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Cell Expansion Market Analysis, COVID-19 Impact,Outlook, Opportunities, Size, Share Forecast and Supply Demand 2021-2027|Trusted Business Insights -…

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Cell Expansion market.

Trusted Business Insights presents an updated and Latest Study on Cell Expansion Market 2020-2029. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Cell Expansion market during the forecast period (2020-2029). It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

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Report Overview: Cell Expansion Market

The global cell expansion market size was estimated at USD 18.1 billion in 2020 is expected to register a compound annual growth rate (CAGR) of 8.3% over the forecast period 2021-2027. Increase in usage of automated solutions in cell expansion applications is one of the market drivers. Automated systems minimize the manpower and cost incurred during the production of Cell Therapy Products (CTP), gene therapies, and other biologics; leading to robust and reliable processes.

Key players engaged in CTP development are expanding their product line to suffice the significant rise in the global demand for these therapy products. For instance, in March 2019, Lonza introduced a comprehensive portfolio of CellBio Services, which includes cell-line expansion and banking services. This solution is designed to fulfill research application requirements, finally accelerating the revenue generation in the market.

The paradigm shift toward Single-Use Systems (SUS) offers substantial production advantages in CTP manufacturing. SUS eliminates concerns of cross-contamination and culture contamination caused due to inappropriate sterilization. SUS also allows the production of CTP with high cell densities as well as offers cost-savings in the long run. Thus, a rise in the adoption of SUS surges the development of CTP, which boosts the market growth.

Cellular therapies are constantly gaining popularity in the life sciences industry. Rise in funding from private & government organizations and initiatives undertaken by them to encourage the development of cellular therapies act as drivers for the market. The International Society for Stem Cell Research is involved in the identification of stem cell-related funding opportunities across biomedical research applications.

Substitution of serum and other incompatible reagents is essential to control the quality of the product in CTP manufacturing. Several research studies have validated that the application of serum-free media controls product quality. Researchers have also developed an optimization platform that integrates high-throughput tools with a differential evolution-based algorithm, which acts as an automated effective optimization strategy for serum-free culture formulations.

Product Insights: Cell Expansion Market

Consumables led the product segment in 2019 and accounted for 46.8% of the overall share. It is estimated to retain its dominant position throughout the forecast years. Availability of a wide range of commercial media and reagent products that are dedicated to specific type of cells contributes to the large revenue share of this segment. In addition, these products are convenient, ready-to-use, and are also available as serum-free formulations.

The instruments segment is expected to register the highest CAGR over the forecast period owing to automation in bioreactors and other expansion platforms to enhance the efficiency of culturing procedures. The advent of automated platforms standardizes the process and facilitates process tracking while reducing the hands-on time; therefore, enables more effective use of the time of skilled personnel.

Continuous commercialization and introduction of automated culturing equipment also drive the revenue generation in the instruments segment. For instance, in March 2019, Hitachi, Ltd. commercialized its automated cell mass culture equipment in Japan. This commercialization allowed manufacture of induced pluripotent stem cells for regenerative medicine applications. Such efforts are expected to accelerate the revenue generation for instruments.

Cell Type Insights: Cell Expansion Market

Mammalian cells segment held the largest revenue share of 58.5% in 2019 and will remain dominant during the forecast period as these culture systems are highly preferred in production of complex protein therapeutics. This is because these systems are pharmacokinetically and functionally relevant to post-translational modifications in humans. Therefore, most of the biopharmaceuticals, including monoclonal antibodies, specific interferons, thrombolytics, and various therapeutic enzymes, are produced using these culture systems.

Differentiated human cells accounted for substantial revenue share as these cells perform a specific function in the body. Differentiated cells, such as fibroblasts, have gained immense importance in the cutaneous wound healing and skin bioengineering, thereby augmenting the segment growth. Moreover, evaluation of 3D Gingival Fibroblast (GF) toroids as a feasible and simple in vitro assay for biomaterial testing has expanded their usage rate.

