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Stem Cell Cryopreservation Equipments Market Size Report: Strategies of Key Manufacturers, Project Investment of New Industries: Chart, Worthington…

By Stell Cell Research

TheStem Cell Cryopreservation Equipments Marketincludes numbers for all the segments at the country level for past, current and coming years. This report provides comprehensive market information through detailed segmentation along with segmental market size and forecasts, growth rates, market dynamics, structure, it also provides an overview of the development of the industry, market situation, and trends.

The report covers a detailed analysis of players and market strategies, a detailed analysis of growth factors that are critical for existing market participants as well as new market players. It provides information on new market opportunities and the future with main drivers and restraints of the market to support the decision in a cost-effective business solution.

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The report introduces Stem Cell Cryopreservation Equipments basics:

Definitions, categories, software, and market reviews; Product Features; Manufacturing process; Cost arrangement, development, and its aftermath. Subsequently, it studied the requirements of the international major Stem Cell Cryopreservation Equipments industry market, for example, the price, profit, capacity, production, distribution, market and demand growth and forecasting of goods, etc. In the long run, the report introduced a new Stem Cell Cryopreservation Equipments SWOT analysis. Feasibility, and revenue investigation.

Stem Cell Cryopreservation Equipments Report by Type, Application with Geography The International Forecast for 2026 is actually an efficient and comprehensive research study on the worlds major regional economy states, focusing on key areas such as the United States, Canada Does, Germany, UK, France, Italy, Spain, Russia, Netherlands, Turkey, Switzerland, Sweden, Poland, Belgium, China, Japan, South Korea, Australia, India, Taiwan, Indonesia, Thailand, Philippines, Malaysia, Brazil, Mexico, Argentina, Colombia, Chile, Saudi Arabia, UAE, Egypt, Nigeria, South Africa and Rest of the World.

This Report covers Leading Companies associated in Stem Cell Cryopreservation Equipments:Chart, Worthington Industries, Cesca Therapeutics, Shengjie Cryogenic Equipment, Sichuan Mountain Vertical, Qingdao Beo

Importance of this Stem Cell Cryopreservation Equipments

1. Industry Synopsis of the International Stem Cell Cryopreservation Equipments Market; 2. Global Stem Cell Cryopreservation Equipments Market Company Manufacturer Overview and Profile; 3. Technical data and economy manufacturing plants; 4. Capacity, revenue, and production analysis; 5. Region, Manufacturers and Types by Cost, Price, Gross, and Gross Fiscal Analysis Stem Cell Cryopreservation Equipments; 6. Sales price analysis of Stem Cell Cryopreservation Equipments business share by volume, price, and valuation of consumption, type, and software; 7. Supply, Import, Export, and Presence Analysis of Stem Cell Cryopreservation Equipments Market; 8. Significant manufacturer analysis of Stem Cell Cryopreservation Equipments market size; 9. Marketing Trader or Distributor Analysis; 10. Industry Chain Analysis; 11. Development Trend Analysis of Stem Cell Cryopreservation Equipments Market Trends; 12. Stem Cell Cryopreservation Equipmentss new project feasibility analysis

By Types

Liquid Phase, Vapor Phas

By Application

Cord Blood Stem Cell Cryopreservation, Other Stem Cell Cryopreservatio

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General studies include Stem Cell Cryopreservation Equipments value chain analysis that provides an understanding of key players from the distribution chain, particularly from manufacturers to end-users. In addition, the report supplies the Stem Cell Cryopreservation Equipments International Economic Competition with the help of Porters analysis of the five forces.

Also, Global Stem Cell Cryopreservation Equipments Market includes the following features with detailed study of each

Key Players: The report provides a company outline for a good number of the leading players of the global Stem Cell Cryopreservation Equipments market. It brings to light their current and future Stem Cell Cryopreservation Equipments market development taking into account their price, gross margin, revenue, production, service sectors, production sites, and other factors.

Stem Cell Cryopreservation Equipments Market Dynamics: The report shares important information on impact factors, Stem Cell Cryopreservation Equipments market drivers, challenges, opportunities, and market trends as part of market dynamics.

Global Stem Cell Cryopreservation Equipments Market Forecast: Readers are provided with production and revenue forecasts for the Stem Cell Cryopreservation Equipments market, production and consumption forecasts for regional markets, production, revenue, and price forecasts for the Stem Cell Cryopreservation Equipments market, and consumption forecasts for the industry by the application.

Regional Stem Cell Cryopreservation Equipments Market Analysis: It can be divided into two different classes one for regional production analysis and the other for regional consumption analysis. Here, analysts share gross margin, price, revenue, production, CAGR, and other factors that indicate the growth of all the regional markets studied in the report.

Stem Cell Cryopreservation Equipments Market Competition: In this section, the report provides information about competitive conditions and trends including mergers and acquisitions and expansions, market shares of three or five players, and market concentration rates. Readers may also be provided by manufacturers with production, revenue, and average price shares.

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Stem Cell Cryopreservation Equipments Market Size Report: Strategies of Key Manufacturers, Project Investment of New Industries: Chart, Worthington...

Seattle Cancer Care Alliance Celebrates 20 Years of Innovation in Cancer Treatment and Care – Business Wire

By Stell Cell Research

SEATTLE--(BUSINESS WIRE)--Seattle Cancer Care Alliance (SCCA) today marked the 20th anniversary of opening the doors of its first clinic in South Lake Union. Established in 2001 to bring together leading research teams and cancer specialists from Fred Hutchinson Cancer Research Center, Seattle Children's and University of Washington Medicine, SCCA began with a singular mission that remains its focus today: the pursuit of better, longer, richer lives for cancer patients. In the last two decades, its teams have served nearly 190,000 cancer patients and conducted more than 1,700 clinical trials, advancing cancer care not only in the Pacific Northwest but also throughout the nation.

SCCA was founded with the unique vision that combining the best in cancer care and research will change the lives of those with cancer, said Dr. Nancy E. Davidson, president and executive director at SCCA and senior vice president of the Clinical Research Division at Fred Hutch. Throughout the past 20 years, our organization has proven the power of this vision. Our teams have helped drive new advances in the delivery of cancer care as well as the development of cutting-edge new therapies and technologies that have improved how we screen for, diagnose and treat many cancers as well as how we support cancer patients and their loved ones. And while we are proud of what we have accomplished the last 20 years, SCCA is ready to continue to build on this foundation for decades to come.

SCCA opened its first outpatient clinic in Seattles South Lake Union neighborhood in 2001. It has grown from a single location to a network of treatment centers in the Puget Sound region encompassing hematology/medical oncology, radiation oncology and infusion outpatient services. It also has network affiliations with hospitals in five states, connecting community-based physicians with the latest cancer research and treatment options from SCCA to elevate cancer care in the community.

