AlloVir’s IPO Raises $276M to Test Cell Therapies in Transplant Patients – Xconomy

Xconomy Boston

AlloVir is the latest biotech company to go public, raising about $276.3 million to pull its cell therapies off the shelf and run multiple clinical trials testing them in stem cell and organ transplant patients.

On Thursday, Cambridge, MA-based AlloVir (NASDAQ: ALVR)priced its offering of 16.25 million shares at $17 apiece, the midpoint of its targeted $16 to $18 per share price range. The company was able to sell more shares than it planned, boosting the size of the stock offering from the initial target of 14.75 million shares. AlloVir shares opened at $20 apiece, up 17.6 percent from the IPO price.

AlloVir is developing cell therapies intended to treat and prevent viral infections in transplant patients. These patients have weak immune systems that make them particularly susceptible to infection. The standard of care includes antiviral drugs. But sometimes these drugs arent enough to stop viruses, and these treatments can damage the kidneys. Instead of taking viruses head on, AlloVirs cell therapies are intended to restore the ability of a patients immune system to tackle them.

The AlloVir cell therapies are called virus-specific T cells, or VSTs. The process for making a VST therapy is similar to the way that CAR-T cancer treatments are produced. Immune cells are removed from the body and multiplied in a lab. But unlike CAR-T therapies, AlloVirs process doesnt engineer the cells in any way. The AlloVir cell therapies also dont start with a patients own immune cells. The allogeneic approach uses the T cells of healthy people who have already been exposed to viruses. That exposure is expected to be enough get these cells to go after these viruses after the therapy has been infused into a transplant patient.

Once produced, AlloVirs therapies can be stored and pulled off the shelf for use as needed. Speaking to Xconomy last year, companyco-founder and Chief Scientific Officer Ann Leen, an immunologist at the Baylor College of Medicine, likened the process to growing an immune system outside of the body, and giving it to transplant patients at the first sign of infection.

The most advanced AlloVir product candidate, Viralym-M, is a VST that targets five viruses: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. The company is testing the cell therapy as a treatment for both stem cell transplant patients and organ transplant patients.

So far, Viralym-M has been evaluated in a Phase 2 clinical trial. According to the prospectus, the experimental therapy was given to 58 stem cell transplant patients whose infections had not responded to earlier antiviral treatments. The study was not designed to show statistical significance or that the VST was superior to antivirals. The proof-of-concept clinical trial was intended to show how the therapy works and whether it can be given safely.

In the study, 93 percent54 patientsresponded to the therapy in some way. A complete response, defined as the viral load returning to the range found in healthy people and resolution of the signs and symptoms of infection, was observed in 17 patients. Another 37 patients showed a partial response, defined as the viral load falling by half.

The therapy was well-tolerated by patients. The serious complications and patient deaths in the study were deemed unrelated to the AlloVir therapy. Graft-versus-host-disease, an immune response that is a known risk of stem cell transplants, was reported in 14 patients. A total of 23 patients in the study died. Preliminary clinical trial data were published in the Journal of Clinical Oncology in 2017.

There are no cell therapies approved for use in the US or Europe for treating or preventing the viral infections that AlloVir is targeting. But the company faces potential competition from South San Francisco-based Atara Biotherapeutics (NASDAQ: ATRA), which has advanced an allogeneic cell therapy called tabelecleucel to Phase 3 testing. That study is evaluating the therapy as a treatment for stem cell and organ transplant patients who develop Epstien-Barr viral infection along with post-operative lymphoproliferative disease, a known complication of transplant procedures.

AlloVir was founded in 2013 based on technology developed in Leens research lab, which is part of the Center for Cell and Gene Therapy at Baylor. The company, which was initially named ViraCyte, is led by CEO David Hallal. Hallal is also the chief executive of ElevateBio, a Waltham, MA-based company that provides manufacturing services for cell and gene therapy companies and also invests in them. ElevateBio is AlloVirs largest institutional shareholder, holding a 21.9 percent post-IPO stake, according to the prospectus. Prior to the IPO, the filing shows that AlloVir had raised $156.3 million, including a $12o million Series B round last year.

According to the prospectus, AlloVir plans to use the IPO cash to advance Viralym-M to a Phase 3 study testing it against hemorrhagic cystitis, inflammation of the bladder that is a virus-associated complication faced by stem cell transplant patients. The drug will also be tested in separate late-stage studies in cytomegalovirus and adenovirus. About $98 million is planned for those Phase 3 tests.

Another $83 million is earmarked for Phase 2 tests of Viralym-M. Those tests will cover the prevention of multiple viral infections in stem cell transplant patients, treating BK virus infection in kidney transplant patients, and treating cytomegalovirus infections in solid organ transplant patients.

The IPO cash will also support two respiratory virus programs. ALVR106 is being developed to treat respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus. That VST is expected to start Phase 1 tests in the second half of this year. The other respiratory virus program, ALVR109, is in development as a treatment for infection by the novel coronavirus SARS-CoV-2. The company plans to advance that program to Phase 1/2 testing.

Image: iStock/Jay_Zynism

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan@xconomy.com.

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AlloVir's IPO Raises $276M to Test Cell Therapies in Transplant Patients - Xconomy

Cerus Corporation Announces a Five-Year Tender Award by the Hong Kong Red Cross Blood Transfusion Service for INTERCEPT Blood Systems – Business Wire

CONCORD, Calif.--(BUSINESS WIRE)--Cerus Corporation (Nasdaq:CERS) today announced it has been awarded a five-year tender by the Hong Kong Red Cross Blood Transfusion Services (BTS) for INTERCEPT Blood System for platelets. The Hong Kong Red Cross BTS is the only public institution providing blood to all hospitals, public and private, in Hong Kong.

Following a thorough selection process, we are pleased to be selected by the Hong Kong Red Cross BTS to supply INTERCEPT Blood System for platelets, said Pascal Maillard, Cerus vice president of commercial operations for the Asia Pacific region. INTERCEPT for platelets provides a robust, broad-spectrum inactivation, reducing the risks of transfusion-transmitted infections and thereby enhancing patient care.

This is an important contract for Cerus, continued Maillard. The Hong Kong Red Cross BTS is a member of the influential Asia Pacific Blood Network (APBN) and a key opinion leader globally. This tender award presents a unique opportunity to expand the use of INTERCEPT in Asia-Pacific, a region that represents a significant market growth opportunity for Cerus.