Human stem cells have gained significant traction and are expected to witness the fastest CAGR from 2020 to 2027. The exponential growth of research in this sector and the huge success of regenerative medicine are among the key factors that can be attributed to the fastest growth rate. Furthermore, implementation of automated, robotic, and closed production systems in the manufacturing of clinical-grade mesenchymal stem cells drives the segment.

Application Insights: Cell Expansion Market

The biopharmaceutical segment captured the largest revenue share of 22.9% in 2019 owing to increase in the approvals of biopharmaceutical products in the past few years. The entry of new biopharmaceutical companies and the proliferation of bioprocessing technologies further drive the development of biopharmaceuticals, which, in turn, boosts the expansion procedures conducted during bio-production.

In addition, single-use technologies are gaining immense traction in manufacturing cellular therapies on a commercial level. The introduction of alternative planar cell expansion technologies, such as compact multi-layer bioreactors, has been witnessed over the years. This sufficed the requirement of closed systems that limits the potential risks associated with contamination and maintains control of a large-scale upstream production and unit production. The vaccine production segment is expected to witness the fastest growth rate during the forecast period due to the expansion of cell-based vaccine production in recent years. The production of these vaccines offers a cost-effective manufacturing solution while accelerating the development process.

End-use Insights: Cell Expansion Market

Biotechnology & biopharmaceutical companies segment held the largest share of 48% in 2019 and will expand further at a significant growth rate. The broadening horizon of cell-based therapeutics in the healthcare industry is one of the major factors contributing to the large share of biopharmaceutical companies. For instance, cellular-based therapies have gained immense popularity in regenerative medicine with constant improvements in injectable cell delivery systems for various clinical applications.

In addition, pharmaceutical & biopharmaceutical companies are also engaged in acquisitions to expand their product portfolio. For instance, in December 2019, Sartorius AG signed an agreement to acquire 50% shares of Biological Industries, an Israeli cell culture media manufacturer. This acquisition expanded the cell culture media portfolio of Sartorius, especially for cellular and gene therapies, regenerative medicine, and other advanced therapies.

Research institutes are anticipated to register the highest CAGR from 2020 to 2027 as researchers are engaged in several studies in the biomedical field. For instance, in April 2019, researchers at the University of Wisconsin, U.S., introduced a new technology for effortless development of the H3N2 vaccine. For this, they developed a new cell line that enhanced the growth of H3N2 for vaccine use. Such research findings are expected to help in introducing new products in this market.

Regional Insights: Cell Expansion Market

North America accounted for the largest share of 43.1% in 2019. The region will retain its leading position over the coming years due to a rise in funding initiatives by the government agencies, which has accelerated the manufacture of stem cells and the development of regenerative medicine and cellular therapy products. This, in turn, drives the demand for cell expansion platforms in this region.

For instance, in October 2019, the FDA and the Medical Technology Enterprise Consortium (MTEC) together provided funding of USD 5.3 million to the Southwest Research Institute (SwRI) (U.S.). The SwRI invested these funds to propagate cells for the development of personalized regenerative medicine. Such investments boost the revenue generation in this region.

Asia Pacific is expected to witness the fastest growth during the forecast period due to increasing efforts laid down by several local pharmaceutical and biotechnology companies to develop and commercialize their cellular therapies. An Indian company, Stempeutics Research Pvt. Ltd., signed an alliance with Kemwell Biopharma in July 2019 for the commercialization of its stem cell-based product, Stempeucel. Such agreements are expected to boost product sales of small- and mid-scale companies.

Key Companies & Market Share Insights: Cell Expansion Market

Key market participants are undertaking several initiatives to expand their market presence and maintain a competitive edge in the space. Moreover, they are involved in collaboration & partnership models, product development, agreements, and business expansion strategies in untapped regions.

