Stem Cell Therapy Market 2020: Size, Professional Survey, and Forecast to 2025 – PRnews Leader

This report on global Stem Cell Therapy market primarily focuses on multi-faceted attributes comprising dynamic untapped market opportunities, end-user profile, vendor landscape, historical and current market status that collectively foster high end growth and sturdy revenue generation with concrete sustenance in global Stem Cell Therapy market. This in-depth research documentation is aimed at unfurling notable development factors and growth propellants that steer growth prospects despite challenging odds and intensified competition.

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To induce a more concrete, real-time synopsis of the current dynamics dominating the global Stem Cell Therapy market, this versatile report attempts to offer a decisive rundown of the major segments comprising of type, application as well as end-user profile, and regional expanse that collectively dominate future growth outlook in global Stem Cell Therapy market.

This future ready report closely monitors and assesses the role of various driving factors and restraints coupled with challenge assessment and elaborate SWOT and PESTEL analysis to arrive at logical deductions. Further in this report, readers are also presented with a 360degree assessment and evaluation of the competition spectrum, emphasizing frontline player activities and investment plans which collectively influence favorable transit in CAGR valuation through the forecast tenure.

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Global Stem Cell Therapy market is segmented based by type, application and region.

Stem Cell Therapy Market segment by Type, the product can be split into

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCs Bone Marrow-Derived Mesenchymal SCs Embryonic SCs Other Sources

Stem Cell Therapy Market segment by Application, split into

Based on therapeutic application, the market has been segmented into,

Musculoskeletal Disorders Wounds & Injuries Cardiovascular Diseases Gastrointestinal Diseases Immune System Diseases Other Applications

The report is designed specifically to entice reader attention towards identification and analysis of the chief growth propellants as well as dominant restraints and associated forces that optimally trace growth trajectory in global Stem Cell Therapy market.

Besides corrective measures, this intricate research report presentation on global Stem Cell Therapy market as compiled by our inhouse research professionals also features details on CAGR percentage, consumption and production patterns, as well as demand and supply gaps that have been immediate outcome of pandemic emergence and its lingering state.

Besides tracing direct implications of COVID-19 on the aforementioned Stem Cell Therapy industry that have affected production, consumption and overall supplychain, this report also shares critical understanding on the effective evolution scheme likely to elevate manufacturers and stakeholders from debilitating pandemic implications.

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Further, our team of research professionals have closely followed primary and secondary research methodologies to scout multi-layered information across disparate sources ranging from corporate sources, analytical solution providers, processing institutions to understand and comprehend value chain developments.

This report on global Stem Cell Therapy market primarily focuses on multi-faceted attributes comprising dynamic untapped market opportunities, end-user profile, vendor landscape, historical and current market status that collectively foster high end growth and sturdy revenue generation with concrete sustenance in global Stem Cell Therapy market. This innovative research documentation is aimed at unfurling notable development factors and growth propellants that steer growth prospects despite challenging odds and intensified competition.

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Stem Cell Therapy Market 2020: Size, Professional Survey, and Forecast to 2025 - PRnews Leader

The Adipose Tissue Derived Stem Cells market to grow in the wake of incorporation of the latest technology – The Think Curiouser

Adipose tissue is rich in multi potent stem cells that have the capability to differentiate into a number of cell types including adipocytes, osteocytes, chondrocytes and others, in vitro. These Adipose Tissue-derived Stem Cells are used for a number of applications including stem cell differentiation studies, regenerative medicine, cell therapy, tissue engineering and development of induced pluripotent stem cell lineage. Adult stem cells such as the Adipose Tissue-derived Stem Cells have a very good potential for regenerative medicine. The Adipose Tissue-derived Stem Cells show higher yields compared with other stem cell sources. Some of the regenerative medicine applications using Adipose Tissue-derived Stem Cells include skin, bone and cartilage regeneration.

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Although, Adipose Tissue-derived Stem Cells have the ability to differentiate into different cell types in vitro, unlike the embryonic stem cells they lack the ability to differentiate into all types of organs and tissues of the body. Derivation of stem cells from adipose tissue have a number of advantages including that fat tissue contain 100 to 1000 times more mesenchymal stem cells than the bone marrow. Furthermore the method of collection of fat tissue is relatively easier and is less invasive than that of bone marrow collection. Although Adipose Tissue-derived Stem Cells have a potential to be used in cell-based therapy, there are a number of challenges the Adipose Tissue-derived Stem Cells market has to face. Some of the challenge include the safety issue for the clinical use of Adipose Tissue-derived Stem Cells, development and differentiation of the cells, delivery of the cells in vivo and immune response after the transplant.