The tender award builds upon the prior experience of the Hong Kong Red Cross BTS with the INTERCEPT Blood System for platelets. The Hong Kong Red Cross BTS, in collaboration with the Queen Mary Hospital, conducted a clinical study successfully transfusing INTERCEPT treated platelet components prepared without leukocyte reduction, without bacterial screening, and without gamma irradiation to allogeneic Hematopoietic Stem Cell Transplant (HSCT) patients (Sim, Tsoi et al. 2019).

This innovative study conducted by the Hong Kong Red Cross BTS and Queen Mary Hospital has the potential to change how patients are supported with platelet transfusions, said Dr. Laurence Corash, Cerus chief scientific officer. The study indicated that INTERCEPT treatment could potentially replace leukocyte filtration, a common practice to reduce the incidence of febrile transfusion reactions, alloimmunization, and cytomegalovirus (CMV) infection. The INTERCEPT platelet system has already demonstrated widespread replacement of bacterial screening, gamma irradiation, and CMV serology in the EU and U.S.

The Hong Kong Red Cross BTS recently completed renovations and is a state-of-the-art facility that manufactures approximately 40,000 platelet doses per year, a portion of which will be treated with INTERCEPT.

Sim, J., W. C. Tsoi, et al. (2019). "Transfusion of pathogen-reduced platelet components without leukoreduction." Transfusion 59(6): 1953-1961.

ABOUT CERUS

Cerus Corporation is dedicated solely to safeguarding the worlds blood supply and aims to become the preeminent global blood products company. Based in Concord, California, our employees are dedicated to deploying and supplying vital technologies and pathogen-protected blood components for blood centers, hospitals and ultimately patients who rely on safe blood. With the INTERCEPT Blood System, we are focused on protecting patients by delivering the full complement of reliable products and expertise for transfusion medicine. Cerus develops and markets the INTERCEPT Blood System and remains the only company in the blood transfusion space to earn both CE Mark and FDA approval for pathogen reduction of both platelet and plasma components. Cerus currently markets and sells the INTERCEPT Blood System in the United States, Europe, the Commonwealth of Independent States, the Middle East and selected countries in other regions around the world. The INTERCEPT Red Blood Cell system is in clinical development. For more information about Cerus, visit http://www.cerus.com and follow us on LinkedIn.

INTERCEPT and the INTERCEPT Blood System are trademarks of Cerus Corporation.

Forward Looking Statements

Except for the historical statements contained herein, this press release contains forward-looking statements concerning Cerus belief that the INTERCEPT Blood System has the potential to change how patients are supported with platelet transfusions and to potentially replace leukocyte filtration, and other statements that are not historical facts. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation: risks related to Cerus ability to demonstrate to the transfusion medicine community and other health care constituencies that pathogen reduction and the INTERCEPT Blood System is safe, effective and economical; the risk that applicable regulatory authorities and healthcare practitioners may disagree with Cerus interpretations of the data from its clinical studies; as well as other risks detailed in Cerus filings with the Securities and Exchange Commission, including Cerus Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, filed with the SEC on May 5, 2020. In addition, to the extent that the COVID-19 pandemic adversely affects Cerus business and financial results, it may also have the effect of heightening many of the other risks and uncertainties described above. Cerus disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release.

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Cerus Corporation Announces a Five-Year Tender Award by the Hong Kong Red Cross Blood Transfusion Service for INTERCEPT Blood Systems - Business Wire

Avacta and Daewoong Pharmaceutical expand partnership to include COVID-19 neutralising Affimer therapy – Cambridge Network

Avacta Group plc (AIM: AVCT), the developer of Affimer biotherapeutics and reagents, is pleased to announce an expansion of its collaboration and licence agreement with Daewoong Pharmaceutical Co. Ltd. (KSX: 069620) and AffyXell Therapeutics, the joint venture established in South Korea by the two companies, to develop stem cell treatments incorporating Avactas neutralising Affimer therapy for the treatment of seriously ill patients with COVID-19 and to also prepare to rapidly develop similar therapies for future global pandemics.

Respiratory diseases such as COVID-19 can cause serious damage to the lungs as a consequence of over-activation of the patient's immune system, resulting in cytokine release syndrome that can potentially lead to multiple organ failure and death. Stem cell therapies offer a very promising approach to repair the damage to lung tissues in these pulmonary diseases by controlling the immune balance.

AffyXell Therapeutics, the next-generation cell and gene therapy joint venture between Avacta and Daewoong Pharmaceutical, is developing a novel class of mesenchymal stem cell (MSC) treatments that are engineered to also produce Affimer therapies in the patient at the site of action.

The expansion of the agreement between Avacta, Daewoong Pharmaceutical and AffyXell announced today extends the scope of the partnership to include Affimer molecules that target viruses, such as coronaviruses, in order to develop therapies that repair the lung damage caused by COVID-19 whilst also producing neutralising Affimer molecules to prevent the progression of the disease.

AffyXell will engineer mesenchymal stem cells to express SARS-COV-2 neutralising Affimer molecules in order to develop treatments for seriously ill COVID-19 patients, and will also prepare for rapid development of next-generation stem cell therapies for future infectious respiratory disease outbreaks.

Dr Alastair Smith, Chief Executive of Avacta Group commented: I am very pleased to have extended our collaboration and licence agreement with Daewoong Pharmaceutical to include the SARS-COV-2 neutralising Affimer molecules for the treatment of COVID-19 and to create the potential to respond very rapidly in future to global virus threats.

"AffyXell will address the need over the coming years for advanced stem cell therapies to treat lung damage caused by cytokine release syndrome suffered by COVID-19 patients and, at the same time, help prevent disease progression in these patients through the action of a neutralising Affimer therapy.

"More broadly, one of the key areas of unmet clinical need that AffyXell can address with its next-generation mesenchymal stem cell therapies is acute pulmonary diseases such as COPD and acute respiratory distress syndrome. We, and our partners in South Korea, are very excited by the potential to develop life improving treatments for patients with these serious respiratory diseases as well as COVID-19.

"I look forward to further updating the market on progress in our ground-breaking programmes with AffyXell in due course, and on progress with our COVID-19 diagnostics programmes shortly.

Seng-ho Jeon, CEO of Daewoong Pharmaceutical and AffyXell Therapeutics, commented: It is very encouraging that AffyXell now has the opportunity to expand the application of its next-generation stem cell platform technology to target viruses. The SARS-COV-2 neutralising Affimer to be developed in combination with AffyXells cell and gene technology is expected to be an innovative solution for COVID-19 patients suffering cytokine release syndrome.