As part of the only National Cancer Institute (NCI)-designated cancer center in Washington state, SCCA has helped to advance cancer research, establish new models of cancer care, connect more cancer patients to clinical trials, and enhance patients access to the best possible cancer care and treatment.

Putting patients first is paramount to everything we do at SCCA. And its because of the commitment and passion of our staff from physicians and nurses to pharmacists and administrative support staff that we have been able to succeed and deliver on that promise, said Aaron Crane, executive vice president at SCCA. Their work has driven SCCA to new heights in cancer care and allowed us to achieve significant growth. With the expected opening of our South Lake Union clinic expansion in 2023, we will be able to conduct more cutting-edge research and provide even more patients world-class cancer care.

SCCA alliance member Fred Hutch is known for its pioneering work in bone marrow (BMT) and stem cell transplantation. Stemming from this legacy work, SCCA is continually recognized for its patients survival rates and has now performed more than 9,000 transplants since 2001, making it among the most experienced cancer centers in this field in the world. SCCA is also noted for its leadership in immunotherapy. In 2016, it opened the Bezos Family Immunotherapy Clinic to support its growing work in this field. This clinic, which has now treated more than 500 patients, has increased the number of clinical trials offering cellular immunotherapies, including CAR-T therapies. SCCA is one of the top providers of CAR-T therapy and among the first cancer centers to offer patients all three FDA-approved cellular immunotherapies.

Through groundbreaking clinical trials, along with alliance partners UW Medicine, Seattle Childrens Hospital and Fred Hutch, SCCA has led the development of new treatment options for patients for a variety of cancers. Over the last two decades, SCCA has opened more than 1,700 trials serving nearly 37,000 participants. A couple of recent examples include a SCCAs trial for a new bladder cancer treatment, which received FDA approval after its results gained international recognition, redefining treatment protocols for that cancer. Another example is a recent clinical trial for lung cancer where SCCA is advancing personalized care through research that assesses the effectiveness of a treatment in real time, allowing providers to give patients treatments based on an individualized care path.

SCCA also has contributed to the development and adoption of molecular testing in cancer care. Its team has expanded known biomarkers that indicate a patients risk for certain cancers including breast, lung and colon cancers and hematologic malignancies as well as determine the best course of treatment. In 2016 and 2018 respectively, SCCA opened the Prostate Cancer Genetics and Hematologic Malignancy Genetics Clinics, which offer genetic testing, counseling, and consultations to help patients better understand their genetic risks and manage their cancers.

SCCA has been a vital part of saving the lives of so many across our region, said Paula Rosput Reynolds, Chair of SCCAs Board of Directors. It has seamlessly combined pioneering research with the best in state-of-the-art clinical patient- and family-centered care. SCCA has made an indelible mark on how many cancers are diagnosed and treated and continues to push boundaries on how cancer care is approached and carried out. We are excited to celebrate its achievements this year and look forward to what is yet to come from this extraordinary organization and its team.

For the past decade, SCCA has also been recognized by U.S. News & World Report as among the Best Cancer Hospitals in the U.S. and the top cancer hospital in the Pacific Northwest.

About Seattle Cancer Care Alliance Seattle Cancer Care Alliance (SCCA) brings together the leading research teams and cancer specialists from Fred Hutch, Seattle Childrens and UW Medicine one extraordinary group whose sole purpose is the pursuit of better, longer, richer lives for our patients. Based in Seattles South Lake Union neighborhood, SCCA is the only National Cancer Institute (NCI)-designated cancer center in Washington state. SCCA has nine clinical care sites in the region, including a medical oncology clinic at EvergreenHealth in Kirkland; hematology/medical oncology and infusion services at Overlake Medical Center in Bellevue, medical and radiation oncology clinics at UW Medical Center - Northwest Seattle and medical oncology services at SCCA Issaquah, as well as Network affiliations with hospitals in five states. For more information about SCCA, visit seattlecca.org.

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Seattle Cancer Care Alliance Celebrates 20 Years of Innovation in Cancer Treatment and Care - Business Wire

Outlook on the CRISPR Gene Editing Global Market to 2030 – Analysis and Forecasts – GlobeNewswire

By Stell Cell Research

February 08, 2021 07:33 ET | Source: Research and Markets

Dublin, Feb. 08, 2021 (GLOBE NEWSWIRE) -- The "Global CRISPR Gene Editing Market: Focus on Products, Applications, End Users, Country Data (16 Countries), and Competitive Landscape - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The global CRISPR gene editing market was valued at $846.2 million in 2019 and is expected to reach $10,825.1 million by 2030, registering a CAGR of 26.86% during the forecast.

The development of genome engineering with potential applications proved to reflect a remarkable impact on the future of the healthcare and life science industry. The high efficiency of the CRISPR-Cas9 system has been demonstrated in various studies for genome editing, which resulted in significant investments within the field of genome engineering. However, there are several limitations, which need consideration before clinical applications. Further, many researchers are working on the limitations of CRISPR gene editing technology for better results. The potential of CRISPR gene editing to alter the human genome and modify the disease conditions is incredible but exists with ethical and social concerns.

The growth is attributed to the increasing demand in the food industry for better products with improved quality and nutrient enrichment and the pharmaceutical industry for targeted treatment for various diseases. Further, the continued significant investments by healthcare companies to meet the industry demand and growing prominence for the gene therapy procedures with less turnaround time are the prominent factors propelling the growth of the global CRISPR gene editing market.

Research organizations, pharmaceutical and biotechnology industries, and institutes are looking for more efficient genome editing technologies to increase the specificity and cost-effectiveness, also to reduce turnaround time and human errors. Further, the evolution of genome editing technologies has enabled wide range of applications in various fields, such as industrial biotech and agricultural research. These advanced methods are simple, super-efficient, cost-effective, provide multiplexing, and high throughput capabilities. The increase in the geriatric population and increasing number of cancer cases, and genetic disorders across the globe are expected to translate into significantly higher demand for CRISPR gene editing market.

Furthermore, the companies are investing huge amounts in the research and development of CRISPR gene editing products, and gene therapies. The clinical trial landscape of various genetic and chronic diseases has been on the rise in recent years, and this will fuel the CRISPR gene editing market in the future.