For instance, in June 2019, Demcon acquired a share in the Scinus Cell Expansion B.V., a manufacturer of stem cell-based therapy equipment headquartered in the Netherlands. This acquisition helped Scinus to expand its business in the stem cell therapy market. Such growth initiatives are expected to enhance the utilization rate of bioreactors used for culturing stem cells, thereby leading to market growth. Some of the prominent players in the cell expansion market include:

Key companies Profiled: Cell Expansion Market Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2016 to 2027. For the purpose of this study, Trusted Business Insights has segmented the global cell expansion market report on the basis of product, cell type, application, end use, and region:

Product Outlook (Revenue, USD Million, 2016 2027)

Cell Type Outlook (Revenue, USD Million, 2016 2027)

Application Outlook (Revenue, USD Million, 2016 2027)

End-use Outlook (Revenue, USD Million, 2016 2027)

Looking for more? Check out our repository for all available reports on Cell Expansion in related sectors.

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University of Alberta research team poised to cure diabetesagain – The Post – Ontario

The team, led by Dr. James Shapiro, has been able to consistently cure diabetes in mice and is now looking to move to human trials

Published on: November 18, 2020 | Last Updated: November 18, 2020 10:56 AM EST

Dr. James Shapiro, a liver transplant surgeon with the University of Alberta and director of the Clinical Islet and Living Donor Liver Transplant programs with Alberta Health Services, inspects the OrganOx Metra portable ex-vitro perfusion device - the first of its kind in North America - at the University of Alberta Hospital on Wednesday, March 18, 2015. PHOTO BY CLAIRE THEOBALD/EDMONTON SUN.

Almost 20 years after a University of Alberta research group made medical history by improving on a treatment for diabetes, the same team is trying to do it again.

Dr. James Shapiro and his research team at the university say theyve been able to cure diabetes in mice, CTV Alberta reports. The team is using a technique that involves stem cells developing into pancreatic cells that can produce insulin. They believe their research can translate into a functional human cure for diabetes.

Weve been working with a company called ViaCyte in San Diego for the last, almost 19 years now, and this company has a cell that is derived from a human embryonic stem cell that makes human insulin in a regulated, perfect way, Dr. Shapiro said in video research update. Weve been able to treat countless thousands of mice with these stem cells and effectively cure mice with diabetes over many years now.

In the late 1990s, Dr. Shapiro and his team in Edmonton improved on the technique of transplanting insulin-producing islet cells from the pancreases of donors into type 1 diabetics. Their technique, which relies on using a large number of the islet cells from as many as three different donors, was published to The New England Journal of Medicinein 2000 and became known as the Edmonton protocol.

Unfortunately, islet cell implantation has major limitations. Transplant recipients are forced to take immunosuppressant anti-rejection drugs that come with a grocery list of side effects, such as high blood pressure and increased risk of infections. Then theres the lack of supply organ donations and the risk that, in most cases, the diabetic patient will slowly have to start reintegrating insulin over the years.

The stem cell therapy that Dr. Shapiro is proposing has none of these drawbacks. The University of Alberta team foresee a one-time injection with possible re-ups later on of insulin-producing cells derived from human stem cells. No need for immunosuppressants or organ donations.

Now, the team is ready to move on to human trials. The only obstacle: money. A small volunteer group, Heading to 2022, wants to raise $22 million by 2022 to help bring Dr. Shapiros new treatment to the next phase of trials.

2022 will be the 100th anniversary of the first successful insulin injection. In 1922, Dr. Frederick Banting and his small team, working out of the University of Toronto, saved the life of a 14-year-old-boy named Leonard Thompson, who was dying from diabetes.

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University of Alberta research team poised to cure diabetesagain - The Post - Ontario

Comprehensive Report on Stem Cell Assay Market 2020 | Size, Growth, Demand, Opportunities & Forecast To 2026 | Bio-Techne Corporation, Promega…

Stem Cell Assay Market research report is the new statistical data source added by A2Z Market Research.

Stem Cell Assay Market is growing at a High CAGR during the forecast period 2020-2026. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

Stem Cell Assay Market research is an intelligence report with meticulous efforts undertaken to study the right and valuable information. The data which has been looked upon is done considering both, the existing top players and the upcoming competitors. Business strategies of the key players and the new entering market industries are studied in detail. Well explained SWOT analysis, revenue share and contact information are shared in this report analysis.