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The global Adipose Tissue-derived Stem Cells market is segmented based on product type and end user. Based on product type the Adipose Tissue-derived Stem Cells can be categorized into cell line and reagent & kits. Cell line can be further classified based on the source of the adipose tissue such as human and rodents. Based on reagents the Adipose Tissue-derived Stem Cells market is further classified as media & sera and kits. Based on application the Adipose Tissue-derived Stem Cells market is classified into regenerative medicine, cell therapy, tissue engineering, and other applications such as cell differentiation studies and other similar research. End users of Adipose Tissue-derived Stem Cells market are biotechnology companies and academic and research institutes.

The Global Adipose Tissue-derived Stem Cells market is classified on the basis of product type, end user and region:

Based on the Product Type, Adipose Tissue-derived Stem Cells market is segmented into following:

Based on the application, Adipose Tissue-derived Stem Cells market is segmented into following:

Based on the end user, Adipose Tissue-derived Stem Cells market is segment as below:

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Growing research activities using stem cells along with the growth of regenerative medicine and cell therapy the global Adipose Tissue-derived Stem Cells market is set to expand considerably during the forecast period. However, ethical concerns and stringent regulations may hinder the growth of the global Adipose Tissue-derived Stem Cells market.

On the basis of geography, global Adipose Tissue-derived Stem Cells market is segmented into six major regions that include North America, Latin America, Europe, Asia-Pacific excluding China, China and Middle East & Africa. North America is expected to be the most lucrative Adipose Tissue-derived Stem Cells market owing to increased research activity of stem cells. Furthermore government support for regenerative and stem cell based studies along with cell therapy studies is driving the growth of the Adipose Tissue-derived Stem Cells market in the region. Changing government regulations in china is supporting the research activity that supports the growth of the adipose tissue-derived stem cell market in the region at a considerable rate.

Key participants operating in the Adipose Tissue-derived Stem Cells market are: Lonza, ThermoFisher Scientific, Celprogen, Inc, American CryoStem, Rexgenero Ltd, iXCells Biotechnologies, Merck KGaA, Lifeline Cell Technology, and others.

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The Adipose Tissue Derived Stem Cells market to grow in the wake of incorporation of the latest technology - The Think Curiouser

Novel Targeted Drugs are Changing the Treatment of Diffuse Large B-Cell Lymphoma – Curetoday.com

Several drugs that work by targeting genetic alterations in cancer cells have won recent approval from the Food and Drug Administration as treatments for patients with diffuse large B-cell lymphoma (DLBCL).

Dr. Germame Ajebo, assistant professor of medicine at Georgia Cancer Center at Augusta University, shared information on novel treatments for the disease during the recent virtual CURE Educated Patient Leukemia & Lymphoma Summit.

In his talk, Ajebo focused on drugs meant for use in disease that has recurred or become resistant to previous treatments.

DLBCL is a usually aggressive form of the blood cancer known as a B-cell non-Hodgkin lymphoma, which affects the immune system. The disease causes rapid growth of tumors in the lymph nodes, spleen, liver, bone marrow or other organs.

Approved by the FDA within the last two years to treat aggressive DLBCL are oral Xpovio (selinexor), Polivy (polatuzumab vedotin-piiq) and Monjuvi (tafasitamab-cxix). In addition, an immunotherapy, the chimeric antigen receptor (CAR)-T cell therapy Yescarta (axi-cel), was approved to treat the disease in 2017, Ajebo reported.

Read more: Monjuvi-Revlimid Combination Approval Fills Unmet Need for Certain Patients with DLBCL.

Xpovio is a nuclear export inhibitor, which prevents cancerous cells from pushing tumor-suppressing proteins out of their nuclei. This results in tumor suppressors accumulating in the nucleus, where they can work to kill the cell.

In the phase 2b clinical trial that led to its approval which administered Xpovio by itself to 134 previously treated older adult patients the partial response rate (including those with tumor shrinkage) was 16%, the complete response rate (including those with no sign of cancer remaining) was 13% and the rate of stable disease (including patients with no progression of cancer) was 8.2%, Ajebo reported. Looking at all patients who had partial or complete responses, 38% responded for at least six months and 15% for at least 12 months.

The most common side effects that were serious or worse were low blood counts, Ajebo summarized. Other serious side effects included nausea, vomiting, diarrhea, weight loss, dizziness and infections.

Polivy is an antibody-drug conjugate that uses a targeted drug to deliver a potent chemotherapy directly to cancer cells.

It was approved based on the results of a phase 2 study of 80 previously treated patients who were divided into equally sized groups to receive the chemotherapy Treanda (bendamustine) and the targeted drug Rituxan (rituximab) with or without Polivy every 21 days for six cycles. At the end of treatment, 40% of those receiving the triplet combination had experienced a complete response, compared with 18% of those receiving Treanda and Rituxan alone. In the 63% of patients who achieved a best overall response at any point in the study while receiving the drug triplet, 48% had a response that lasted at least 12 months and 64% responded for at least six months, Ajebo noted.

Major side effects, he said, included tingling or weakness in the extremities, low blood counts, liver toxicity and tumor lysis syndrome, a condition that can damage organs due to blood chemistry issues arising from the quick destruction of tumor cells. The most common side effects of any severity included low blood counts, fatigue, diarrhea and fever.

