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Catalent and BrainStorm Cell Therapeutics Announce Partnership for the Manufacture of Mesenchymal Stem Cell Platform Therapy NurOwn – GlobeNewswire

By daniellenierenberg

SOMERSET, N.J. and NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Catalent (NYSE: CTLT), the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, and BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced an agreement for the manufacture of NurOwn, BrainStorms autologous cellular therapy being investigated for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease or motor neuron disease.

NurOwn induces mesenchymal stem cells (MSCs) to secrete high levels of neurotrophic factors (NTFs) known to promote the survival of neurons and neuroprotection. The therapy has received Fast Track status from the U.S. FDA for ALS and has also been granted Orphan Drug Status for ALS by both the FDA and the European Medicines Agency. BrainStorm is currently completing a 200-patient, double-blind, placebo-controlled, repeat-dosing NurOwn Phase 3 study in the U.S.

As part of its commitment, Catalent will undertake the transfer of the manufacturing process to, and provide future CGMP clinical supply of NurOwn from, its new, 32,000 square-foot cell therapy manufacturing facility in Houston, Texas. On completion of the clinical trials and in anticipation of potential approval of NurOwn, the companies will look to extend the partnership to include commercial supply from the Houston facility.

We are proud to have a partner in Catalent whose excellence in manufacturing quality therapies will support commercial supply of NurOwn, said Chaim Lebovits, Chief Executive Officer of BrainStorm Cell Therapeutics. We know that ALS patients are in urgent need of a new treatment option. If NurOwn is successful in the current clinical trials, this agreement will be integral to ensuring rapid access for patients.

Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy, said, Our experience in cell therapy development, and the manufacturing capabilities that our newly constructed, state-of-the-art facility in Houston offers, position us to best support BrainStorm, with its leading therapeutic candidate for ALS treatment. We look forward to partnering with BrainStorm and providing our stem cell manufacturing expertise as we work to optimize production and streamline the products path towards commercial launch.

About Catalent Cell & Gene Therapy

With deep experience in viral vector scale-up and production, Catalent Cell & Gene Therapy is a full-service partner for adeno-associated virus (AAV) and lentiviral vectors, and CAR-T immunotherapies. When it acquired MaSTherCell, Catalent added expertise in autologous and allogeneic cell therapy development and manufacturing to position it as a premier technology, development and manufacturing partner for innovators across the entire field of advanced biotherapeutics. Catalent has a global cell and gene therapy network of dedicated, large-scale clinical and commercial manufacturing facilities, and fill-finish and packaging capabilities located in both the U.S. and Europe. An experienced partner, Catalent Cell & Gene Therapy has worked with industry leaders across 70+ clinical and commercial programs.

About Catalent

Catalent is the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable global clinical and commercial product supply. Catalent employs approximately 14,000 people, including around 2,400 scientists and technicians, at more than 45 facilities, and in fiscal year 2020 generated over $3 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey. For more information, visit http://www.catalent.com

More products. Better treatments. Reliably supplied.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) completed enrollment inAugust 2020. For more information, visit the company's website at http://www.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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Randomized Controlled Study Using Direct Injection of Remestemcel-L Into Inflamed Gut of Patients With Crohn’s Disease and Ulcerative Colitis -…

By daniellenierenberg

NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that a randomized, controlled study of remestemcel-L delivered by an endoscope directly to the areas of inflammation and tissue injury in up to 48 patients with medically refractory Crohns disease and ulcerative colitis has commenced at Cleveland Clinic.

Mesoblast Chief Medical Officer Dr Fred Grossman said: Inflammation of the gut in Crohns disease and ulcerative colitis closely resembles the most severe manifestation of advanced-stage, life-threatening acute graft versus host disease (aGVHD). Mesoblasts objective is to confirm the potential for remestemcel-L to induce luminal healing and early remission in a wider spectrum of diseases with severe inflammation of the gut, in addition to steroid-refractory aGVHD.

Mesenchymal stem cells (MSCs) promote healing of inflamed gut tissue by downregulating gut mucosal effector T-cell activity and promoting regulatory T-cell formation.1 MSCs have been tested in clinical trials of Crohns disease using two different modalities: intravenous infusions of MSCs to treat the primary inflammation of Crohns disease and local injections of MSCs to treat fistulae complicating Crohns disease.

A third modality, endoscopic delivery of MSCs, has been successful in preclinical experimental models of colitis, reducing the excessive cytokine storm in the inflamed gut and resulting in tissue healing.2-3 The study at Cleveland Clinic will be the first in humans using local delivery of MSCs in the gut, and will enable Mesoblast to compare clinical outcomes using this delivery method with results from an ongoing randomized, placebo-controlled trial in patients with biologic-refractory Crohns disease where remestemcel-L was administered intravenously.

The studys lead investigator Dr Amy L. Lightner, Associate Professor of Surgery in the Department of Colon and Rectal Surgery at Cleveland Clinic, stated: We are aiming to establish a new treatment paradigm by administering remestemcel-L at one of two escalating doses, or placebo, directly to inflamed gut tissue in patients with medically refractory Crohns disease and ulcerative colitis, both highly debilitating conditions with significant, unmet medical needs.

According to recent estimates, more than three million people (1.3%) in the US alone have inflammatory bowel disease, with more than 33,000 new cases of Crohns disease and 38,000 new cases of ulcerative colitis diagnosed every year.4-6 Despite recent advances, approximately 30% of patients are primarily unresponsive to anti-TNF agents and even among responders, up to 10% will lose their response to the drug every year. Up to 80% of patients with medically-refractory Crohns disease eventually require surgical treatment of their disease,7 which can have a devastating impact on quality of life.

References1.Mayne C and Williams C. Induced and natural regulatory T cells in the development of inflammatory bowel disease. Inflamm Bowel Dis 2013; 19: 17721788.2.Molendijk I et al. Intraluminal Injection of Mesenchymal Stromal Cells in Spheroids Attenuates Experimental Colitis. Journal of Crohn's and Colitis, 2016, 9539643.Pak S eta al. Endoscopic Transplantation of Mesenchymal Stem Cell Sheets in Experimental Colitis in Rats. Scientific Reports | (2018) 8:11314 | DOI:10.1038/s41598-018-296174.CDC Facts and Figures 20155.Globaldata Pharmapoint 20186.Dahlhamer JM, MMWR Morb Mortal Wkly Rep. 2016;65(42):11661169.7.Crohns and Colitis Foundation

About Remestemcel-LMesoblasts lead product candidate, remestemcel-L, is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. Remestemcel-L is thought to have immunomodulatory properties to counteract severe inflammatory processes by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

About MesoblastMesoblast Limited (Nasdaq:MESO; ASX:MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblast has a strong and extensive global intellectual property (IP) portfolio with protection extending through to at least 2040 in all major markets. The Companys proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid-refractory acute graft versus host disease and moderate to severe acute respiratory distress syndrome. Mesoblast is completing Phase 3 trials for its product candidates for advanced heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, likely, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions and variations thereof. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. The risks, uncertainties and other factors that may impact our forward-looking statements include, but are not limited to: statements about the initiation, timing, progress and results of Mesoblast and its collaborators clinical studies; Mesoblast and its collaborators ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblasts product candidates, if approved; the potential benefits of strategic collaboration agreements and Mesoblasts ability to maintain established strategic collaborations; Mesoblasts ability to establish and maintain intellectual property on its product candidates and Mesoblasts ability to successfully defend these in cases of alleged infringement. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. Unless required by law, we do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

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British Society for Haematology Guideline Update for the Diagnosis and Management of Chronic Myeloid Leukemia – Cancer Therapy Advisor

By daniellenierenberg

The development of tyrosine kinase inhibitors (TKIs) has revolutionized the treatment of chronic myeloid leukemia (CML).1,2 However, despite effectively inducing remission and prolonging survival in patients with CML, TKI therapy does not eradicate leukemia stem cells (LSCs), which are responsible for drug resistance, relapse, and disease progression.2 Given recent changes to the treatment paradigm, updated clinical practice guidelines are essential to ensure optimal clinical care is provided.2

The British Society for Haematology (BSH) published a guideline update for the investigation and management of CML in adults and children in the British Journal of Haematology.1 Lead author of the guidelines, Graeme Smith, MD, of St Jamess University Hospital in the United Kingdom, and coauthors, developed the evidence-based recommendations to provide clinical practitioners with clear guidance on the diagnosis and treatment of adults and children with CML (Tables 1 and 2).

Diagnosis and Key Investigations

The diagnosis of CML is established based on findings from a peripheral blood smear and bone marrow aspirate showing positivity for BCR-ABL1, and the presence of the Philadelphia (Ph) chromosome. The Ph chromosome, or a variant, is present in approximately 95% of CML cases. Other cases include a cryptic BCR-ABL1 fusion, commonly detected by reverse transcriptase polymerase chain reaction (RT-PCR), or fluorescence in situ hybridization (FISH). Additional findings from bone marrow aspirate include the presence of other cytogenetic abnormalities, including isochromosome 17q or trisomy 19, and trisomy 8, suggesting a higher risk of progression to accelerated phase or blast crisis in adults.