Within the research report, the market is segmented based on product type, application, end-user, and region. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Key Companies Profiled

Abcam, Inc., Applied StemCell, Inc., Agilent Technologies, Inc., Cellecta, Inc., CRISPR Therapeutics AG, Thermo Fisher Scientific, Inc., GeneCopoeia, Inc., GeneScript Biotech Corporation, Horizon Discovery Group PLC, Integrated DNA Technologies, Inc., Merck KGaA, New England Biolabs, Inc., Origene Technologies, Inc., Rockland Immunochemicals, Inc., Synthego Corporation, System Biosciences LLC, ToolGen, Inc., Takara Bio

Key Questions Answered in this Report:

Key Topics Covered:

1 Technology Definition

2 Research Scope

3 Research Methodology

4 Market Overview 4.1 Introduction 4.2 CRISPR Gene Editing Market Approach 4.3 Milestones in CRISPR Gene Editing 4.4 CRISPR Gene Editing: Delivery Systems 4.5 CRISPR Technology: A Potential Tool for Gene Editing 4.6 CRISPR Gene Editing Current Scenario 4.7 CRISPR Gene Editing Market: Future Potential Application Areas

5 Global CRISPR Gene Editing Market, $Million, 2020-2030 5.1 Pipeline Analysis 5.2 CRISPR Gene Editing Market and Growth Potential, 2020-2030 5.3 Impact of COVID-19 on CRISPR Gene Editing Market 5.3.1 Impact of COVID-19 on Global CRISPR Gene Editing Market Growth Rate 5.3.1. Impact on CRISPR Gene Editing Companies 5.3.2 Clinical Trial Disruptions and Resumptions 5.3.3 Application of CRISPR Gene Editing in COVID-19

6 Market Dynamics 6.1 Impact Analysis 6.2 Market Drivers 6.2.1 Prevalence of Genetic Disorders and Use of Genome Editing 6.2.2 Government and Private Funding 6.2.3 Technology Advancement in CRISPR Gene Editing 6.3 Market Restraints 6.3.1 CRISPR Gene Editing: Off Target Effects and Delivery 6.3.2 Ethical Concerns and Implications With Respect to Human Genome Editing 6.4 Market Opportunities 6.4.1 Expanding Gene and Cell Therapy Area 6.4.2 CRISPR Gene Editing Scope in Agriculture

7 Industry Insights 7.1 Introduction 7.2 Funding Scenario 7.3 Regulatory Scenario of CRISPR Gene Editing Market 7.4 Pricing of CRISPR Gene Editing 7.5 Reimbursement of CRISPR Gene Editing 7.5.1 CRISPR Gene Editing: Insurance Coverage in the U.S.

8 CRISPR Gene Editing Patent Landscape 8.1 Overview 8.2 CRISPR Gene Editing Market Patent Landscape: By Application 8.3 CRISPR Gene Editing Market Patent Landscape: By Region 8.4 CRISPR Gene Editing Market Patent Landscape: By Year

9 Global CRISPR Gene Editing Market (by Product Type), $Million 9.1 Overview 9.2 CRISPR Products 9.2.1 Kits and Enzymes 9.2.1.1 Vector-Based Cas9 9.2.1.2 DNA-Free Cas9 9.2.2 Libraries 9.2.3 Design Tools 9.2.4 Antibodies 9.2.5 Other Products 9.3 CRISPR Services 9.3.1 gRNA Design and Vector Construction 9.3.2 Cell Line and Engineering 9.3.3 Screening Services 9.3.4 Other Services

10 CRISPR Gene Editing Market (by Application), $Million 10.1 Overview 10.2 Agriculture 10.3 Biomedical 10.3.1 Gene Therapy 10.3.2 Drug Discovery 10.3.3 Diagnostics 10.4 Industrial 10.5 Other Applications

11 Global CRISPR Gene Editing Market (by End User) 11.1 Academic Institutions and Research Centers 11.2 Biotechnology Companies 11.3 Contract Research Organizations (CROs) 11.4 Pharmaceutical and Biopharmaceutical Companies

12 Global CRISPR Gene Editing Market (by Region) 12.1 Introduction 12.2 North America 12.3 Europe 12.4 Asia-Pacific 12.5 Latin America

13 Competitive Landscape 13.1 Key Developments and Strategies 13.1.1 Overview 13.1.1.1 Regulatory and Legal Developments 13.1.1.2 Synergistic Activities 13.1.1.3 M&A Activities 13.1.1.4 Funding Activities 13.2 Market Share Analysis 13.3 Growth Share Analysis

14 Company Profiles 14.1 Overview 14.2 Abcam, Inc. 14.2.1 Company Overview 14.2.2 Role of Abcam, Inc. in the Global CRISPR Gene Editing Market 14.2.3 Financials 14.2.4 SWOT Analysis 14.3 Applied StemCell, Inc. 14.3.1 Company Overview 14.3.2 Role of Applied StemCell, Inc. in the Global CRISPR Gene Editing Market 14.3.3 SWOT Analysis 14.4 Agilent Technologies, Inc. 14.4.1 Company Overview 14.4.2 Role of Agilent Technologies, Inc. in the Global CRISPR Gene Editing Market 14.4.3 Financials 14.4.4 R&D Expenditure, 2017-2019 14.4.5 SWOT Analysis 14.5 Cellecta, Inc. 14.5.1 Company Overview 14.5.2 Role of Cellecta, Inc. in the Global CRISPR Gene Editing Market 14.5.3 SWOT Analysis 14.6 CRISPR Therapeutics AG 14.6.1 Company Overview 14.6.2 Role of CRISPR Therapeutics AG in the Global CRISPR Gene Editing Market 14.6.3 Financials 14.6.4 R&D Expenditure, 2017-2019 14.6.5 SWOT Analysis 14.7 Thermo Fisher Scientific, Inc. INC 14.7.1 Company Overview 14.7.2 Role of Thermo Fisher Scientific, Inc. in the Global CRISPR Gene Editing Market 14.7.3 Financials 14.7.4 R&D Expenditure, 2017-2019 14.7.5 SWOT Analysis 14.8 GeneCopoeia, Inc. 14.8.1 Company Overview 14.8.2 Role of GeneCopoeia, Inc. in the Global CRISPR Gene Editing Market 14.8.3 SWOT Analysis 14.9 GeneScript Biotech Corporation 14.9.1 Company Overview 14.9.2 Role of GenScript Biotech in the Global CRISPR Gene Editing Market 14.9.3 Financials 14.9.4 SWOT Analysis 14.1 Horizon Discovery Group PLC 14.10.1 Company Overview 14.10.2 Role of Horizon Discovery Group PLC in the Global CRISPR Gene Editing Market 14.10.3 Financials 14.10.4 SWOT Analysis 14.11 Integrated DNA Technologies, Inc. 14.11.1 Company Overview 14.11.2 Role of Integrated DNA Technologies, Inc. in the Global CRISPR Gene Editing Market 14.11.3 SWOT Analysis 14.12 Merck KGaA 14.12.1 Company Overview 14.12.2 Role of Merck KGaA in the Global CRISPR Gene Editing Market 14.12.3 Financials 14.12.4 SWOT Analysis 14.13 New England Biolabs, Inc. 14.13.1 Company Overview 14.13.2 Role of Integrated DNA Technologies, Inc. in the Global CRISPR Gene Editing Market 14.13.3 SWOT Analysis 14.14 Origene Technologies, Inc. 14.14.1 Company Overview 14.14.2 Role of Origene Technologies, Inc. in the Global CRISPR Gene Editing Market 14.14.3 SWOT Analysis 14.15 Rockland Immunochemicals, Inc. 14.15.1 Company Overview 14.15.2 Role of Rockland Immunochemicals, Inc. in the Global CRISPR Gene Editing Market 14.15.3 SWOT Analysis 14.16 Synthego Corporation 14.16.1 Company Overview 14.16.2 Role of Synthego Corporation in the Global CRISPR Gene Editing Market 14.16.3 SWOT Analysis 14.17 System Biosciences LLC 14.17.1 Company Overview 14.17.2 Role of System Biosciences LLC in the Global CRISPR Gene Editing Market 14.17.3 SWOT Analysis 14.18 ToolGen, Inc. 14.18.1 Company Overview 14.18.2 Role of ToolGen, Inc. in the Global CRISPR Gene Editing Market 14.18.3 SWOT Analysis 14.19 Takara Bio 14.19.1 Company Overview 14.19.2 Role of Takara Bio in the Global CRISPR Gene Editing Market 14.19.3 Financials 14.19.4 SWOT Analysis

