacy and expertise in both immunology and hematology to deliver an important treatment option for patients in a disease with high unmet need.

Takeda Pharmaceutical Company Limited (NYSE: TAK) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of intravenous (IV) vedolizumab for the treatment of adult patients with moderately to severely active chronic pouchitis, who have undergone proctocolectomy and ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC), and have had an inadequate response with or lost response to antibiotic therapy. The CHMP opinion will now be reviewed by the European Commission. If approved, vedolizumab will become the first treatment indicated for active chronic pouchitis across the European Union.

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates Financialnewsmedia.com and MarketNewsUpdates.com, is a third- party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM was compensated twenty five hundred dollars for news coverage of current press release issued by: Hoth Therapeutics, Inc. by a non-affiliated third party.

FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

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Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options - PRNewswire

Bone Marrow Stem Cells Comments Off on Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options – PRNewswire | December 23rd, 2021

Image Credit: Supplied

American Hospital Dubai launches the first and only autologous stem cell transplant department in Dubai. It is the first private hospital in the UAE to offer in-housestem cell transplant of patients stem cells, without the need for a donor. The services include laboratory diagnostics, chemotherapy, stem cell mobilisation, collection, storage and re-infusion with individualised care in specialised rooms.

stem cell transplant of patients stem cells, without the need for a donor. The services include laboratory diagnostics, chemotherapy, stem cell mobilisation, collection, storage and re-infusion with individualised care in specialised rooms.

The DHA-licensed stem cell unit is another step forward for American Hospital Dubais comprehensive cancer care programme, established more than 12 years ago.

The unit has a team of European- and US-qualified medical consultants, subspecialists and allied staff, with an international affiliation for multidisciplinary case review and discussions.

The units nurses are highly skilled in Bone Marrow Transplant (BMT) procedures, with experience in apheresis (separating blood components), cellular therapy, and post-transplant care.

The non-surgical transplant procedure is akin to a blood transfusion. It involves stimulating the stem cells, present mainly in bone marrow, by medication to travel out into the blood. This process, called Peripheral Blood Stem Cell collection, is more common in stem cell transplants for cancer treatment than harvesting stem cells directly from the bone marrow for a BMT.

Welcoming the launch, Dr Tarek Dufan, Chief Medical Officer, American Hospital Dubai, said, The stem cell transplant unit is another milestone in American Hospital Dubais commitment to delivering the most advanced healthcare to UAE and the region. Our cutting-edge Cancer Care Department has been a leader in oncology, and the stem cell unit expands our expertise in offering the latest cancer treatments and management.

Dr Maroun El Khoury, Director of Cancer Centre, said, American Hospital Dubais autologous stem cell transplant unit is the only one of its kind in the UAE. We have highly trained staff specialised in stem cell transplant and care management, excellent in-house laboratory services, radiation facilities, and psychological support systems to deliver a complete and compassionate care experience for patients.

The unit, led by Dr Shabeeha K. Rana, Consultant Haematologist and Director of Stem Cell Transplantation and Cellular Therapy at American Hospital Dubai, includes Dr Maroun El Khoury, Director of Cancer Centre; Dr Faraz Khan, Consultant Haematologist/Oncologist; Dr Julieta Zuluaga, Specialist Haematology and Stem Cell Transplantation; Dr Mona Tareen, Pain Management/Palliative Care Consultant; and Dr Melanie Schlatter, Clinical Psychologist.

The unit will treat haematological cancers such as multiple myeloma, lymphoma, certain types of leukaemia and amyloidosis (build-up of a rare protein called amyloid in the body). In addition, it will treat non-haematological conditions such as germ cell tumours and autoimmune diseases such as multiple sclerosis, Crohns, and ulcerative colitis.

The unit provides patients with support groups who have undergone stem cell transplants as an invaluable psychological tool. Every opportunity is made available to patients to provide feedback, ask questions, and inform and educate themselves with written material resources and emotional support for pre-and post-treatment phases.

American Hospital Dubais stem cell transplant unit follows strict selection criteria before accepting patients to ensure the highest adherence to care quality, safety and efficacy.

This content comes from Reach by Gulf News, which is the branded content team of GN Media.

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American Hospital Dubai launches first and only autologous stem cell transplant centre in the UAE - Gulf News

Bone Marrow Stem Cells Comments Off on American Hospital Dubai launches first and only autologous stem cell transplant centre in the UAE – Gulf News | December 10th, 2021

JSP191 is well tolerated with no treatment-related adverse events in dose-escalation study

Single-agent conditioning with JSP191 is associated with engraftment, immune reconstitution, and clinical benefit

REDWOOD CITY, Calif., Dec. 08, 2021 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced that data on JSP191 showing long-term benefits of hematopoietic stem cells (HSC) engraftment following targeted single-agent JSP191 conditioning in the treatment of severe combined immunodeficiency (SCID) will be presented at the 2021 American Society of Hematology (ASH) Annual Meeting.

The accepted abstract is published and available on the ASH website here.

Title: JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Nave Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)Session: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities; Novel Conditioning Approaches. Hematology Disease Topics & Pathways:Abstract: 554Date and Time: Sunday, December 12, 2021, 4.45 p.m. ET

Our ongoing study shows JSP191 to be well tolerated with no treatment-related adverse events across multiple patients ranging from 3 months to 38 years old, said Kevin N. Heller, M.D., Executive Vice President, Research and Development. In this study six of nine non-IL2RG patients with prior hematopoietic cell transplant (HCT), dosed in the initial JSP191 dose escalation (0.1, 0.3, 0.6 and 1.0 mg/kg), achieved HSC engraftment, nave donor T lymphocyte production, and demonstrated clinical improvement. As this trial continues to enroll, the 0.6 mg/kg dose will continue to be evaluated as the potential recommended Phase 2 dose (RP2D) based on HSC engraftment, clinical outcomes and an optimal half-life allowing for integration within existing transplant protocols. We believe that with these initial successful clinical findings, we are one step closer, and uniquely positioned to deliver a targeted non-genotoxic conditioning agent to patients with SCID.

Story continues

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical mRNA engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

Forward-Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements the proposed business combination between AMHC and Jasper Therapeutics, the estimated or anticipated future results and benefits of the combined company following the business combination, including Jasper Therapeutics business strategy, expected cash resources of the combined company and the expected uses thereof, current and prospective product candidates, planned clinical trials and preclinical activities and potential product approvals, as well as the potential for market acceptance of any approved products and the related market opportunity. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of the respective management teams of Jasper Therapeutics and AMHC and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Jasper Therapeutics and AMHC. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions the outcome of any legal proceedings that may be instituted against the parties regarding the Business Combination; the risk that the potential product candidates that Jasper Therapeutics develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Jasper Therapeutics product candidates; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper Therapeutics will be unable to successfully market or gain market acceptance of its product candidates; the risk that Jasper Therapeutics product candidates may not be beneficial to patients or successfully commercialized; the risk that Jasper Therapeutics has overestimated the size of the target patient population, their willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper Therapeutics business; the risk that third parties on which Jasper Therapeutics depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper Therapeutics business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic; the risk that Jasper Therapeutics will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; the potential inability of the parties to successfully or timely consummate the proposed transaction; the risk of failure to realize the anticipated benefits of the proposed transaction and other risks and uncertainties indicated from time to time in AMHCs public filings, including its most recent Annual Report on Form 10-K for the year ended December 31, 2020 and the proxy statement/prospectus relating to the proposed transaction, including those under Risk Factors therein, and in AMHCs other filings with the SEC. If any of these risks materialize or AMHCs and Jasper Therapeutics assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that neither AMHC nor Jasper Therapeutics presently know, or that AMHC or Jasper Therapeutics currently believe are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. In addition, forward-looking statements reflect AMHCs and Jasper Therapeutics expectations, plans or forecasts of future events and views as of the date of this press release. AMHC and Jasper Therapeutics anticipate that subsequent events and developments will cause AMHCs and Jasper Therapeutics assessments to change. However, while AMHC and Jasper Therapeutics may elect to update these forward-looking statements at some point in the future, AMHC and Jasper Therapeutics specifically disclaim any obligation to do so. These forward-looking statements should not be relied upon as representing AMHCs and Jasper Therapeutics assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Contacts:John Mullaly (investors)LifeSci Advisors617-429-3548jmullaly@lifesciadvisors.com

Lily Eng (media)Real Chemistry206-661-8627leng@realchemistry.com

Jeet Mahal (investors)Jasper Therapeutics650-549-1403jmahal@jaspertherapeutics.com

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Jasper Therapeutics to Present Data on JSP191 Conditioning in SCID patients at the 2021 American Society of Hematology Annual Meeting - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics to Present Data on JSP191 Conditioning in SCID patients at the 2021 American Society of Hematology Annual Meeting – Yahoo Finance | December 10th, 2021

Fifty years ago a boy was born whose brief life would bring hope for thousands of people diagnosed with blood disease.

Anthony Nolans struggle with deadly Wiskott Aldrich Syndrome and his mum Shirleys tireless campaign to save him by finding a suitable bone marrow donor moved the world.

Shirley established the worlds first register of volunteer donors here in the UK.

Tragically, she never did find a suitable match for Anthony and he died when he was seven years old.

But the register became his legacy, recruiting donors around the world. Their bone marrow and stem cells have saved more than 20,000 patients with leukaemia and other blood disorders.

Alan Corby spent six months in the next isolation room to Anthony at Westminster Childrens Hospital. Neither were expected to survive.

Alan said: Much of the time he was the only person I could see. We would talk and play card games like Twist through the glass.

When I was well enough I moved to my local hospital. I went back to see Anthony a few months later, but he had passed away.