Table 1. Selected Recommendations by the BSH Guideline Panel on the Diagnosis of CML1

Table 2. ELTS Score Calculation1

This article originally appeared on Hematology Advisor

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British Society for Haematology Guideline Update for the Diagnosis and Management of Chronic Myeloid Leukemia - Cancer Therapy Advisor

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Some Patients With AML Secondary to MPN May Benefit From Venetoclax in Combination With a Hypomethylating Agent – Oncology Nurse Advisor

By daniellenierenberg

The combination of the BCL2 inhibitor venetoclax with a hypomethylating agent (HMA) may be a treatment option for patients who develop acute myeloid leukemia (AML) secondary to a myeloproliferative neoplasm (MPN), according to results of a small, retrospective cohort study published in Leukemia Research.1

The Philadelphia chromosome-negative MPNs, which include polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF), are clonal disorders resulting in the proliferation of myeloid cells in the bone marrow. Both PV and ET can progress to secondary myelofibrosis which, along with PMF, can progress to secondary AML, also known as MPN-blast phase (MPN-BP).

Furthermore, MPN-BP, defined in this study as being associated with peripheral or bone marrow blasts of at least 20%, is not sensitive to intensive chemotherapy, and clinical outcomes for patients with this disease are very poor. Median overall survival is only approximately 3 to 5 months, and allogeneic hematopoietic stem cell transplantation (HSCT) is considered the only curative option for these patients.

Venetoclax in combination with an HMA, azacitidine, decitabine, or low-dose cytarabine recently received regular US Food and Drug Administration (FDA) approval for the treatment of adults with newly diagnosed AML who are at least 75 years old or unable to tolerate intensive chemotherapy.2 However, patients with MPN-BP were excluded from the VIALE-A (ClinicalTrial.gov Identifier: NCT02993523) and VIALE-C (ClinicalTrial.gov Identifier: NCT03069352) phase 3 studies evaluating venetoclax in combination with azacitadine and low-dose cytarabine, respectively, in newly diagnosed AML.

This study included 8 patients with MPN-BP and 1 with MPN-accelerated phase (MPN-AP), defined as peripheral or bone marrow blasts of 10% to 19%, which was associated with very high-risk cytogenetics, who were treated at the Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai in New York, New York. Most of the patients in this cohort had relapsed/refractory disease, and had been treated with prior therapies.

A key study finding included the achievement of either a complete response (CR) or a CR associated with incomplete hematologic recovery (CRi) in 3 patients treated with the combination of venetoclax plus decitabine or azacitidine. In addition, stable disease as best response was achieved by 2 additional patients who received this treatment.

Of note, 2 of the 3 patients who achieved a CR/CRi had experienced disease relapse on prior HMA therapy.

This suggests a synergy with the combination that is not precluded by prior HMA exposure, the study authors remarked.

Perhaps more striking was the finding that when this therapeutic approach was used as a bridge to HSCT in 3 patients who achieved CR, CRi, or stable disease, all of them were alive at a median follow-up of 8.5 months compared with 4.2 months for the overall cohort.

However, high rates of grade 3 or higher bleeding and infection were observed in this patient cohort, and occurred in 5 and 7 patients, respectively.

Given the propensity for prolonged cytopenias with resultant complications, caution should be used in patients with baseline cytopenias, study authors noted.

In closing, the study authors stated, This is the largest report of venetoclax use in patients with MPN-AP/BP and suggests that this therapeutic strategy is a viable treatment option in this adverse risk group eligible for HSCT.

They further added that prospective clinical trial evaluation of combination HMA and venetoclax in MPN-BP is warranted.

Disclosures: Multiple authors declared affiliations with industry. Please refer to the original article for a full list of disclosures.

References

1. Tremblay D, Feld J, Dougherty M, et al. Venetoclax and hypomethylating agent combination therapy in acute myeloid leukemia secondary to a myeloproliferative neoplasm. Leuk Res. Published online September 22, 2020. doi:10.1016/j.leukres.2020.106456

2. U.S. Food and Drug Administration. FDA grants regular approval to venetoclax in combination for untreated acute myeloid leukemia [news release]. U.S. Food and Drug Administration; October 16, 2020. Accessed October 19, 2020. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-grants-regular-approval-venetoclax-combination-untreated-acute-myeloid-leukemia

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Be Biopharma debuts with $52M to advance engineered B-cell therapies – FierceBiotech

By daniellenierenberg

You may have heard of T cells, but Aleks Radovic-Moreno, Ph.D., Be Biopharmas co-founder, president and director, is betting on B cells as the future of cell therapies.

Our mission is to develop what we see as a new class of cell medicines that have a broad new pharmacology, he said of B cells potential. We think it's a big new white space that's enabled by the rich biology of these cells.

The Cambridge, Massachusetts-based company is capitalizingearly on research by scientists at the University of Washington School of Medicine. With a $52 million series A round in the bank, it'smaking a beeline for the clinic.

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Why the enthusiasm around B cells? The wayRadovic-Moreno sees it, they'rethe cellular gadget, if you will, that's really good at making large amounts of protein, and they also traffic to where you want them to go."

When we think about it from a drug development standpoint, now you have a system that can make a protein that you want in high quantities in places where you want it to be made, he added.

B cells may also be useful for targeting specific tissues and modulating microenvironments, or [talking] to the cells that are nearby, he said.

One of the biggest challenges to bringing Be Bio to fruition was making the products themselves. Theyre harder to engineer than other cell types thanksto their intrinsic biology, Radovic-Moreno said. Theyre also hard to make correctly and in large quantities, challenges the company only recently overcame.

Those two are the final two bottlenecks that were preventing B cells from being a viable stem cell therapy modality, he said.

RELATED: Q32 debuts with $46M to 'rebalance' innate and adaptive immunity

The applications of B cells include everything from autoimmune diseases to cancer and monogenic disorders, which are caused by variation in a single gene. B-cell therapy could eliminate the need for patients with monogenic disorders who are missing proteins to get biweekly four-hour infusions.

And that's not all. It couldalso eliminate the need for bone marrow transplants in these patients, as well asthe need for a pre-therapy round of chemotherapy, otherwise known as conditioning. For cancer patients who need conditioningahead of a stem cell treatment, the regimencan be deadly up to 10% of the time.

That's extraordinary if you think about a therapy killing patients 10% of the time, Radovic-Moreno said.

Beyond pushing Be'spipeline toward the clinic, the new fundingfrom Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund and other investorswill bankroll potential partnerships and build out the company's team.

The most important thing is to build a great company, hire the best people. We want to be the best B-cell engineers in the world and in history, Radovic-Moreno said. We want to fully capitalize on the timing of this, given that it's a very kind of unusual place to be in this time and age of biotech, where you're sitting right in front of this massive blue wave, big blue ocean of possibilities so big.

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Potential impact of Covid-19 on Rheumatoid Arthritis Stem Cell Therapy Market Growth and Demand, Concludes Fact.MR – The Cloud Tribune

By daniellenierenberg

The globalRheumatoid Arthritis Stem Cell Therapy marketstudy presents an all in all compilation of the historical, current and future outlook of the market as well as the factors responsible for such a growth. With SWOT analysis, the business study highlights the strengths, weaknesses, opportunities and threats of each Rheumatoid Arthritis Stem Cell Therapy market player in a comprehensive way. Further, the Rheumatoid Arthritis Stem Cell Therapy market report emphasizes the adoption pattern of the Rheumatoid Arthritis Stem Cell Therapy across various industries.Request Sample Reporthttps://www.factmr.com/connectus/sample?flag=S&rep_id=1001The Rheumatoid Arthritis Stem Cell Therapy market report highlights the following players:The global market for rheumatoid arthritis stem cell therapy is highly fragmented. Examples of some of the key players operating in the global rheumatoid arthritis stem cell therapy market include Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc, International Stem Cell Corporation, TiGenix and others.

The Rheumatoid Arthritis Stem Cell Therapy market report examines the operating pattern of each player new product launches, partnerships, and acquisitions has been examined in detail.Important regions covered in the Rheumatoid Arthritis Stem Cell Therapy market report include:

North America (U.S., Canada)Latin America (Mexico, Brazil)Western Europe (Germany, Italy, U.K., Spain, France, Nordic countries, BENELUX)Eastern Europe (Russia, Poland, Rest Of Eastern Europe)Asia Pacific Excluding Japan (China, India, Australia & New Zealand)JapanMiddle East and Africa (GCC, S. Africa, Rest Of MEA)

The Rheumatoid Arthritis Stem Cell Therapy market report takes into consideration the following segments by treatment type:

Allogeneic Mesenchymal stem cellsBone marrow TransplantAdipose Tissue Stem Cells

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HospitalsAmbulatory Surgical CentersSpecialty ClinicsHave Any Query? Ask our Industry Experts-https://www.factmr.com/connectus/sample?flag=AE&rep_id=1001

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The Rheumatoid Arthritis Stem Cell Therapy market report offers a plethora of insights which include:

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The Rheumatoid Arthritis Stem Cell Therapy market report answers important questions which include:

