Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy – GlobeNewswire

July 09, 2020 08:00 ET | Source: Fate Therapeutics, Inc.

FT819 CAR T-cell Product Candidate Derived from Clonal Master iPSC Line with Novel CD19-specific 1XX CAR Integrated into TRAC Locus

Phase 1 Clinical Study will Evaluate FT819 for Patients with Advanced B-cell Leukemias and Lymphomas

SAN DIEGO, July 09, 2020 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for FT819, an off-the-shelf allogeneic chimeric antigen receptor (CAR) T-cell therapy targeting CD19+ malignancies. FT819 is the first-ever CAR T-cell therapy derived from a clonal master induced pluripotent stem cell (iPSC) line, and is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy. The Company plans to initiateclinical investigation of FT819for the treatment of patients with relapsed / refractory B-cell malignancies, including chronic lymphocytic leukemia (CLL), acute lymphoblastic leukemia (ALL), and non-Hodgkin lymphoma (NHL).

The clearance of our IND application for FT819 is a ground-breaking milestone in the field of cell-based cancer immunotherapy. Our unique ability to produce CAR T cells from a clonal master engineered iPSC line creates a pathway for more patients to gain timely access to therapies with curative potential, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. Four years ago, we first set out under our partnership with Memorial Sloan Kettering led by Dr. Michel Sadelain to improve on the revolutionary success of patient-derived CAR T-cell therapy and bring an off-the-shelf paradigm to patients, and we are very excited to advance FT819 into clinical development.

FT819 was designed to specifically address several limitations associated with the current generation of patient- and donor-derived CAR T-cell therapies. Under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering, and Head, Gene Expression and Gene Transfer Laboratory at MSK, the Company incorporated several first-of-kind features into FT819 including:

The multi-center Phase 1 clinical trial of FT819 is designed to determine the maximum tolerated dose of FT819 and assess its safety and clinical activity in up to 297 adult patients across three types of B-cell malignancies (CLL, ALL, and NHL). Each indication will enroll independently and evaluate three dose-escalating treatment regimens: Regimen A as a single dose of FT819; Regimen B as a single dose of FT819 with IL-2 cytokine support; and Regimen C as three fractionated doses of FT819. For each indication and regimen, dose-expansion cohorts of up to 15 patients may be enrolled to further evaluate the clinical activity of FT819.

At the American Association for Cancer Research (AACR) Virtual 2020 Meeting, the Company presented preclinical data demonstrating FT819 is comprised of CD8 T cells with uniform 1XX CAR expression and complete elimination of endogenous TCR expression. Additionally, data from functional assessments showed FT819 has antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines that is comparable to that of healthy donor-derived CAR T cells, and persists and maintains tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia.

Fate Therapeutics has an exclusive license for all human therapeutic use to U.S. Patent No. 10,370,452 pursuant to its license agreement with MSK1, which patent covers compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an iPSC. In addition to the patent rights licensed from MSK, the Company owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control.

1 Fate Therapeutics haslicensedintellectual propertyfrom MSK on which Dr. Sadelain is aninventor.As a result of the licensing arrangement, MSK has financial interests related to Fate Therapeutics.

About Fate Therapeutics iPSC Product Platform The Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

About Fate Therapeutics, Inc. Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Companys immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking Statements This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of and plans related to the Company's product candidates and clinical studies, the Companys progress, plans and timelines for the clinical investigation of its product candidates, the therapeutic potential of the Companys product candidates including FT819, and the Companys clinical development strategy for FT819. These and any other forward-looking statements in this release are based on management's 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, the risk of difficulties or delay in the initiation of any planned clinical studies, or in the enrollment or evaluation of subjects in any ongoing or future clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that results observed in preclinical studies of FT819 may not be replicated in ongoing or future clinical trials or studies, and the risk that FT819 may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact: Christina Tartaglia Stern Investor Relations, Inc. 212.362.1200 christina@sternir.com

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Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy - GlobeNewswire

Growing Focus on Product Innovation Likely to Impact the Growth of the Amniotic Fluid Stem Cell Therapy Market 2018 2026 – Jewish Life News

A new intelligence report Amniotic Fluid Stem Cell Therapy Market has been recently added to Adroit Market Research collection of top-line market research reports. Global Amniotic Fluid Stem Cell Therapy Market report is a meticulous all-inclusive analysis of the market that offers access to direct first-hand insights on the growth trail of market in near term and long term. On the basis of factual information sourced from authentic industry experts and extensive primary industry research, the report offers insights on the historic growth pattern of Amniotic Fluid Stem Cell Therapy Market and current market scenario. It then provides short- and long-term market growth projections.