Image:

His life may have been short, but it had an incredible impact. Thousands have been given a second chance of life thanks to him and his mum.

On what would have been Anthonys 50th birthday, the Mirror meets three boys given hope of beating deadly blood diseases by his legacy.

Visit anthonynolan.org for more information or to join the donor register.

Georgie McAvoy knows the heartache Shirley Nolan endured searching for a donor to save her son.

Because her little boy Daniel was born with the same rare disease that killed Anthony.

Daniel, two, has Wiskott Aldrich Syndrome which prevents his blood cells from fighting infection and clotting properly. His only hope is a bone marrow transplant to reset his immune system.

A first transplant in June last year failed as Daniel body rejected his donor cells and relapsed.

Image:

He is now preparing to undergo another gruelling course of chemotherapy, followed by a second transplant and will spend Christmas recovering in hospital.

Georgie, 31, said: We have been through so much, but Daniel is still fighting. He has coped with everything that has been thrown at him and he keeps smiling.

We are so grateful for the donor register and everything that Shirley Nolan did it is the reason that Daniel is still alive.

Daniels parents realised something was wrong when he began suffering nosebleeds and they found blood in his nappy when he was three weeks old.

He then developed sepsis and spent 11 days fighting for his life in intensive care.

Georgie and dad Andrew, 38, even asked the hospital chaplain to christen Daniel is his cot as they feared he might not survive.

Image:

Georgie, from Huntingdon in Cambridge, said: The doctors told us he needed a bone marrow transplant to save his life, but that some children didnt make it to transplant.

It was devastating. We didnt know if he would start to walk or go to school. I remember thinking, I need him to be christened in case something happens.

It was emotional. Daniels big sister Holly wore a christening gown made from my wedding dress and Id planned for Daniel to wear it too, but that obviously wasnt possible.

Daniel was eventually diagnosed with Wiskott-Aldrich syndrome, a rare genetic disorder that affects one in every one million boys, in May last year after an unrelated hernia operation.

Neither his parents nor Holly, four, were a suitable match, so their only hope was to find a donor through the stem cell register, which the charity Anthony Nolan managed within two months.

Image:

Daniel was due to undergo the transplant in March last year, but his procedure was postponed after the Covid pandemic began for fear there would be a shortage of doctors, nurses, or beds.

Georgie said: That was really scary. Daniel had been through all his preparation and we were ready to go, then everything blew up before our eyes. We didnt know what would happen.

Daniel continued to deteriorate, picking up more infections until his transplant finally went ahead at Great Ormond Street Hospital at the end of June as doctors could not risk waiting any longer.

He was only allowed one parent with him as he underwent chemotherapy to remove his immune system ahead of the transplant.

Image:

Daniel returned home in August but suffered a drug relating seizure, then graft versus host disease as his body tried to reject the donor cells and spent last Christmas in hospital.

Georgie said: The last two relapses have been particularly difficult. During the last one he began vomiting digested blood. His stomach had to be drained constantly.

At that point they said there were no more options, we had to do another transplant and we needed a different donor as his body had rejected the first.

It will be hard spending another Christmas in hospital, but we feel so lucky to have found another donor to give Daniel a second chance. That wouldnt have happened without Anthony Nolan.

Time is running out for Alife Pinckney to find a lifesaving stem cell donor.

The eight year-old from Exeter relies on weekly blood transfusions to top up his critically low levels of platelets. That has bought Alfie more time, but his condition is getting worse.

His desperate family know his only hope is a transplant, but his mixed British and Chinese heritage makes it harder to find a matching tissue type to prevent his body rejecting the donor cells.

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Alfies mum Lily, said: Its so hard to watch your child in pain and be incapable of helping. Its tearing me apart. Our only hope is to encourage as many people as possible to join the register.

Alfie developed Aplastic Anaemia when he was five years-old. It means his body cannot produce the platelets he needs for his blood to clot properly and he cannot fight infection.

At the time his British-born parents Lily and Charles were living and working in Hong Kong. They returned to the UK just before lockdown last year to be near family and step up Alfies treatment just like Shirley Nolan moved home from Australia to search for a donor for Anthony.

They hoped they had found a donor earlier this year when a woman in Brazil was confirmed as a matching tissue type, but that fell through.

Image:

Since then, Alfie has continued to deteriorate as his body burns through the weekly platelet transfusions and he suffered a terrifying haemorrhage.

Dad Charles said: He had a huge, uncontrollable nosebleed and bleeding from the gums. He was clutching the kitchen bin, vomiting blood, screaming Daddy, help me.

We rushed him into the high dependency unit and I was mopping blood of his arms, face, and torso as several doctors and nurses tried to keep him alive. It was harrowing.

Its so easy to join the register. It only takes three minutes to swab your checks, then you can get on with your life. But that could help to save Mason or another childs life.

Katie Jordan got the devastating news that little Mason had blood cancer on Christmas Eve last year.

Image:

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The only cure for his Juvenile Myelomonocytic Leukaemia was a bone marrow transplant but mum Katie was not a good match, nor was anyone on the donor register.

Most children with the disease only survive for 12 months after diagnosis. So Katie, a single mum like Shirley, launched her own campaign to save her son.

Masons Mission raised nearly 54,000 for Anthony Nolan, helping the charity to test the backlog of 25,000 swab samples that built up during the pandemic and add them to the donor register.

Katie, from Stockton-on-Tees said: I was living my worst nightmare. It was heartbreaking to think that Christmas could have been our last together.

I would give my life for Mason, but I wasnt a match. So I did everything I could to find a donor.

Image:

Thankfully Anthony Nolan did find a suitable donor two months later and Mason had a successful stem cell transplant in March this year.

He was rushed back to hospital over the summer after developing blisters all over his body and spent a week fighting for his life in intensive care before they subsided.

But the two year-old made a full recovery and is now looking forward to a happy, healthy Christmas.

Katie said: We were lucky that we found a donor so quickly. When they told us, I couldnt stop crying. I would love to meet his donor one day and thank them."

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The Anthony Nolan legacy: Three boys given hope of beating deadly blood diseases - The Mirror

Bone Marrow Stem Cells Comments Off on The Anthony Nolan legacy: Three boys given hope of beating deadly blood diseases – The Mirror | December 10th, 2021

Report Ocean presents a new report on Mesenchymal Stem Cells Market size, share, growth, industry trends, and forecast 2026, covering various industry elements and growth trends helpful for predicting the markets future. The global mesenchymal stem cells market size to reach USD 2,518.5 Million by 2026, growing at a CAGR of 7.0% during forecast period, according to a new research report published . The report Mesenchymal Stem Cells Market, [By Source (Bone Marrow, Umbilical Cord Blood, Peripheral Blood, Lung Tissue, Synovial Tissues, Amniotic Fluids, Adipose Tissues); By Application (Injuries, Drug Discovery, Cardiovascular Infraction, Others); By Region]: Market Size & Forecast, 2018 2026 provides an extensive analysis of present market dynamics and predicted future trends. The market was valued at USD 1,335.1 million in 2017. In 2017, the drug discovery application dominated the market, in terms of revenue. North America region is observed to be the leading contributor in the global market revenue in 2017.

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In order to produce a holistic assessment of the market, a variety of factors is considered, including demographics, business cycles, and microeconomic factors specific to the market under study. Mesenchymal Stem Cells Market report 2021 also contains a comprehensive business analysis of the state of the business, which analyzes innovative ways for business growth and describes critical factors such as prime manufacturers, production value, key regions, and growth rate.

The Centers for Medicare and Medicaid Services report that US healthcare expenditures grew by 4.6% to US$ 3.8 trillion in 2019, or US$ 11,582 per person, and accounted for 17.7% of GDP. Also, the federal government accounted for 29.0% of the total health expenditures, followed by households (28.4%). State and local governments accounted for 16.1% of total health care expenditures, while other private revenues accounted for 7.5%.

This study aims to define market sizes and forecast the values for different segments and countries in the coming eight years. The study aims to include qualitative and quantitative perspectives about the industry within the regions and countries covered in the report. The report also outlines the significant factors, such as driving factors and challenges, that will determine the markets future growth.

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These stem cells mainly function for the replacement of damaged cell and tissues. The potential of these cell is to heal the damaged tissue with no pain to the individual. Scientists are majorly focusing on developing new and innovative treatment options for the various chronic diseases like cancer. Additionally, the local governments have also taken various steps for promoting the use of these stem cells.

The significant aspects that are increasing the development in market for mesenchymal stem cells consist of enhancing need for these stem cells as an efficient therapy option for knee replacement. Raising senior populace throughout the world, as well as increasing frequency of numerous persistent conditions consisting of cancer cells, autoimmune illness, bone and cartilage diseases are elements anticipated to enhance the market development throughout the forecast period. The mesenchymal stem cells market is obtaining favorable assistance by the reliable federal government policies, as well as funding for R&D activities which is anticipated to influence the market growth over coming years. According to the reports released by world health organization (WHO), by 2050 individuals aged over 60 will certainly make up greater than 20% of the globes population. Of that 20%, a traditional quote of 15% is estimated to have symptomatic OA, as well as one-third of these individuals are expected to be influenced by extreme specials needs. Taking into consideration all these aspects, the market for mesenchymal stem cells will certainly witness a substantial development in the future.

Increasing demand for better healthcare facilities, rising geriatric population across the globe, and continuous research and development activities in this area by the key players is expected to have a positive impact on the growth of Mesenchymal Stem Cells market. North America generated the highest revenue in 2017, and is expected to be the leading region globally during the forecast period. The Asia Pacific market is also expected to witness significant market growth in coming years. Developing healthcare infrastructure among countries such as China, India in this region is observed to be the major factor promoting the growth of this market during the forecast period.