"Daewoong has been also conducting several research programs and clinical trial for COVID-19. As the COVID-19 crisis has caused great difficulties worldwide, we will continue to focus our efforts to develop a novel therapeutic agent for COVID-19.

This announcement contains information which, prior to its disclosure, was considered inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 (MAR).

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Avacta and Daewoong Pharmaceutical expand partnership to include COVID-19 neutralising Affimer therapy - Cambridge Network

Global Cell Isolation/Cell Separation Market Industry Analysis and Forecast (2019-2026), By-Product, Cell Type, Cell Source, Technique, Application,…

Global Cell Isolation/Cell Separation Market was valued US$ XX Bn in 2018 and is expected to reach US$ 17.92 Bn by 2026, at a CAGR of around XX % during a forecast period.

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The report covers all the trends and technologies playing a major role in the growth of the Cell Isolation/Cell Separation market during the forecast period. It highlights the drivers, restraints, and opportunities expected to influence the market growth during 2019-2026.

Some of the market drivers for the cell isolation/cell separation market are increasing incidences & prevalence of chronic diseases with the aging population, technological advancement in cell isolation, growing demand for bio-pharmaceuticals, personalized medicine, and increasing stem cell research. Cell isolation or separation is a tool used to sort cells into a specific population from a heterogeneous group of cells without contamination. The use of cell isolation techniques helps to open the door of cell-based therapies and thereby improve the quality of treatment and clinical outcome.

However, the ethical issues regarding the isolation of embryonic stem cells and the high cost of cell separation instruments are expected to restrict the growth of this market during the forecast period.

Based on cell type, the human cell segment is expected to register a major revenue share in the cell isolation/cell separation market globally. Owing to increasing investments by public and private organizations for research on human cells, growing application areas of human stem cells, and the high frequency and growing incidence of diseases such as cancer.

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Based on the product, the consumables segment is expected to witness the fastest growth during the forecast period. Because of the increasing investments by companies to develop advanced products and the rising government initiatives for improving cell-based research are driving the growth of this segment.

North America region is expected to grow at a XX % rate of CAGR during the forecast period owing to increasing government support for cancer and stem cell research, the expanding biotechnology and biopharmaceutical industries and the increasing prevalence of chronic and infectious diseases in which cell isolation is required for diagnosis and treatment. Which results in, increase in demand for cell isolation products.

The objective of the report is to present a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, industry-validated market data and projections with a suitable set of assumptions and methodology. The report also helps in understanding Global Cell Isolation/Cell Separation Market dynamics, structure by identifying and analyzing the market segments and project the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. The report also provides PEST analysis, PORTERs analysis, and SWOT analysis to address the question of shareholders to prioritizing the efforts and investment in the near future to the emerging segment in Global Cell Isolation/Cell Separation Market. Scope of the Global Cell Isolation/Cell Separation Market

Global Cell Isolation/Cell Separation Market, By Product

Consumables o Reagents, Kits, Media, and Sera o Beads o Disposables Instruments o Centrifuges o Flow Cytometers o Magnetic-activated Cell Separator Systems o Filtration Systems Global Cell Isolation/Cell Separation Market, By Cell Type

Human Cells o Differentiated Cells o Stem Cells Animal Cells Global Cell Isolation/Cell Separation Market, By Cell Source

Adipose Tissue Bone Marrow Cord Blood/Embryonic Stem Cells Global Cell Isolation/Cell Separation Market, By Technique

Centrifugation-based Cell Isolation Surface Marker-based Cell Isolation Filtration-based Cell Isolation Global Cell Isolation/Cell Separation Market, By Application

Biomolecule Isolation Cancer Research Stem Cell Research Tissue Regeneration & Regenerative Medicine In Vitro Diagnostics Global Cell Isolation/Cell Separation Market, By End user

Research Laboratories and Institutes Hospitals and Diagnostic Laboratories Biotechnology and Biopharmaceutical Companies Other End Users Global Cell Isolation/Cell Separation Market, By Region

North America Europe Asia Pacific Middle East & Africa South America Key players operating in the Global Cell Isolation/Cell Separation Market

Thermo Fisher Scientific Beckman Coulter Becton, Dickinson and Company GE Healthcare Merck KgaA Miltenyi Biotech pluriSelect STEMCELL Technologies Inc. Terumo BCT Bio-Rad Laboratories Inc.

MAJOR TOC OF THE REPORT

Chapter One: Cell Isolation/Cell Separation Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Isolation/Cell Separation Market Competition, by Players

Chapter Four: Global Cell Isolation/Cell Separation Market Size by Regions

Chapter Five: North America Cell Isolation/Cell Separation Revenue by Countries

Chapter Six: Europe Cell Isolation/Cell Separation Revenue by Countries

Chapter Seven: Asia-Pacific Cell Isolation/Cell Separation Revenue by Countries

Chapter Eight: South America Cell Isolation/Cell Separation Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Isolation/Cell Separation by Countries

Chapter Ten: Global Cell Isolation/Cell Separation Market Segment by Type

Chapter Eleven: Global Cell Isolation/Cell Separation Market Segment by Application

Chapter Twelve: Global Cell Isolation/Cell Separation Market Size Forecast (2019-2026)

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Celsius Therapeutics, Twist Bioscience, And More: News From July 2020 – Bio-IT World

July 30, 2020 |July featured exciting new, products, and partnerships from around the bio-IT community from innovating companies, organizations, and universities, including Celsius Therapeutics, Twist Bioscience, and more.