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Amniotic Fluid Stem Cell Therapy Market Table of Contents

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Growing Focus on Product Innovation Likely to Impact the Growth of the Amniotic Fluid Stem Cell Therapy Market 2018 2026 - Jewish Life News

Rheumatoid Arthritis Stem Cell Therapy Market Trends, Outlook and Opportunity Analysis 2018 to 2028 – Cole of Duty

Global Rheumatoid Arthritis Stem Cell Therapy Market Growth Projection

The new report on the global Rheumatoid Arthritis Stem Cell Therapy market is an extensive study on the overall prospects of the Rheumatoid Arthritis Stem Cell Therapy market over the assessment period. Further, the report provides a thorough understanding of the key dynamics of the Rheumatoid Arthritis Stem Cell Therapy market including the current trends, opportunities, drivers, and restraints. The report introspects the micro and macro-economic factors that are expected to nurture the growth of the Rheumatoid Arthritis Stem Cell Therapy market in the upcoming years.

The report suggests that the global Rheumatoid Arthritis Stem Cell Therapy market is projected to reach a value of ~US$XX by the end of 2029 and grow at a CAGR of ~XX% through the forecast period (2019-2029). The key indicators such as the year-on-year (Y-o-Y) growth and CAGR growth of the Rheumatoid Arthritis Stem Cell Therapy market are discussed in detail in the presented report. This data is likely to provide readers an understanding of qualitative and quantitative growth prospects of the Rheumatoid Arthritis Stem Cell Therapy market over the considered assessment period.

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Rheumatoid Arthritis Stem Cell Therapy Market Trends, Outlook and Opportunity Analysis 2018 to 2028 - Cole of Duty

Ziopharm Oncology Announces Initiation of Phase 1 Trial Evaluating Rapid Personalized Manufacturing CAR-T Technology in Patients with Relapsed CD19+…

July 09, 2020 16:15 ET | Source: ZIOPHARM Oncology Inc

BOSTON, July 09, 2020 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (Ziopharm or the Company) (Nasdaq:ZIOP), today announced the initiation of a phase 1 clinical trial to evaluate CD19-specific CAR-T, using its Rapid Personalized Manufacturing (RPM) technology, as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas. The trial is now open for enrollment at The University of Texas MD Anderson Cancer Center.

In this trial, the Company utilizes its non-viral Sleeping Beauty genetic engineering technology to infuse CAR-T the day after electroporation. Ziopharms RPM CD19-specific CAR-T therapy results from the stable, non-viral insertion of DNA into the genome of resting T cells to co-express the chimeric antigen receptor (CAR), membrane-bound IL-15 (mbIL15) and a safety switch.

We are pleased to expand the scope of our clinical development with MD Anderson, as we seek to evaluate our RPM technology using CD19-specific CAR-T cells, said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. RPM is a promising manufacturing solution, as T cells from the bloodstream are genetically reprogramed with DNA plasmids from the Sleeping Beauty system and then simply administered the next day.

Our CAR-T therapy can be administered at low cell doses, which may control cytokine release syndrome and is appealing for the treatment of patients including those with CD19-expressing malignancies that have relapsed after allogeneic bone marrow transplantation (BMT). There are limited effective treatment options for such patients as evidenced by the low rate of remission and poor long-term survival, Dr. Cooper added.

Up to 24 patients with advanced CD19+ leukemias and lymphomas who have relapsed after allogeneic BMT will be enrolled in this investigator-initiated trial (NCT03579888). The primary endpoint of the study is to determine the safety and maximum tolerated dose of donor-derived genetically modified CD19-specific T cells manufactured using the RPM process. An additional study is planned through Ziopharms joint venture with Eden BioCell to evaluate the RPM technology using patient-derived (autologous) CD19-specific CAR-T in Greater China.