The major key players operating in the industry are Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem cell technologies Inc., Celprogen, Inc. BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc. These companies launch new products and undertake strategic collaboration and partnerships with other companies in this market to expand presence and to meet the increasing needs and requirements of consumers.

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Polaris Market Research has segmented the global mesenchymal stem cells market on the basis of source type, application and region:

Mesenchymal Stem Cells Source Type Outlook (Revenue, USD Million, 2015 2026)

Bone Marrow

Umbilical Cord Blood

Peripheral Blood

Lung Tissue

Synovial Tissues

Amniotic Fluids

Adipose Tissues

Mesenchymal Stem Cells Application Outlook (Revenue, USD Million, 2015 2026)

Injuries

Drug Discovery

Cardiovascular Infraction

Others

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Mesenchymal Stem Cells Regional Outlook (Revenue, USD Million, 2015 2026)

North America

U.S.

Canada

Europe

Germany

UK

France

Italy

Spain

Russia

Rest of Europe

Asia-Pacific

China

India

Japan

Singapore

Malaysia

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Argentina

Rest of LATAM

Middle East & Africa

What are the aspects of this report that relate to regional analysis?

The reports geographical regions include North America, Europe, Asia Pacific, Latin America, the Middle East, and Africa.

The report provides a comprehensive analysis of market trends, including information on usage and consumption at the regional level.

Reports on the market include the growth rates of each region, which includes their countries, over the coming years.

How are the key players in the market assessed?

This report provides a comprehensive analysis of leading competitors in the market.

The report includes information about the key vendors in the market.

The report provides a complete overview of each company, including its profile, revenue generation, cost of goods, and products manufactured.

The report presents the facts and figures about market competitors, alongside the viewpoints of leading market players.

A market report includes details on recent market developments, mergers, and acquisitions involving the key players mentioned.

What is the key information extracted from the report?

Extensive information on factors estimated to affect the Market growth and market share during the forecast period is presented in the report.The report offers the present scenario and future growth prospects Market in various geographical regions.The competitive landscape analysis on the market as well as the qualitative and quantitative information is delivered.The SWOT analysis is conducted along with Porters Five Force analysis.The in-depth analysis provides an insight into the Market, underlining the growth rate and opportunities offered in the business.

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Mesenchymal Stem Cells Market Growth Drivers 2021, Industry Share-Size, Global Demand, Emerging Trends, Opportunities in Grooming Regions, Key Players...

Bone Marrow Stem Cells Comments Off on Mesenchymal Stem Cells Market Growth Drivers 2021, Industry Share-Size, Global Demand, Emerging Trends, Opportunities in Grooming Regions, Key Players… | December 10th, 2021

What happened

Shares of Longeveron (NASDAQ:LGVN), a clinical-stage biotechnology company headquartered in Miami, are soaring in response to good news from the U.S. Food and Drug Administration (FDA). Investors excited about an orphan drug designation for the company's stem cell treatment pushed the stock 36.7% higher as of 10:37 a.m. ET on Friday.

Longeveron is developing bone marrow-derived mesenchymal stromal cells for the treatment of a variety of age-related disorders including Alzheimer's disease. Today, the FDA granted the company's lead candidate, Lomecel-B, an orphan designation for the treatment of a rare condition called hypoplastic left heart syndrome.

Image source: Getty Images.

Generally, orphan drug designations aren't something to get excited about. The FDA hands them out like candy to just about anyone that intends to develop a drug for underserved patients with a rare condition.

If approved to treat hypoplastic left heart syndrome, this orphan drug designation can begin assisting Longeveron with a handful of useful benefits. The most important one is seven years of market exclusivity.

There isn't much of a difference between the bone marrow-derived stem cells in Lomecel-B and the stem cells biomedical scientists have been researching for decades. They've been renamed medical signaling cells in recent years, but evidence of a significant benefit for specific indications remains elusive.

Longeveron is now a good stock to buy, and betting against it could prove disastrous, too. In the first quarter of 2022, Longeveron intends to begin clinical trials with Lomecel-B as a treatment for Alzheimer's disease.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Here's Why Longeveron Is Surging Today - Motley Fool

Bone Marrow Stem Cells Comments Off on Here’s Why Longeveron Is Surging Today – Motley Fool | December 10th, 2021

Sanford Health is continuing to explore the potential of orthobiologics and regenerative medicine with a study that may advance treatment for those living with osteoarthritis.

This trial known as ENDURE, which is run in full compliance with the FDA, will examine the safety and effectiveness of adult, adipose (fat)-derived stem cells.

Im really excited to get the trial started, said Donella Herman, M.D., a specialist in sports medicine at Sanford Health. Im even more excited about a year from now because I feel like were just going to learn so much about how effective this treatment is and what it is effective for.

Learn more: Orthopedics regenerative medicine at Sanford Health

Essentially, orthobiologics and regenerative medicine tap into the bodys natural healing ability.

Orthobiologics, a part of regenerative medicine, refers to the use of biological substances found in the body to help treat muscle and bone issues.

This may include injuries to muscles, tendons, and ligaments and as the focus of this trial more chronic conditions such as osteoarthritis.

When used properly by qualified providers, cell therapies are proving to be effective treatment options.

Our goals are to collect data for the different branches of orthobiologics, Dr. Herman said. Do people with knee osteoarthritis respond better with PRP (platelet rich plasma) or bone-marrow derived cells or fat-derived stem cells? Which works best? Which works for the longest time? Thats the kind of information were looking for. Its that kind of ratio that we just dont have right now.

One of the obstacles in regenerative medicine at this point, Dr. Herman said, is that its often being marketed by people with dubious clinical qualifications. They use underdeveloped research and make unfounded claims.

Its become a little bit of a wild, wild west with stem cells and PRP, Dr. Herman said. There are pop-up shops all over the place. Its one thing to have access to these things and allow access to people, but if youre not doing that in a responsible way and using that opportunity to build on a knowledge base, its kind of smoke and mirrors.

In sharp contrast to that scenario, this trial aims to add to the bank of knowledge in regenerative medicine.

We know we can tell people what were putting in their knee or their hip or their shoulder because were testing, Dr. Herman said. A lot of other places theyre just injecting it. They dont know if the stem cells are viable or not.

A typical candidate for the study, as described by Dr. Herman, might have osteoarthritis in their wrist and would not want to sacrifice a loss of motion that could come with surgery. They also would likely have exhausted the steroid injections treatment options that offer temporary relief.

We think of regenerative medicine as a possible alternative therapy that may help people put off surgery, Dr. Herman said. I dont ever tell people it means you will never need surgery but what it does is hopefully buy a little time until youre ready for that. There are lots of times where well do steroid injections until those fail and then they have to get a new joint. Were hoping for this to give us a bridge.

Once a patient is approved to be part of the ENDURE study, the procedure includes:

Follow-ups are then completed in person and over the phone.

Qualified candidates must be 18 or older and:

The cost associated with the study is not currently covered by insurance. By enrolling in the ENDURE study, however, participants may be playing a role in advancing regenerative cell research that could have the potential to improve care for future generations.

Those interested in hearing more about possible eligibility in the study can call (605) 328-3700.

Posted In Innovations, Orthopedics, Research, Specialty Care, Sports Medicine

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Sanford Health opens study of cell injections for arthritis - Sanford Health News

Bone Marrow Stem Cells Comments Off on Sanford Health opens study of cell injections for arthritis – Sanford Health News | December 10th, 2021

Significance

The recently introduced RNA velocity methods, by leveraging the intrinsic RNA splicing process, have shown their unique capability of identifying the directionality of the cell differentiation trajectory. However, due to the minimal amount of unspliced RNA contents, the estimation of RNA velocity suffers from high noise and may result in less reliable trajectories. Here, we present Velocity Autoencoder (VeloAE), a tailored autoencoder to denoise RNA velocity for more accurate quantification of cell transitions. Through various biological systems, we demonstrate its effectiveness for correcting the inferred trajectory and its interpretability for linking the learned dimensions to underlying biological processes.

RNA velocity is a promising technique for quantifying cellular transitions from single-cell transcriptome experiments and revealing transient cellular dynamics among a heterogeneous cell population. However, the cell transitions estimated from high-dimensional RNA velocity are often unstable or inaccurate, partly due to the high technical noise and less informative projection. Here, we present Velocity Autoencoder (VeloAE), a tailored representation learning method, to learn a low-dimensional representation of RNA velocity on which cellular transitions can be robustly estimated. On various experimental datasets, we show that VeloAE can both accurately identify stimulation dynamics in time-series designs and effectively capture expected cellular differentiation in different biological systems. VeloAE, therefore, enhances the usefulness of RNA velocity for studying a wide range of biological processes.

Author contributions: C.Q. and Y.H. designed research; C.Q. performed research; C.Q. contributed new reagents/analytic tools; C.Q. analyzed data; and C.Q. and Y.H. wrote the paper.

The authors declare no competing interest.

This article is a PNAS Direct Submission.

This article contains supporting information online at https://www.pnas.org/lookup/suppl/doi:10.1073/pnas.2105859118/-/DCSupplemental.

VeloAE is an open-source Python package available at GitHub, https://github.com/qiaochen/VeloAE. All the analysis notebooks for reproducing the results are also available in this repository. Previously published data were used for this work (8, 10, 1925).

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Representation learning of RNA velocity reveals robust cell transitions - pnas.org

Bone Marrow Stem Cells Comments Off on Representation learning of RNA velocity reveals robust cell transitions – pnas.org | December 10th, 2021

Jack Stevens was diagnosed with juvenile myelomonocytic leukaemia and underwent stem cell treatment but has sadly lost his battle as his father's former clubs pay tribute

The young son of former England footballer Gary Stevens has died tragically aged four.