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Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

Top Key Players Profiled in this report are:

Bio-Techne Corporation, Promega Corporation, Merck KGaA, STEMCELL Technologies, GE Healthcare, Thermo Fisher Scientific, Cellular Dynamics International, Bio-Rad Laboratories, Hemogenix, Cell Biolabs

The key questions answered in this report:

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Stem Cell Assay market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Stem Cell Assay markets trajectory between forecast periods.

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Global Stem Cell Assay Market Segmentation:

Market Segmentation by Type:

Viability/Cytotoxicity Isolation & Purification Cell Identification Proliferation Differentiation Function Apoptosis

Market Segmentation by Application:

Regenerative Medicine & Therapy Development Drug Discovery and Development Clinical Research

Regions Covered in the Global Stem Cell Assay Market Report 2020: The Middle East and Africa(GCC Countries and Egypt) North America(the United States, Mexico, and Canada) South America(Brazil etc.) Europe(Turkey, Germany, Russia UK, Italy, France, etc.) Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The report provides insights on the following pointers:

Table of Contents

Global Stem Cell Assay Market Research Report 2020 2026

Chapter 1 Stem Cell Assay Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Stem Cell Assay Market Forecast

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Asia Pacific ultra-low temperature freezer market is anticipated to reach US$ 159.04 million by 2027 from US$ 109.02 million in 2019 – GlobeNewswire

November 18, 2020 10:51 ET | Source: ReportLinker

New York, Nov. 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Asia Pacific Ultra-Low Temperature Freezer Market to 2027 Regional Analysis and Forecasts by Type ; End User ; and, Country" - https://www.reportlinker.com/p05953753/?utm_source=GNW Growing R&D activities to introduce new drug compounds, is likely to damage the growth of the market in the coming years.

On the other hand, development in the healthcare market is expected to have a positive impact on the growth of the Asia Pacific ultra-low temperature freezer market in the coming years. Ultra-low temperature freezers are designed for storage of biological materials such as virus, bacteria, eukaryotic cells, blood, and semen. These freezers are used in blood banks, hospitals, epidemic prevention services, research institutes, and biomedical engineering facilities, among others. Pharmaceutical companies focus on R&D to introduce new molecules for various therapeutic applications with enhanced medical and commercial potential. The companies invest majorly on R&D activities with an aim to delivering high quality and innovative products in the market. R&D spending by biopharmaceutical companies has been increasing over the years. According to the report of Pharmaceutical Research and Manufacturers of America (PhRMA), the R&D expenditure of the biopharmaceutical companies has increased from US$ 49.6 billion in 2012 to US$ 58.8 billion in 2015. Among the worlds top 50 companies by total R&D investment in the fiscal year 2014/2015 were 16 pharmaceutical companies. Novartis, Roche, Johnson & Johnson, and Pfizer are among the top 10 leading R&D investing companies worldwide. Temperature accuracy and monitoring are the major aspects in medical science to preserve and store temperature-sensitive biomedical components that are frequently used for new drug development. Thus, the growing R&D expenditure to introduce new drug compounds will require reliable storage; this, in turn, is expected to drive the growth of the ultra-low temperature freezers market during the forecast period. As per the S&P Global ratings, the COVID-19 outbreak is expected to wipe approximately US$ 200 billion of Asia Pacific economies this year, sending growth rate to its lowest level.The outbreak has spread to at least 85 countries in the region and is expected to hamper the market growth as investors fret over its economic impact.

The total reported COVID-19 cases in the major countries of APAC are: china (82,295), Japan (8,100), and India (11,555).The pharmaceutical companies in the region are working on drug discovery and clinical testing processes on a large scale.