For more information about this report visit https://www.researchandmarkets.com/r/c7om7t

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Outlook on the CRISPR Gene Editing Global Market to 2030 - Analysis and Forecasts - GlobeNewswire

Manageable Safety Profile Observed in Phase 1 Studies Examining UCART19 for Pediatric and Adult Patients with B-Cell ALL – Cancer Network

By Adult Stem Cells

UCART19 produced a manageable safety profile in 2 separate phase 1 studies examining heavily pretreated pediatric and adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), according to data published in The Lancet.

For the first time, these studies support the feasibility of UCART19 and other genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor (CAR) T-cells to treat this group of patients with aggressive forms of ALL.

Phase 1 trials in paediatric and adult patients with late-stage relapsed or refractory B-cell acute lymphoblastic leukaemia have shown the feasibility, safety, and activity of UCART19, an off-the-shelf CAR T-cell product, wrote the investigative team. The results of these trials represent a substantial step forward in the development of CAR T cells and could herald a new, effective, and easily accessible cell therapy for patients with B-cell acute lymphoblastic leukaemia.

The results determined that the most common adverse event between both phase 1 studies was cytokine release syndrome (CRS), observed in 19 patients (91%). Three patients (14%) experienced grade 3/4 CRS.

More, 8 patients (38%) experienced grades 1/2 neurotoxicity, 2 (10%) experienced grade 1 acute skin graft-versus-host disease, and 6 (32%) had grade 4 prolonged cytopenia.

The research team recorded 2 treatment-related deaths between the 2 studies. The first was caused by neutropenic sepsis in a patient with concurrent CRS and the other was from pulmonary hemorrhage in a patient with persistent cytopenia.

Overall, 14 of 21 patients (67%) experienced a complete response or complete response with incomplete hematological recovery at 28 days following infusion. Median duration of response was recorded at 4.1 months, with 10 of 14 adult patients (71%) progressing to subsequent allogeneic stem cell transplant. The progression-free survival rate at 6 months was 27%, with an overall survival rate of 55%.

The adverse effects observed with UCART19 to date seem similar to those reported for autologous anti-CD19 CAR T cells, wrote the investigators. Cytokine release syndrome was encountered in the majority of patients in whom UCART19 expansion was detected and appeared no more severe than with approved autologous products.

The 2 ongoing, multicenter, clinical trials (NCT02808442 and NCT02746952) enrolled 7 pediatric and 14 adult patients from June 3, 2016, through October 23, 2018, to examine the safety profile and antileukemic activity of UCART19.

The dose-escalation studies began with patients undergoing lymphodepletion with fludarabine and cyclophosphamide, with or without alemtuzumab (Lemtrada), followed by different doses of UCART19 for adults and children. The primary end point of the data was adverse events.

The small sample size for the investigation is the leading limitation for the research, but the research team also mentioned the differing trial designs, lymphodepletion regimens, and UCART19 cell doses to be among limitations of both trials.

The results [of these studies] are an encouraging step forward for the field of allogeneic CAR T cells, and UCART19 offers the opportunity to treat patients with rapidly progressive disease and where autologous CAR T-cell therapy is unavailable, wrote the investigators.

Reference:

Benjamin R, Graham C, Yallop D, et al. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5

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Manageable Safety Profile Observed in Phase 1 Studies Examining UCART19 for Pediatric and Adult Patients with B-Cell ALL - Cancer Network

The Very First Signs of an Immune Response Have Been Filmed in a Developing Embryo – ScienceAlert

By Adult Stem Cells

Even as a hollow ball of embryonic cells, developing fish and mammals are not entirely defenceless.

The very first tissue, formed on the surface of a vertebrate blastula, has been shown to possess an innate immune response.

Incredible new research has shown that long before the development of organs or specialized immune cells, this simple protective layer, known as the epithelium, can reach out with its arm-like protrusions and detect, ingest, and destroy defective cells - helping to increase the embryo's chance of survival.

This 'surprisingly' efficient process, which was filmed in zebrafish and later confirmed in mice, is the earliest sign of an immune response in vertebrates.

Better understanding how it works could help researchers figure out why some embryos fail to form in those earliest states, potentially lead to new approaches for treating infertility or early miscarriages.

"Here we propose a new evolutionarily conserved function for epithelia as efficient scavengers of dying cells in the earliest stages of vertebrate embryogenesis," says cell biologist Verena Ruprecht from the Centre for Genomic Regulation.

"Our work may have important clinical applications by one day leading to improved screening methods and embryo quality assessment standards used in fertility clinics."

In developing animals, it's not uncommon for embryos to produce cellular errors during rapid cell division, and these can cause the whole embryo to fail if not taken care of. In fact, such mistakes are thought to be a leading reason for why embryos do not survive to reach implantation.

Scientists have long suspected there is an innate immune response at play, keeping fragile young embryos from threats such as sporadic cell death, inflammation, and infectious agents.

Recent research has revealed such innate immune responses in both mouse and human embryonic stem cells. But up until now, no one had ever seen it in action at the earliest stages.

This newest study is the first to explain how 'garbage collectors' like apoptotic cells are cleared out of the blastula without a specialised immune system. As you can see in the footage below, it looks a little like PAC-MAN.

So how does it work?

The blastula is a hollow ball, one cell thick, and the first stage of embryogenesis. The next stage includes further division into three germ layers, known as the gastrula.

In both these preliminary stages, researchers found evidence for the clearance of apoptotic cells, which initiate cell death.

Using four dimensional in vivo imaging of mice and zebrafish embryos, the authors show two types of epithelial 'arms' that seem to gobble up and destroy these apoptotic cells.

The first protrusion is called a phagocytic cup, and it helps scoop up and swallow the apoptotic target, a process known as phagocytosis. This structure is not unlike what we see in adult organisms, where epithelial phagocytosis keep organs and tissues healthy from infection and inflammation.