Stevens son Jack was diagnosed with a rare form of blood cancer.

Just a year ago his family were hopeful of a recovery after Jack underwent a stem cell transplant following his diagnosis with juvenile myelomonocytic leukaemia.

The stem cells Jack received from older brother Oliver had engrafted, meaning they had entered Jacks bone marrow, enabling him to produce his own white blood cells.

The transplant procedure was brought forward after Jack responded to two rounds of preparatory chemotherapy, negating an original plan for five cycles of treatment.

He was discharged to a nearby apartment and during the week lived with mum Louise.

Stevens a brilliant right-back in Howard Kendalls magical mid-1980s Everton team and the couples other sons, Oliver and Josh, would join Jack and Louise at weekends.

This morning Everton said: "Everyone at Everton is deeply saddened to learn that Gary Stevens four-year-old son, Jack, has passed away following his courageous battle with leukaemia.

"Our thoughts are with Gary and his family at this incredibly sad time."

Image:

Stevens, 59, began his football career with Everton, representing the Toffees for six seasons and making over 200 appearances between 1982-1988.

He then transferred to Glasgow Rangers where he played a part in their treble-winning season in 1993.

After another six-year stint, he returned to Merseyside to finish his career plying his trade for Tranmere Rovers.

Stevens earned 46 caps for England during his playing days before moving into physiotherapy after his retirement.

Tranmere shared Everton's post and issued their own touching message, which read: "The thoughts of everyone at Tranmere Rovers are with Gary Stevens and his family at this sad time."

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Former England and Everton footballer Gary Stevens' son tragically dies aged four - Mirror.co.uk

Bone Marrow Stem Cells Comments Off on Former England and Everton footballer Gary Stevens’ son tragically dies aged four – Mirror.co.uk | November 22nd, 2021

PRC Clinical to Provide Start-up CRO Services for BRTX-100 Phase 2 Clinical Trial

MELVILLE, N.Y., Nov. 19, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company" or BioRestorative) (NASDAQ:BRTX), a life sciences company focused on adult stem cell-based therapies, today announced that it has entered into a letter of intent with PRC Clinical, a CRO specializing in clinical trial management, with regard to PRC Clinical providing startup clinical project management activities for the Companys BRTX-100 Phase 2 clinical trial to treat chronic lumbar disc disease.

We are pleasedto announce that we have entered into a letter of intent for PRC Clinical to provide startup activities for our Phase 2 study. PRC has extensive experience and expertise in managing clinical studies in the stem cell and regenerative medicine space. They also have theexperienced and professionalnetwork of clinicians and study sites streamlining patient enrollment, site monitoring and management. Additionally, we have been working with and familiarizing ourselves with PRCs team and capabilities since 2019. We are thrilled to finally be in a position to begin the process of validating our technology through the FDA process, while keeping shareholders updated along the regulatory pathway, said Lance Alstodt, CEO of BioRestorative.

PRC Clinical has provided specialty CRO services for nearly 20 years. Their innovative approach to executing studies for biotech and pharmaceutical companies combines high-touch human elements and cutting-edge technology with extensive experience and deep therapeutic knowledge. PRC Clinical is an all inclusive CRO and has specialized expertise across regenerative medicine, CNS, ophthalmology, pulmonary and COVID-19, rare and orphan disease and more complex indications.

PRC Clinical is pleased to begin start-up CRO activities for BRTX-100. We look forward to being able to bring our stem cell experience to this trial. We are committed to supporting BioRestoratives development of BRTX-100 and its clinical application, said Curtis Head, CEO of PRC Clinical.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:

Email: ir@biorestorative.com

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BioRestorative Therapies Enters into Letter of Intent with PRC Clinical - GlobeNewswire

Bone Marrow Stem Cells Comments Off on BioRestorative Therapies Enters into Letter of Intent with PRC Clinical – GlobeNewswire | November 22nd, 2021

About five years ago, UGBC President Jack Bracher joined the registry to become a potential stem cell donor. A few years later, Bracher found out that he had a match.

I got a call from the nonprofit that Id done it through saying that I was a match for a patient with leukemia, so I had started going through the process of being able to donate to him, Bracher, MCAS 22, said. Fortunately he went into remission, so my donation wasnt needed, but I wanted to find a way to bring back the stem cell drive to Boston Colleges campus and register more students to donate.

The Undergraduate Government of Boston College (UGBC), Student Athlete Advisory Committee (SAAC), and Project Life Movement teamed up on Thursday and Friday to encourage students to join the global registry of potential bone marrow and stem cell donors.

Project Life Movement ambassador Luke Kuechly, former linebacker for the Carolina Panthers and BC 11, returned to campus to encourage students to get swabbed.

The University last hosted a Project Life Movement bone marrow registration event when BC retired Kuechlys jersey in 2016. Over 800 people joined the registry. This year, 777 people registered, according to Bracher, with 480 students getting swabbed on the first day. Bracher said this was the most swabs Project Life Movement got on a single day at a college campus.

Steve Luquire, a co-chair of Project Life Movement, said the number of students swabbed at BC exceeds what it defines as a good number for most colleges.

On most college campuses where we go to do these drives, 200 is a good drive, Luquire said. Were gonna be here today because of SAAC and the student government, and weve already done I think close to 250 in less than two hours.

College campuses are the best place to find a healthy, diverse group willing to join the registry, according to Luquire.

My wife of 41 years died of myelodysplasia syndrome, Luquire said. Her only match was her brother, who was 60 years old, and frankly, it works so much better if you have a person who is 18 to 35. And theres no place better than a college campus to find people who are willing to look at the vision and mission and join us.

Kuechly, who met Luquire in 2013, said they have been hosting drives together since then to raise awareness and improve the chances of finding donor matches.

We just know this little bit of time that we spend today and tomorrow and having you guys come by, we can raise awareness to potentially have a match for somebody that needs it, Kuechly said.

Finding even a few donor matches, Kuechly said, is a huge deal.

You might have five to 500 to 1000 people here, but if you can get a couple donors that match, thats whats powerful, Kuechly said.

Students joining the registry, Kuechly said, is a perfect example of BC students being men and women for others.

The big pillar in the Jesuit community is how can you help other people by being selfless with your time, and this is a perfect example of it, he said.

The impact of becoming a donor goes beyond just saving a persons life, according to Ann Henegar, executive director of Project Life Movement.

When you donate your stem cells or your bone marrow to a patient, youre not only saving that persons life, youre affecting a community, youre affecting a family, you know, a workplace, a campus, Henegar said.

Henegar said she encourages people to think about the impact they can make by registering to be a donor.

This is what I tell everybody If it were your sister, your boyfriend, your girlfriend, your aunt, your child, wouldnt you want someone to say yes? she said.

For Lubens Benjamin, CSOM 23, joining the registry is a great way of fulfilling BCs mission.

I think part of being someone who goes to BC is being a person for others and, like, this is right along with that mission, Benjamin said. If I could be a match for someone, if I can help someone extend their life, thats just something great to be a part of and I dont see why Id say no to that.

Jostine Rozenich, MCAS 25, spoke to the importance of taking time out of the day to join the registry.

Its such a crucial and important thing, even if it only takes a few minutes and it can save lives, Rozenich said. I think thats all about finding ways to put service into your daily life.

Rozenich said she has family members who have needed various transplants that rely on others to donate, which has shaped her perspective on joining the registry.

Why not go ahead and do that and save the life of somebody? she said. That is such a scary feeling to not know whether or not youre going to get a match.

Ultimately, it is a privilege to be part of a drive like this, Bracher said, and hosting the event just before the Red Bandanna Gamean annual football game that commemorates Welles Crowther, the BC alumnus credited with saving the lives of about a dozen people during the Sept. 11 terrorist attacksis a great way of uniting the mission of Project Life Movement and Crowthers story.

I think its just a great privilege to be able to work with Project Life and Luke, as well as the Student Athlete Advisory Committee, who hosted the drive that I was a match for, and for all that to come full circle, Bracher said. And for us to of course be doing it on Red Bandanna weekend of all weekends means a lot.

Featured Image by Ikram Ali / Heights Editor

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Kuechly Returns to Campus for Stem Cell and Bone Marrow Registry Drive The Heights - The Heights

Bone Marrow Stem Cells Comments Off on Kuechly Returns to Campus for Stem Cell and Bone Marrow Registry Drive The Heights – The Heights | November 8th, 2021

PlateletBio, a company developing a new class of cell therapies based on the biology of platelets, has raised $75.5 million to advance its drug pipeline, including a lead candidate for a rare bleeding disorder on track to reach the clinic next year.

Platelets are components of blood best known for their role forming clots that stop bleeding. But Watertown, Massachusetts-based PlateletBio notes that platelets have other properties, including a role delivering growth factors and proteins throughout the body. PlateletBio is developing therapies that take advantage of these properties, but rather than using platelets from a patient or healthy donors, the startup makes them.

In the body, platelets are formed in bone marrow. PlateletBio produces its platelet-like cells, or PLCs, inside a bioreactor that mimics bone marrow conditions. The source material for its PLCs are stem cells, which have the ability to become almost any cell or tissue in the body.

Platelets are technically not cells. They dont have a nucleus, but thats an advantage for therapeutic applications. Since a PlateletBio therapy wont introduce DNA into a patients body, the potential risks that come from introducing foreign genetic material are avoided. PlateletBio says it can produce PLCs with new features and therapeutic payloads that include antibodies, signaling proteins, therapeutic proteins, and nucleic acids.