However, China and Japan have been frontiers in the research activities including the development of stem cell therapies, cell therapies, and regenerative medicine. Therefore, the ultra-low temperature freezer market in APAC is anticipated to be less affected by the pandemic situation. In terms of type, the upright ULT freezers segment accounted for the largest share of the Asia Pacific ultra-low temperature freezer market in 2019 and is estimated to mark the highest CAGR in the market during the forecast period, owing to the factors such as easier to organize because of having shelves. Also, the convenience of use of upright freezers has led to its dominance during 2019 and is expected to witness similar trend over the coming years. Centers for Disease Control and Prevention (CDC), Biomedical Research Centers (BRCs), Society of Infectious and Tropical Diseases (SIMIT), and International Society for Stem Cell Application (ISSCA) are among the significant primary and secondary sources for ultra-low temperature freezer included in the report. Read the full report: https://www.reportlinker.com/p05953753/?utm_source=GNW

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Asia Pacific ultra-low temperature freezer market is anticipated to reach US$ 159.04 million by 2027 from US$ 109.02 million in 2019 - GlobeNewswire

Karnataka:Community cord blood banking emerges as lifesaver – United News of India

More News 18 Nov 2020 | 11:29 PM

Hyderabad, Nov 18 (UNI) AICC Spokesperson Dr Sravan Dasoju slams the State Election Commission (SEC) for taking a U-turn on the distribution of flood relief funds to the victims.

Hyderabad, Nov 18 (UNI) A couple who allegedly cheated a bank of Rs 5.3 crore by submitting false documents to get loans, was arrested by the Cyberabad Police on Wednesday.

Hyderabad, Nov 18 (UNI) A 50-year-old woman waiting in queue at a Meeseva centre to apply for Rs 10,000 flood relief from the Telangana Government, collapsed and died at Tolichowki near here on Wednesday even as the stampede-like situation prevailed at Mee Seva centres in Hyderabad and surrounding areas where thousands of persons in queued up for applying to for flood relief.

Kochi, Nov 18 (UNI) Opposition Leader in Kerala Assembly Ramesh Chennithala on Wednesday alleged that the arrest of former PWD Minister VK Ebrahim Kunju was politically-motivated.

Bengaluru, Nov 18(UNI) Karnataka on Wednesday reported 1791 fresh cases of COVID-19 on Wednesday, taking the total number of infections in the state to 865931.

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Karnataka:Community cord blood banking emerges as lifesaver - United News of India

Human sperm stem cells grown in lab, an early step toward infertility treatment – Jill Lopez

Infertility affects one in seven men of reproductive age worldwide. One idea for treating male sterility is spermatogonial stem cell (SSC) therapy. In this approach, sperm stem cells in the testis are transferred to a test tube, cultured and nudged into becoming fully fledged sperm. However, a key bottleneck has been identifying just the right conditions to get human SSCs to grow in the lab. There have been many attempts, but in most reported cases it was not clear whether the cells being cultured were actually SSCs, and no previously published method is routinely used.

Researchers at University of California San Diego School of Medicine have now developed a reliable method for culturing cells with the characteristics of human SSCs. Their work is published in the July 13, 2020 issue ofProceedings of the National Academy of Sciences.

"We think our approach -- which is backed up by several techniques, including single-cell RNA-sequencing analysis -- is a significant step toward bringing SSC therapy into the clinic," said senior author Miles Wilkinson, PhD, Distinguished Professor in the Department of Obstetrics, Gynecology and Reproductive Sciences at UC San Diego School of Medicine.

SSCs are what make it possible for men to father children beyond the age of 65. These specialized cells continually self-renew, making more SSCs, and develop into sperm so prolifically that men (and some transgender, non-binary and gender fluid people) produce more than 1,000 new sperm every few seconds.

Progress in the field has been hindered by the fact that it's extremely difficult to distinguish SSCs from other cells in the testes. It was a major step forward when several laboratories, including the Wilkinson team,recently useda technique called single-cell RNA sequencing to define the likely molecular characteristics specific to human SSCs.

In their latest effort, the Wilkinson team used its single-cell RNA sequencing information to purify what it thought might be human SSCs. Using a method called germ-cell transplantation, it showed that the cells it purified were indeed highly enriched in human SSCs. The team then gathered the profile of genes expressed in these human SSCs to make guesses as to the conditions that might best support their growth in the lab. Using more than 30 human testis biopsies, the researchers determined just the right conditions needed to culture immature germ cells with the characteristics of SSCs.