Celsius Therapeuticsannounced the signing of three collaboration agreements with the University of Oxford, Cleveland Clinic, and the LMU University Hospital Munich. In each agreement, Celsius will partner with leading academic scientists to access patient tissue samples and apply its proprietary single-cell genomics platform and machine learning capabilities to discover new insights into inflammatory bowel disease (IBD). Under these collaborations, Celsius will evaluate a large number of longitudinal (both pre- and post-treatment) samples, including biopsies from patients who respond to or fail anti-TNFa therapy (University of Oxford); Crohns disease patients who develop disease recurrence following intestinal resection (Cleveland Clinic); and children with very early onset IBD (LMU University Hospital Munich). Celsius will integrate single-cell RNA sequencing data from these samples with clinical data provided by its partners to identify novel drug targets and patient stratification approaches for specific patient subsets. Under the terms of each agreement, Celsius retains the ability to incorporate both the de-identified clinical information and longitudinal single-cell genomics data generated from these studies into its large and continuously growing database. Celsius plans to combine the knowledge generated by these agreements with other work using its single-cell genomics and machine learning algorithms to elucidate this complicated disease biology. Further, Celsius will combine these insights with orthogonal data sets derived from functional genomics and other approaches to verify biological mechanisms and validate potential drug targets. Press release

Twist Bioscienceannounced a partnership withTakeda for access to proprietary phage display libraries for the discovery, validation and optimization of antibodies in Takedas pipeline of biologics for oncology, rare diseases, neuroscience, and gastroenterology. Under the terms of the agreement, Twist Biopharma, a division of Twist Bioscience, will license to Takeda its Library of Libraries, a panel of synthetic antibody phage display libraries derived only from sequences that exist in the human body. Together, the companies will work to discover, validate and optimize new antibody candidates. In return, Takeda will pay Twist annual technology licensing fees, as well as milestones and royalties for all compounds discovered from the Twist phage display libraries. We look forward to applying our unique ability to generate robust, diverse and cutting-edge libraries together with Takedas deep insight into therapeutic drug discovery and development to truly forge new frontiers in bringing personalized therapies to patients worldwide, Emily M. Leproust, CEO and co-founder of Twist Bioscience, said in an official statement. Press release

LabVantage Solutionsannounced that the newly-released 8.5 edition of its LabVantageplatform offers a fully integrated Scientific Data Management System (SDMS). LabVantage SDMS ensures security and control against data loss or modification. The optional SDMS seamlessly interfaces with all elements of the LabVantage platform, including LIMS (laboratory information management system), ELN (electronic laboratory notebook), and LES (laboratory execution system). LabVantage 8.5 also includes a new and comprehensive Configuration Management and Transfer (CMT) feature for greater LIMS administration productivity and better control, along with a number of enhancements that make the LabVantage ELN easier to use. During these challenging times, we are especially proud to release LabVantage 8.5, which represents a major advance reflecting our commitment to making the advantages of digital transformation fully accessible to our customers, said John Heiser, CEO of LabVantage, in a press release. LabVantage 8.5s fully integrated SDMS is specifically designed for the LabVantage platform and provides major benefits to laboratory managers, with the aim of making their operations more efficient, productive, and secure. Our user-friendly, all-in-one SDMS offers seamless, continuous data integrity. Importantly, it allows managers to secure all of their data, not just the final results. The integrated SDMS automatically collects and protects all instrument and other relevant laboratory data as it is generated, which provides a rich informatics and analytical resource that enables better lab management, improved quality, and enhanced security. Full integration means that all relevant lab-generated data is collected automatically and rapidly made available to laboratory staff. Importantly, integration ensures data integrity and security by eliminating the need to transfer data between system components or to leave it with the instrument system, typically a weak link in the process. Press release

The Josep Carreras Leukaemia Research Institutehas created the OneChain Immunotherapeutics (OCI)spin-off, the aim of which is to develop new immuno-oncological therapeutic tools with various preclinical candidates, based on CAR-T technology for different tumors, such as cortical T-cell acute lymphoblastic leukemia (coT-ALL), a rare subtype of leukemia that mainly affects children, and which has a poor prognosis. The spin-off is being funded initially through a round of seed capital provided by Invivo Ventures, the Ministry of Industry, through CDTI-Innvierte, and the Josep Carreras Foundation, and it clearly makes the case for bringing the research carried out by Dr. Pablo Menndez's group closer to patients. Dr. Mendez is an ICREA research professor and the scientific director at the Josep Carreras Leukaemia Research Institute's Hospital Clnic-UB Campus. "OCI is the vehicle that will enable us to take all the work we have been doing over all these years and apply it to clinical use, Dr. Menndez said in an official statement. At the academic level it is very complicated to develop these strategies and manage all the regulatory mechanisms associated with the development of a product. OCI will enable us to carry out all the necessary steps so that all our knowledge can be available to patients." Press release

VantAIand TARA Biosystemsannounced the launch of a biology-driven, AI-enabled collaboration for accelerated cardiac drug development. The partnership will leverage TARAs state-of-the-artin vitrohuman biology models and VantAIs leading computational drug discovery capabilities to identify and develop new therapies to fight cardiac disease. Combining the power of VantAIs machine learning-enabled gene-disease mapping techniques with the rich, high-fidelity phenotypic data generated from TARAsin vitrohuman cardiac disease models, TARA and VantAI will identify novel drug targets linked to cardiac disease. Then, the team will use VantAIsin silicoplatform to produce novel molecules that effectively modulate these targets, many of which have been previously considered undruggable. VantAIs platform will prioritize modality strategies and use generative approaches to design precise chemistry that is effective while also displaying favorable pharmaceutical properties. The functional response of these novel molecules will then be evaluated in TARAs induced pluripotent stem cell-derived human cardiac tissue models, which include a repertoire of healthy, gene-edited, patient-derived, and drug-induced phenotypes of human disease. The TARA models represent a highly versatile platform for capturing robust physiologic endpoints of human cardiac function, including contractility, electrophysiology, calcium signaling, structure, as well as genomic, proteomic, and metabolic profiles. These comprehensive experimental results will be fed back to VantAIsin silicoplatform to both power further refinement of lead candidates and expand the VantAI-TARA joint data graph view of cardiac disease systems. Press release

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Celsius Therapeutics, Twist Bioscience, And More: News From July 2020 - Bio-IT World

Global Covid-19 impact on Stem Cell Storage Market Insights and Upcoming Trends to Forecast till 2025| CCBC, CBR, ViaCord, Esperite, Vcanbio – Owned

Global Stem Cell Storage Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2026 (Based on 2020 COVID-19 Worldwide Spread)

Chicago, United States: The report comes out as an intelligent and thorough assessment tool as well as a great resource that will help you to secure a position of strength in the global Stem Cell Storage Market. It includes Porters Five Forces and PESTLE analysis to equip your business with critical information and comparative data about the Global Stem Cell Storage Market. We have provided deep analysis of the vendor landscape to give you a complete picture of current and future competitive scenarios of the global Stem Cell Storage market. Our analysts use the latest primary and secondary research techniques and tools to prepare comprehensive and accurate market research reports.