The second protrusion is a previously undescribed structure that is fast and can mechanically push apoptotic targets around, herding them into manageable positions.

"The cells cooperate mechanically," explains developmental biologist Esteban Hoijman, "like people distributing food around the dining table before tucking into their meal, we found that epithelial cells push defective cells towards other epithelial cells, speeding up the removal of dying cells."

Three dimensional tracking of these defective cells show they actually accumulate inside the epithelium, which suggests this protective layer is singling out certain cells specifically and gulping them up.

Even in conditions with abundant apoptosis, or cell death, occurring, zebrafish embryos were able to survive, which suggests this immune response is a highly efficient one.

Within two hours, in fact, the authors found the embryonic epithelium could remove 68 apoptotic particles.

Even when programmed cell death was triggered in the blastula using only two photons of illumination, the embryo showed epithelial clearance, indicating an impressive level of sensitivity.

"Together, these observations establish epithelial clearance as an error-correction mechanism that is present at the blastula stages of embryonic development," the authors conclude.

Zebrafish are model organisms for studying embryonic development, but to see whether this 'epithelial scavenging' also stood in mammals, the authors investigated what cell death looks like in mouse blastocysts.

Through time lapse imaging, the results reveal several apoptotic events, whereby cells are forced out of the blastocyst cavity and later ingested by the trophoblast. This is a tissue on the outside of the mammalian embryo that later forms a large part of the placenta. It also shows some level of innate immune response.

When mouse blastocysts were transplanted with apoptotic embryonic stem cells, the authors observed trophoblast cells eating up the targets.

Similar functions have also been documented in the human trophectoderm, which suggests the phagocytic epithelium has also been conserved in mammals and doesn't just appear in fish.

Knowing how mammal embryos survive from blastocyst to implantation could not only allow scientists to develop better fertility treatments, it could also teach us something about the early immune system - a power we could possibly try to replicate in adult tissues.

"Here we show that during early vertebrate development, epithelial cells specialize to perform phagocytic immune functions in the complete absence of immune cells," the authors write.

"At later developmental stages, professional phagocytes differentiate and can share their phagocytic tasks with mesenchymal or epithelial cells."

Future research will determine if the same innate immune process is also observed in invertebrates.

The study was published in Nature.

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The Very First Signs of an Immune Response Have Been Filmed in a Developing Embryo - ScienceAlert

Stem Cell Therapy Market: Top 5 trends fueling the industry revenue through 2025 – BioSpace

By Stem Cell Medicine

The global stem therapy market growth will be driven by extensive R&D activities aimed at developing novel therapies and personalized medicines. For example, to treat patients who have suffered a myocardial infarction (heart attack), scientists are emphasizing on developing techniques to regenerate healthy heart from placental stem cells.

As per the Centers for Disease Control and Prevention (CDC), heart diseases in the U.S. cause nearly 655,000 deaths each year. The development of novel therapies will aid in treating patients suffering from cardiovascular disease more effectively and reducing mortality rates. Ongoing examination of the different aspects of stem cell therapy by researchers to explore its application in neurological disorders can help in the treatment of complex diseases.

According to a Global Market Insights, Inc., report, stem cell therapy market size will surpass US$15 billion by 2025.

Described below are some key factors driving the stem cell therapy market trends.

Higher risk of degenerative diseases among the elderly

The substantial rise in the geriatric population and increasing chronic ailments would boost the demand for stem cell therapies. Older people happen to be more vulnerable to degenerative diseases such as Alzheimers and Parkinsons disorders. As compared to traditional therapeutic methods, the various benefits of stem cell therapy can make it preferable for the treatment of degenerative diseases. Newer solutions are based on minimally invasive techniques that generate healthy cells by replacing the defectives cells in the host.

Use of allogenic stem cell therapy in leukemia treatment

Allogenic transplant is used to completely replace host cells with the donor cells. It also possesses the potential to eliminate viral reservoirs and genetic alterations in host cells. Because of these capabilities, allogenic stem cell therapy is used for treating patients suffering from leukemia and other chronic disorders.

Reportedly, the annual valuation of allogenic stem cell therapy market was estimated to be worth US$3 billion in 2018 and it will grow substantially in the forthcoming years.

To access sample pages of this report titled, Stem Cell Therapy Market Size By Type (Allogenic Stem Cell Therapy, Autologous Stem Cell Therapy), By Application (Oncology, Orthopedic, Cardiovascular, Neurology), End-users (Hospitals, Clinics) Industry Analysis Report, Regional Outlook, Technology Potential, Competitive Market Share & Forecast, 2019 - 2025 in detail along with the table of contents, please click on the link below:

https://www.gminsights.com/request-sample/detail/3331

Global increase in trauma cases and bone-joint injuries

Rising incidences of accidents and trauma cases across the globe will augment demand for orthopedic care solutions. Stem cell therapy has a high success rate in the treatment of bone-joint injuries such as ligament tendon, osteogenesis imperfecta, femoral head, spinal, and fractured bone defects.

Additionally, the growing preference for mesenchymal stem cell therapy in nursing orthopedic diseases such as osteoporosis and arthritis can be attributed to its ability differentiate bones and cartilage Notably, the share of orthopedic application segment of stem cell therapy is estimated to rise at a significant CAGR of 9% up to 2025.

Availability of advanced stem cell therapies in clinical settings

The availability of technologically advanced medical equipment and highly skilled professionals in clinical facilities enable the delivery of safe and superior quality stem cell therapies. Clinics can provide specialized stem cell treatment for a wide range of applications including orthopedics, oncology, and cardiovascular disorders. Reportedly, clinics accounted for more than 35% revenue share of stem cell therapy market in 2018.

Increasing burden of chronic diseases in Europe

Europes stem cell therapy market is projected to experience a CAGR of nearly 10% up to 2025. The escalating prevalence of various chronic diseases will drive the demand for advanced therapeutic solutions in the region. Ongoing efforts from regulatory bodies to create awareness regarding stem cell therapy and its advantages among people will bolster the regional industry outlook.

The presence of stringent regulations pertaining to the quality control of these therapies could affect its adoption rate slightly. However, their high success rate in the treatment of complex diseases will fuel global stem cell therapy market expansion in near future.

Prominent firms involved in the development of novel stem cell therapy solutions include Takeda Pharmaceuticals, ReNeuron Group, Celyad, Capricor Therapeutics, Gamida Cell, Novadip Biosciences, Cellular Dynamics, DiscGenics, CESCA Therapeutics, Mesoblast, OxStem, Cellectis, and Astellas Pharma. These companies are focusing on several strategies such as acquisitions, mergers, and new product development to sustain their market position.

About Global Market Insights, Inc.