Which regulatory authorities have granted approval to the application of Rheumatoid Arthritis Stem Cell Therapy in Health industry?How will the global Rheumatoid Arthritis Stem Cell Therapy market grow over the forecast period?Which end use industry is set to become the leading consumer of Rheumatoid Arthritis Stem Cell Therapy by 2028?What manufacturing techniques are involved in the production of the Rheumatoid Arthritis Stem Cell Therapy?Which regions are the Rheumatoid Arthritis Stem Cell Therapy market players targeting to channelize their production portfolio?Get Full Access of the Report @https://www.factmr.com/report/1001/rheumatoid-arthritis-stem-cell-therapy-market

Pertinent aspects this study on the Rheumatoid Arthritis Stem Cell Therapy market tries to answer exhaustively are:

What is the forecast size (revenue/volumes) of the most lucrative regional market? What is the share of the dominant product/technology segment in the Rheumatoid Arthritis Stem Cell Therapy market? What regions are likely to witness sizable investments in research and development funding? What are Covid 19 implication on Rheumatoid Arthritis Stem Cell Therapy market and learn how businesses can respond, manage and mitigate the risks? Which countries will be the next destination for industry leaders in order to tap new revenue streams? Which new regulations might cause disruption in industry sentiments in near future? Which is the share of the dominant end user? Which region is expected to rise at the most dominant growth rate? Which technologies will have massive impact of new avenues in the Rheumatoid Arthritis Stem Cell Therapy market? Which key end-use industry trends are expected to shape the growth prospects of the Rheumatoid Arthritis Stem Cell Therapy market? What factors will promote new entrants in the Rheumatoid Arthritis Stem Cell Therapy market? What is the degree of fragmentation in the Rheumatoid Arthritis Stem Cell Therapy market, and will it increase in coming years?Why Choose Fact.MR?

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

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Potential impact of Covid-19 on Rheumatoid Arthritis Stem Cell Therapy Market Growth and Demand, Concludes Fact.MR - The Cloud Tribune

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Bragar Eagel & Squire, PC Reminds Investors That Class Action Lawsuits Have Been Filed Against Mesoblast, Loop Industries, Turquoise Hill…

By daniellenierenberg

NEW YORK, Oct. 21, 2020 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized shareholder rights law firm, reminds investors that class actions have been commenced on behalf of stockholders of Mesoblast Limited (NASDAQ: MESO), Loop Industries, Inc. (NASDAQ: LOOP), Turquoise Hill Resources Ltd. (NYSE: TRQ), and Reata Pharmaceuticals, Inc. (NASDAQ: RETA). Stockholders have until the deadlines below to petition the court to serve as lead plaintiff. Additional information about each case can be found at the link provided.

Mesoblast Limited (NASDAQ: MESO)

Class Period: April 16, 2019 to October 1, 2020

Lead Plaintiff Deadline: December 7, 2020

Mesoblast develops allogeneic cellular medicines using its proprietary mesenchymal lineage cell therapy platform. Its lead product candidate, RYONCIL (remestemcel-L), is an investigational therapy comprising mesenchymal stem cells derived from bone marrow. In February 2018, the Company announced that remestemcel-L met its primary endpoint in a Phase 3 trial to treat children with steroid refractory acute graft versus host disease (aGVHD).

In early 2020, Mesoblast completed its rolling submission of its Biologics License Application (BLA) with the FDA to secure marketing authorization to commercialize remestemcel-L for children with steroid refractory aGVHD.

On August 11, 2020, the FDA released briefing materials for its Oncologic Drugs Advisory Committee (ODAC) meeting to be held on August 13, 2020. Therein, the FDA stated that Mesoblast provided post hoc analyses of other studies to further establish the appropriateness of 45% as the null Day-28 ORR for its primary endpoint. The briefing materials stated that, due to design differences between these historical studies and Mesoblasts submitted study, it is unclear that these study results are relevant to the proposed indication.

On this news, the Companys share price fell $6.09, or approximately 35%, to close at $11.33 per share on August 11, 2020.

On October 1, 2020, Mesoblast disclosed that it had received a Complete Response Letter (CRL) from the FDA regarding its marketing application for remestemcel-L for treatment of SR-aGVHD in pediatric patients. According to the CRL, the FDA recommended that the Company conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD. The CRL also identified a need for further scientific rationale to demonstrate the relationship of potency measurements to the products biologic activity.

On this news, the Companys share price fell $6.56, or 35%, to close at $12.03 per share on October 2, 2020.

The complaint, filed on October 8, 2020, alleges that throughout the Class Period defendants made materially false and/or misleading statements, as well as failed to disclose material adverse facts about the Companys business, operations, and prospects. Specifically, defendants failed to disclose to investors: (1) that comparative analyses between Mesoblasts Phase 3 trial and three historical studies did not support the effectiveness of remestemcel-L for steroid refractory aGVHD due to design differences between the four studies; (2) that, as a result, the FDA was reasonably likely to require further clinical studies; (3) that, as a result, the commercialization of remestemcel-L in the U.S. was likely to be delayed; and (4) that, as a result of the foregoing, defendants positive statements about the Companys business, operations, and prospects were materially misleading and/or lacked a reasonable basis.

For more information on the Mesoblast class action go to: https://bespc.com/MESO

Loop Industries, Inc. (NASDAQ: LOOP)

Class Period: September 24, 2018 to October 12, 2020

Lead Plaintiff Deadline: December 14, 2020

On October 13, 2020, Hindenburg Research published a report alleging, among other things, that Loops scientists, under pressure from CEO Daniel Solomita, were tacitly encouraged to lie about the results of the companys process internally. The report also stated that Loops previous claims of breaking PET down to its base chemicals at a recovery rate of 100% were technically and industrially impossible, according to a former employee. Moreover, the report alleged that Executives from a division of key partner Thyssenkrupp, who Loop entered into a global alliance agreement with in December 2018, told us their partnership is on indefinite hold and that Loop underestimated both costs and complexities of its process.

On this news, the Companys share price fell $3.78, or over 32%, to close at $7.83 per share on October 13, 2020.

The complaint, filed on October 13, 2020, alleges that throughout the Class Period defendants made materially false and/or misleading statements, as well as failed to disclose material adverse facts about the Companys business, operations, and prospects. Specifically, defendants failed to disclose to investors: (1) that Loop scientists were encouraged to misrepresent the results of Loops purportedly proprietary process; (2) that Loop did not have the technology to break PET down to its base chemicals at a recovery rate of 100%; (3) that, as a result, the Company was unlikely to realize the purported benefits of Loops announced partnerships with Indorama and Thyssenkrupp; and (4) that, as a result of the foregoing, defendants positive statements about the Companys business, operations, and prospects were materially misleading and/or lacked a reasonable basis.

For more information on the Loop class action go to: https://bespc.com/Loop

Turquoise Hill Resources Ltd. (NYSE: TRQ)

Class Period: July 17, 2018 to July 31, 2019

Lead Plaintiff Deadline: December 14, 2020

Turquoise Hill is an international mining company focused on the operation and development of the Oyu Tolgoi copper-gold mine in Southern Mongolia (Oyu Tolgoi), which is the Companys principal and only material resource property. Turquoise Hills subsidiary, Oyu Tolgoi LLC, holds a 66% interest in Oyu Tolgoi, and the remainder is held by the Government of Mongolia.

Rio Tinto plc and Rio Tinto Limited are operated and managed together as single economic unit and engage in mining and metals operations in approximately 35 countries. Through their subsidiaries, Rio Tinto owns 50.8% of Turquoise Hill. A Rio Tinto subsidiary, Rio Tinto International Holdings, Inc. (Rio Tinto International or RTIH; and collectively with Rio Tinto plc and Rio Tinto Limited, Rio Tinto), is also the manager of the Oyu Tolgoi project, including having responsibility for its development and construction.

On July 31, 2019, Turquoise Hill issued a press release and Management Discussion & Analysis (MD&A) making further disclosures about the status of the project, including that Turquoise Hill took a $600 million impairment charge and a substantial deferred income tax recognition adjustment tied to the Oyu Tolgoi project, and that it suffered a loss in the second quarter. The next day, before the market open, Rio Tinto issued a release concerning in part the project status, including that it had also taken an impairment charge related to the Oyu Tolgoi project, of $800 million.

Following this news, on August 1, 2019, Turquoise Hills common stock price closed at $0.53 per share, down 8.62% from the prior days closing price of $0.58 per share.

The complaint, filed on October 15, 2020, alleges that throughout the Class Period defendants made materially false and misleading statements and omitted to disclose material facts regarding the Companys business and operations. Specifically, defendants made false and or misleading statements and/or failed to disclose that: (i) the progress of underground development of Oyu Tolgoi was not proceeding as planned; (ii) there were significant undisclosed underground stability issues that called into question the design of the mine, the projected cost and timing of production; (iii) the Companys publicly disclosed estimates of the cost, date of completion and dates for production from the underground mine were not achievable; (iv) the development capital required for the underground development of Oyu Tolgoi would cost substantially more than a billion dollars over what the Company had represented; and (v) Turquoise Hill would require additional financing and/or equity to complete the project.

For more information on the Turquoise Hill class action go to: https://bespc.com/TRQ

Reata Pharmaceuticals, Inc. (NASDAQ: RETA)

Class Period: October 15, 2019 to August 7, 2020

Lead Plaintiff Deadline: December 14, 2020

Reata is a clinical stage biopharmaceutical company that develops novel therapeutics for patients with serious or life-threatening diseases by targeting molecular pathways that regulate cellular metabolism and inflammation.