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Stem Cell Storage Market competition by top manufacturers/Key player Profiled: CCBC, CBR, ViaCord, Esperite, Vcanbio, Boyalife, LifeCell, Crioestaminal, RMS Regrow, Cordlife Group, PBKM FamiCord, cells4life, Beikebiotech, StemCyte, Cryo-cell, Cellsafe Biotech Group, PacifiCord, Americord, Krio, Familycord, Cryo Stemcell, Stemade Biotech

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Global Stem Cell Storage Market is estimated to reach xxx million USD in 2020 and projected to grow at the CAGR of xx% during the 2021-2026. The report analyses the global Stem Cell Storage market, the market size and growth, as well as the major market participants. The analysis includes market size, upstream situation, market segmentation, market segmentation, price & cost and industry environment. In addition, the report outlines the factors driving industry growth and the description of market channels.The report begins from overview of industrial chain structure, and describes the upstream. Besides, the report analyses market size and forecast in different geographies, type and end-use segment, in addition, the report introduces market competition overview among the major companies and companies profiles, besides, market price and channel features are covered in the report.

Segmentation by Product:

Umbilical Cord Blood Stem Cell Embryonic Stem Cell Adult Stem Cell Other

Segmentation by Application:

Diseases Therapy Healthcare

Competitive Analysis:

Global Stem Cell Storage Market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of Stem Cell Storage Market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Scope of the Report: The all-encompassing research weighs up on various aspects including but not limited to important industry definition, product applications, and product types. The pro-active approach towards analysis of investment feasibility, significant return on investment, supply chain management, import and export status, consumption volume and end-use offers more value to the overall statistics on the Stem Cell Storage Market. All factors that help business owners identify the next leg for growth are presented through self-explanatory resources such as charts, tables, and graphic images.

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North America(United States, Canada and Mexico)

Europe (Germany, France, UK, Russia and Italy)

Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia)

South America (Brazil, Argentina, Colombia)

Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

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Table of Contents

Report Overview:It includes major players of the global Stem Cell Storage Market covered in the research study, research scope, and Market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends:This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the global Stem Cell Storage Market. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the global Stem Cell Storage Market are discussed.

Market Share by Manufacturers:Here, the report provides details about revenue by manufacturers, production and capacity by manufacturers, price by manufacturers, expansion plans, mergers and acquisitions, and products, market entry dates, distribution, and market areas of key manufacturers.

Market Size by Type:This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

Market Size by Application:Besides an overview of the global Stem Cell Storage Market by application, it gives a study on the consumption in the global Stem Cell Storage Market by application.

Production by Region:Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the global Stem Cell Storage Market are profiled in this section. The analysts have provided information about their recent developments in the global Stem Cell Storage Market, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the global Stem Cell Storage Market as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the global Stem Cell Storage Market as well as for key regional markets.

Value Chain and Sales Analysis:It deeply analyzes customers, distributors, sales channels, and value chain of the global Stem Cell Storage Market.

Key Findings: This section gives a quick look at important findings of the research study.

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Global Covid-19 impact on Stem Cell Storage Market Insights and Upcoming Trends to Forecast till 2025| CCBC, CBR, ViaCord, Esperite, Vcanbio - Owned

Trending Now: Stem Cell Source Market Growth by Top Companies, Trends by Types and Application, Forecast to 2026| BD Bioscience, Beckman Coulter, Ge…

LOS ANGELES, United States: QY Research has recently published a report, titled Global Stem Cell Source Market Research Report 2020. The research report gives the potential headway openings that prevails in the global market. The report is amalgamated depending on research procured from primary and secondary information. The global Stem Cell Source market is relied upon to develop generously and succeed in volume and value during the predicted time period. Moreover, the report gives nitty gritty data on different manufacturers, region, and products which are important to totally understanding the market.

Key Companies/Manufacturers operating in the global Stem Cell Source market include: , BD Bioscience, Beckman Coulter, Ge Healthcare, Merck Millipore, Miltenyi Biotec, Pluriselect Life Science, Sigma-Aldrich Corporation, Stemcell Technologies, Terumo BCT, Thermo Fisher ScientificCompany 13

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Segmental Analysis

Both developed and emerging regions are deeply studied by the authors of the report. The regional analysis section of the report offers a comprehensive analysis of the global Stem Cell Source market on the basis of region. Each region is exhaustively researched about so that players can use the analysis to tap into unexplored markets and plan powerful strategies to gain a foothold in lucrative markets.

Global Stem Cell Source Market Segment By Type:

Reagent Instrument Others

Global Stem Cell Source Market Segment By Application:

Hospital Biotechnology Research Center Others

Competitive Landscape

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the global Stem Cell Source market.

Key companies operating in the global Stem Cell Source market include , BD Bioscience, Beckman Coulter, Ge Healthcare, Merck Millipore, Miltenyi Biotec, Pluriselect Life Science, Sigma-Aldrich Corporation, Stemcell Technologies, Terumo BCT, Thermo Fisher ScientificCompany 13

Key questions answered in the report:

