Optimized Tandem CAR T-Cell Therapy Targeted CD19/CD20 Appears Feasible in B-NHL – Targeted Oncology

Optimized tandem CD19/CD20 chimeric antigen receptor (CAR) T-cell therapy induced potent and durable anti-tumor responses as treatment of patients with relapsed/refractory B cell non-Hodgkin lymphoma (B-NHL) with good control of cytokine release syndrome (CRS) and CAR T-related encephalopathy syndrome, according to a phase 1/2 clinical trial presented in a poster at the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Program.

After a median of 13.5 months of follow-up (IQR, 33.2-3.3), 84% of patients had an objective response, and 74% had a complete response (CR). The duration of overall response rate (ORR) at 6 months was 94% and 74% at 12 months.

Both the median progression-free and overall survivals had not been reached for patients at the time of data cut-off. At 6 months, the progression-free survival rate was 76%, and at 12 months, it was 59%.

Sixty-two patients experienced CRS (71%), which was grade 1 or 2 in 61% of patients and grade 3 or greater in 10%. The median time to the onset of CRS after infusion was 1 day (range, 1-5). The median duration of CRS was 6 days (range, 1-9). Investigators also noted that the median time to the onset of grade 3 CRS was 1 day (range, 1-2).

The most common adverse events within 1 month of the IV infusion were leukopenia, pyrexia, and anorexia. Only 2 patients (2%) experienced CAR T-cell-related encephalopathy syndrome of grade 3 severity.

Three treatment-related deaths occurred in the study, 2 due to pulmonary infection and 1 due to deposition of CAR T cells in pulmonary alveoli.

Ninety-nine patients were screened for the study, of which 87 received the infusion and 74 were followed for at least 3 months before the data cutoff date. Patients underwent leukapheresis and conditioning chemotherapy, which was followed by a single intravenous infusion of tandem CD19/20 CAR T cells on day 0 at a dose of 0.5x106x106 per kg of body weight.

The majority of patients in the study were under the age of 60 years (82%) and female (53%). Overall, 62% of patients had an ECOG performance status of 0 or 1 versus 38% who had a 2. Upon study entry, 85% had stage III or IV disease and 15% had stage I or II. Patients were diagnosed with either diffuse large B-cell lymphoma (66%), follicular lymphoma (15%), transformed follicular lymphoma (7%), primary mediastinal B-cell lymphoma (6%) or other (6%).

Fifty-six percent of patients had 3 to 5 prior lines of anti-neoplastic therapy, while 27% had 2 or less and 17% had 6 or more. The majority of patients had a lesion diameter less than 10 cm (78%) and had tumor burden SPD of 100 cm2 or more (55%). Eighty percent of the patients were refractory, 20% had relapsed to second-line or later therapy, 14% became refractory after stem cell transplant, and 10% had relapsed after a prior CD19 CAR-T cell therapy.

To be eligible for the study, patients had to be between the ages of 16 and 70 years, and they could not have received prior anti-CD20 monoclonal antibody and anthracycline treatment. Patients had to have an ECOG performance status of 0 to 2 with a life expectancy greater than 3 months and adequate organ function to enroll in the study. Patients also had to have measurable disease according to the IWG Response Criteria for Malignant Lymphoma.

Patients who had a CR with no evidence of disease were ineligible to enroll. If they had definite involvement of the gastrointestinal tract, negative tumor puncture detection in both CD19 and CD20, or had serious uncontrolled medical disorders or active infections, they also could not enroll. Patients were also excluded from the study if they were deemed unsuitable for the trial based on clinical judgement or were pregnant or lactating.

CD19-targeted CAR T cells have been highly effective in the treatment landscape of hematologic malignancies, but the recurrence rate appears high, which is a major obstacle to durable remissions with this therapy. This study aimed to evaluate the safety and tolerability of intravenous tandem CD19/20 CAR T cells among patients with relapsed/refractory NHL.

Secondary objectives of this study also included assessment of efficacy of the study treatment defined by ORR and evaluation of the duration of overall response, progression-free and overall survival. An exploratory objective of the study was to determine in vivo expansion and persistence of the Tandem CD19/CD20 CAR T cells.