PlateletBios lead cell therapy candidate is being developed to treat immune thrombocytopenia, a blood disorder in which the immune system mistakenly sees a patients platelets as foreign and destroys them. Immune thrombocytopenia patients have dangerously low platelet counts that make them susceptible to bleeding.

There is no FDA-approved treatment for the underlying cause of immune thrombocytopenia, but corticosteroids are used to try to dampen the immune systems attack on platelets. Platelet transfusions are another option, but the National Organization for Rare Disorders notes that these treatments are usually reserved for emergencies because the platelets are likely to be destroyed by antibodies produced by the patient.

Patients who have not responded to earlier treatments have two FDA-approved small molecule options: Tavalisse, from Rigel Pharmaceuticals, and the Swedish Orphan Biovitrum drug Doptelet. Sanofi aims to treat the disease with a small molecule called rilzabrutinib. That drug is designed to block Brutons tyrosine kinase, a protein that plays a role in the development of a B cells, a type of immune cell. Sanofi acquired the molecule last year via its $3.7 billion acquisition of Principia Biopharma.

The lead disease target for the Principia drug was multiple sclerosis. In September, Sanofi reported that rilzabrutinib failed that Phase 3 study. A separate Phase 3 test in immune thrombocytopenia is ongoing, as is a mid-stage clinical trial in another autoimmune condition called IgG4-related disease.

PlateletBio isnt the only company trying to turn a component of the blood into a new type of cell therapy. Cambridge, Massachusetts-based Rubius Therapeutics is developing cell therapies based on red blood cells. After disappointing early clinical trial results in the rare disease phenylketonuria last year, Rubius shifted its focus to cancer and immune system disorders. PlateletBios PLCs would represent an entirely new approach to treating immune thrombocytopenia. According to PlateletBios website, the company plans to file an investigational new drug application for its therapeutic candidate in the first half of next year.

PlateletBio is based on the research of Harvard University scientist Joseph Italiano, who co-founded the company under the name Platelet BioGenesis. When the startup emerged in 2017, it was developing platelets that could address the platelet shortage problem facing blood donation centers. Two years ago, the startup expanded its Series A round with $26 million in additional financing and plans to make its platelets into cell therapies. Besides immune thrombocytopenia, other diseases the biotech aims to treat include osteoarthritis and liver fibrosis.

PlateletBios latest financing, a Series B round, adds new investors SymBiosis, K2 HealthVentures, and Oxford Finance. Earlier investors Ziff Capital Partners and Qiming Venture Partners also participated in the new round.

This is a major milestone for PlateletBio, adding capital and resources needed to advance our innovative platelet-like cell therapy science and manufacturing platform and support key corporate initiatives over the next 18 to 24 months, Sam Rasty, the startups president and CEO, said in a prepared statement.

Photo by Flickr user Marco Verch via a Creative Commons license

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Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test - MedCity News

Bone Marrow Stem Cells Comments Off on Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test – MedCity News | November 8th, 2021

TORONTO -- Owen Snider was given as little as three months to live. His blood cancer had returned and the prognosis was not good. The news, delivered over the phone during the height of the early pandemic lockdown in spring 2020, was devastating.

The Ottawa-area retiree began putting his affairs in order, preparing for what appeared to be inevitable.

It was terrible, his wife Judith Snider told CTV News. But we finally decided that what we had to do was to live each day not to look forward to the end, but to look forward to tomorrow.

And yet, a year later, Snider is alive -- transformed, even -- and his non-Hodgkins lymphoma is in remission. His second chance is all thanks to a promising, Canadian-run program for cancer treatment called CAR T-cell therapy.

Snider became one of the first patients to participate in a national research program that is assessing whether this experimental treatment can be done safely in Canada and cheaper than in the U.S., where costs can run upwards of half a million dollars per patient.

I think I am a pretty successful lab rat, Snider, who previously endured chemotherapy treatments and a stem cell transplant, said in an interview.

Thirty days after treatment, the lymphoma was gone. So how can you not be happy about that?

CAR T-cell therapy is a type of gene therapy that trains or engineers a patients own immune system to recognize cancerous cells. A type of white blood cell, called a T-cell, is a key component of a bodys immune system. They are developed from stem cells in the bone marrow and help fight infection and disease by searching and targeting specific foreign substances, known as antigens, in the body.

The protein receptors on T cells bind to the protein antigens on the surfaces of foreign particles that fit those receptors, like a lock and key. The foreign substance is eradicated once their antigens are bound to a T-cell. But blood cancer cells are normal cells that undergo mutations, so they are not recognized as a foreign threat to the body. In other words, T-cells generally do not have the right receptor key to fit with the antigens of a cancer cell.

CAR T-cell therapy modifies the cells so they are able to identify the cancer cells and destroy them. Its a labour-intensive process that involves taking blood from a patient and separating the T-cells. Then scientists add a gene to the cells that gives them instructions to develop an artificial receptor called a chimeric antigen receptor, or CAR.

We actually take the T-cells out and we modify them in the lab and put them back into the patient. So now they're able to recognize the cancer and kill it off, explained Dr. Kevin Hay, Medical Director for Clinical Cell Therapy with BC Cancer.

I think we're just at the cusp of really understanding what this is going to do for patients in the future.

The therapy is a labour-intensive process -- Snider's cells were shipped to Victoria, B.C to be processed in a special lab facility, then shipped back to Ottawa about a week later, where they were infused back into his body.

The treatment is still being studied, but is already available for some cancers in the U.S. and Canada at a steep price.

Researchers began trials in Canada in 2019 to see if it could be done domestically at a lower cost, highlighting the importance of having key medical production and therapies available in Canada.

We knew we had to do domestic manufacturing and if we've learned anything from COVID-19, it's that domestic capability is really important when it comes to science and medicine, and this is a perfect example of that, said Dr. Natasha Kekre, a hematologist and lead researcher on the trail based at the Ottawa Hospital.

Progress was impacted slightly by the pandemic, but Snider was fortunate enough to participate and is the first patient to come forward to discuss their experience and why he hopes the program will expand across Canada to help others dealing with otherwise untreatable forms of cancer.

Scientists are hoping to release more data in the coming months -- more than 20 patients have been treated so far, according to Dr. Kekre.

This is hopefully just the beginning for us. So this first trial was a foundation to prove that we could actually manufacture T cells, that we could do this in a clinical trial. And so this trial will remain open for patients who are in need, she said.

So absolutely we feel like were opening a door.

Snider's first experience with cancer treatment was more than a decade ago, in 2010, when he underwent a powerful and aggressive chemotherapy regimen that helped him stay cancer-free for six years.

But the treatment was so harsh that when his cancer came back in 2016, doctors told him he could not go through that kind of chemotherapy again. Instead, Snider underwent a stem cell transplant, which gave him another four years without cancer, until April 2020.

This time the outlook was grim, so doctors decided to try and get him into the CAR T-cell trials that started just before the pandemic hit. The study was specifically for patients with acute lymphoblastic leukemia and non-Hodgkins lymphoma who were not responding to other treatments.

Snider said the entire process was a walk in the park compared to what he had gone through before. He was given a mild chemotherapy treatment for three days while his T-cells were being modified in a lab on the other side of the country.

[The T-cells] went to work right away. There's a period of time where there's a lot going on inside fighting each other and that sort of thing. You don't feel great or you don't really know how you feel, Snider described. The treatment was met with outstanding success.

And in 30 days, there was no lymphoma. I couldn't believe it.

For Dr. Kekre, the results bring hope. Snider has done quite well and does not have any evidence of lymphoma at the moment, she said.

I'm unfortunately in a business where I often have to give bad news, and it is really motivating and exciting to be able to offer therapies to patients who didn't have options and to make them better, she said.

The trial is currently at the stage where scientists are making sure the product remains safe. Side-effects can include neurotoxicity, which harms the nervous system, and cytokine release syndrome, which triggers an acute system-wide inflammatory response that can result in organs not functioning properly. But so far researchers have, for the most part, been able to manage and reverse any side effects.

With such promising outcomes for patients who otherwise had no options left, researchers are talking about expanding these studies across Canada and to other forms of cancer. For now, the lab in Victoria is the only facility equipped to make these cell modifications.

I think its really going to be revolutionary with how we treat cancer in the future, not just blood cancers, but all cancers, said Dr. Hay.

Today, Snider is healthy and strong, even able to chop wood at his home near Ottawa. He and his wife Judith, a retired federal judge, are enjoying life anew.

It certainly has given us a future that we didnt know we had, she said.

The treatment not only bought Snider extra time, but also significantly improved his quality of life.

What was given to me is practically a normal life, he added.

It's really just transformed, not just extended, but transformed my life.

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CAR T-cell therapy: Hear from a Canadian patient - CTV News

Bone Marrow Stem Cells Comments Off on CAR T-cell therapy: Hear from a Canadian patient – CTV News | November 8th, 2021

Two kidney transplant patients who received a stem cell therapy developed by Talaris Therapeutics were able to come off all immunosuppressant drugs within a year, without any evidence of graft rejection.

The first findings from Talaris phase 3 trial of the cell therapy called FCR001 suggest it may be possible to eliminate the need entirely for patients to take what may be dozens of tablets daily after organ transplants, according to the US biotech.

While still preliminary, the experience with the two patients back up Talaris hope that giving a one-shot dose of FCR001 the day after an organ transplant could stimulate immune tolerance in the recipient, and avoid the side effects of current drug treatments such as infections, heart disease, and some forms of cancer.

The companys approach relies on administering haematopoietic stem cells from the individual who donated the organ, in order to generate what Talaris refers to as chimerism, with both donor and recipient cells present in the bone marrow. That allows the immune system to see the transplanted organ as self rather than foreign.