Research reveals three-year survival for adults with CD19+ acute lymphoblastic leukemia after allogeneic BMT ranges from 30% to 65%.1 For patients with other CD19+ cancers, allogeneic BMT can provide three-year survival rates between 30% to 75%.1 Few patients experience a durable remission following allogeneic BMT, regardless of the treatment modality, with some having a median survival of only 2 to 3 months.2

About Ziopharm Oncology, Inc. Ziopharm is developing non-viral and cytokine-driven cell and gene therapies that weaponize the bodys immune system to treat the millions of people globally diagnosed with a solid tumor each year. With its multiplatform approach, Ziopharm is at the forefront of immuno-oncology with a goal to treat any type of solid tumor. Ziopharms pipeline is built for commercially scalable, cost effective T-cell receptor T-cell therapies based on its non-viral Sleeping Beauty gene transfer platform, a precisely controlled IL-12 gene therapy, and rapidly manufactured Sleeping Beauty-enabled CD19-specific CAR-T program. The Company has clinical and strategic collaborations with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and Regeneron Pharmaceuticals. For more information, please visit http://www.ziopharm.com.

Forward-Looking Statements Disclaimer This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the progress, design and timing of the Company's research and development programs, the potential benefits of the Companys therapies, and the Companys expectations regarding the number of patients in its clinical trials. Although Ziopharms management team believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Ziopharm, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, changes in our operating plans that may impact our cash expenditures, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Ziopharms product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Ziopharms intellectual property rights; competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Ziopharm, including those risks and uncertainties listed in Ziopharms Quarterly Report on Form 10-Q filed by Ziopharm with the Securities and Exchange Commission. We are providing this information as of the date of this press release, and Ziopharm does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason.

Investor Relations Contacts:Ziopharm Oncology: Chris Taylor VP, Investor Relations and Corporate Communications T: 617.502.1881 E: ctaylor@ziopharm.com

LifeSci Advisors: Mike Moyer Managing Director T: 617.308.4306 E: mmoyer@lifesciadvisors.com

Media Relations Contact:LifeSci Communications: Patrick Bursey T: 646.876.4932 E: pbursey@lifescicomms.com

1 D'Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2018. Available at https://www.cibmtr.org

2 Keil F, Prinz E, Kalhs P, et al. Treatment of leukemic relapse after allogeneic stem cell transplantation with cytotoreductive chemotherapy and/or immunotherapy or second transplants. Leukemia 2001; 15:355-361.

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Ziopharm Oncology Announces Initiation of Phase 1 Trial Evaluating Rapid Personalized Manufacturing CAR-T Technology in Patients with Relapsed CD19+...

UK researchers use mini retinas to test gene therapy for RP – AOP

Scientists report promising results from a study that aims to develop a new approach to treating the most common inherited eye condition

Pixabay/Arek Socha

The study, which was published in Stem Cell Reports, involved using a viral vector to carry a replacement gene to malfunctioning cells in retinal organoids.

Following treatment, analysis revealed that the mini retinas had begun to produce a protein that is essential for vision.

Professor Mike Cheetham, from UCL, helped to develop the mini retinas that were used to test the gene therapy.

He highlighted that the mini retinas allow researchers to reproduce many different elements of inherited disease.

It makes it possible for us to study in detail why people go blind and try to find ways to prevent blindness. Its exciting that the gene therapy seems to be so effective for this form of retinitis pigmentosa, Professor Cheetham said.

The research was part-funded by Fight for Sight. The charitys chief executive, Sherine Krause, described the findings as incredibly promising.

Professor Cheetham and his teams work in collaboration with Trinity College Dublin represents a significant breakthrough for eye research and shows the importance of science to find new treatments for the prevailing causes of sight loss, she said.

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UK researchers use mini retinas to test gene therapy for RP - AOP

Stem Cell Therapy Market 2020-2029 Size, Share and Trends By Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell -…

The Global Stem Cell Therapy Market study report presents an in depth study about the market on the basis of key segments such as product type, application, key companies and key regions, end users and others. Thus the study report offers the comprehensive analysis of market size across the globe as regional and country level market size analysis, estimation of market growth during the forecast period, competitive landscape and sales analysis. On the basis of historic data and current data available, research report offers comprehensive and accurate study of Stem Cell Therapy market. The global Stem Cell Therapy market was valued at $XX million in 2019, and analysts predict the global market size will reach $XX million by the end of 2029, growing at a CAGR of XX% between 2019 and 2029.