Among Reatas drug candidates under development is omaveloxolone, which is in Phase 2 clinical development to treat Friedreich's ataxia (FA). Following the announcement of positive data from the MOXIe Part 2 study of omaveloxolone for FA inOctober 2019, the Company represented that it would seek submission for marketing approval of omaveloxolone for the treatment of FA in the U.S. with the U.S. Food and Drug Administration (FDA).

OnAugust 10, 2020, Reata issued a press release announcing its second quarter 2020 financial results, wherein it disclosed that the FDA is not convinced that the MOXIe Part 2 results of the Company's study assessing omaveloxolone for the treatment of FA will support a single study approval without additional evidence that lends persuasiveness to the results, and that, [i]n preliminary comments for [a] meeting, the FDA stated that [Defendants] will need to conduct a second pivotal trial that confirms the mFARS [modified Friedreich's Ataxia Rating Scale] results of the MOXIe Part 2 study with a similar magnitude of effect.

On this news, Reatas stock price fell$51.79per share, or 33.16%, to close at$104.41per share onAugust 10, 2020.

The Complaint, filed on October 15, 2020, alleges that throughout the Class Period defendants made materially false and misleading statements regarding the Companys business. Specifically, defendants made false and/or misleading statements and/or failed to disclose that: (i) the MOXIe Part 2 study results were insufficient to support a single study marketing approval of omaveloxolone for the treatment of FA in the U.S. without additional evidence; (ii) as a result, it was foreseeable that the FDA would not accept marketing approval of omaveloxolone for the treatment of FA in the U.S. based on the MOXIe Part 2 study results; and (iii) as a result, the Company's public statements were materially false and misleading at all relevant times.

For more information on the Reata class action go to: https://bespc.com/REATA

About Bragar Eagel & Squire, P.C.:Bragar Eagel & Squire, P.C. is a nationally recognized law firm with offices in New York and California. The firm represents individual and institutional investors in commercial, securities, derivative, and other complex litigation in state and federal courts across the country. For more information about the firm, please visit http://www.bespc.com. Attorney advertising. Prior results do not guarantee similar outcomes.

Contact Information:Bragar Eagel & Squire, P.C.Brandon Walker, Esq. Melissa Fortunato, Esq.Marion Passmore, Esq.(212) 355-4648investigations@bespc.comwww.bespc.com

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Bragar Eagel & Squire, PC Reminds Investors That Class Action Lawsuits Have Been Filed Against Mesoblast, Loop Industries, Turquoise Hill...

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categoriaBone Marrow Stem Cells commentoComments Off on Bragar Eagel & Squire, PC Reminds Investors That Class Action Lawsuits Have Been Filed Against Mesoblast, Loop Industries, Turquoise Hill… | dataOctober 23rd, 2020
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Genmab Announces IFM, HOVON and Janssen Achieve Positive Topline Results in Second Part of Phase 3 CASSIOPEIA Study of Daratumumab in Multiple Myeloma…

By daniellenierenberg

Company Announcement

Copenhagen, Denmark; October 21, 2020 Genmab A/S (Nasdaq: GMAB) announced today positive topline results from the second part of the Phase 3 CASSIOPEIA (MMY3006) study of daratumumab monotherapy as maintenance treatment versus observation (no treatment) for patients with newly diagnosed multiple myeloma eligible for autologous stem cell transplant (ASCT). The second part of the study, which is being conducted by the French Intergroupe Francophone du Myelome (IFM) in collaboration with the Dutch-Belgian Cooperative Trial Group for Hematology Oncology (HOVON) and Janssen Research & Development, LLC (Janssen), met the primary endpoint of improving progression free survival (PFS) at a pre-planned interim analysis (Hazard Ratio (HR) = 0.53 (95% CI 0.42 0.68), p < 0.0001) resulting in a 47% reduction in the risk of progression or death in patients treated with daratumumab. The safety profile observed in this study was consistent with the known safety profile of daratumumab and no new safety signals were observed.

Based on the results at the pre-planned interim analysis conducted by an Independent Data Monitoring Committee (IDMC), it was recommended to unblind the study results. Janssen Biotech, Inc., which licensed daratumumab from Genmab in 2012, plans to discuss the potential for a regulatory submission for this indication with health authorities, and plans to submit the data to an upcoming medical conference and for publication in a peer-reviewed journal.

Following the positive data from the first part of the CASSIOPEIA study, we are very pleased to see this benefit. We are appreciative of the efforts of the IFM, of HOVON and of Janssen for their work on this study, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the CASSIOPEIA (MMY3006) StudyThis Phase 3 study is a randomized, open-label, multicenter study, conducted by the IFM in collaboration with the HOVON and Janssen, which includes 1,085 newly diagnosed subjects with previously untreated symptomatic multiple myeloma who were eligible for high dose chemotherapy and ASCT. In the first part of the study, patients were randomized to receive induction and consolidation treatment with daratumumab combined with bortezomib, thalidomide and dexamethasone (VTd) or VTd alone. The primary endpoint was the number of patients that achieved a stringent complete response (sCR). In the second part of the study, patients that achieved a response underwent a second randomization to either receive maintenance treatment of daratumumab 16 mg/kg every 8 weeks for up to 2 years versus no further treatment (observation). The primary endpoint of this part of the study is progression free survival.

About Multiple MyelomaMultiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 26,000 new patients were expected to be diagnosed with multiple myeloma and approximately 13,650 people were expected to die from the disease in the U.S. in 2018.3 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

About DARZALEX (daratumumab)DARZALEX (daratumumab) has become a backbone therapy in the treatment of multiple myeloma. DARZALEX intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with carfilzomib and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma who have received one to three previous lines of therapy; in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma.

DARZALEX is indicated for the treatment of adult patients in Europe via intravenous infusion or subcutaneous administration: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy7. Daratumumab is the first subcutaneous CD38 antibody approved in Europe for the treatment of multiple myeloma. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S.

In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, is approved in the United States for the treatment of adult patients with multiple myeloma: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for ASCT; in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy; and as monotherapy, in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.8 DARZALEX FASPRO is the first subcutaneous CD38 antibody approved in the U.S. for the treatment of multiple myeloma.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,9,10,11,12

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase 3 studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis and T-cell acute lymphocytic leukemia (ALL). Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company is the creator of the following approved antibodies: DARZALEX (daratumumab, under agreement with Janssen Biotech, Inc.) for the treatment of certain multiple myeloma indications in territories including the U.S., Europe and Japan, Kesimpta (subcutaneous ofatumumab, under agreement with Novartis AG), for the treatment of adults with relapsing forms of multiple sclerosis in the U.S. and TEPEZZA (teprotumumab, under agreement with Roche granting sublicense to Horizon Therapeutics plc) for the treatment of thyroid eye disease in the U.S. A subcutaneous formulation of daratumumab, known as DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) in the U.S., has been approved in the U.S. and Europe for the treatment of adult patients with certain multiple myeloma indications. The first approved Genmab created therapy, Arzerra (ofatumumab, under agreement with Novartis AG), approved for the treatment of certain chronic lymphocytic leukemia indications, is available in Japan and is also available in other territories via compassionate use or oncology access programs. Daratumumab is in clinical development by Janssen for the treatment of additional multiple myeloma indications, other blood cancers and amyloidosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with sites in Utrecht, the Netherlands, Princeton, New Jersey, U.S. and Tokyo, Japan.

Contact: Marisol Peron, Corporate Vice President, Communications & Investor Relations T: +1 609 524 0065; E: mmp@genmab.com

For Investor Relations: Andrew Carlsen, Senior Director, Investor RelationsT: +45 3377 9558; E: acn@genmab.com

This Company Announcement contains forward looking statements. The words believe, expect, anticipate, intend and plan and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with pre-clinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmabs most recent financial reports, which are available on http://www.genmab.com and the risk factors included in Genmabs most recent Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at http://www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab; the Y-shaped Genmab logo; Genmab in combination with the Y-shaped Genmab logo; HuMax; DuoBody; DuoBody in combination with the DuoBody logo; HexaBody; HexaBody in combination with the HexaBody logo; DuoHexaBody; HexElect; and UniBody. Arzerra and Kesimpta are trademarks of Novartis AG or its affiliates. DARZALEX and DARZALEX FASPRO are trademarks of Janssen Pharmaceutica NV. TEPEZZA is a trademark of Horizon Therapeutics plc.

1 American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma.Accessed June 2016.2 National Cancer Institute. "A Snapshot of Myeloma." Available at http://www.cancer.gov/research/progress/snapshots/myeloma. Accessed June 2016. 3 Globocan 2018. United States of America Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/840-united-states-of-america-fact-sheets.pdf.4 Globocan 2018. World Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-sheets.pdf. Accessed December 2018.5 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed June 20166 DARZALEX Prescribing information, August 2020 https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/761036s029lbl.pdf Last accessed August 20207 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed June 20208 DARZALEX FASPRO Prescribing information, May 2020. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/761145s000lbl.pdf Last accessed May 20209 De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.10 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.11 Krejcik, MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.12 Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974.