Monjuvi, a targeted drug that inhibits the activity of the DLBCL-fueling protein CD19, was approved based on results of the phase 2 L-MIND study that demonstrated a 43% complete response rate and an 18% partial response rate in 80 previously treated adult patients who were prescribed the drug along with the targeted medication Revlimid (lenalidomide), with a median duration of response of 21.7 months. The drug is approved in combination with Revlimid for patients with recurrent or resistant DLBCL who are not eligible for, or did not agree to undergo, bone marrow transplant using their own stem cells, Ajebo said.

Serious side effects occurred in 52% of patients, he said, and included low blood counts and infections. The drug caused fatal reactions in 5% of patients, including stroke, respiratory failure, progressive multifocal leukoencephalopathy (a virus that infects the brain) and sudden death.

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Novel Targeted Drugs are Changing the Treatment of Diffuse Large B-Cell Lymphoma - Curetoday.com

Equillium Announces Positive Interim Data of Itolizumab in the First-line Treatment of Acute Graft-Versus-Host Disease – GlobeNewswire

November 06, 2020 08:35 ET | Source: Equillium

100% overall response rate in dose cohort 3 and 80% overall response rate across all cohorts to date

Complete response observed in seven of eight responding patients

EQUATE Phase 1b topline data expected during first half 2021

LA JOLLA, Calif., Nov. 06, 2020 (GLOBE NEWSWIRE) -- Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company developing itolizumab to treat severe autoimmune and inflammatory disorders, today announced positive interim data from the third cohort of the Phase 1b open label, dose escalation study of itolizumab in the first-line treatment of acute graft-versus-host disease (aGVHD). The EQUATE trial is evaluating itolizumab in severe aGVHD patients concomitant with standard of care, which is typically comprised of high dose corticosteroids, as no other therapeutics are currently approved for this indication. Equillium anticipates reporting topline data across all cohorts from the Phase 1b portion of the EQUATE trial during the first half of 2021 and is accelerating plans for further development of itolizumab in graft-versus-host disease.

In the EQUATE trial, the overall response rate across the first three dose cohorts was 80%, and seven of eight patients responding achieved a complete response (CR) and one patient achieved a very good partial response (VGPR) by Day 29 (VGPR approximates the clinical benefit of CR). Responses observed have been rapid, with most patients achieving a CR within the first 15 days, and durable as patients in the first two cohorts have maintained responses through Day 57. To date, adverse events reported with the EQUATE trial have been consistent with the safety profile previously reported for itolizumab and those common in the aGVHD patient population. In review of the totality of safety, efficacy and pharmacodynamic data, the independent data monitoring committee has recommended to expand cohort 3 (1.6 mg/kg dose) and proceed forward with enrollment.

We continue to accrue compelling data in the EQUATE trial the rapid and durable response rates in patients treated with itolizumab meaningfully exceeds what has been observed in patients with severe aGVHD treated with steroids alone, said Bruce Steel, chief executive officer of Equillium. We plan to engage the U.S. Food and Drug Administration (FDA) to explore expedited regulatory pathways to advance itolizumab for the first-line treatment of aGVHD. Further, this data suggests opportunities to expand the potential therapeutic application of itolizumab for patients with chronic GVHD and as a potential preventative treatment for patients who have undergone hematopoietic stem cell transplantation. We believe itolizumab has the potential to be a life-saving medicine for aGVHD patients, a severe and life-threatening illness for which there are currently no approved therapeutics.

Equillium has received fast track designation from the FDA for the treatment of itolizumab in patients with aGVHD and orphan drug designations from the FDA for both the prevention and treatment of aGVHD. Equillium plans to provide additional updates on the program at its upcoming analyst day on December 4, 2020.

About Graft-Versus-Host Disease GVHD is a multisystem disorder that is a common complication of allogeneic hematopoietic stem cell transplants (allo-HSCT) caused by the transplanted immune system recognizing and attacking the recipients body. Symptoms of GVHD include rash, itching, skin discoloration, nausea, vomiting, diarrhea, and jaundice, as well as eye dryness and irritation.

GVHD is the leading cause of non-relapse mortality in cancer patients receiving allo-HSCT, and the risk of GVHD limits the number and type of patients receiving HSCT. GVHD results in very high morbidity and mortality, with five-year survival of approximately 53% in patients who respond to steroid treatment and mortality as high as 95% in patients who do not respond to steroids. In the first-line aGVHD setting, published literature (MacMillan et al., 2015) describes background response rates to high-dose steroid administration in less severe standard risk patients as 69% overall response rate (ORR) and 48% CR, whereas in more severe high-risk patients response rates observed were 43% ORR and 27% CR.