Global Market Insights, Inc., headquartered in Delaware, U.S., is a global market research and consulting service provider, offering syndicated and custom research reports along with growth consulting services. Our business intelligence and industry research reports offer clients with penetrative insights and actionable market data specially designed and presented to aid strategic decision making. These exhaustive reports are designed via a proprietary research methodology and are available for key industries such as chemicals, advanced materials, technology, renewable energy, and biotechnology.

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Arun Hegde Corporate Sales, USA Global Market Insights, Inc. Phone:1-302-846-7766 Toll Free:1-888-689-0688 Email:sales@gminsights.com Website:https://www.gminsights.com

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Stem Cell Therapy Market: Top 5 trends fueling the industry revenue through 2025 - BioSpace

Magenta Therapeutics to Present Additional Data from Phase 1 MGTA-145 Stem Cell Mobilization Program and Preclinical Updates on Targeting Conditioning…

By Stem Cell Medicine

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune and blood systems reset via stem cell transplant to more patients, today announced data presentations across its stem cell mobilization and targeted conditioning programs at the Transplantation and Cellular Therapy (TCT) Annual Meeting, to be held virtually on February 8-12, 2021.

Magenta continues to generate encouraging data across our pipeline, furthering our commitment to patients to expand eligibility and improve the clinical outcomes with stem cell transplant, said John Davis Jr., M.D., M.P.H., M.S., Magentas Head of Research & Development and Chief Medical Officer. Our presentations this year at TCT highlight the potential wide-ranging utility of our portfolio, and we are particularly excited to share these results, and to continue our progress in the year ahead.

Oral Presentations Showcasing Clinical Data of MGTA-145 Stem Cell Mobilization Program

Magenta is developing MGTA-145 in combination with plerixafor utilizing complementary mechanisms to mobilize hematopoietic stem cells (HSCs) for collection and transplantation. This combination has the potential to be the preferred mobilization regimen for rapid, reliable, predictable and safe collection of high numbers of functional blood stem cells to improve outcomes across autologous and allogeneic stem cell transplantation, which also includes stem cells necessary for all HSC-based gene therapies.

Title: MGTA-145 / Plerixafor-Mediated HSC Mobilization and Intravenous HDAd5/35++ Vector Injection into Mice Allows for Efficient in vivo HSC Transduction and Stable Gene Marking in Peripheral Blood Cells (Oral Abstract, #16) Presenting Author: Chang Li, Ph.D., Division of Medical Genetics, Department of Medicine, University of Washington Date and Time of Presentation: Session B Transplantation for Non-Malignant Disease; Monday, February 8, 2021, 3:15PM CST / 4:15PM EST

Data from this preclinical study demonstrate the potential of MGTA-145 plus plerixafor to serve as an efficient, single-dose mobilization regimen for in vivo HSC gene therapy where stem cells could be gene corrected or edited without having to remove them from the body. This could potentially replace current mobilization regimens that rely on ex vivo gene therapy approaches to treat genetic diseases.

Title: MGTA-145, in Combination with Plerixafor in a Phase 1 Clinical Study, Mobilizes Large Numbers of Hematopoietic Stem Cells and a Graft with Potent Immunosuppressive Properties for Autologous and Allogeneic Transplant (Oral Abstract, #35) Presenting Author: Kevin Goncalves, Ph.D., Magenta Therapeutics Date and Time of Presentation: Session E Consider the Source: Stem Cell Grafts and Donors; Tuesday, February 9, 2021, 4:00PM CST / 5:00PM EST

Data from this Phase 1 clinical trial with healthy volunteers further underscore the potential utility of MGTA-145 plus plerixafor as an effective, single-day mobilization and collection regimen for autologous and allogeneic HSC transplant. MGTA-145 plus plerixafor mobilized high numbers of HSCs and showed durable engraftment, successful gene-modification and immunosuppressive properties by reducing Graft-versus-Host disease (GvHD) in preclinical models.

Oral Presentation Showcasing Preclinical Study of MGTA-117 Targeted ADC Conditioning Program

Magenta is developing a suite of novel antibody-drug conjugates (ADCs) for conditioning, a step in the transplant process that currently relies on the use of systemic chemotherapy agents and radiation. Magentas targeted conditioning programs are designed to selectively eliminate stem cells and/or immune cells from a patient prior to transplant or gene therapy, and to reduce or potentially eliminate the need for high dose or high intensity chemotherapy-based treatments. These programs focus on developing targeted products that remove specific cell types, with an approach that is tailored to the patients disease and transplant requirements.

MGTA-117, Magentas most advanced conditioning program, is a CD117-targeted ADC designed to precisely deplete hematopoietic stem and progenitor cells to clear space in the bone marrow prior to transplant, and to support long-term engraftment and improved disease outcomes in patients. MGTA-117 has shown to be highly selective with potent activity, efficacy and tolerability in preclinical models.

Title: A Single Dose of a Novel Anti-Human CD117-Amanitin Antibody Drug Conjugate (ADC) Engineered for a Short Half-life Provides Dual Conditioning and Anti-Leukemia Activity and Extends Survival Compared to Standard of Care in Multiple Pre-clinical Models of Acute Myeloid Leukemia (AML) (Oral Abstract, #53) Presenting Author: Leanne Lanieri, M.S., Magenta Therapeutics Date and Time of Presentation: Session H Novel Conditioning Regimens & Transplantation for Aged Populations, Wednesday, February 10, 2021, 4:00PM CST / 5:00PM EST

Hematopoietic stem cell transplant (HSCT) can often be a curative treatment for patients with acute myeloid leukemia (AML). There is currently a need for safer and more effective targeted conditioning agents, as current conditioning regimens are associated with severe toxicities and high post-transplant relapse or graft failure. MGTA-117 was studied in multiple human leukemic xenograft murine models to mimic untreated and refractory AML. In preclinical models, MGTA-117 significantly increased median survival versus a multi-day standard-of-care regimen using cytarabine. Data from this study demonstrate MGTA-117s potential as a potent, targeted HSCT conditioning agent with anti-leukemic activity, emphasizing its potential to improve HSCT outcomes in AML by reducing the risk of post-transplant relapse.

Poster Presentation Highlighting Preclinical Data of CD45-ADC Targeted Conditioning Program

Magentas other ADC-based conditioning program, CD45-ADC, targets both patient HSCs and disease-causing immune cells. The programs lead target is CD45, a cell surface molecule broadly expressed throughout the hematopoietic and immune systems. CD45-ADC has the potential to significantly increase the number of patients eligible to receive a stem cell transplant, particularly those patients with autoimmune diseases and acute leukemias.

Developing a broad targeting approach for safer patient conditioning prior to HSCT could bring the curative potential of allogeneic HSCT to more patients with both malignant and non-malignant disorders. Current conditioning regimens limit accessibility of this procedure due to toxicity.