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Since the COVID-19 virus outbreak in December 2019, the disease has spread to over 210 countries and territories around the world and 2 international conveyances. The global impacts of COVID-19 are already starting to be felt, and will significantly affect this industry in 2020. This report analyses the impact of COVID-19 on this industry. COVID-19 can affect the global market in 3 ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on enterprises and financial markets. The research report of global Stem Cell Therapy market provides the information about the top most manufacturers which are presently functioning in this industry and which have good market region wise. This section is important as it sheds light on the sales growth of various country level and regional level Stem Cell Therapy market. The end users of the global Stem Cell Therapy market can be categorized on the basis of size of the enterprise. As the industry analysts estimates and extracts the data which are affecting the growth of market for the estimated forecast period.

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Stem Cell Therapy Market Segmentation by Type:

Autologous Allogeneic

Stem Cell Therapy Market Segmentation by Application:

Musculoskeletal Disorder Wounds & Injuries Cornea Cardiovascular Diseases Others

Market segment by Region/Country including: North America (United States, Canada and Mexico) Europe (Germany, UK, France, Italy, Russia and Spain etc.) Asia-Pacific (China, Japan, Korea, India, Australia and Southeast Asia etc.) South America Brazil, Argentina, Colombia and Chile etc.) Middle East & Africa (South Africa, Egypt, Nigeria and Saudi Arabia etc.)

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Stem Cell Therapy Market 2020-2029 Size, Share and Trends By Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell -...

Lysosomal Disease Treatment Market 2020 Size By Product Types, End-Users, Regional Outlook, Growth Potential, Price Trends And Forecast To 2026 |…

The Global Lysosomal Disease Treatment Market report by IndustryGrowthInsights.com provides a detailed analysis of the area marketplace expanding; competitive landscape; global, regional, and country-level market size; impact market players; market growth analysis; market share; opportunities analysis; product launches; recent developments; sales analysis; segmentation growth; technological innovations; and value chain optimization. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report.

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Market Segmentation

The Global Lysosomal Disease Treatment Market has been divided into product types, application, and regions. These segments provide accurate calculations and forecasts for sales in terms of volume and value. This analysis can help customers increase their business and take calculated decisions.

By Product Types, Hematopoietic Stem Cell Transplantation Enzyme Replacement Therapy Substrate Reduction Chaperone Therapies Lysosomal Disease Treatmen

By Applications, Hospitals Clinics Stem Cell Transplant Center Research Organizations Others

By Regions and Countries, Asia Pacific: China, Japan, India, and Rest of Asia Pacific Europe: Germany, the UK, France, and Rest of Europe North America: The US, Mexico, and Canada Latin America: Brazil and Rest of Latin America Middle East & Africa: GCC Countries and Rest of Middle East & Africa

The regional analysis segment is a highly comprehensive part of the report on the global Lysosomal Disease Treatment market. This section offers information on the sales growth in these regions on a country-level Lysosomal Disease Treatment market.

The historical and forecast information provided in the report span between 2018 and 2026. The report provides detailed volume analysis and region-wise market size analysis of the market.

Competitive Landscape of the Lysosomal Disease Treatment Market

The chapter on competitive landscape provides information about key company overview, global presence, sales and revenue generated, market share, prices, and strategies used.

Major players in the global Lysosomal Disease Treatment Market include Astellas Pharma Astrazeneca Actelion Pharmaceuticals Ltd. Eli Lilly and Co. Merck & Co. Novo Nordisk A/S Shire Pfizer Sanofi BioMarin Lysosomal Disease Treatmen

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Lysosomal Disease Treatment Market 2020 Size By Product Types, End-Users, Regional Outlook, Growth Potential, Price Trends And Forecast To 2026 |...

Stem Cell Therapy Market Outlook, Opportunity, Demand Analysis And Forecast 2020 2026 – Owned

New Jersey, United States,- Latest update on Stem Cell Therapy Market Analysis report published with extensive market research, Stem Cell Therapy Market growth analysis, and forecast by 2026. this report is highly predictive as it holds the overall market analysis of topmost companies into the Stem Cell Therapy industry. With the classified Stem Cell Therapy market research based on various growing regions, this report provides leading players portfolio along with sales, growth, market share, and so on.

The research report of the Stem Cell Therapy market is predicted to accrue a significant remuneration portfolio by the end of the predicted time period. It includes parameters with respect to the Stem Cell Therapy market dynamics incorporating varied driving forces affecting the commercialization graph of this business vertical and risks prevailing in the sphere. In addition, it also speaks about the Stem Cell Therapy Market growth opportunities in the industry.