Company Announcement no. 45CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122

Genmab A/SKalvebod Brygge 431560 Copenhagen VDenmark

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Jeff Bridges is one of the 85,000-plus lymphoma cases expected in the U.S. this year – MarketWatch

By daniellenierenberg

Careful, man, theres a beloved actor here.

Jeff Bridges revealed that he has lymphoma, which is the most common type of blood cancer. And this sobering news has spurred celebrities and fans to send their best wishes to the star best known for playing the Dude, the White Russiandrinking bowler and casual-wear icon from the Coen brothers 1998 cult classic, The Big Lebowski.

But the Dude abides, and Bridges suggested that his outlook looks just as promising.

As the Dude would say.. New S**T has come to light, tweeted Bridges, 70, on Monday. I have been diagnosed with Lymphoma. Although it is a serious disease, I feel fortunate that I have a great team of doctors and the prognosis is good.

Celebrities such as Cary Elwes, John Lithgow, Patricia Arquette and George Takei posted encouraging words and prayers to Bridges, who is the son of Lloyd and Dorothy Bridges, and has starred in more than 70 films including Starman, True Grit and The Last Picture Show. He won an Academy Award in 2010 for Crazy Heart, and was honored with the Cecil B. DeMille lifetime-achievement award during the 2019 Golden Globes.

And he is now one of the most high-profile cases of lymphoma, a cancer of the bodys infection-fighting lymphatic system that affects the blood and bone marrow. And more than 85,000 new cases of lymphoma are expected to be diagnosed in the U.S. this year, according to American Cancer Society data shared by the Leukemia & Lymphoma Society, with some 791,550 people currently living with lymphoma or in remission from the disease in the U.S.

Many different types of lymphoma exist, and Bridges did not share any more details about his diagnosis or treatment. But his disclosure is an opportunity to share more information about lymphoma, the risk factors and symptoms to be aware of, as well as treatment options.

What is lymphoma?

Lymphoma is a type of cancer that starts in cells that are part of the bodys immune system, specifically the lymphocytes, which are a type of white blood cell that fights germs. So these cancers can affect the blood and bone marrow, as well as the other tissues and organs that produce, store and carry white blood cells including the spleen.

Doctors still dont know what specifically causes lymphoma, but at some point a lymphocyte mutates and begins to reproduce rapidly. The mutated, abnormal cells live longer than the normal cells would, and in time, the diseased and ineffective lymphocytes outnumber the healthy cells, which causes the lymph nodes, liver and spleen to swell.

There are two main types of lymphoma, the CDC explains, including:

Hodgkin lymphoma (HL), which spreads in an orderly manner from one group of lymph nodes to another.

Non-Hodgkin lymphoma (NHL), which spreads through the lymphatic system in a non-orderly manner.

What are the symptoms?

Signs and symptoms of lymphoma may include:

These symptoms can be signs of other health conditions, of course, so its recommended that anyone experiencing them should see a doctor to determine the cause.

How is it treated?

There are many different types of lymphoma including 90 different types of non-Hodgkin lymphoma and treatment varies depending on the type and severity. Generally, lymphoma treatment involves chemotherapy, radiation therapy and immunotherapy medication. The Mayo Clinic, which is an international authority on lymphoma research, explains that the goal of treatment is to destroy as many cancer cells as possible to bring the disease into remission. A bone marrow or stem cell transplant may be performed in some cases to help rebuild healthy bone marrow after chemo and radiation has suppressed the diseased bone marrow.

Bridges didnt specify his own treatment, only saying that he is beginning treatment and will keep the public posted on his recovery.

Treatment can be very expensive, however, with almost 60% of patients covered by Medicare telling the Leukemia & Lymphoma Society in a 2019 study that they decided to delay or forego treatment, largely due to steep out-of-pocket costs. It noted that some traditional Medicare lymphoma patients getting anti-cancer therapy though infusions experienced out-of-pocket costs of more than $19,000 in their first year. And costs can extend two or three years beyond a blood cancer diagnosis.

Who is most at risk?

While children, teens and adults can all develop lymphoma, some types are more common in certain age groups. The CDC notes that rates of Hodgkin lymphoma are highest among teens and young adults (ages 15 to 39) as well as among older adults (ages 75 and older). But non-Hodgkin lymphoma becomes more common as people get older.

Men are also slightly more likely to develop lymphoma than women, the CDC adds, and white people are more likely than Black people to develop non-Hodgkin lymphoma.

Cases have also been more common in people who are immunocompromised, including those who take drugs to suppress their immune systems. And some infections such as HIV and the Epstein-Barr virus are also associated with an increased lymphoma risk.

And like many other cancers, family history has been linked with a higher risk of Hodgkin lymphoma.

What is the survival rate?

The good news is, Hodgkin lymphoma is now considered to be one of the most curable forms of cancer, according to the Leukemia & Lymphoma Society, with a five-year survival rate of 94.4% among patients younger than 45 at diagnosis. And the five-year relative survival rate for those with Hodgkin lymphoma more than doubled from 40% in whites in 1960 to 1963 (the only data available) to 88.5% for all races from 2009 to 2015.

And the five-year relative survival rate for people with non-Hodgkin lymphoma rose from 31% in whites from 1960 to 1963 (the only data available) to 74.7% for all races from 2009 to 2015.

Still, an estimated 20,910 Americans are expected to die from lymphoma this year, including 19,940 with non-Hodgkin lymphoma and 970 with Hodgkin lymphoma.

How does COVID-19 complicate things?

While the medical community is still learning about COVID-19, the general consensus is that people with cancer, who are in active cancer treatment or have previously been treated for cancer, may be at higher risk of severe illness and death if they get the coronavirus. So its important that these folks lower their risk of exposure to COVID-19 by avoiding large crowds and non-essential travel; working from home, if possible; staying at least six feet away from people outside their household; wearing a face mask when they cant socially distance; as well as washing their hands frequently, and not touching their eyes, nose or mouth.

Where can I find more information or support?

Visit the CDC and American Cancer Society pages on lymphoma.

The Mayo Clinic also outlines its lymphoma research and treatment strategies on its website.

The Leukemia & Lymphoma Society and the Lymphoma Research Foundation also provide valuable information and support.

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Jeff Bridges is one of the 85,000-plus lymphoma cases expected in the U.S. this year - MarketWatch

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The Myelofibrosis Treatment market to turn out to be Sublime between 2016 and 2022 – PRnews Leader

By daniellenierenberg

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

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

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

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

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

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

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

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Stem Cell Therapy Market 2020-2026 | XX% CAGR Projection Over the Next Five Years, Predicts Market Research Future with Market Size & Growth Key…

By daniellenierenberg

Stem Cell Therapy Market report would come handy to understand the competitors in the market and give an insight into sales, volumes, revenues in the Stem Cell Therapy Industry & will also assists in making strategic decisions. The report also helps to decide corporate product & marketing strategies. It reduces the risks involved in making decisions as well as strategies for companies and individuals interested in the Stem Cell Therapy industry. Both established and new players in Stem Cell Therapy industries can use the report to understand the Stem Cell Therapy market.

In Global Market, the Following Companies Are Covered:

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Analysis of the Market:

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

In the last several years, global stem cell therapy market developed fast at a average growth rate of 46.81%.

Market Analysis and Insights: Global Stem Cell Therapy Market

In 2019, the global Stem Cell Therapy market size was USD 403.6 million and it is expected to reach USD 1439.9 million by the end of 2026, with a CAGR of 19.7% during 2021-2026.

Global Stem Cell Therapy Scope and Market Size

Stem Cell Therapy market is segmented by Type, and by Application. Players, stakeholders, and other participants in the global Stem Cell Therapy market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast by Type and by Application in terms of revenue and forecast for the period 2015-2026.

Segment by Type, the Stem Cell Therapy market is segmented into Autologous, Allogeneic, etc.

Segment by Application, the Stem Cell Therapy market is segmented into Musculoskeletal Disorder, Wounds & Injuries, Cornea, Cardiovascular Diseases, Others, etc.

Regional and Country-level Analysis

The Stem Cell Therapy market is analysed and market size information is provided by regions (countries).

The key regions covered in the Stem Cell Therapy market report are North America, Europe, China, Japan, Southeast Asia, India and Central & South America, etc.

The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by Type, and by Application segment in terms of revenue for the period 2015-2026.

Competitive Landscape and Stem Cell Therapy Market Share Analysis

Stem Cell Therapy market competitive landscape provides details and data information by vendors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on revenue (global and regional level) by player for the period 2015-2020. Details included are company description, major business, company total revenue and the revenue generated in Stem Cell Therapy business, the date to enter into the Stem Cell Therapy market, Stem Cell Therapy product introduction, recent developments, etc.

The major vendors include Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell, Medi-post, Anterogen, Molmed, Takeda (TiGenix), etc.

This report focuses on the global Stem Cell Therapy status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Stem Cell Therapy development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.

Stem Cell Therapy Market Breakdown by Types:

Stem Cell Therapy Market Breakdown by Application:

Critical highlights covered in the Global Stem Cell Therapy market include:

The information available in the Stem Cell Therapy Market report is segmented for proper understanding. The Table of contents contains Market outline, Market characteristics, Market segmentation analysis, Market sizing, customer landscape & Regional landscape. For further improving the understand ability various exhibits (Tabular Data & Pie Charts) has also been used in the Stem Cell Therapy Market report.