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TOC

1 Stem Cell Source Market Overview 1.1 Product Overview and Scope of Stem Cell Source 1.2 Stem Cell Source Segment by Type 1.2.1 Global Stem Cell Source Sales Growth Rate Comparison by Type (2021-2026) 1.2.2 Reagent 1.2.3 Instrument 1.2.4 Others 1.3 Stem Cell Source Segment by Application 1.3.1 Stem Cell Source Sales Comparison by Application: 2020 VS 2026 1.3.2 Hospital 1.3.3 Biotechnology Research Center 1.3.4 Others 1.4 Global Stem Cell Source Market Size Estimates and Forecasts 1.4.1 Global Stem Cell Source Revenue 2015-2026 1.4.2 Global Stem Cell Source Sales 2015-2026 1.4.3 Stem Cell Source Market Size by Region: 2020 Versus 2026 1.5 Stem Cell Source Industry 1.6 Stem Cell Source Market Trends 2 Global Stem Cell Source Market Competition by Manufacturers 2.1 Global Stem Cell Source Sales Market Share by Manufacturers (2015-2020) 2.2 Global Stem Cell Source Revenue Share by Manufacturers (2015-2020) 2.3 Global Stem Cell Source Average Price by Manufacturers (2015-2020) 2.4 Manufacturers Stem Cell Source Manufacturing Sites, Area Served, Product Type 2.5 Stem Cell Source Market Competitive Situation and Trends 2.5.1 Stem Cell Source Market Concentration Rate 2.5.2 Global Top 5 and Top 10 Players Market Share by Revenue 2.5.3 Market Share by Company Type (Tier 1, Tier 2 and Tier 3) 2.6 Manufacturers Mergers & Acquisitions, Expansion Plans 2.7 Primary Interviews with Key Stem Cell Source Players (Opinion Leaders) 3 Stem Cell Source Retrospective Market Scenario by Region 3.1 Global Stem Cell Source Retrospective Market Scenario in Sales by Region: 2015-2020 3.2 Global Stem Cell Source Retrospective Market Scenario in Revenue by Region: 2015-2020 3.3 North America Stem Cell Source Market Facts & Figures by Country 3.3.1 North America Stem Cell Source Sales by Country 3.3.2 North America Stem Cell Source Sales by Country 3.3.3 U.S. 3.3.4 Canada 3.4 Europe Stem Cell Source Market Facts & Figures by Country 3.4.1 Europe Stem Cell Source Sales by Country 3.4.2 Europe Stem Cell Source Sales by Country 3.4.3 Germany 3.4.4 France 3.4.5 U.K. 3.4.6 Italy 3.4.7 Russia 3.5 Asia Pacific Stem Cell Source Market Facts & Figures by Region 3.5.1 Asia Pacific Stem Cell Source Sales by Region 3.5.2 Asia Pacific Stem Cell Source Sales by Region 3.5.3 China 3.5.4 Japan 3.5.5 South Korea 3.5.6 India 3.5.7 Australia 3.5.8 Taiwan 3.5.9 Indonesia 3.5.10 Thailand 3.5.11 Malaysia 3.5.12 Philippines 3.5.13 Vietnam 3.6 Latin America Stem Cell Source Market Facts & Figures by Country 3.6.1 Latin America Stem Cell Source Sales by Country 3.6.2 Latin America Stem Cell Source Sales by Country 3.6.3 Mexico 3.6.3 Brazil 3.6.3 Argentina 3.7 Middle East and Africa Stem Cell Source Market Facts & Figures by Country 3.7.1 Middle East and Africa Stem Cell Source Sales by Country 3.7.2 Middle East and Africa Stem Cell Source Sales by Country 3.7.3 Turkey 3.7.4 Saudi Arabia 3.7.5 U.A.E 4 Global Stem Cell Source Historic Market Analysis by Type 4.1 Global Stem Cell Source Sales Market Share by Type (2015-2020) 4.2 Global Stem Cell Source Revenue Market Share by Type (2015-2020) 4.3 Global Stem Cell Source Price Market Share by Type (2015-2020) 4.4 Global Stem Cell Source Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End 5 Global Stem Cell Source Historic Market Analysis by Application 5.1 Global Stem Cell Source Sales Market Share by Application (2015-2020) 5.2 Global Stem Cell Source Revenue Market Share by Application (2015-2020) 5.3 Global Stem Cell Source Price by Application (2015-2020) 6 Company Profiles and Key Figures in Stem Cell Source Business 6.1 BD Bioscience 6.1.1 Corporation Information 6.1.2 BD Bioscience Description, Business Overview and Total Revenue 6.1.3 BD Bioscience Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.1.4 BD Bioscience Products Offered 6.1.5 BD Bioscience Recent Development 6.2 Beckman Coulter 6.2.1 Beckman Coulter Corporation Information 6.2.2 Beckman Coulter Description, Business Overview and Total Revenue 6.2.3 Beckman Coulter Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.2.4 Beckman Coulter Products Offered 6.2.5 Beckman Coulter Recent Development 6.3 Ge Healthcare 6.3.1 Ge Healthcare Corporation Information 6.3.2 Ge Healthcare Description, Business Overview and Total Revenue 6.3.3 Ge Healthcare Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.3.4 Ge Healthcare Products Offered 6.3.5 Ge Healthcare Recent Development 6.4 Merck Millipore 6.4.1 Merck Millipore Corporation Information 6.4.2 Merck Millipore Description, Business Overview and Total Revenue 6.4.3 Merck Millipore Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.4.4 Merck Millipore Products Offered 6.4.5 Merck Millipore Recent Development 6.5 Miltenyi Biotec 6.5.1 Miltenyi Biotec Corporation Information 6.5.2 Miltenyi Biotec Description, Business Overview and Total Revenue 6.5.3 Miltenyi Biotec Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.5.4 Miltenyi Biotec Products Offered 6.5.5 Miltenyi Biotec Recent Development 6.6 Pluriselect Life Science 6.6.1 Pluriselect Life Science Corporation Information 6.6.2 Pluriselect Life Science Description, Business Overview and Total Revenue 6.6.3 Pluriselect Life Science Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.6.4 Pluriselect Life Science Products Offered 6.6.5 Pluriselect Life Science Recent Development 6.7 Sigma-Aldrich Corporation 6.6.1 Sigma-Aldrich Corporation Corporation Information 6.6.2 Sigma-Aldrich Corporation Description, Business Overview and Total Revenue 6.6.3 Sigma-Aldrich Corporation Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.4.4 Sigma-Aldrich Corporation Products Offered 6.7.5 Sigma-Aldrich Corporation Recent Development 6.8 Stemcell Technologies 6.8.1 Stemcell Technologies Corporation Information 6.8.2 Stemcell Technologies Description, Business Overview and Total Revenue 6.8.3 Stemcell Technologies Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.8.4 Stemcell Technologies Products Offered 6.8.5 Stemcell Technologies Recent Development 6.9 Terumo BCT 6.9.1 Terumo BCT Corporation Information 6.9.2 Terumo BCT Description, Business Overview and Total Revenue 6.9.3 Terumo BCT Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.9.4 Terumo BCT Products Offered 6.9.5 Terumo BCT Recent Development 6.10 Thermo Fisher ScientificCompany 13 6.10.1 Thermo Fisher ScientificCompany 13 Corporation Information 6.10.2 Thermo Fisher ScientificCompany 13 Description, Business Overview and Total Revenue 6.10.3 Thermo Fisher ScientificCompany 13 Stem Cell Source Sales, Revenue and Gross Margin (2015-2020) 6.10.4 Thermo Fisher ScientificCompany 13 Products Offered 6.10.5 Thermo Fisher ScientificCompany 13 Recent Development 7 Stem Cell Source Manufacturing Cost Analysis 7.1 Stem Cell Source Key Raw Materials Analysis 7.1.1 Key Raw Materials 7.1.2 Key Raw Materials Price Trend 7.1.3 Key Suppliers of Raw Materials 7.2 Proportion of Manufacturing Cost Structure 7.3 Manufacturing Process Analysis of Stem Cell Source 7.4 Stem Cell Source Industrial Chain Analysis 8 Marketing Channel, Distributors and Customers 8.1 Marketing Channel 8.2 Stem Cell Source Distributors List 8.3 Stem Cell Source Customers 9 Market Dynamics 9.1 Market Trends 9.2 Opportunities and Drivers 9.3 Challenges 9.4 Porters Five Forces Analysis 10 Global Market Forecast 10.1 Global Stem Cell Source Market Estimates and Projections by Type 10.1.1 Global Forecasted Sales of Stem Cell Source by Type (2021-2026) 10.1.2 Global Forecasted Revenue of Stem Cell Source by Type (2021-2026) 10.2 Stem Cell Source Market Estimates and Projections by Application 10.2.1 Global Forecasted Sales of Stem Cell Source by Application (2021-2026) 10.2.2 Global Forecasted Revenue of Stem Cell Source by Application (2021-2026) 10.3 Stem Cell Source Market Estimates and Projections by Region 10.3.1 Global Forecasted Sales of Stem Cell Source by Region (2021-2026) 10.3.2 Global Forecasted Revenue of Stem Cell Source by Region (2021-2026) 10.4 North America Stem Cell Source Estimates and Projections (2021-2026) 10.5 Europe Stem Cell Source Estimates and Projections (2021-2026) 10.6 Asia Pacific Stem Cell Source Estimates and Projections (2021-2026) 10.7 Latin America Stem Cell Source Estimates and Projections (2021-2026) 10.8 Middle East and Africa Stem Cell Source Estimates and Projections (2021-2026) 11 Research Finding and Conclusion 12 Methodology and Data Source 12.1 Methodology/Research Approach 12.1.1 Research Programs/Design 12.1.2 Market Size Estimation 12.1.3 Market Breakdown and Data Triangulation 12.2 Data Source 12.2.1 Secondary Sources 12.2.2 Primary Sources 12.3 Author List 12.4 Disclaimer