Stem Cell Therapy Market Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution etc., these data help the consumer know about the competitors better. This report also covers all the regions and countries of the world, which shows a regional development status, including Stem Cell Therapy market size, volume and value, as well as price data.

Stem Cell Therapy Market competition by top Manufacturers:

Stem Cell Therapy Market Classification by Types:

Bytype1

Stem Cell Therapy Market Size by End-user Application:

Byapplication1

Listing a few pointers from the report:

Objective of Stem Cell Therapy Market Report:

Cataloging the competitive terrain of the Stem Cell Therapy market:

Unveiling the geographical penetration of the Stem Cell Therapy market:

The report of the Stem Cell Therapy market is an in-depth analysis of the business vertical projected to record a commendable annual growth rate over the estimated time period. It also comprises of a precise evaluation of the dynamics related to this marketplace. The purpose of the Stem Cell Therapy Market report is to provide important information related to the industry deliverables such as market size, valuation forecast, sales volume, etc.

Major Highlights from Table of contents are listed below for quick look up into Stem Cell Therapy Market report

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Stem Cell Therapy Market Outlook, Opportunity, Demand Analysis And Forecast 2020 2026 - Owned

Hematopoietic Stem Cells Transplantation (HSCT) Market: Future Innovation Ways That, Growth & Profit Analysis, Forecast By 2026 – 3rd Watch News

Hematopoietic Stem Cells Transplantation (HSCT) Marketreport provides in-depth COVID19 impact analysis ofMarket Overview, Product Scope, Market Drivers, Trends, Opportunities,Market Driving Force and Market Risks. It also profile the topmost prime manufacturers (Kite Pharma, Thermo Fisher Scientific, CellGenix Technologie Transfer, Cesca Therapeutics, R&D Systems) are analyzed emphatically by competitive landscape contrast, with respect toPrice, Sales,Capacity, Import, Export, Consumption, Gross, Gross Margin, Revenue and Market Share. Hematopoietic Stem Cells Transplantation (HSCT) industry breakdown data are shown at the regional level, to show the sales, revenue and growth by regions.Hematopoietic Stem Cells Transplantation (HSCT) Market describe Hematopoietic Stem Cells Transplantation (HSCT) Sales Channel,Distributors, Customers, Research Findings and Conclusion, Appendix and Data Source.

Key Target Audience of Hematopoietic Stem Cells Transplantation (HSCT) Market:Manufacturers of Hematopoietic Stem Cells Transplantation (HSCT), Raw material suppliers, Market research and consulting firms, Government bodies such as regulating authorities and policy makers, Organizations, forums and alliances related to Hematopoietic Stem Cells Transplantation (HSCT) market.

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In-Depth Qualitative Analyses Include Identification and Investigation Of The Following Aspects:Hematopoietic Stem Cells Transplantation (HSCT) Market Structure, Growth Drivers, Restraints and Challenges, Emerging Product Trends & Market Opportunities, Porters Fiver Forces.

Summary of Hematopoietic Stem Cells Transplantation (HSCT) Market:Hematopoietic stem cell transplants (HSCT) present to a valid treatment for several congenital and other hematopoietic system disorders, post chemotherapy, and immune sensitive diseases. HSCT is also preferred for replacement of cellular components and deficient cells. The indications for HSCT thus are wide; the most frequent indication as per reported by Worldwide Network for Blood and Marrow Transplantation Group (WNBT) (2013) is lymphoproliferative disorder (53.2% of all HSCT), 12% of whom received allogeneic and the rest received autologous transplant. Plasma cell disorders are the most frequent indication in this group. A multitude of literature published by researchers and organizations demonstrate that autologous transplant own a greater edge against allogeneic HSCT.

Over 30 years of studies in the field of blood-forming stem cells i.e. hematopoietic stem cells (HSC), researchers have developed significant understanding to use HSCs as a therapy. At present, no type of stem cell, adult, embryonic or fetal has attained such sufficient status. Hematopoietic stem cell transplantation (HSCT) is now routinely used for treating patients with malignant and non-malignant disorders of blood and the immune system. Currently, researchers have observed that through animal studies HSCs have the ability to form other cells such as blood vessels, muscles, and bone. Further application of this approach it may eventually be able to treat a wide array of conditions and replace ailing tissues. However, despite the vast experience with HSCs, researchers face major barriers in expanding their use beyond the replacement of immune and blood cells.