The first two recipients in Talaris FREEDOM-1 phase 3 trial had received FCR001 at least 12 months earlier, and showed stable kidney function, according to Talaris.

A larger group of five patients who were at least three months from the cell therapy maintained more than 50% chimerism in their T cells, which the biotech said was a sign of long-term, immunosuppression-free tolerance to the donated kidney in its phase 2 trials.

The FREEDOM-1 results reported at the American Society of Nephrology (ASN) meeting this week were accompanied by updated results from Talaris phase 2 study, in which all 26 patients originally weaned off immunosuppressants have continued to remain off them without rejecting their donated kidney.

Some transplant patients treated with Talaris therapy in earlier trials have now been off all immunosuppression for more than 12 years without signs of kidney rejection.

Talaris intends to enrol 120 subjects into the phase 3 trial, which is scheduled to generate results in 2023.

Earlier this year, Talaris raised $150 million via a Nasdaq listing that will be used to take FCR001 through the phase 3 programme in organ transplantation and as a treatment for rare autoimmune disease scleroderma.

It also recently started a phase 2 trial of the cell therapy to see if it is able to induce immune tolerance to a transplanted kidney in patients who received the transplant from a living donor up to a year prior to administration of FCR001.

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Talaris therapy ends need for immune drugs in transplant patients - - pharmaphorum

Bone Marrow Stem Cells Comments Off on Talaris therapy ends need for immune drugs in transplant patients – – pharmaphorum | November 8th, 2021

TEL AVIV, Israel, Nov. 4, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical Company focused on oncology, today announced an oral presentation and three poster presentations at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, which is being held December 11-14, 2021 in Atlanta, GA, and virtually.

The oral presentation will elaborate on the successful results of the Company's GENESIS Phase 3 pivotal trial. The study showed highly significant and clinically meaningful results supporting the use of Motixafortide on top of G-CSF for mobilization of stem cells for subsequent collection and transplantation in patients with multiple myeloma. In addition, the poster presentations will show that extended inhibition of the CXCR4 receptor by Motixafortide results in the mobilization of high numbers of stem cells, including specific sub-populations, which were correlated with reduced time to engraftment when infused in high numbers.

The Company is also presenting findings from in-vivo and in-vitro pre-clinical studies demonstrating that Motixafortide acts as an immunomodulator by affecting the biology of regulatory T cells (Tregs), supporting biomarker findings from the Company's COMBAT Phase 2 study in pancreatic cancer patients.

"We are very pleased with the breadth of our oral and poster presentations at this year's ASH meeting, which reflect the versatility of Motixafortide as the potential backbone of promising new treatments for both hematological and solid tumor cancers," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Of particular note is the oral presentation on the outstanding results from our GENESIS Phase 3 pivotal study in stem cell mobilization demonstrating that Motixafortide effectively mobilizes a high number of cells enabling ~90% of patients to undergo transplantation following a single administration of Motixafortide and a single apheresis session. In addition, the high number of cells mobilized by Motixafortide enables infusion of an optimal number of cells, which could result in faster time to engraftment, and also allows for cryopreservation for future transplantation(s). These results, together with our recently completed successful pharmacoeconomic study, strongly support our view that Motixafortide on top of G-CSF can become the new standard of care in SCM, if approved, to the benefit of patients and payers alike. We look forward to submitting an NDA in the first half of next year, as previously communicated."

Further details of the presentations are provided below.

Oral Presentation

Title: Motixafortide (BL-8040) and G-CSF Versus Placebo and G-CSF to Mobilize Hematopoietic Stem Cells for Autologous Stem Cell Transplantation in Patients with Multiple Myeloma: The GENESIS Trial

Date: Sunday, December 12, 2021

Time: 12:00 PM

Location: Georgia World Congress Center, Hall A1

This oral presentation describes the GENESIS Phase 3 pivotal trial design, endpoints and results. The GENESIS study was a double blind, placebo controlled, multicenter trial, in which 122 patients were randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for stem cell mobilization prior to stem cell transplant in multiple myeloma patients. Total CD34+ cells/kg were analyzed on site to determine if patients mobilized to the goal and all samples were subsequently sent for assessment by a central laboratory. The number of CD34+ cells infused was determined independently by each investigator according to local practice.

The study concluded that a single administration of Motixafortide on top of G-CSF significantly increased the proportion of patients mobilizing 6x106 CD34+ cells/kg for stem cell transplantation (92.5%) vs G-CSF alone (26.2%) in up to two apheresis days (p<0.0001), while enabling 88.8% to collect 6x106 CD34+ cells/kg in just one apheresis day (vs 9.5% with G-CSF alone; p<0.0001). In addition, the median number of hematopoietic stem cells mobilized in one apheresis day with Motixafortide + G-CSF was 10.8x106 CD34+cells/kg vs 2.1x106 CD34+ cells/kg with G-CSF alone.

Poster Presentations

Title:Autologous Hematopoietic Cell Transplantation with Higher Doses of CD34+ Cells and Specific CD34+ Subsets Mobilized with Motixafortide and/or G-CSF is Associated with Rapid Engraftment A Post-hoc Analysis of the GENESIS Trial

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

The CD34+ hematopoietic stem and progenitor cell (HSPC) dose infused during stem cell transplantation remains one of the most reliable clinical parameters to predict quality of engraftment. A minimum stem cell dose of 2-2.5x106 CD34+ cells/kg is considered necessary for reliable engraftment, while optimal doses of 5-6x106 CD34+ cells/kg are associated with faster engraftment, as well as fewer transfusions, infections, and antibiotic days.

An analysis was performed using pooled data from all patients in the GENESIS trial to evaluate time to engraftment based on the total number of CD34+ cells/kg infused, as well as specific numbers of CD34+ cell sub-populations infused.

The addition of Motixafortide to G-CSF enabled significantly more CD34+ cells to be collected in one apheresis (median 10.8x106 CD34+ cells/kg) compared to G-CSF alone (2.1x106 CD34+ cells/kg), as well as 3.5-5.6 fold higher numbers of hematopoietic stem cells (HSCs), multipotent progenitors (MPPs), common myeloid progenitors (CMPs) and granulocyte and macrophage progenitors (GMPs) (all p-values <0.0004). A dose response was observed with a significant correlation between faster time to engraftment and infusion of higher number of total CD34+ HSPC doses (6x106 CD34+ cells/kg) and combined HSC, MPP, CMP and GMP subsets. The high number of CD34+ cells/kg mobilized with Motixafortide on top of G-CSF enables the potential infusion of 6x106 CD34+ cells/kg, as well as cryopreservation of cells for later use.

Title: Immunophenotypic and Single-Cell Transcriptional Profiling of CD34+ Hematopoietic Stem and Progenitor Cells Mobilized with Motixafortide (BL-8040) and G-CSF Versus Plerixafor and GCSF Versus Placebo and G-CSF: A Correlative Study of the GENESIS Trial

Date: Monday, December 13, 2021

Time: 6:00 PM - 8:00 PM

CD34 expression remains the most common immunophenotypic cell surface marker defining human hematopoietic stem and progenitor cells (HSPCs). The addition of CXCR4 inhibitors to G-CSF has increased mobilization of CD34+ HSPCs for stem cell transplantation; yet the effect of CXCR4 inhibition, with or without G-CSF, on mobilization of specific immunophenotypic and transcriptional CD34+ HSPC subsets is not well-characterized.

Motixafortide is a novel cyclic peptide CXCR4 inhibitor with a low receptor-off rate and extended in vivo action when compared to plerixafor. GENESIS Phase 3 trial patients were prospectively randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for HSPC mobilization. Demographically similar multiple myeloma patients undergoing mobilization with plerixafor + G-CSF prior to stem cell transplant were prospectively enrolled in a separate tissue banking protocol.

Extended CXCR4 inhibition with Motixafortide + G-CSF mobilized significantly higher numbers of combined CD34+ HSCs, MPPs and CMPs compared to plerixafor + G-CSF or G-CSF alone (p<0.05). Additionally, Motixafortide + G-CSF mobilized a 10.5 fold higher number of immunophenotypically primitive CD34+ HSCs capable of broad multilineage hematopoietic reconstitution compared to G-CSF alone (p<0.0001) and similar numbers compared to plerixafor + G-CSF. Furthermore, lack of CXCR4 inhibition resulted in mobilization of more-differentiated HCSs, whereas extended CXCR4 inhibition with Motixafortide + G-CSF (but not plerixafor + G-CSF) mobilized a unique MPP-III subset expressing genes specifically related to leukocyte differentiation.

Title: The High Affinity CXCR4 Inhibitor, BL-8040, Impairs the Infiltration, Migration, Viability and Differentiation of Regulatory T Cells

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

This poster describes results of pre-clinical in-vivo and in-vitro studies demonstrating that Motixafortide potentially acts as an immunomodulator by affecting the biology of regulatory T cells. Motixafortide reduced the amount of infiltrating Tregs into the tumors, impaired the migration of Tregs toward CXCL12 and induced Tregs cell death. Furthermore, Motixafortide was found to inhibit the differentiation of nave CD4 T cells toward Tregs.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, has reported positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:

Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx Announces an Oral Presentation and Three Poster Presentations at the 63rd American Society of Hematology (ASH) Annual Meeting &...

Bone Marrow Stem Cells Comments Off on BioLineRx Announces an Oral Presentation and Three Poster Presentations at the 63rd American Society of Hematology (ASH) Annual Meeting &… | November 8th, 2021

Rachel B. Salit, MD, discussed the case of a 48-year-old patient with graft-versus-host-disease.

Rachel B. Salit, MD, associate professor, Clinical Research Division, Fred Hutchinson Cancer Research Center at the University of Washington School of Medicine in Seattle, WA, discussed the case of a 48-year-old patient with graft-versus-host-disease.