About the EQUATE Study The EQUATE study is a Phase 1b/2 trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical activity of itolizumab for first-line treatment in patients who present with aGVHD (NCT 03763318). The Phase 1b part of the trial is an open-label dose escalation study in adult patients who present with severe aGVHD (Grades III and IV) and typically respond poorly to steroids. The Phase 1b data will inform selection of the dose to be used in the next phase of development for the program.

About Itolizumab Itolizumab is a clinical-stage, first-in-class monoclonal antibody that selectively targets the CD6-ALCAM pathway. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. Itolizumab is currently being evaluated in multiple clinical trials in patients with severe diseases, including aGVHD, lupus nephritis, uncontrolled asthma, and will soon be evaluated in a clinical trial of patients with COVID-19. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited. Itolizumab is marketed in India under the trade name ALZUMAb-L for the treatment of chronic plaque psoriasis and has received emergency use approval in India to treat cytokine release syndrome in COVID-19 patients with moderate to severe acute respiratory distress syndrome.

About Equillium Equillium is a clinical-stage biotechnology company leveraging deep understanding of immunobiology to develop novel products to treat severe autoimmune and inflammatory disorders with high unmet medical need. Equillium is developing itolizumab for multiple severe immuno-inflammatory diseases, including COVID-19, aGVHD, lupus nephritis and uncontrolled asthma.

For more information, visitwww.equilliumbio.com.

Forward Looking Statements Statementscontained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limitedto, the potential benefit of treating aGVHD or chronic GVHD patients, as well as patients who have undergone hematopoietic stem cell transplantation, with itolizumab, Equilliums business strategy, Equilliums plans and expected timing for developing itolizumab, including theexpected timingofcompletion of theEQUATE study and initiating a clinical trial in patients with COVID-19,the potential for interim data results to be consistent with final results, once available,the potential benefits of itolizumab, the potential for any ofEquilliumsongoingor plannedclinical trials to show safety or efficacy, and the impact of the COVID-19 pandemic. Risks that contribute to the uncertain nature of the forward-looking statements include:the risk that interim results of a clinical trial do not necessarily predict final results and that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; potential delays in the commencement, enrollment and completion of clinical trials and the reporting of data therefrom;the risk thatstudies willnot becompletedas planned;uncertainties related to Equilliums capital requirements; Equilliums plans and product development, including the initiation, restarting and completion of clinical trials; uncertainties related to the actual impacts and length of such impacts caused by the COVID-19 pandemic; uncertainties caused by the recent restarting of the EQUIP and EQUALISE clinical trials after a pause; whether the results from clinical trials will validate and support the safety and efficacy of itolizumab;andhaving to use cash in ways or on timing other than expected and the impact of market volatility on cash reserves. These and other risks and uncertainties are described more fully under the caption "Risk Factors" and elsewhere in Equillium's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Equillium undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor Contact Christine Zedelmayer, Chief Operating Officer +1-858-412-5302 ir@equilliumbio.com

Media Contact Katherine Carlyle Smith Senior Account Associate Canale Communications 805-907-2497 katherine.smith@canalecomm.com

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Equillium Announces Positive Interim Data of Itolizumab in the First-line Treatment of Acute Graft-Versus-Host Disease - GlobeNewswire

Orchard Therapeutics Announces New OTL-201 Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA) Accepted for Oral Presentation at 62nd American…

November 05, 2020 07:00 ET | Source: Orchard Therapeutics (Europe) Limited

BOSTONandLONDON, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced the presentation of new clinical data at the upcoming 62nd American Society of Hematology (ASH) Annual Meeting to be held virtually December 5-8, 2020. The oral presentation will highlight data from the first patient treated in the ongoing proof-of-concept study of OTL-201, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy being studied for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA).

MPS-IIIA is a progressive, life-threatening metabolic disease with no approved treatment options, said Professor Robert Wynn, chief investigator at The Royal Manchester Childrens Hospital, part of Manchester University NHS Foundation Trust. We are pleased to see encouraging initial results, including evidence of engraftment of gene-modified cells, an important first step in the investigation of whether OTL-201 could address critical unmet needs for patients with MPS-IIIA. We look forward to continuing to advance this program to add to the growing body of evidence supporting the use of HSC gene therapy to treat severe neurometabolic conditions.

Preliminary results from the first patient treated with OTL-201 show evidence of engraftment of gene-modified cells, supra-physiological N-sulphoglucosamine sulphohydrolase (SGSH) enzyme expression in multiple lineages, and reduction of heparan sulfate in plasma, cerebrospinal fluid and urine over an initial three-month follow-up period. Additional follow-up data and an update on the trial status will be shared at the time of the oral presentation.

Oral Presentation Details:

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress NCT04201405 Publication number: 676 Session: 801. Gene Editing, Therapy and Transfer I Date and time: Monday, December 7, 2020; 12:45 p.m. PT

Abstracts are available online at the ASH Annual Meeting website.