Hematopoietic stem cells are unable to proliferate and differentiate in-vitro. Researchers have yet to evolve an accurate method to differentiate stem cells from other cells derived from blood or bone marrow. Once such technical barriers are overcome, the avenues for realizing the full potential of HSCT. The type of transplant a person receives depends on several different factors, including the type and course of the disease, availability of suitable donors, and the patients overall health. There are three different sources of hematopoietic stem cells such as bone marrow, peripheral blood stem cells, and umbilical cord blood. The stem cell source used for a given transplant depends upon the underlying disease, the type of transplant (allogeneic or autologous), and size of the patient.

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate of Hematopoietic Stem Cells Transplantation (HSCT) market foreach application, including-

Leukemia Lymphoproliferative Disorders Solid Tumors Non-Malignant Disorders Others

On the basis of product,this report displays the sales volume, revenue (Million USD), product price, market share and growth rate ofeach type, primarily split into-

Autologous Transplant Allogenic Transplant

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Hematopoietic Stem Cells Transplantation (HSCT) Market: Future Innovation Ways That, Growth & Profit Analysis, Forecast By 2026 - 3rd Watch News

Vor bags $110M to move engineered cell therapy into the clinic – FierceBiotech

CAR-T treatments and other immunotherapies have changed the treatment of some blood cancers, but they can target healthy cells as well as cancer cells, causing nasty side effects. Vor Biopharma is working on an engineered stem cell solution, and its raised $110 million to move its lead program into the clinic.

The Cambridge, Massachusetts-based company is developing the treatment, VOR33, for patients with acute myeloid leukemia (AML) whose disease has worsened despite undergoing chemotherapy or a stem cell transplant.

Thats the setting in which a lot of targeted agents are used. The trouble is, a great number of the targeted agents tend to failnot because they are not efficacious, but because the drug is too toxic for the patients bone marrow, Vor CEO Robert Ang told Fierce Biotech.

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The drugs home in on healthy cells as well as cancerous ones because they express the same proteins. This leads to myelosuppression, which means the bone marrow doesnt make enough white blood cells, platelets or red blood cells for the patient to survive, Ang said.

RELATED: Neon CBO Robert Ang jumps ship to take the helm at Vor Biopharma

Vors treatment is made from hematopoietic, or blood-forming, stem cells from healthy donors. The company uses gene editing to get rid of cancer drug targets in those cells that are biologically redundant, which means deleting them doesnt cause any harm. That target is CD33, in the case of VOR33.

Were trying to make the marrow treatment resistant such that the only cells that are expressing CD33 should be cancer cells, Ang said. We should be able to target them much more specifically, while minimizing the collateral damage that typically happens with these drugs.

RELATED: 5AM, JJDC get in on $42M series A round for cell therapy player Vor Biopharma

Vor believes its treatments could boost the reach of targeted therapies by improving their efficacy and increasing the amount of time patients can undergo those treatments.

And thats not all. In addition to protecting these transplants and the blood cells they produce from targeted drugs, Vor thinks its approach could change the way we think about bone marrow transplant.

To some degree, transplants have been relatively decentralized and less controlled A lot of hospitals develop their own unique practices as to what they think works and how to handle cells and process them, Ang said. Our product will be regulated by the FDA, so we will be able to provide controls and the proper manufacturing steps to ensure were making the best quality product for patients.

The series B will push VOR33 into clinical trials in the first half of 2021, a target the companys on track to meet despite the COVID-19 pandemic. And Vor plans to expand its portfolio beyond CD33, starting with an umbrella of targets in the myeloid space, namely in acute myeloid leukemia, myelodysplastic syndromes and related diseases.

But we are also looking beyond that to other cancers where there are similar potentially biologically redundant targets we could pursue, said Ang, who took the companys helm in August 2019.

Since then, Vor has grown from a staff of six to 50, and its about to move into new digs in west Cambridge as it moves VOR33 toward the clinic. Its got the backing of RA Capital Management, Fidelity, 5AM Ventures, Johnson & Johnson Innovation, Osage University Partners, PureTech Health, the Pagliuca Family Office and Alexandria Venture Investments to do it all.

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Vor bags $110M to move engineered cell therapy into the clinic - FierceBiotech