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In the end, Stem Cell Therapy Industry report provides the main region, market conditions with the product price,profit, capacity, production, supply, demand and market growth rateand forecast etc. This report also Present newproject SWOT analysis,investment feasibility analysis, andinvestment return analysis.

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Stem Cell Therapy Market 2020-2026 | XX% CAGR Projection Over the Next Five Years, Predicts Market Research Future with Market Size & Growth Key...

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Stem Cell Assay Market In-Depth Analysis & Forecast 2017-2025 – The Think Curiouser

By daniellenierenberg

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues andtumors, wherein their toxicity, impurity, and other aspects are studied.

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With the growing number of successfulstem cell therapytreatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

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Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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Stem Cell Assay Market In-Depth Analysis & Forecast 2017-2025 - The Think Curiouser

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The Anthony Nolan and NHS Stem Cell Registry – The Hippocratic Post

By daniellenierenberg

More than 2 million people have registered to become stem cell donors the UK, new figures released today reveal. The UK stem cell register had an immensely successful year in 2019/20, with 326,756 new donors added over 100,000 more than the previous year.

The UK stem cell register is known as the Anthony Nolan and NHS Stem Cell Registry and is made up of donors recruited by NHS Blood and Transplant, the Welsh Blood Service, DKMS and Anthony Nolan. The UK donor registers are urging young men, and people from black, Asian and minority ethnic backgrounds to register and ensure that all patients in need of a stem cell transplant can find a, potentially, lifesaving match.

If a patient has a condition that affects their bone marrow or blood, then a stem cell transplant may be their best chance of survival. Doctors will give new, healthy stem cells to the patient via their bloodstream, where they begin to grow and create healthy red blood cells, white blood cells and platelets.

In 2019/20 62 per cent of people who donated stem cells or bone marrow to patients in the UK were men under 30. They are the demographic most likely to be chosen to donate, but make up just 19 per cent of the UK stem cell register.

The percentage of all donors from minority ethnic backgrounds has remained steady at 13 per cent in 2019/20, highlighting the importance of raising awareness of their lifesaving potential amongst this group. Patients from Black, Asian or other minority backgrounds have a 20 per cent chance of finding the best possible stem cell match from an unrelated donor, compared to 69 per cent for northern European backgrounds.

Henny Braund, Chief Executive of Anthony Nolan, said:

Nobody could have foreseen the challenges this year would bring to building a healthy, diverse stem cell register. But weve adapted and weve innovated as patients cant wait and were thrilled that in 2020, weve collectively recruited two million donors onto the stem cell register. Each donor represents hope, a potential cure for blood cancer.

I thank colleagues and partners for their commitment helping us reach this point. I am also immensely grateful for the two million selfless individuals who signed up to the registry, making themselves available whenever they are needed.

The two million milestone means increased chances for many of finding an unrelated donor match. But were still far from our goal of finding a match for everyone who needs one.

I would urge anyone thinking of joining the stem cell register, especially young men, who are the most likely to be chosen, to do so today. You could be someones lifesaver, without you there is no cure.

Christopher Harvey, Head of the Welsh Bone Marrow Registry, said:

Its incredibly heart-warming to know there are two million people in the UK who are willing to donate stem cells should they be the match for someone in need of their potentially lifesaving donation.

We see in our roles the difference stem cells make, for lots of patients receiving stem cells is the final treatment option.

Despite this great news we still have more to do. Unfortunately, there are still patients who are unable to find a match. Thats why were committed to ensuring every patient has the best possible chance of finding that one lifesaving donor in their time of need.

Guy Parkes, Head of Stem Cell Donation & Transplantation at NHS Blood and Transplant, said:

We want all patients in need of a transplant to be able to find a lifesaving match. Each time a person joins this register it brings fresh hope to patients of a match.

This register is used by hospitals across the UK to find suitable matches for patients and it has helped to save and improve the lives of thousands of people since its creation 33 years ago its amazing that we now have over 2 million people on the register, putting the chances of matching donors to patients at a record high.

Donating stem cells is an altruistic, lifesaving act and its an amazing thing to do. We will continue to expand the UK register to help patients in need. We particularly need more young men to join.

Jonathan Pearce, CEO of DKMS UK said:

Were delighted to have reached such an amazing milestone and are grateful to those two million people who are actively registered and waiting to help give someone living with blood cancer or a blood disorder a second chance of life.

At any one time there are around 2,000 people in the UK in need of a blood stem cell transplant, so whilst we recognise this achievement it goes without saying that we need to continue to encourage everyone that can register to do so. This will help to grow the numbers and diversify the registry further in order to improve the odds for those who currently have less chance of finding a matching donor.

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The Anthony Nolan and NHS Stem Cell Registry - The Hippocratic Post

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SA becomes 2nd country to allow 16 and 17-year-olds to donate bone marrow – News24

By daniellenierenberg

"Young people today are often drivers of social change movements and we look forward to engaging them."

South African youth aged 16 and 17 will be able to make history, alongside their peers in the UK, as the worlds youngest bone marrow donors.

The South African Bone Marrow Registry (SABMR) received the nod from its Clinical Governance Committee and board members, as well as the National Health Department to allow 16 and 17-year-old teens to become bone marrow, stem-cell donors.

Recent changes in legislation and advances in stem cell donation have allowed registries to reduce the age limit of donors. South Africa now joins the UK in this move. The latter became one of the first countries to do so.

Dr Charlotte Ingram, Medical Director of the SA Bone Marrow Registry (SABMR) the largest registry in the country says it's a landmark moment as the change in joining policy will contribute to saving more lives.

"In general, young people make better donors. Research shows that younger donors are associated with better survival rates for patients following a stem cell transplant."

"It's a step towards further enhancing the registry towards a younger and more ethnically diverse pool for blood cancer patients and others in need of a bone marrow transplant."

Previously, teenagers had to wait until they were 18 to join the SA Bone Marrow Registry. Now they can join by following the same procedure as others would.

While it is not required, it is important for the SABMR to involve parents and address any questions or concerns they may have re the procedure and what it entails.

Once youth have applied online, they will be contacted to discuss the easiest way of dispatching and collecting swab kits. The only initial sample that is required is a cheek swab.

Currently, 18-25-year olds only account for 6.8% of the SABMR registry but with increased awareness of bone marrow donation among young people, the figure should increase substantially.

Read:Knowledge is key: What you need to know about the most common childhood cancer in SA

"Studies tell us that generation WE (aged 14-20) and generation Z (21-25) are a lot more self-aware, socially-responsible and globally-minded than previous generations. They are more concerned about tackling social issues and want to roll up their sleeves and make a difference. Young people today are often drivers of social change movements and we look forward to engaging them."

She says there is no greater way to help another than to potentially save a life.

"So many lives are lost if there is a delay in finding a donor match. While we have 74 000 donors on our registry, we often discover that many older donors can no longer donate stem cells as they have developed hypertension, heart disease or diabetes."

"When this happens, we have to start the search process all over again, which prolongs the agonising wait for a patient, who doesn't have time to waste."

"By opening up the donor pool to a younger audience, means doctors and donors can choose the healthiest matches that substantially increases a patients chance of survival."

For now, social media will serve as the primary channel to create awareness among youth, but physical donor drives at schools and other initiatives, which encourage collaboration between learners, peers and patients are in the pipeline for 2021.

If you are between the ages of 16 and 45 and want to become a donor, contact the SABMR on 021 447 8638 or email: donors@sabmr.co.za.

Financial donations can also be made via http://www.sabmr.co.za/donate.

Submitted to Parent24 by theSA Bone Marrow Registry

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SA becomes 2nd country to allow 16 and 17-year-olds to donate bone marrow - News24

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YOUR HEALTH: Saving an unborn baby breaking apart in the womb – WQAD.com

By daniellenierenberg

DENVER A baby broken.

Most doctors gave little unborn Payton Calvillo any hope she would survive.

But through strong faith and the help of a team of medical experts, she is thriving today.

"She's a complete miracle baby," said Payton's mother, Ahna Calvillo.

When Ahna was just five months pregnant, she was told her unborn baby would probably not survive birth.

"It was pretty much a death sentence from the beginning."

Payton's bones were breaking and bending inside the womb.

"She likely had a problem where she couldn't make alkaline phosphatase properly," explained Dr. Sunil Nayak, a pediatric endocrinologist at Rocky Mountain Hospital for Children.

Alkaline phosphatase is needed for bones to grow and strengthen.

There was little anyone could do.

19 different specialists were on hand for the C-section delivery

"They even asked us the question that morning, how far do you want us to go?" Ahna remembered.

"'Do you want a ventilator on her?', you know, 'How far do you want us to prolong her life?'"

"Our ultimate hope and goal was that she would come out and breathe on her own."

"She just came out screaming," said Ahna.

"She came out crying. She breathed on her own right away. She was perfect."

Payton was diagnosed with hypophosphatasia, a disorder that weakens bones.

She was immediately placed on a new FDA-approved medicine.