About OTL-201 and MPS-IIIA Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease. People with MPS-IIIA are born with a mutation in the N-sulphoglucosamine sulphohydrolase (SGSH) gene, which, when healthy, helps the body break down sugar molecules called mucopolysaccharides, including heparan sulfate. The buildup of mucopolysaccharides in the brain and other tissues leads to intellectual disability and loss of motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years.1 There are currently no approved treatment options for MPS-IIIA. OTL-201 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-IIIA. It uses a modified virus to insert a functional copy of the SGSH gene into a patients cells.

About Orchard Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

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

About Manchester University NHS Foundation Trust Manchester University NHS Foundation Trust is one of the largest NHS trusts in England and a leading provider of specialist healthcare services. Its nine hospitals are home to hundreds of world class clinicians and academic staff committed to finding patients the best care and treatments. More information is available at http://www.mft.nhs.uk.

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

Forward-Looking Statements This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, and the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

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

Media Molly Cameron Manager, Corporate Communications +1 978-339-3378 media@orchard-tx.com

1Lavery, C., Hendriksz, C.J. & Jones, S.A. Mortality in patients with Sanfilippo syndrome. Orphanet J Rare Dis 12, 168 (2017) doi:10.1186/s13023-017-0717-y

Excerpt from:
Orchard Therapeutics Announces New OTL-201 Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA) Accepted for Oral Presentation at 62nd American...

bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from its gene and cell therapy programs for sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT) and multiple myeloma (MM) will be presented, including seven oral presentations, at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.

Updated results from patients in Group C of the companys Phase 1/2 HGB-206 study of LentiGlobin for SCD gene therapy (bb1111) will be presented.

bluebird bio will also present updated long-term efficacy and safety results from the LTF-303 follow-up study; outcomes across genotypes; and outcomes in pediatric patients from Phase 3 studies HGB-207 and HGB-212 of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for -thalassemia) in TDT.

Data from across the companys multiple myeloma program will be presented. Presentations will include updated safety and efficacy results from the Phase 1 CRB-401 clinical study of idecabtagene vicleucel (ide-cel, bb2121) and preliminary data from the ongoing Phase 1 CRB-402 clinical study of bb21217, as well as subgroup analyses of the pivotal Phase 2 KarMMa study of ide-cel. Ide-cel and bb21217 are investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immune therapies being studied, in partnership with Bristol-Myers Squibb, for the treatment of adult patients with MM.

Sickle Cell Disease Data at ASH

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy Presenting Author: Julie Kanter, MD, University of Alabama at Birmingham, Birmingham, AL Date/Time: Oral #365, Sunday, December 6, 2020, 9:45 am PST

Resolution of Serious Vaso-occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy Presenting Author: Alexis A. Thompson, MD, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital, Chicago, IL Date/Time: Oral #677, Monday, December 7, 2020, 1:30 pm PST

The GRNDaD Registry: Contemporary Natural History data and an analysis of real-world patterns of use and limitations of Disease Modifying Therapy in adults with SCD Presenting Author: Alexandra Boye-Doe, MD, University of North Carolina School of Medicine, Chapel Hill, NC Date/Time: Poster #1730, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Transfusion-Dependent -Thalassemia Data at ASH

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent -Thalassemia: Results in Patients with up to 6 Years of Follow-up Presenting Author: Janet L. Kwiatkowski, MD, MSCE, Director, Thalassemia Center at Children's Hospital of Philadelphia, Philadelphia, PA Date/Time: Oral #153, Saturday, December 5, 2020, 12:00 pm PST

Favorable Outcomes in Pediatric Patients in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) Studies of betibeglogene autotemcel Gene Therapy for the Treatment of Transfusion-dependent -thalassemia Presenting Author: Alexis A. Thompson, MD, MPH, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, IL Date/Time: Oral #154, Saturday, December 5, 2020, 12:15 pm PST

Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent -Thalassemia in the Phase 3 HGB-207 Study Presenting Author: John B. Porter, MA, MD, FRCP, FRCPath, Head of Red Cell Unit, University College London Hospital, London, UK Date/Time: Poster #776, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Response of patients with transfusion-dependent -thalassemia (TDT) to betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy based on HBB genotype and disease genetic modifiers Presenting Author: Mark C. Walters MD, Medical Director, Jordan Family Center for BMT & Cellular Therapies Research, UCSF Benioff Childrens Hospital Oakland, Oakland, CA Date/Time: Poster #1699, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Multiple Myeloma Data at ASH

Updated results from the Phase I CRB-402 study of anti-BCMA CAR-T cell therapy bb21217 in patients with relapsed and refractory myeloma: correlation of expansion and duration of response with T cell phenotypes Presenting Author: Melissa Alsina, MD, Department of Blood and Marrow Transplantation and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL Date/Time: Oral #130, Saturday, December 5, 2020, 9:45 am PST

Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study Presenting Author: Yi Lin, MD, PhD, Division of Hematology, Mayo Clinic, Rochester, MN Date/Time: Oral #131, Saturday, December 5, 2020, 10:00 am PST