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Trending Now: Stem Cell Source Market Growth by Top Companies, Trends by Types and Application, Forecast to 2026| BD Bioscience, Beckman Coulter, Ge...

Covid-19 Impact on Global Stem Cell Drugs Market by Technology, Solutions, Application, Price, Demand Analysis and Growth Opportunities to 2026 -…

The report on Stem Cell Drugs, gives an in-depth analysis of Global Stem Cell Drugs Market based on aspects that are very important for the market study. Factors like production, market share, revenue rate, regions and key players define a market study start to end. This report gives an overview of market valued in the year 2019 and its growth in the coming years till 2026. The report is based on the in-depth view of Stem Cell Drugs industry on the basis of market growth, market size, development plans and opportunities offered by the global Stem Cell Drugs market. The energetic aspects studied in this report include SWOT analysis, feasibility and forecast information.

Request a sample of Stem Cell Drugs Market report @ https://www.orbispharmareports.com/sample-request/42149

For the consumers to gain the in-depth analysis of the global Stem Cell Drugs market and further growth of the market, the report offers significant statistics and information. Stem Cell Drugs report studies the current state of the market to analyze the future opportunities and risks. Stem Cell Drugs report provides a 360-degree global market state. Primarily, the report delivers Stem Cell Drugs introduction, overview, market objectives, market definition, scope, and market size valuation.

Major companies of this report:

JCR Pharmaceuticals Co., Ltd MEDIPOST Anterogen CORESTEM, Inc New York Blood Center Pharmicell Co., Ltd Takeda Chiesi Pharmaceuticals

Make an enquiry of this report @ https://www.orbispharmareports.com/enquiry-before-buying/42149

Moreover, the report provides historical information with future forecast over the forecast period. Some of the important aspects analyzed in the report includes market share, production, key regions, revenue rate as well as key players. This Stem Cell Drugs report also provides the readers with detailed figures at which the Stem Cell Drugs market was valued in the historical year and its expected growth in upcoming years. Besides, analysis also forecasts the CAGR at which the Stem Cell Drugs is expected to mount and major factors driving markets growth. The study on global Stem Cell Drugs market, offers deep insights about the Stem Cell Drugs market covering all the crucial aspects of the market.

Stem Cell Drugs Market Segmentation by Type:

Cord Blood-Derived Adipose-Derived Bone Marrow-Derived Others

In addition, the report include deep dive analysis of the market, which is one of the most important features of the market. Furthermore, the need for making an impact is likely to boost the demand for the experts which are working in the market. Moreover, an in depth analysis of the competitors is also done to have an estimate for the market. The Stem Cell Drugs market has its impact all over the globe. On global level Stem Cell Drugs industry is segmented on the basis of product type, applications, and regions. It also focusses on market dynamics, Stem Cell Drugs growth drivers, developing market segments and the market growth curve is offered based on past, present and future market data. The industry plans, news, and policies are presented at a global and regional level.

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About Us :

At Orbispharma we curate the most relevant news stories, features, analysis and research reports on the important challenges undertaken by the pharmaceutical and related sectors. Our editorial philosophy is to bring you sharp, focused and informed perspective of industries, the end users and application of all upcoming trends into the pharma sector. Orbispharma believes in conversations that can bring a change in one of the most crucial economic sectors in the world. With these conversations we wish our customers to make sound business decisions with right business intelligence.

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Covid-19 Impact on Global Stem Cell Drugs Market by Technology, Solutions, Application, Price, Demand Analysis and Growth Opportunities to 2026 -...

Global Cell Expansion Market, Forecast to 2025 – Increasing Incidence of Chronic Diseases and Government Investments for Cell-based Research – Yahoo…

DUBLIN, July 30, 2020 /PRNewswire/ -- The "Cell Expansion Market by Product (Reagent, Media, Flow Cytometer, Centrifuge, Bioreactor), Cell Type (Human, Animal), Application (Regenerative Medicine & Stem Cell Research, Cancer & Cell-based Research), End-User, and Region - Global Forecast to 2025" report has been added to ResearchAndMarkets.com's offering.

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The global cell expansion market is projected to reach USD 30.06 billion by 2025 from an estimated USD 14.91 billion in 2020, at a CAGR of 15.1%.