The key ingredient was an inhibitor of the AKT pathway, a cellular system that controls cell division and survival. The Wilkinson team determined that AKT inhibition maintains human SSCs by inhibiting development of later-stage sperm precursors. Several AKT inhibitors are currently used to treat cancer.

With that approach, the researchers were able to favor the culture of human cells with the molecular characteristics of SSCs for two-to-four weeks.

"Next, our main goal is to learn how to maintain and expand human SSCs longer so they might be clinically useful," Wilkinson said.

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Human sperm stem cells grown in lab, an early step toward infertility treatment - Jill Lopez

Global Stem Cell and Primary Cell Culture Medium Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts Research…

IndustryGrowthInsights has published a detailed report on the Stem Cell and Primary Cell Culture Medium market. This market research report was prepared after considering the COVID-19 impacts and monitoring the market for a minimum of five years. The report provides you with growing market opportunities, revenue drivers, challenges, pricing trends & factors, and future market assessments. Our research team has implemented a robust research methodology that includes SWOT analysis, Porters 5 Force analysis, and real-time analysis. Furthermore, they have conducted interviews with the industry experts to offer a report that helps the clients to formulate strategies accordingly.

The Global Stem Cell and Primary Cell Culture Medium Market has outlined the supply and demand scenario in the industry and provided a detailed analysis of the product developments, technology advancements, and competitive analysis in the market. It offers an in-depth analysis and all the information required by the new entrants and emerging players to stay ahead in the competition. This report includes information on the latest government policies, norms, and regulations that have and can affect the dynamics of the 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.

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IGI has laid out a dedicated section for the prominent companies in the market which provides information on their revenue drivers, product innovation, and challenges they are facing during in the industry. This company profiling section includes industry players mergers, acquisitions, and collaborations which have helped them to leverage or impacted their market position. Besides this, the report is fragmented on the basis of the products, applications, and region-based analysis which imparts a holistic view and scope of the market.

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Market Segmentation

The Stem Cell and Primary Cell Culture Medium market report is fragmented into product types, applications, and regional analysis. In this report, the product flow, distribution, and possible future innovations are bestowed in a detailed manner. It also provides accurate calculations for product sales in terms of volume and value.

The applications of the products are discussed in a coherent way which includes potential future applications.

The Stem Cell and Primary Cell Culture Medium market is classified into:

The Study is segmented by following Product Type:

Liquid Media Powder Media

Major applications/end-users industry are as follows:

Biopharmaceutical Manufacturing Tissue Engineering & Regenerative Medicine Gene Therapy Other

Geographical Analysis:

Major Companies in the Market:

Merck STEMCELL Technologies Irvinesci Cell Applications, Inc Biological Industries Miltenyi Biotec Swiss Medica Clinic Promocell Creative Biolabs Lifeline Cell Technology ScienCell Research Laboratories Osiris Therapeutics NuVasive Chiesi Pharmaceuticals JCR Pharmaceutical Pharmicell Medi-post Anterogen Molmed Takeda (TiGenix)

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Updated: FDA gives Cellectis all clear after patient death halted off-the-shelf CAR-T trial – Endpoints News

Four months after a patient death forced Cellectis to halt one of their off-the-shelf CAR-T trials, the FDA has given them the OK to start dosing patients again.

The Paris-based biotech said they changed the studys protocol to accommodate the agencys concerns, although they will still have to work with investigators to obtain local approval to restart the trial and start recruiting patients again. Prior its halt, the Phase I had sites in New York, New Jersey and Texas.

Cellectis did not disclose what the changes were, but after the initial hold, analysts suggested the company might monitor patient cytokine levels more closely, swap out of one of the chemotherapy drugs used or prevent patients who have previously received an experimental multiple myeloma CAR-T from enrolling.

The companys stock {CLLS} rose 5.6% on the news, from $19.70 to $20.80.