Secondary Quality-of-Life Domains in Patients With Relapsed and Refractory Multiple Myeloma Treated With the BCMA-Directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel; bb2121): Results from the KarMMa Clinical Trial Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA Date/Time: Oral #437, Sunday, December 6, 2020, 12:15 pm PST

Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients with Relapsed/Refractory Multiple Myeloma: KarMMa Subgroup Analysis Presenting Author: Jess Berdeja, MD, Sarah Cannon Research Institute and Tennessee Oncology, Nashville, TN Date/Time: Poster #1367, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Characterization of Cytokine Release Syndrome in the KarMMa Study of Idecabtagene Vicleucel (ide-cel, bb2121) For Relapsed and Refractory Multiple Myeloma Presenting Author: Ankit Kansagra, MD, Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, TX Date/Time: Poster #1378, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Molecular and Phenotypic Profiling of Drug Product and Post-infusion Samples from CRB-402, an Ongoing: Phase I Clinical Study of bb21217 a BCMA-directed CAR T Cell Therapy Presenting Author: Olivia Finney, PhD, Associate Director, Immunotherapy, bluebird bio Date/Time: Poster #1401, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Effects of Prior Alkylating Therapies on Preinfusion Patient Characteristics and Starting Material for CAR T Cell Product Manufacturing in Late-Line Multiple Myeloma Presenting Author: Julie Rytlewski, PhD, Bristol Myers Squibb, Princeton, NJ Date/Time: Poster #1405, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

KarMMa-4: Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy, in High-Risk Newly Diagnosed Multiple Myeloma Presenting Author: Saad Z. Usmani, MD, Director, Clinical Research in Hematologic Malignancies, Levine Cancer Institute/Atrium Health, Charlotte, NC Date/Time: Poster #1418, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Healthcare Resource Utilization and Cost of Cytokine Release Syndrome and Neurologic Events in Patients with Relapsed and Refractory Multiple Myeloma Receiving the BCMA-directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel, bb2121) in the KarMMa Trial Presenting Author: Parmeswaran Hari, MD, Medical College of Wisconsin, Milwaukee, WI Date/Time: Poster #1598, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

A Matching-Adjusted Indirect Comparison of Efficacy Outcomes for Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T Cell Therapy Versus Conventional Care in Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Presenting Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA Date/Time: Poster #1653, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) Responses Are Characterized by Early and Temporally Consistent Activation and Expansion of CAR T Cells With a T Effector Phenotype Presenting Author: Nathan Martin, PhD, Bristol Myers Squibb, Princeton, NJ Date/Time: Poster #2315, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

KarMMa-3: A Phase 3 Study of Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy Versus Standard Regimens in Relapsed and Refractory Multiple Myeloma Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium Date/Time: Poster #2323, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) in Relapsed and Refractory Multiple Myeloma: Analyses of High-Risk Subgroups in the KarMMa Study Presenting Author: Noopur S. Raje, MD, Massachusetts General Hospital, Boston, MA Date/Time: Poster #3234, Monday, December 7, 2020, 7:00 am 3:00 pm PST

Health State Utility Valuation in Patients with Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Treated with the BCMAdirected CAR T Cell Therapy, Idecabtagene Vicleucel (idecel, bb2121): Results from the KarMMa Trial Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium Date/Time: Poster #3465, Monday, December 7, 2020, 7:00 am 3:00pm PST

Abstracts outlining bluebird bios accepted data at ASH are available on the ASH conference website.

About LentiGlobin for SCD (bb1111)

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD (bb1111) is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

LentiGlobin for SCD was designed to add functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once patients have the A-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin (HbA-T87Q) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As of March 3, 2020, a total of 37 patients have been treated with LentiGlobin for SCD to-date in the HGB-205 (n=3) and HGB-206 (n=34) clinical studies. The HGB-206 total includes: Group A (n=7), B (n=2) and C (n=25).

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

The U.S. Food and Drug Administration (FDA) granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD. LentiGlobin for SCD continues to be evaluated in the ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About betibeglogene autotemcel

Transfusion dependent beta-thalassemia (TDT) is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT require chronic blood transfusions to maintain adequate Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel (beti-cel) adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbA-T87Q, which is gene therapy-derived adult hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

As of March 3, 2020, a total of 60 pediatric, adolescent and adult patients, including 11 patients with at least 5 years of follow-up, across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. In studies of beti-cel, patients were assessed for transfusion independence, defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. FDA granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the United States. Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of beti-cel.

About idecabtagene vicleucel (ide-cel, bb2121)

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio. Ide-cel was granted accelerated assessment by the European Medicines Agency (EMA) on March 26, 2020, and the Marketing Authorization Application (MAA) was validated by the EMA on May 20, 2020. The FDA accepted the ide-cel Biologics License Application (BLA) for priority review on September 22, 2020.