Targeted OncologyTM: What are your thoughts on the currently accepted options for acute GVHD (aGVHD) prophylaxis?

SALIT: Between calcineurin inhibitors, if we have a choice, my preference is usually tacrolimus. Tacrolimus is better tolerated [than cyclosporin] in terms of adverse events [AEs], blood pressure, kidney function, and [even] the smell.

Methotrexate is a tried-and-true prophylaxis, especially in the myeloablative or high-intensity transplant setting. [In contrast], mycophenolate mofetil [MMF]; [CellCept] is usually used in the nonmyeloablative or reduced-intensity setting. When calcineurin inhibitors were used with MMF as prophylaxis for GVHD, the GVHD was higher. Thats why we [use] methotrexate [instead of MMF].1

Sirolimus [Rapamune] is often combined with a calcineurin inhibitor and MMF, or with a calcineurin inhibitor and methotrexate. Sirolimus is very well tolerated, except for some triglyceride AEs. Additionally, the combination of sirolimus plus MMF and a calcineurin inhibitor has been shown to significantly decrease GVHD in the reduced-intensity setting compared with [the effect observed with] MMF and a calcineurin inhibitor alone.2

CAR [chimeric antigen receptor] T-cellantibody therapy plus antithymocyte globulin [ATG] and alemtuzumab [Lemtrada] are more frequently used in Europe [than in the United States]. There have been mixed results, and there is some concern of increased relapse with [anti-thymocyte globulin (ATG) therapy]. Ex vivo T-cell depletion and CD34-positive cell selection [are] also uncommon in the United States.

Posttransplant cyclophosphamide [Cytoxan] [is becoming more common], and it was originally [used in the setting of] haploidentical transplants. Now it is increasingly used in the unrelated donor setting, and Im sure it will be translated to the sibling setting, too. [This regimen] has been shown to decrease effector T cells.3 Moreover, chronic GVHD is [reduced by this regimen], but aGVHD is not changed.

A recent study retrospectively compared many patients [with haploidentical donors] to a smaller number of patients [who had] unrelated donors and who received posttransplant cyclophosphamide. The data showed that the patients with unrelated donors and posttransplant cyclophosphamide had better overall survival [OS] and decreased relapse compared with the patients with haploidentical donors.4

For a long time, [most trials that compared GVHD and OS between patients with haploidentical] vs unrelated or sibling donors have shown that posttransplant [cyclophosphamide in the setting of haploidentical] transplants is associated with reduced chronic GVHD, but the other outcomes were the same. Is this result attributable to the fact that the transplant is haploidentical, or is it attributable to the posttransplant cyclophosphamide? I think that question will be answered within the next [few] years.

What risk factors for GVHD do you notice in the case described?

There are multiple risk factors. The fact that the donor is multiparous puts the recipient at higher risk for GVHD. The patients high intensity, myeloablative conditioning regimen increases the risk for GVHD, as do the donors CMV seropositivity and the fact that the patient and donor are not sex matched. The risk of GVHD also increases with donor age.

Risk for GVHD is also increased by major human leukocyte antigen [HLA] disparity. We look at class I [HLA-A, -B, and -E] and class II [HLA-DR and -DQ] antigens, with a 10 out of 10 score constituting a match. There are data coming out that show that the class II antigen HLA-DP also matters in certain cases5; a match that includes this antigen [a 12 out of 12 (score)] is better than a 10 out of 10 [score]. [This patients donor was HLA-matched, but] minor HLA mismatches can increase the risk of GVHD [in patients like this one whose donor is unrelated].

Stem cell source and graft composition are other considerations, but this patient received peripheral blood, which confers a higher risk of GVHD than does bone marrow. Peripheral blood has a higher CD34-positive cell count, therefore a higher T-cell dose; both factors increase GVHD risk. At our center, we dont often cap CD34-positive cell count or T-cell dose, except in the haploidentical setting.

I would not include ABO blood type as a risk factor. There are mixed data regarding whether major and minor ABO mismatches lead to increased GVHD.6

What standardized guidelines exist for organ staging and grading in the context of aGVHD?

[According to Mount Sinai Acute GVHD International Consortium], the skin, the liver, and gastrointestinal [GI] tract are the 3 organs included in aGVHD staging. The skin is [described in terms of] the percentage of body surface area [BSA] affected. Stage 0 is no rash, stage 1 is a rash covering less than 25% of the BSA, stage 2 is a rash covering 25% to 50% of the BSA, stage 3 is a rash covering greater than 50% of the BSA, and stage 4 is generalized erythroderma.7

According to the liver status [bilirubin level], staging starts at stage 0 [less than 2 mg/dL] and progresses through stage 1 [2-3 mg/dL], stage 2 [3.1-6 mg/dL], and stage 3 [6.1-15 mg/dL] to a final stage of 4 [greater than 15 mg/dL]. The lower GI staging system [counts] the number of episodes per day of liquid stool output. Stage 0 is fewer than 3 episodes [of stool output], stage 1 is 3 to 4 episodes, stage 2 is 5 to 7 episodes, and stage 3 is greater than 7 episodes. If you have an inpatient, then you can use these exact quantities. If you have an outpatient, you can use these values as rough markers. Regarding the upper GI staging system, in stage 0, nausea, vomiting, or anorexia are absent or intermittent, but in stage 1, they are persistent.

The other thing I often look at [to judge] severity [of GI involvement] is the electrolytes. For example, if the patient says they are having 5 episodes of stool a day, but their potassium and magnesium are normal and theyre not becoming acidemic, then you [might consider that] these stools are only of small volume. If the patient starts to have electrolyte abnormalities or starts to become acidemic, then you [should consider that] maybe theyre having more diarrhea than theyre [telling you].

When we grade according to most severe target organ involvement, grade I reflects the presence only of stage 1 to 2 skin involvement. Any GI or liver involvement is automatically [at least grade] II, the point at which you would consider treating symptoms topically. Grade IIA indicates upper GI involvement, and grade IIB indicates lower GI involvement.8 Once the patient gets to grade III, they almost always [require] systemic therapy.7

What stage and grade would you give this patient?

With 60% skin involvement, he would have a skin stage of 3, and with 4 episodes of diarrhea per day, he would have a lower GI stage of 1. [He would have an overall clinical grade of IIB.]

What are GVHD and aGVHD biomarkers, and how are they used?

Biomarkers of GVHD are markers of inflammation found in the blood that will tell you the patient is at a higher risk for developing GVHD. These biomarkers include elafin, IL-2 receptor-, IL-8, tumor necrosis factor receptor-1, hepatocyte growth factor, and regenerating islet-derived 3- [NCT00224874].9 The use of biomarkers [to predict patients risk of developing GVHD could guide physicians as they choose] a starting steroid dosage, eg, 2 mg/kg vs 1 mg/kg.

What data guide your decisions about steroid therapy in GVHD?

Although the concept of GVHD and aGVHD risk stratification is not generally used in practice, high-risk GVHD vs standard-risk GVHD has been shown to be associated with a lower rate of complete response to steroids [27% vs 48%, respectively; P < .001] and higher treatment-related mortality [incidence at 6 months after steroid therapy onset, 44% vs 22%, respectively; P < .001].10 If a patient has a higher grade of GVHD, they are more likely to be steroid refractory.

The steroid response of GVHD is classified as steroid refractory or resistant if GVHD progresses within the first 3 to 5 days of prednisone therapy onset [ 2 mg/kg per day], fails to improve within 5 to 7 days of treatment initiation at 1 mg/kg or shows an incomplete response after more than 28 days [of immunosuppressive treatment including steroids]. Steroid dependence [means that either] the prednisone cannot be tapered below 2 mg/kg daily or the GVHD recurs during the steroid taper.11

You cant really [know] who is going to respond to steroids without [trying]. Our initial treatment for any patient with GVHD is steroids. There are no data to suggest that we [should] add something other than steroids as the first line or that we [should] add double therapy for the first line. Its going to be different for every individual.

Also, regarding steroid therapy, the question has been raised: If patients receive higher doses sooner, will that result in a lower [total] exposure to steroids? In the study we did at our institution, we found that when patients with skin GVHD were randomly assigned [to receive] 1 mg/kg vs 0.5 mg/kg, patients [who received the lower dose] had a longer and higher overall exposure to steroids.12 [In cases of skin GVHD], we tend to undertreat patients, and it may help to give them at least 1 mg/kg, but for GI GVHD, we usually give 1 mg/kg. It may not help to give 2 mg/kg unless the GVHD is severe.

Other than steroids, what therapy options exist for aGVHD, according to the National Comprehensive Cancer Network (NCCN)?

Ruxolitinib [Jakafi] is the only approved therapy, and it is supported by category 1 evidence. Some other therapies, such as MMF and sirolimus, are [relatively] benign. Other treatments, like ATG, are more toxic, whereas extracorporeal photopheresis [ECP] doesnt have a lot of data [to support it]. However, we do use a lot of ECP, [primarily for] steroid-dependent GVHD of the skin.13

What data support the use of ruxolitinib for aGVHD?