Though partnered with the new upstarts such as Arie Belldegruns Allogene, Cellectis, a 21-year-old company, has been working on off-the-shelf versions to the first CAR-Ts since before those CAR-Ts were approved.

One of three different solo projects now in the clinic, the halted study, MELANI-01, is testing a cell therapy for multiple myeloma. If effective, it could serve as a potentially easier way to manufacture and administer an alternative to the multiple myeloma CAR-Ts now nearing approval from J&J and Legend Biotech and from bluebird bio and Bristol Myers Squibb.

When they announced the hold, Cellectis CMO Carrie Brownstein said that the company had already independently decided to move forward with dose level 1 in the study. The patient who died had been the first to receive dose level 2, an infusion of 3 million cells per kilogram. They developed cytokine release syndrome, the hallmark side effect of CAR-T therapy, and died of cardiac arrest 25 days later.

The dose 1 level involves 1 million cells per kilogram.

The FDA has been on high alert for safety issues in cell therapy trials, and this wasnt the first time theyve paused a Cellectis study. Back in 2017, another patient death paused development of UCART123.

After some protocol adjustments, the therapy was allowed to go back into patients. It is now in a Phase I for acute myeloid leukemia.

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Updated: FDA gives Cellectis all clear after patient death halted off-the-shelf CAR-T trial - Endpoints News

Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency – Health Europa

For the first time, researchers at the Center for Cell-Based Therapy (CTC) in Ribeiro Preto, Brazil, have identified a non-hereditary mutation in blood cells from a patient with GATA2 deficiency.

GATA2 deficiency is a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2), which regulates the expression of genes that play a role in developmental processes and cell renewal.

An article on the study is publishedin the journalBlood.

The non-hereditary mutation may have acted as a natural gene therapy which prevented the disease from damaging the process of blood cell renewal. This meant that the patient did not develop such typical clinical manifestations as bone marrow failure, hearing loss, and lymphedema.

The researchers say that the findings pave the way for the use of gene therapy and changes to the process of checking family medical history and medical records for families with the hereditary disorder.

Luiz Fernando Bazzo Catto, first author of the article, said: When a germline [inherited] mutation in GATA2 is detected, the patients family has to be investigated because there may be silent cases.

The discovery was made when two sons were receiving medical treatment at the blood centre of the hospital run by FMRP-USP, both of which, in post-mortem DNA sequencing, showed germline mutations and GATA2 deficiency diagnosis. The researchers used next generation sequencing to estimate the proportion of normal blood cells in the fathers bone marrow, preventing clinical manifestations of GATA2 deficiency, and of cells similar to his childrens showing that 93% of his leukocytes had the mutation that protects from the clinical manifestations of GATA2 deficiency.

Following the sequencing of the fathers T-lymphocytes, the researchers found that the mutation occurred early in their lives and in the development of hematopoietic stem cells, which have the potential to form blood.

They also measured the activity of the blood cells, to see if they could maintain the activity of inducing normal cell production for a long time, by measuring the telomeres of his peripheral blood leukocytes. Telomeres are repetitive sequences of non-coding DNA at the tip of chromosomes that protect them from damage. Each time cells divide, their telomeres become shorter. They eventually become so short that division is no longer possible, and the cells die or become senescent.

The telomeres analysed by the researchers were long, indicating that the cells can remain active for a long time.

The researchers hypothesised that the existence of the somatic mutation in the fathers blood cells, and its restoration of the blood cell renewal process, may have contributed to the non-manifestation of extra-haematological symptoms of GATA2 deficiency such as deafness, lymphedema, and thrombosis.

Professor Rodrigo Calado, a corresponding author of the article, said: A sort of natural gene therapy occurred in this patient. Its as if he embodied an experiment and a medium-term prospect of analogous gene therapy treatment in patients with GATA2 deficiency.

The findings help us understand better how stem cells can recover by repairing an initial genetic defect.

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Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency - Health Europa

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Cell Therapy Market Attractiveness, Competitive Landscape and Key Players MEDIPOST, Kolon TissueGene, NuVasive, Fibrocell Science - The Think...