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with RRMM in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other secondary endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

CRB-401 (NCT02658929) is an open-label Phase 1 study evaluating the preliminary safety and efficacy of ide-cel in patients with relapsed and refractory multiple myeloma (RRMM). The primary endpoint of the study is safety. CRB-401 was designed as a two-part (dose escalation and dose expansion) study to determine the maximum tolerated dose and further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose; these findings established the recommended dose of the Phase 2 KarMMa trial. All patients have been treated in the study and follow-up is ongoing.

In addition to the pivotal KarMMa and CRB-401 trials, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) exploring ide-cel combinations and activity in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

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

About bb21217

bb21217 is an investigational BCMA-targeted CAR T cell therapy that uses the ide-cel CAR molecule and is cultured with the PI3 kinase inhibitor (bb007) to enrich for T cells displaying a memory-like phenotype with the intention to increase the in vivo persistence of CAR T cells. bb21217 is being studied for patients with multiple myeloma in partnership with Bristol Myers Squibb.

bluebird bios clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with relapsed and refractory multiple myeloma (RRMM), designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM. For more information visit: clinicaltrials.gov using identifier NCT03274219.

bb21217 is not approved for any indication in any geography.

About bluebird bio, Inc.

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

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

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

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: regarding the potential for betibeglogene autotemcel to treat transfusion-dependent -thalassemia and the potential for LentiGlobin for sickle cell disease (SCD) to treat SCD, and the potential for idecabtagene vicleucel and bb21217 to treat multiple myeloma; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -...

Voters approve stem cell research bonds, but other health care measures defeated – State of Reform – State of Reform

Shawna De La Rosa | Nov 4, 2020

Californians voted on several health-related ballot measures including Proposition 23 which would require kidney dialysis clinics to have an on-site physician present while patients were being treated. The proposition was defeated in the election with a 64% no vote. The rejection of the measure is similar to the response to California Prop 8, which would have required dialysis clinics to issue refunds to patients for profits above 115% of the costs of direct patient care and health care improvements.

Proposition 14, which unofficially passed at 51% to 48.9%, will issue $5.5 billion in general obligation bonds for the California Institute for Regenerative Medicine, which was created to fund stem cell research. Proposition 71, which passed in 2004, issued $3 billion in bonds to finance CIRM. The current proposition requires that CIRM spends no more than 7.5% of bond funds on operational costs. The remaining funds will be spent on grants to groups that conduct trials, programs and research on stem cell treatments and for start-up costs for facilities. Californians for Stem Cell Research, Treatment and Cures led the campaign which received $16.62 million as of Oct. 17. Its largest donor was Robert N. Klein II, a real estate investor and stem cell research advocate.

Proposition 22, allows ride-hailing companies like Uber, Lyft, Doordash and Instacart to continue to treat its employees as independent contractors and not offer them health care and other benefits. The measure was approved by the voters by a 58% in favor and 42% opposed.

Proposition 15 would increase commercial property owners taxes for holdings of $3 million. The funds would be used for public health infrastructure, but the measure is still to close to call. The measureis trailing at 52% opposed with 100% of the precincts reporting. Community clinics, nurses and Planned Parenthood supported the measure hoping for funds to help rebuild the underfunded health care system.

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Voters approve stem cell research bonds, but other health care measures defeated - State of Reform - State of Reform

Stem cells key to ALS therapy – Agoura Hills Acorn

By The Acorn Staff | on November 05, 2020

Twenty years ago, when stem cell therapy was highly regulated in the United States and other countries, it was well underway in the Hadassah Hospital labs in Jerusalem.

Never would we have imagined that the U.S. expansion of one of the key clinical trials conducted in our labs in Israel would be later funded by Californias Stem Cell Institute.

In 2004, California had the foresight to advance this critical area of research with the passage of Prop. 71 in 2004.

Stem cells replace damaged or diseased tissue. In this way, treatments or cures for diseases like age-related macular degeneration, ALS, MS, Parkinsons, Alzheimers and diabetes could be a reality in the foreseeable future.

I come to this subject from a place of personal sorrow. I watched my father-in-law, Irv, suffer for 12 years with ALS, a man I loved as if he were my own father. He fought hard. He made every minute of his battle meaningful, to soak up as much life as he could until he couldnt.

Hadassah researchers conducted the worlds first clinical trial using the patients own bone marrow stem cells to treat ALS. Expanded stem cell trials are now taking place here in California to treat ALS.

The California Stem Cell Agencys ALS funding has awarded a total of $79 million in grants to understand ALS and then to translate those discoveries into treatments and therapies. Two have already reached the clinical trial phase.

A Phase 1 clinical trial at Cedars-Sinai was funded to investigate the safety and efficacy of ways in which surviving neurons can be protected in people with ALS.

The second, a Phase 3 clinical trial at Brainstorm Cell Therapeutics, first began in Israel. The approach is to use mesenchymal stem cells derived from bone marrow boosted with protective factors to support and protect the neurons of ALS patients.