The report segments the cell expansion market based on region (Asia Pacific, Europe, North America, and RoW), product (consumables and instruments), cell type (human cells and animal cells), application (regenerative medicine and stem cell research, cancer and cell-based research and other applications), and end user (research institutes, biotechnology and biopharmaceutical companies, cell banks, and other end users). The report also provides a comprehensive review of market drivers, restraints, and opportunities in the cell expansion market.

Increasing incidence of chronic diseases and government investments for cell-based research are set to drive the cell expansion market

Growth in this market is largely driven by the increasing incidence of chronic diseases, government investments for cell-based research, growing focus on personalized medicine, increasing focus on R&D for cell-based therapies, and increasing GMP certifications for cell therapy production facilities. On the other hand, ethical concerns regarding research in cell biology are expected to limit market growth to a certain extent in the coming years.

By instruments type, the cell expansion supporting equipment accounted for the fasted growing product segment of the cell expansion market

The instruments segment includes cell expansion supporting equipment, bioreactors, and automated cell expansion systems. The cell expansion supporting equipment market includes flow cytometers, cell counters and hemocytometers, centrifuges, and other supporting equipment. They are used in cell culture processes for isolating, culturing, scaling-up, and extracting biological products. These instruments are essential in laboratories and institutes for conducting research and analyzing the cell structure and function for cell therapy research.

By cell type, the human cells segment accounted for the largest share of the cell expansion market

Based on cell type, the cell expansion market is segmented into human cells and animal cells. The human cells segment includes stem cells and differentiated cells. The stem cells segment is further classified into adult stem cells, ESCs, and iPSCs. The human cells segment accounted for the larger share of the cell expansion market majorly due to the increasing investments by public and private organizations for research on human cells, growing application areas of human stem cells, and the growing incidence of diseases such as cancer.

Asia Pacific: The fastest-growing region in the cell expansion market

The Asia Pacific market is projected to grow at the highest CAGR during the forecast period, mainly due to the increasing focus of players on emerging Asian markets, increasing incidence of chronic and infectious diseases, rising geriatric population, and government initiatives for infrastructural improvements of healthcare facilities are driving the growth of the cell expansion market in this region.

North America: the largest share of the cell expansion market

North America accounted for the largest share of the cell expansion market. The large share of this segment can primarily be attributed to the rising incidence of cancer, increasing government funding, rising research activates on stem cell therapies, growing awareness regarding advanced treatment methods, growing geriatric population, and the strong presence of industry players in the region.

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Global Cell Expansion Market, Forecast to 2025 - Increasing Incidence of Chronic Diseases and Government Investments for Cell-based Research - Yahoo...

UW researchers devise approach to treat rare, incurable form of blindness – University of Wisconsin-Madison

Scientists at the University of WisconsinMadison have published a proof-of-concept method to correct an inherited form of macular degeneration that causes blindness, and that is currently untreatable.

Andrew Hellpap 608-225-5024 ahellpap@uwhealth.org

The researchers were able to correct the disease in stem cells from patients with BEST1 mutations by overwhelming broken copies of the gene with many functional copies of BEST1. This approach worked for most, but not all, of the BEST1 mutations that they tested. As an alternative approach for mutations that did not respond to this gene augmentation method, the team used CRISPR-Cas9 gene editing to target and correct the mutations.

A paper chronicling the research, co-led by David Gamm, MD, PhD, professor of ophthalmology and visual sciences in the School of Medicine and Public Health, was published online July 23 in the American Journal of Human Genetics. The study was also led by Kris Saha, PhD, associate professor of biomedical engineering and Wisconsin Institute for Discovery, and Bikash Pattnaik, PhD, assistant professor of pediatrics.

This BEST1 gene encodes a protein that regulates the movement of chloride across a layer of the retina called the retinal pigment epithelium (RPE). Best disease is dominant, meaning that people who inherit only one faulty copy of the BEST1 gene from either their mother or their father will develop the disorder. Mutations in BEST1 cause the retinal layer to break down, resulting in blurred central vision that progresses to irreversible vision loss.

People with Best disease have a wide range of mutations that can affect different parts of the protein, all of which were thought to require complex, individualized gene therapies to fix them, Gamm said. We found that many of these mutations were actually very sensitive to a broader gene therapy method that is already established for other retinal diseases.

Fixing a dominant genetic disease via gene therapy typically requires precise removal or repair of the nonfunctional gene without causing harm to the functional gene a difficult task that is frequently unsuccessful. In contrast, recessive genetic diseases that arise when a person inherits two nonfunctional genes one from each parent can be corrected by a technique called gene augmentation. This well-established process introduces a functional copy of the gene to fill the void.

To use another analogy, dominant mutations produce workers that actively look to sabotage the efforts of their capable coworkers, whereas recessive mutations produce proteins that never show up for work at all, Gamm said. As it turns out, the latter situation is usually simpler to treat than the former.

A team of researchers at the McPherson Eye Research Institute, which Gamm directs, hypothesized that it may be possible to adequately dilute the influence of the nonfunctional BEST1 protein by counter-balancing it with many functional copies of BEST1 protein through gene augmentation.

In the lab, the approach worked in RPE cells derived from induced pluripotent stem cells of patients with most, but not all, of the BEST1 gene mutations they tested. Where gene augmentation did not succeed, the team was able to correct the dysfunction using CRISPR-Cas9 gene editing.

The research was carried out in large part by Divya Sinha, PhD, an assistant scientist in Gamms lab, Ben Steyer, a former MD-PhD student in Sahas lab, and Pawan Shahi, PhD, postdoctoral research associate in Pattnaiks lab. The research team also included Sushmita Roy, PhD, associate professor of biostatics and medical informatics at the UW School of Medicine and Public Health and Wisconsin Institute for Discovery.

The scientists demonstrated that their two-pronged gene therapy strategy may hold potential to treat all Best disease mutations in a highly effective manner.

We were able to reverse the disease in all the cell lines using one method or the other, Gamm said. We were also able to determine which mutations were likely to respond to the first-line gene augmentation strategy, and which would be better served with the second-line gene editing approach.

An additional benefit came into focus as this research progressed, according to Gamm.

Our findings also could be applicable to some dominant genetic mutations that affect tissues elsewhere in the body, he said. Its very exciting.

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UW researchers devise approach to treat rare, incurable form of blindness - University of Wisconsin-Madison