"Here we are just one year later at one year of age and you see a dramatic difference in the shape," said Dr. Jared Riley, a pediatric orthopedic surgeon at Rocky Mountain Hospital.

Before the medicine, 75% of all patients died by the age of five.

Now there is a 97% chance Payton will live a normal life.

"My baby was broken and that's what I needed God to do was a miracle," said Ahna.

One was also treated with bone fragments and cultured osteoblasts, which are bone-forming cells.

"Cultured" refers to cells that are grown under specific conditions outside of the natural environment (the body) and within a laboratory.

Both patients showed significant, but incomplete improvement, although no more formal studies have been conducted.

Then, the drug teriparatide (parathyroid hormone 1-34) has been given "off-label" to several adults with HPP with metatarsal stress fractures or femoral pseudo fractures, resulting in healing.

The drug is not permitted for use in children.

More research is necessary to determine the long-term safety and effectiveness of teriparatide in the treatment of HPP.

Every year eight million babies are born with genetic disorders passed down from generation to generation.

Payton will stay on the new medication for the next few years and then doctors will re-evaluate whether she needs to continue.

Payton's family didn't even know they carried the problematic HPP gene until an ultrasound revealed it in their unborn baby.

After being genetically tested, Payton's mother and grandfather are positive.

Neither one has ever suffered from weak or broken bones.

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

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Stem Cell Therapy Market To Observe Exponential Growth By 2020-2027 | Verified Market Research – Stock Market Vista

By daniellenierenberg

Global Stem Cell Therapy Market was valued at USD 117.66 million in 2019 and is projected to reach USD 255.37 million by 2027, growing at a CAGR of 10.97% from 2020 to 2027.

For top companies in United States, European Union and China, this report investigates and analyzes the production, value, price, market share and growth rate for the top manufacturers, key data from 2020 to 2027.

The Stem Cell Therapy market report firstly introduced the basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the Stem Cell Therapy market report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

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Competitive Analysis:

The competitive analysis covers key players and the innovations and business strategies undertaken by them. The report captures the best long term growth opportunities for the sector and includes the latest process and product developments. The report includes basic information of the companies along with their market position, historical background, and market capitalization and revenue. The report covers revenue figures, market growth rate, and gross profit margin of each player based on regional classification and overall market position. The report provides a separate analysis of the recent business strategies such as mergers, acquisitions, product launches, joint ventures, partnerships, and collaborations.

Key features of the Report:

Leading players of Stem Cell Therapy including:

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Market Breakdown:

The market breakdown provides market segmentation data based on the availability of the data and information. The market is segmented on the basis of types and applications.

1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

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To understand the global Stem Cell Therapy market dynamics, the market is analyzed across major global regions and countries. Market Research Intellect provides customized specific regional and country-wise analysis of the key geographical regions as follows:

North America: USA, Canada, Mexico

Latin America: Argentina, Chile, Brazil, Peru, and Rest of Latin America

Europe:K., Germany, Spain, Italy, and Rest of EU

Asia-Pacific: India, China, Japan, South Korea, Australia, and Rest of APAC

Middle East & Africa: Saudi Arabia, South Africa, U.A.E., and Rest of MEA

The report considers:

Historical Years: 2017-2018

Base Year: 2019

Estimated Year: 2020

Forecast Years: 2020-2027

Benefits of Stem Cell Therapy Market Report:

Report Overview with TOC:

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Thank you for reading our report. Customization of the report is also available on the basis of region and countries. Kindly get in touch with us for more information regarding the report and its customization. Our team will ensure the report is best suited according to your needs.

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Stem Cell Therapy Market To Observe Exponential Growth By 2020-2027 | Verified Market Research - Stock Market Vista

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BrainStorm Announces Financial Results for the Third Quarter of 2020 and Provides a Corporate Update – PRNewswire

By daniellenierenberg

NEW YORK, Oct. 15, 2020 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced today financial results for the third quarter ended September 30, 2020, and provided a corporate update.

"The most important near-term event for BrainStorm will be the upcoming top-line data readout for the NurOwn Phase 3 trial in ALS, expected by the end of November. A successful outcome will set us on the path to filing a Biologic License Application (BLA) for what we believe will be a valuable new treatment for ALS," said Chaim Lebovits, Chief Executive Officer of BrainStorm Cell Therapeutics. "In parallel to our preparations for upcoming data read out, we are very busy planning and executing on other pre-BLA activities. On the management front, we appointed William K. White and Dr. Anthony Waclawski, adding valuable commercial and regulatory expertise to our leadership team. This expertise will be crucial as we work towards obtaining regulatory approval for NurOwn and ensuring that, if approved, it will be readily accessible to ALS patients in need of new treatment options for this devastating disease."

NurOwn has an innovative mechanism of action that is broadly applicable across neurodegenerative diseases and BrainStorm continues to invest in clinical trials evaluating the product in conditions beyond ALS to maximize value creation for its various stakeholders. The company remains on track to complete dosing in its Phase 2 clinical trial in progressive multiple sclerosis (PMS) by the end of 2020. In addition, the Company recently unveiled a clinical development program in Alzheimer's' disease (AD) and is planning a Phase 2 proof-of-concept clinical trial at several leading AD centers in the Netherlands and France.

Third Quarter 2020 and Recent Corporate Highlights:

Presented at the following Investor Conferences:

Cash and Liquidity as of October 14, 2020

Total available funding as of October 14, 2020, which includes cash, cash equivalents and short-term bank deposits of approximately $33.1 million as well as remaining non-dilutive funding from CIRM, IIA and other grants, amounts to approximately $36 million.

Financial Results for the Three Months Ended September 30, 2020

Conference Call & WebcastThursday, October 15, 2020 at 8 a.m. Eastern TimeFrom the US:877-407-9205International: 201-689-8054Webcast:https://www.webcaster4.com/Webcast/Page/2354/37811

Replays, available through October 29, 2020From the US:877-481-4010International: 919-882-2331Replay Passcode: 37811

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from theU.S. Food and Drug Administration(FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and completed enrollment inAugust 2020.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(FDA) and theEuropean Medicines Agency(EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at sixU.S.sites supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a filing forU.S.FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently receivedU.S.FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) completed enrollment inAugust 2020. For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

ContactsInvestor Relations:Corey Davis, Ph.D.LifeSci Advisors, LLCPhone: +1 646-465-1138[emailprotected]

Media:Paul TyahlaSmithSolvePhone: + 1.973.713.3768[emailprotected]

BRAINSTORM CELL THERAPEUTICS INC. AND SUBSIDIARIESINTERIM CONDENSED CONSOLIDATED BALANCE SHEETSU.S. dollars in thousands(Except share data)

September30,

December31,

2020

2019

U.S.$ inthousands

Unaudited

Audited

ASSETS

Current Assets:

Cash and cash equivalents

$

24,770

$

536

Short-term deposit (Note 4)

4,038

33

Other accounts receivable

1,473

2,359

Prepaid expenses and other current assets (Note 5)

56

432

Total current assets

30,337

3,360

Long-Term Assets:

Prepaid expenses and other long-term assets

27

32

Operating lease right of use asset (Note 6)

1,377

2,182

Property and Equipment, Net

950

960

Total Long-Term Assets

2,354

3,174

Total assets

$

32,691

$

6,534

LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT)

Current Liabilities:

Accounts payable

$

3,283

$

14,677

Accrued expenses

917

1,000

Operating lease liability (Note 6)

1,216

1,263

Other accounts payable

1,013

714

Total current liabilities

6,429

17,654

Long-Term Liabilities:

Operating lease liability (Note 6)

284

1,103

Total long-term liabilities

284

1,103

Total liabilities

$

6,713

$

18,757

Stockholders' Equity (deficit):

Stock capital: (Note 7)

12

11

Common Stock of $0.00005 par value - Authorized: 100,000,000 shares at September 30, 2020 and December 31, 2019 respectively; Issued and outstanding: 31,567,592 and 23,174,228 shares at September 30,2020 and December31,2019 respectively.

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BrainStorm Announces Financial Results for the Third Quarter of 2020 and Provides a Corporate Update - PRNewswire

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An Indian Baby ‘Savior Sibling’ Just Gave Her Brother Bone Marrow. But Is It Ethical? – The Swaddle

By daniellenierenberg

India just conducted its first successful experiment with savior sibling therapy, in which a baby was conceived through in-vitro fertilization for the purposes of donating bone marrow to an older ailing brother struggling with thalassemia, a condition characterized by low levels of hemoglobin in the blood that requires frequent blood transfusions. While the doctors involved in the therapy celebrated their success this week, some on social media challenged the ethics of such a therapy, in which a baby was essentially birthed to save her sibling.

In this case, the child with the genetic disorder needed a bone marrow transplant to cure his disease, and the chances of a successful cure are higher if coming from a person whose proteins (human leukocyte antigens, or HLA) exactly match those of the child. None of the childs existing family members was a match, further complicating the process of getting a bone marrow transplant an already difficult process to execute. The parents, in an effort to create a perfect bone marrow match for their child, underwent three cycles of in-vitro fertilization, out of which 18 embryos were created, and one perfectly matched that of the child, and was disease-free, using a technique called pre-implantation genetic diagnosis (PGD). The embryo was then implanted in the mothers uterus, carried to term, and a baby girl was born.