Stacey Dorenfeld Agoura Hills

Dorenfeld is the National State Advocacy co-chair and the Hadassah Southern California advocacy chair.

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Stem cells key to ALS therapy - Agoura Hills Acorn

Development and validation of a novel stem cell subtype for bladder cancer based on stem genomic profiling. – UroToday

Bladder cancer (BLCA) is the fifth most common type of cancer worldwide, with high recurrence and progression rates. Although considerable progress has been made in the treatment of BLCA through accurate typing of molecular characteristics, little is known regarding the various genetic and epigenetic changes that have evolved in stem and progenitor cells. To address this issue, we have developed a novel stem cell typing method.

Based on six published genomic datasets, we used 26 stem cell gene sets to classify each dataset. Unsupervised and supervised machine learning methods were used to perform the classification.

We classified BLCA into three subtypes-high stem cell enrichment (SCE_H), medium stem cell enrichment (SCE_M), and low stem cell enrichment (SCE_L)-based on multiple cross-platform datasets. The stability and reliability of the classification were verified. Compared with the other subtypes, SCE_H had the highest degree of cancer stem cell concentration, highest level of immune cell infiltration, and highest sensitivity not only to predicted anti-PD-1 immunosuppressive therapy but also to conventional chemotherapeutic agents such as cisplatin, sunitinib, and vinblastine; however, this group had the worst prognosis. Comparison of gene set enrichment analysis results for pathway enrichment of various subtypes reveals that the SCE_H subtype activates the important pathways regulating cancer occurrence, development, and even poor prognosis, including epithelial-mesenchymal transition, hypoxia, angiogenesis, KRAS signal upregulation, interleukin 6-mediated JAK-STAT signaling pathway, and inflammatory response. Two identified pairs of transcription factors, GRHL2 and GATA6 and IRF5 and GATA3, possibly have opposite regulatory effects on SCE_H and SCE_L, respectively.

The identification of BLCA subtypes based on cancer stem cell gene sets revealed the complex mechanism of carcinogenesis of BLCA and provides a new direction for the diagnosis and treatment of BLCA.

Stem cell research & therapy. 2020 Oct 28*** epublish ***

Chaozhi Tang, Jiakang Ma, Xiuli Liu, Zhengchun Liu

Department of Urology, The First Affiliated Hospital of China Medical University, Shenyang, 110001, China., Department of Oncology, The Second Affiliated Hospital of Zhengzhou University, Zhengzhou, 450014, China., Department of Oncology, Affiliated Hospital of Guilin Medical University, Guilin, 541001, China., Department of Radiation Oncology, Affiliated Hospital of Guilin Medical University, Guilin, 541001, China. .

PubMed http://www.ncbi.nlm.nih.gov/pubmed/33115513

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Development and validation of a novel stem cell subtype for bladder cancer based on stem genomic profiling. - UroToday

New-onset Post-transplant Diabetes and Therapy in Long-term Survivors After Allogeneic Hematopoietic Stem Cell Transplantation – DocWire News

This article was originally published here

In Vivo. 2020 Nov-Dec;34(6):3545-3549. doi: 10.21873/invivo.12197.

ABSTRACT

BACKGROUND: Survival after allogeneic hematopoietic stem cell transplantation (allo-HSCT) has increased but so have long-term sequelae. New-onset post-transplant diabetes mellitus (PTDM) occurs frequently following allo-HSCT.

PATIENTS AND METHODS: Study endpoints were incidence and risk factors of PDTM. We studied 599 adult patients suffering from either acute myeloid leukemia n=220), acute lymphoblastic leukemia (n=79), chronic myeloid leukemia (n=22), myelodysplastic syndrome/myeloproliferative neoplasm (n=105), chronic lymphocytic leukemia (n=37), lymphoma/myeloma (n=116, or non-malignant disorders (e.g. bone marrow failure, hemoglobinopathies) (n=20) who underwent myeloablative (466; 77.8%) or non-myeloablative (131; 21.9%) allo-HSCT between 2006 and 2016.

RESULTS: Altogether, 39 patients (6.5%) developed PTDM. In a competing-risk analysis, time to PTDM was associated with acute grade 2-4 graft-versus-host-disease (p=0.017). Further cardiovascular risk factors were hypertension (n=145; 24.2%), coronary artery disease (n=36, 6%), dyslipidemia (n=139; 23.3%), and stroke (n=12; 2%).

CONCLUSION: After allo-HSCT, a significant number of patients developed PTDM and patients with acute graft-versus-host-disease were found to have a higher risk for PTDM. Long-term and continuous follow-up for diabetes and cardiovascular risk factors after HSCT is important in order to be able to provide timely and appropriate treatment.

PMID:33144466 | DOI:10.21873/invivo.12197

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New-onset Post-transplant Diabetes and Therapy in Long-term Survivors After Allogeneic Hematopoietic Stem Cell Transplantation - DocWire News