Title: Targeted CD45 Antibody Drug Conjugate Enables Full Mismatch Allogeneic Hematopoietic Stem Cell Transplantation in a Murine HSCT Model as a Single Agent (AML) (Poster #242) Lead Author: Sharon Hyzy, M.S., Magenta Therapeutics

Data from this study showed conditioning with single agent CD45-ADC enabled complete chimerism in a full mismatch allogeneic HSCT model.

Oral Presentation of MGTA-456 Stem Cell Therapy Expansion Program in Patients with Blood Cancer

Magenta is continuing long-term patient follow up to evaluate MGTA-456 in blood cancers through the investigator-initiated Phase 2 trial in blood cancers at the University of Minnesota and will assess best next steps for the program. Magenta previously announced in June 2020 it had discontinued enrollment in the Phase 2 trial of MGTA-456 in patients with inherited metabolic disorders.

Title: MGTA-456, A CD34 Expanded Cord Blood Product, Permits Selection of Better HLA Matched Units and Results in Rapid Hematopoietic Recovery, Uniform Engraftment and Reduced Graft-Versus-Host Disease in Adults with High-Risk Hematologic Malignancies (Oral Abstract, #31) Presenting Author: Heather Stefanski, M.D., Ph.D., Assistant Professor, Department of Pediatrics, University of Minnesota Date and Time of Oral Presentation: Session E Consider the Source: Stem Cell Grafts and Donors; Tuesday, February 9, 2021, 3:00PM CST / 4:00PM EST

Twenty-two patients were enrolled in the study, with 18 transplanted with MGTA-456. Compared to transplant patients who had undergone the same conditioning, GvHD prophylaxis and supportive care, patients who received MGTA-456 showed faster neutrophil recovery (median of 17 days compared to 23 days) and better platelet recovery (median 36 days compared to 59 days). Additionally, incidence of grade 2-4 acute GvHD was lower (24% compared to 46%), likely because of the ability to find a better matched cord unit.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with blood cancer, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant community to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Magentas future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting clinical data, projections regarding our long-term growth, cash, cash equivalents and marketable securities, the anticipated timing of our clinical trials and regulatory filings, the development of our product candidates and advancement of our clinical programs, the timing, progress and success of our collaborations, as well as other statements containing words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions that can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from pre-clinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials or to market products; whether Magenta's cash resources will be sufficient to fund Magenta's foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Magentas business, operations, strategy, goals and anticipated timelines, Magentas ongoing and planned pre-clinical activities, Magentas ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Magentas timelines for regulatory submissions and Magentas financial position; and other risks concerning Magenta's programs and operations set forth under the caption Risk Factors in Magentas Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics to Present Additional Data from Phase 1 MGTA-145 Stem Cell Mobilization Program and Preclinical Updates on Targeting Conditioning...

Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of…

By Stem Cell Medicine

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced the results of a Phase 3 clinical study of omidubicel presented in an oral session at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR), or the TCT Meetings. Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies.

This clinical data set was from the international, multi-center, randomized Phase 3 study of omidubicel that was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. This is the first presentation of these data in a peer-reviewed conference. The full presentation is available on the Gamida Cell website.

The results of this global Phase 3 study of omidubicel in patients with hematologic malignancies show that omidubicel resulted in faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital, all of which are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant, said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. The comparator, a transplant with umbilical cord blood, has been historically shown to result in low incidence of graft versus host disease (GvHD) in relation to other graft sources, and in this study, omidubicel demonstrated a GvHD profile similar to the comparator. Moreover, previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study receiving their transplant more than 10 years ago. The data presented at this meeting indicate that omidubicel has the potential to be considered a new standard of care for patients who are in need of stem cell transplantation but do not have access to a matched donor.

Details of Phase 3 Efficacy and Safety Results Shared at the TCT Meetings

Patient demographics including racial and ethnic diversity and baseline characteristics were well-balanced across the two study groups. The studys intent-to-treat analysis included 125 patients aged 1365 years with a median age of 41. Diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma. Patients were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America.

Gamida Cell previously reported in May 2020 that the study achieved its primary endpoint, showing that omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment, a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and a key milestone in a patients recovery from a bone marrow transplant. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001).

All three secondary endpoints demonstrated a statistically significant improvement among patients who were randomized to omidubicel in relation to patients randomized to the comparator group (intent-to-treat). Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.027). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 60.5 days, compared to 48.0 days for the comparator (p = 0.005). The details of these data were first reported in December 2020.

Previously unpublished data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the studys primary and secondary endpoints. There was no statistically significant difference between the two patient groups related to grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).

These clinical data results will form the basis of a Biologics License Application (BLA) that Gamida Cell expects to submit to the U.S. Food and Drug Administration (FDA) in the second half of 2021.

We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor, said Julian Adams, Ph.D., chief executive officer of Gamida Cell. Sharing the results of the Phase 3 study of omidubicel with the transplant community is a major moment for Gamida Cell, and we are forever grateful to the patients who participated in this study, their caregivers, and the work of the investigators and their teams.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of anticipated regulatory submissions, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the progress and expansion of Gamida Cells manufacturing capabilities and other commercialization efforts and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, its Report on Form 6-K filed with the SEC on August 12, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

1 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.

2 Gamida Cell press release, Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies, issued May 12, 2020. Last accessed August 31, 2020.

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Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of...

Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with…

By Stem Cell Medicine

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced positive preliminary findings from its ongoing multicenter Phase 1 clinical trial of JSP191, a first-in-class anti-CD117 (stem cell factor receptor) monoclonal antibody, as a conditioning agent in older patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) undergoing hematopoietic (blood) cell transplantation.

Data from the first six patients who received a single dose of JSP191 prior to transplantation showed successful engraftment in all six patients. Complete donor myeloid chimerism (equal or greater than 95%) was observed in five of six evaluable patients at 28 days, and all three evaluable patients had total donor chimerism equal or greater than 95% observed at day 90. In addition, at 28 days, three of five evaluable patients showed complete eradication of measurable residual disease (MRD) as measured by next-generation sequencing. Two of the five evaluable patients showed substantial reductions in MRD. No treatment-related serious adverse events were reported.

The findings were presented by lead investigator Lori Muffly, M.D., M.S., Assistant Professor of Medicine (Blood and Bone Marrow Transplantation) at Stanford Medicine, as a late-breaking abstract at the 2021 Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).

These early clinical results are the first to demonstrate that JSP191 administered in combination with a standard non-myeloablative regimen of low-dose radiation and fludarabine is well tolerated and can clear measurable residual disease in older adults with MDS or AML undergoing hematopoietic cell transplantation a patient population with historically few options, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. These patients could be cured by hematopoietic cell transplantation, but the standard-of-care myeloablative conditioning regimens used today are highly toxic and associated with high rates of morbidity and mortality particularly in older adults. Traditional lower intensity transplant conditioning regimens are better tolerated in older adults, but are associated with higher rates of relapse in MDS/AML patients with measurable residual disease. JSP191, a well-tolerated biologic conditioning agent that targets and depletes both normal hematopoietic stem cells and those that initiate MDS and AML, has the potential to be a curative option for these patients.