In the REACH1 study [NCT02953678], patients with steroid refractory grade 2 to 4 aGVHD received ruxolitinib, 5 mg twice a day. Later, patients could increase to 10 mg twice a day.14,15

The overall response rate [ORR] at day 28 was about 55%. The best ORR at any time during treatment was 73%. Time to response was about 7 days [range, 6-49]. The median duration of response was almost a year. Death from causes other than malignancy relapse was found in about 50% of patients. The median OS was about 5 months, whereas median OS for steroid refractory GVHD was 1 month, [but median OS for day 28 responders was not reached].16,17 The overall response rate [ORR] at day 28 was about 55%. The best ORR at any time during treatment was 73%. Time to response was about 7 days [range, 6-49]. The median duration of response was almost a year. Death from causes other than malignancy relapse was found in about 50% of patients. The median OS was about 5 months, whereas median OS for steroid-refractory GVHD was 1 month, [but median OS for day 28 responders was not reached].15

The ORR at day 28 was 62% in the ruxolitinib group vs 39% in the control group [odds ratio (OR), 2.64; 95% CI, 1.65-4.22; P < .001]; the durable ORR at day 56 was 40% in the ruxolitinib group vs 22% in the control group [OR, 2.38; 95% CI, 1.43-3.94; P < .001].18 These results led to the FDA approval of ruxolitinib for second-line therapy for steroid-refractory aGVHD.19

[Separate analyses were conducted of] GI and skin GVHD. In the ruxolitinib group, aGVHD staging of the lower GI was stage 3 and 4 for most patients at baseline. This was reduced in most patients to stage 0, 1, and 2 by day 28. In contrast, most patients treated with BAT still presented with stage 2 to 4 GVHD by day 28. Likewise for the skin, the GVHD stage was more likely to decrease following treatment with ruxolitinib than with BAT.19

Median failure-free survival was 5 months in the ruxolitinib group vs 1 month in the BAT group [HR, 0.46; 95% CI, 0.35-0.60]; 5 months was a big achievement compared with our previous standard. After 1 year, 40% of the patients in the experimental group were still alive. Regarding AEs associated with ruxolitinib, the most difficult [AE to manage] is thrombocytopenia [in REACH2, affecting 33% of the ruxolitinib group vs 18% of the BAT group]. Infections with ruxolitinib [in the context of GVHD] probably are equivalent to [those observed with] any other immune suppression drug [for cytomegalovirus, 26% in the ruxolitinib group, 21% in the BAT group].19

REFERENCES:

1. Yoshida S, Ohno Y, Nagafuji K, et al. Comparison of calcineurin inhibitors in combination with conventional methotrexate, reduced methotrexate, or mycophenolate mofetil for prophylaxis of graft-versus-host disease after umbilical cord blood transplantation. Ann Hematol. 2019;98(11):2579-2591. doi:10.1007/s00277-019-03801-z

2. Bejanyan N, Rogosheske J, DeFor TE, et al. Sirolimus and mycophenolate mofetil as calcineurin inhibitor-free graft-versus-host disease prophylaxis for reduced-intensity conditioning umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2016;22(11):2025-2030. doi:10.1016/j. bbmt.2016.08.005

3. Wodarczyk M, Ograczyk E, Kowalewicz-Kulbat M, Druszczyska M, Rudnicka W, Fol M. Effect of cyclophosphamide treatment on central and effector memory T cells in mice. Int J Toxicol. 2018;37(5):373-382.

4. Shaw BE. Related haploidentical donors are a better choice than matched unrelated donors: counterpoint. Blood Adv. 2017;1(6):401-406. doi:10.1182/bloodadvances.2016002188

5. Zachary AA, Leffell MS. HLA mismatching strategies for solid organ transplantation - a balancing act. Front Immunol. 2016;7:575. doi:10.3389/ fimmu.2016.00575

6. Brierley CK, Littlewood TJ, Peniket AJ, et al. Impact of ABO blood group mismatch in alemtuzumab-based reduced-intensity conditioned haematopoietic SCT. Bone Marrow Transplant. 2015;50(7):931-938. doi:10.1038/bmt.2015.51

7. Harris AC, Young R, Devine S, et al. International, multicenter standardization of acute graft-vs-host disease clinical data collection: a report from the Mount Sinai Acute GVHD International Consortium. Biol Blood Marrow Transplant. 2016;22(1):4-10. doi:10.1016/j.bbmt.2015.09.001

8. Lee SJ. Classification systems for chronic graft-versus-host disease. Blood. 2017;129(1):30-37. doi:10.1182/blood-2016-07-686642

9. Levine JE, Logan BR, Wu J, et al. Acute graft-vs-host disease biomarkers measured during therapy can predict treatment outcomes: a Blood and Marrow Transplant Clinical Trials Network study. Blood. 2012;119(16):3854-3860. doi:10.1182/blood-2012-01-403063

10. MacMillan ML, Robin M, Harris AC, et al. A refined risk score for acute graft-vs-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biol Blood Marrow Transplant. 2015;21(4):761-767. doi:10.1016/j.bbmt.2015.01.001

11. Schoemans HM, Lee SJ, Ferrara JL, et al; European Society for Blood and Marrow Transplantation [EBMT] Transplant Complications Working Party; EBMT-National Institutes of Health [NIH]-Center for International Blood and Marrow Transplant Research [CIBMTR] GVHD Task Force. EBMT-NIH-CIBMTR Task Force position statement on standardized terminology & guidance for graft-vs-host disease assessment. Bone Marrow Transplant. 2018;53(11):1401-1415. doi:10.1038/s41409-018-0204-7

12. Mielcarek M, Furlong T, Storer BE, et al. Effectiveness and safety of lower dose prednisone for initial treatment of acute graft-versus-host disease: a randomized controlled trial. Haematologica. 2015;100(6):842-848. doi:10.3324/haematol.2014.118471

13. NCCN. Clinical Practice Guidelines in Oncology. Hematopoietic cell transplantation, version 5.2021. Accessed October 13, 2021. https://www.nccn.org/professionals/physician_gls/pdf/hct.pdf

14. Chao N. Finally, a successful randomized trial for GVHD. N Engl J Med. 2020;382(19):1853-1854. doi:10.1056/NEJMe2003331

15. Jagasia M, Zeiser R, Arbushites M, Delaite P, Gadbaw B, von Bubnoff N. Ruxolitinib for the treatment of patients with steroid-refractory GVHD: an introduction to the REACH trials. Immunotherapy. 2018;10(5):391-402. doi:10.2217/ imt-2017-0156

16. Jagasia M, Perales MA, Schroeder MA, et al. Ruxolitinib for the treatment of steroid-refractory acute GVHD (REACH1): a multicenter, open-label phase 2 trial. Blood. 2020;135(20):1739-1749. doi:10.1182/blood.2020004823

17. Jagasia M, Ali H, Schroeder MA, et al. Ruxolitinib in combination with corticosteroids for the treatment of steroid-refractory acute graft-vs-host disease: results from the phase 2 REACH1 trial. Biol Blood Marrow Transplant. 2019;25(suppl 3):S52. doi:10.1016/j.bbmt.2018.12.130

18. Zeiser R, von Bubnoff N, Butler J, et al; REACH2 Trial Group. Ruxolitinib for glucocorticoid-refractory acute graft-vs-host disease. N Engl J Med. 2020;382(19):1800-1810. doi:10.1056/NEJMoa1917635

19. Przepiorka D, Luo L, Subramaniam S, et al. FDA approval summary: ruxolitinib for treatment of steroid-refractory acute graft-versus-host disease. Oncologist. 2020;25(2):e328-e334. doi:10.1634/theoncologist.2019-0627

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Salit Discusses the Use of Staging and Grading for Patients With GVHD to Choose Appropriate Treatment - Targeted Oncology

Bone Marrow Stem Cells Comments Off on Salit Discusses the Use of Staging and Grading for Patients With GVHD to Choose Appropriate Treatment – Targeted Oncology | November 8th, 2021

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, 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

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - KULR-TV

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent … – KULR-TV | November 8th, 2021

Our bone marrow harbors thousandsof rare hematopoietic stem cells tiny shape shifters that can morph into red or white blood cells. But conditions like sickle cell anemia or immune deficiency can damage these cells, and treatments for blood cancers may destroy them altogether.

To rebuild bone marrow, researchers have perfected the art of transplanting stem cells either those belonging to the patient or ones that have been donated.

You only have so many of these cells, says Jennifer Gillette, PhD, associate professor inThe University of New Mexico Department of Pathology. These are cells that are with us our entire life. They stay turned off when youre healthy and things are moving along. But in a stress condition, these are cells that get turned on and rev up and then go quiescent again.

Gillette has been devising ways to trick donors bone marrow into releasing more stem cells into the bloodstream so they can be harvested and transfused.

Ina new paper published onlinein the journalStem Cell Reports, Gillette and her colleagues describe manipulating a protein called CD82 that sits on the surface of each stem cell and helps them migrate into and out of the bone marrow.

Gillettes team found that when CD82 disruption is coupled with existing medications used to stimulate stem cell release into the bloodstream, the process is significantly amplified, causing more stem cells to find their way into circulation.

One of those medications AMD3100 costs $10,000 for a single dose, she says, and it is hoped that treatment with antibodies might be less expensive.

Patients diagnosed with leukemia or lymphoma may be treated with chemotherapy and/or radiation, which destroy cancerous cells in the bone marrow along with stem cells.

It wipes out everything, Gillette says. For leukemia or blood cancers youre trying to get rid of that malignant population and replace it with healthy cells.

Patients may bank some of their stem cells beforehand for autologous transplants, or they could receive allograft transplants from suitable donors.

There are some patients that just dont mobilize well, she says. Were looking for any way that we can enhance cell release, because the number and quality of cells going in really enhance the response of the patients.

Gillette has applied for a provisional patent to repurpose the antibodies originally developed to study CD82 functioning as a clinical treatment. The next step is to see exactly how that antibody is working, she says.

Originally posted here:
UNM Scientist Jennifer Gillette Tricks the Bone Marrow ...

Bone Marrow Stem Cells Comments Off on UNM Scientist Jennifer Gillette Tricks the Bone Marrow … | October 28th, 2021