We had to wait for the baby to grow. She had to weigh 10 kg before we could draw bone marrow, Deepa Trivedi, program director of Sankalp Bone Marrow Unit in Ahmedabad, told The Hindu. Its been approximately seven months since the transplant, and the older sibling has not needed any more blood transfusions, indicating he has been cured of his thalassemia, his doctors announced.

Savior sibling therapy has already been used in countries such as the United Kingdom, the U.S.A., New Zealand, and France. Its mainly used to cure genetic blood disorders in children, such as sickle cell anemia or as seen in the Indian case, thalassemia major. The main way this is done, which is a departure from the Indian case, is by harvesting stem cells from a newborns umbilical cord, which are then injected into the bone marrow of the sibling with the disease, a practice that works 90% of the time. In case it doesnt, doctors can take bone marrow from the savior sibling as they grow, in a process that is painful but not known to be dangerous.

Related on The Swaddle:

Designer Babies Are Far From Reality, Even After the Gene-Edited Babies in China

The first ethical concern with this practice is treating a baby as a source for spare parts, as a means to an end, as a commodity. A study of the bioethics of savior sibling therapy, published in the Journal of Medical Ethics, surmised that treating a baby as a means to an end was not by itself a concern that devalued the utility of savior sibling therapy, as long as theyre also treated as human beings. Bioethicists surmise that using cord blood, something that is frequently discarded after birth, cannot endanger a newborn, or prove to be an ethical quandary used against the therapy.

But what has happened in the most recent case in India actually complicates the issue, because its not the umbilical cord blood that was harvested from the savior sibling at birth, but bone marrow 10 months into her life, which makes her an organ donor. This traverses thorny territory, as governments strictly regulate organ donation by minors due to issues related to consent. Can a baby consent to donating bone marrow to their sibling, or a 10-year-old consent to donating a kidney to their parent? It depends on where the individual resides, and how old the person being asked to donate is. In India, for example, it was only recently that the Delhi High Court ruled that minors could donate organs or tissues, as long as the procedure didnt pose a danger to their lives, and only in exceptional circumstances. However, where minors are mostly dependent upon their families, an element of coercion can also manifest. Also, determining whether a child rationally consented to donate an organ to their parent, for example, becomes difficult when we factor in the emotional element of their relationship that can perhaps override their judgments about their own safety.

Another concern is the well-being of the savior sibling throughout their life, both physical and psychological. Whats to stop a parent from asking the savior sibling to be on standby for their entire lives for their siblings health, available to be tapped for tissues and organs at any point in their lives? This is the plot of Jodi Picoults My Sisters Keeper (also turned into a film of the same name starring Cameron Diaz), but it is an unlikely scenario in real life, ethics experts have said. The aforementioned organ donation rules can prevent such an exploitative situation from arising, they say, with governments around the world tasked with ensuring the consent of the donor remains at the forefront of organ donation.

The third issue with savior sibling therapy arises out of the process itself if a parent can select an embryo that perfectly matches their child, whats to stop them from selecting an embryo for intelligence, or athleticism? This wades into the territory of the production of designer babies, which is an ethical slippery slope that critics have said goes against the natural reproductive order. However, the bioethics study asserts that the connection between savior sibling therapy and the production of designer babies is less of a slippery slope and more of a reach, as the technology might be similar, but the utility of both poles apart the former is used to save childrens lives, while the latter is a superficial, hypothetical fantasy.

For now, the world of savior sibling therapy, and its perception, remains similar to when parents first selected an embryo to create a savior sibling in the U.S. in 2000. As appeared in a New York Times article at the time, It is the kind of talk heard with every scientific breakthrough, from the first heart transplant to the first cloned sheep. We talk like this because we are both exhilarated and terrified by what we can do, and we wonder, with each step, whether we have gone too far. But though society may ask, How could you? the only question patients and families ask is, How could we not? 20 years later, savior sibling therapy still centers the children that can be saved, while government stipulations around the world try to ensure the savior siblings are protected, cared for, and treated as human beings, like any other child.

While a few critics argue for a ban, the bioethics study sums up the dilemma, and perhaps a solution to this ethical debate given that a ban will be fatal for a section of the population, the onus of proof rests clearly with the prohibitionists who must demonstrate that these childrens deaths are less terrible than the consequences of allowing this particular use of PGD.

You have got to have a very powerful reason to resist the means by which a childs life can be saved.

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An Indian Baby 'Savior Sibling' Just Gave Her Brother Bone Marrow. But Is It Ethical? - The Swaddle

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Child Conceived To Donate Bone Marrow Saves 6-Year-Old Brother’s Life – NDTV

By daniellenierenberg

A one-year-old girl donated her bone marrow to her brother. (Representational)

A one-year-old girl, conceived by her parents through IVF technology specifically for the purpose of donating her bone marrow to their thalassemic son, has succeeded in saving her six-year-old brother's life.

Baby Kavya was born a year ago through In-Vitro Fertilisation (IVF) technique in Ahmedabad, under the concept known as "saviour sibling".

Her bone marrow was successfully transplanted to her brother Abhijeet Solanki in March this year and the boy is now "risk-free", doctors said on Thursday.

Abhijeet, the second child of Sahdev Singh Solanki and Alpa Solanki, was diagnosed with Thalassemia-major, a blood disorder, and was dependent on blood transfusion every month, they said.

Thalassemia-major patients require frequent blood transfusions and their life expectancy is also less.

His parents were advised bone marrow transplant as the last resort to treat the child, but they could not find the required HLA (human leukocyte antigen) match.

"Due to unavailability of matching HLA donors for the transplant, we opted for IVF with HLA matching," city-based Nova IVF Fertility's medical director Dr Manish Banker told PTI.

This process of HLA typing is an established method for conceiving a child, who may donate cord blood or hematopoietic stem cells for transplantation to save a sibling with a critical illness.

Abhijeet's father approached Mr Banker after he found that the bone marrow of his family members, including the boy's elder sister, was not matching.

"Bone marrow transplant from an HLA-identical donor is the best therapeutic option for thalassemia major patients. We took the challenge and created a healthy savior sibling to save her elder brother," Mr Banker said.

With the help of IVF, Abhijeet's mother delivered a healthy baby girl Kavya a year ago, who was found to be the HLA match for the sibling.

In March this year, after Kavya gained the required weight, a successful bone marrow transplant was done for Abhijeet at the CIMS Hospital, Mr Banker said.

"Now, Abhijeet is risk-free and doesn't require blood transfusion," Mr Banker said.

"This is the first case in India when an HLA matching baby was born through IVF specifically to save the thalassemia-major sibling," he said.

The siblings' father, who himself researched well about this technique, thanked Mr Banker and other expert doctorsfor saving his son.

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Looking to save lives? Here’s how – The Mancunion

By daniellenierenberg

Cecelia Ahern, the author of P.S. I love you, once beautifully said: Moments are precious; sometimes they linger and other times theyre fleeting, and yet so much could be done in them; you could change a mind, you could save a life and you could even fall in love.

Helping save lives is what we decided to dedicate some of our lives to at Manchester Marrow.More specifically, we are the student-ran arm of the charity Anthony Nolan, which signs up students/young people (aged 16-30 years) to the stem cell register. This is required in finding matches for patients suffering from blood cancers and blood disorders who desperately need transplants. The more people we sign up for this register, the higher the chance of finding a blood stem cell or bone marrow match.

Anthony Nolan was initially founded by Shirley Nolan in 1974, realising the hardships associated with requiring an urgent bone marrow transplant. This was due to her three-year-old son suffering from a rare blood disorder known as Wiskott-Aldrich Syndrome. This inspired her to set up the worlds first register to match donors with people in desperate need. Today, there are over 800,000 people on Anthony Nolans UK register list, and each of these people could be a potential donor and save a life.

Although there are many resources at hand, without you, theres no cure! In Marrow, we have three important missions: raise awareness of Anthony Nolan and blood cancer within UK universities through our events, encourage every student to join stem cell register through our donor recruitment opportunities, and lastly, raise funds to help support this vital work.

As a student, in addition to signing up to the register, you have the amazing opportunity of volunteering for us and to save a life! One of our most outstanding achievements is signing up over 100,000 people to the Anthony Nolan register and raising over 92,000 in a year. Additionally, 1 in 4 people who go on to donate stem cells is recruited via Marrow!

Being a volunteer for us is no hard work. You could do many things, including spreading the word and talking to people about why they should sign up to the register. Furthermore, you need to inform them what the donation involves if they ever found a match, checking medical backgrounds for donor eligibility, assisting them with cheek swabs, and filling out an application form.

If youre interested in this opportunity, there will be several volunteer training sessions held throughout the year. Unfortunately, due to the current situation, all these events will be held online. We can assure you, however, that were doing our best to make the most of it.

To sign up to the register visit the Anthony Nolan website!

Make sure to follow Manchester Marrows social media accounts to keep updated with all the news and events:

Facebook: @ManchesterMarrow

Facebook: Manchester Marrow Volunteers Group

Instagram: @manchestermarrow

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Looking to save lives? Here's how - The Mancunion

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