The open-label, multicenter Phase 1 study (JSP-CP-003) is evaluating the safety, tolerability and efficacy of adding JSP191 to the standard conditioning regimen of low-dose radiation and fludarabine among patients age 65 to 74 years with MDS or AML undergoing hematopoietic cell transplantation. Patients were ineligible for full myeloablative conditioning. The primary outcome measure of the study is the safety and tolerability of JSP191 as a conditioning regimen up to one year following a donor cell transplant.

We designed JSP191 to be given as outpatient conditioning and to have both the efficacy and safety profile required for use in newborn patients and older patients for successful outcomes, said Wendy Pang, M.D., Ph.D. Executive Director, Research and Translational Medicine, of Jasper Therapeutics. We are enthusiastic about the reduction of measurable residual disease seen in these patients, especially given that it is associated with improved relapse-free survival. We are excited to continue our research in MDS/AML, with plans for an expanded study. We are evaluating JSP191, the only antibody of its kind, in two ongoing clinical studies and are encouraged by the positive clinical data seen to date.

About MDS and AML

Myelodysplastic syndromes (MDS) are a group of disorders in which immature blood-forming cells in the bone marrow become abnormal and do not make new blood cells or make defective blood cells, leading to low numbers of normal blood cells, especially red blood cells.1 In about one in three patients, MDS can progress to acute myeloid leukemia (AML), a rapidly progressing cancer of the bone marrow cells.1 Both are diseases of the elderly with high mortality. Each year, about 5,000 patients with MDS and 8,000 people with AML in the G7 countries receive hematopoietic cell transplants. These transplants are curative but are underused due to the toxicity of the current high-intensity conditioning regimen, which includes the chemotherapy agents busulfan and fludarabine.

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplantation. A Phase 1/2 dose-escalation and expansion trial is evaluating JSP191 as a sole conditioning agent to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplantation for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. Data presented at the 62nd American Society of Hematology (ASH) Annual Meeting showed that a single dose of JSP191 administered prior to stem cell transplantation in a 6-month-old infant was effective in establishing sustained donor chimerism followed by development of B, T and NK immune cells. No treatment-related adverse events were reported. A Phase 1 clinical study is evaluating JSP191 in combination with another low-intensity conditioning regimen in patients with MDS or AML undergoing hematopoietic cell transplantation. For more information about the design of these two ongoing clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

1https://www.cancer.org/cancer/myelodysplastic-syndrome/about/what-is-mds.html

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Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with...

Creative Medical Technology Holdings Recruits Internationally Renowned Kidney Expert to Scientific Advisory Board – PRNewswire

By Stem Cell Medicine

PHOENIX, Feb. 8, 2021 /PRNewswire/ --(OTC-CELZ) Creative Medical Technology Holdings Inc. announced today recruitment of Dr. Caigan Du, Associate Professor at the University of British Columbia to the Company's Scientific Advisory Board.

Dr. Du is a top researcher in the area of molecular and immunological understanding of kidney failure and transplant rejection. Dr. Du is funded by numerous national and international organizations including the Kidney Foundation and the Canadian Institutes of Health Research.

"I am honored to work with Creative Medical Technology Holdings in this fascinating field of leveraging reprogrammed immune cells for regenerating injured kidneys." Said Dr. Du. "To date people think about regenerative medicine and immunology as separate fields. It is very exciting to consider the possibility that immune cells can act as a catalyst for regenerative processes: this is the basis of the ImmCelz product."

ImmCelz is a personalized cell therapy generated by incubation of patient cells with allogeneic JadiCell stem cells under proprietary conditions. The JadiCell possess potent ability to reprogram the immune system, as exemplified in part by their ability to significantly extend survival of COVID patients in an FDA double blind, placebo controlled, clinical trial1. ImmCelz has been demonstrated effective in animal models of rheumatoid arthritis2, liver failure3, stroke4, type 1 diabetes5 and kidney failure6. Scientific studies suggest ImmCelz functions through secretion of a fundamentally important molecule called Hepatocyte Growth Factor7, as well as stimulation of T regulatory cells, a type of immune system cell that suppresses pathological immunity8.

"As a clinical-stage biotechnology company, having already commercialized other stem cell products, we understand the key to any success is based on the ability to attract scientific key opinion leaders." Said Timothy Warbington, President and CEO of Creative Medical Technology Holdings. "Dr. Du is a visionary and pioneer in understanding of kidney diseases and we wholeheartedly look forward to him joining our scientific advisory board."

The Advisory Board of Creative Medical Technology Holdings includes internationally renowned neurologist Santosh Kesari MD, Ph.D, the former head of cardiology at Cedar Sinai Medical Center Timothy Henry, MD and our Director Dr. Amit Patel, inventor of the JadiCell and the first physician to have implanted stem cells into the human heart.

About Creative Medical Technology Holdings Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in regenerative medicine/stem cell technology in the fields of immunotherapy, urology, neurology and orthopedics and is listed on the OTC under the ticker symbol CELZ. For further information about the company, please visitwww.creativemedicaltechnology.com.

Forward Looking Statements OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website atwww.sec.gov.

Creativemedicaltechnology.comwww.StemSpine.comwww.Caverstem.comwww.Femcelz.com ImmCelz.com

1 Umbilical cord mesenchymal stem cells for COVID19 acute respiratory distress syndrome: A doubleblind, phase 1/2a, randomized controlled trial - Lanzoni - - STEM CELLS Translational Medicine - Wiley Online Library 2 Creative Medical Technology Holdings Reports Positive Preclinical Data on ImmCelz Immunotherapy Product in Rheumatoid Arthritis Model | BioSpace 3 Creative Medical Technology Holdings Announces Reversion of Liver Failure Using ImmCelz Personalized Cellular Immunotherapy in Preclinical Model | Nasdaq 4 Creative Medical Technology Holdings Identifies Mechanism of Action of ImmCelz Stroke Regenerative Activity (prnewswire.com) 5 Creative Medical Technology Holdings Announces Positive Data and Patent Filing Using ImmCelz to Treat Type 1 Diabetes (prnewswire.com) 6 Creative Medical Technology Holdings Files Patent based on Positive Data on Renal Failure using ImmCelz Regenerative Immunotherapy (prnewswire.com) 7 Creative Medical Technology Holdings Identifies and Files Patent on Novel Mechanism of ImmCelz Therapeutic Activity (apnews.com) 8 Creative Medical Technology Holdings Identifies Mechanism of Action of ImmCelz Stroke Regenerative Activity (prnewswire.com)

SOURCE Creative Medical Technology Holdings, Inc.

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