The rationale for this study was to address the high recurrence rate observed with CAR T-cell therapy, which often prevents durable remission after treatment. Overall the optimized tandem CAR T treatment appeared feasible in patients with B-NHL. Although many of the patients in the study had heavy tumor burden, were in poor physical condition, or had highly aggressive characteristics, they were still able to achieve a satisfactory ORR and CR rate with the tandem CD19/CD20 CAR-engineered T-cell therapy.

Reference

Ja-Jing Z, Yao W, Zhi-Qiang Wu, et al. Safety and Efficacy of Optimized Tandem CD19/CD20 CAR-Engineered T Cells in Patients with Relapsed/Refractory Non-Hodgkin Lymphoma. J Clin Oncol. 38: 2020 (suppl; abstr 3034). doi: 10.1200/JCO.2020.38.15_suppl.3034

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Optimized Tandem CAR T-Cell Therapy Targeted CD19/CD20 Appears Feasible in B-NHL - Targeted Oncology

ReNeuron encouraged by progress in stroke and RP treatments – Proactive Investors UK

What the company does

Human retinal progenitor cells (hRPC)

Human retinal progenitor cells differentiate into components of the retina.

Reneuron has developed the ability to scale up the manufacturing of hRPCs using a patented low-oxygen cell expansion technology.

The hRPC cell therapy candidate is being evaluated in an ongoing phase I/IIa clinical trial in the US in subjects with a blindness-causing inherited retinal disease, retinitis pigmentosa (RP).

CTX Cells

CTX cell therapy candidate is a treatment for patients left disabled by the effects of a stroke.

Reneurons product is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting with the diseases targeted, without the need for additional immunosuppressive drug treatments.

Data from the Phase II PISCES trial indicated CTX therapy was safe and well-tolerated and produced clinically meaningful and sustained improvement in the level of disability and dependence as well as motor function.

Exosome platform

Exosomes are nanoparticles, released by cells, and contain a number of active proteins and micro RNAs, which are short non-coding RNAs capable of regulating gene expression, that arebelieved to play a key role in cell-to-cell communication.

ExoPr0, Reneurons first CTX-derived exosome therapeutic candidate, has demonstrated potential as both a novel therapeutic candidate as well as a drug delivery vehicle

hRPC

's ()human retinal progenitor cells (hRPC) have scored some early success.

A Phase I/II assessment of a very small group of sufferers of a blindness-causing disease called retinitis pigmentosa saw a significant improvement in vision after treatment.

Six months after treatment there was a mean improvement of 18.5per treated eye, with a mean improvement of 12 letters per treated eye after nine months, whereasinexorable disease progression is the norm for this disease.

With a total of 22 patients now treated and the study still ongoing, ReNeuron said the efficacy in subsequent patients was seen but at a lower rate and magnitude, with improvement in visual acuity ranging from +5 to +11 letters in the treated eye threemonths after treatment.

In February, clinicaldata from the PISCES II clinical trial were published in peer-reviewedJournal of Neurology, Neurosurgery, and Psychiatry.

CTX

A peer journal review published in May indicated a CTX human neural stem cell line can rescue deficits associated with an accepted animal model of Huntington's disease, a progressive genetic brain disorder.

ReNeuron has previously presented data demonstrating that its CTX stem cell line, currently undergoing clinical evaluation for the treatment of stroke disability, can cause functional and behavioural recovery in animal models of ischemic (restriction of blood supply) injury.

The new data showed that implantation of CTX cells into a model of Huntington's disease can reduce inflammation, glial scar formation and induce host neurogenesis (the generation of new brain cells) leading to a recovery in behavioural deficits.

Coronavirus

In April, Reneuron said it haddeveloped a line of the human exosomes that can deliver a medically relevant payload: Viral vaccines thatmight help in the fight against coronavirus.

The stem cell specialist added that the disruption from lockdowns would inevitably lead to delays in the recruitment of patients for trials of its treatments for stroke disability and retinitis pigmentosa (RP).

It said it will update on how this will affect the release of top-line data from the two studies once it knows the full impact of the restrictions.

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ReNeuron encouraged by progress in stroke and RP treatments - Proactive Investors UK

NexImmune to Present at the Jefferies Virtual Healthcare Conference – GlobeNewswire

GAITHERSBURG, Md., June 01, 2020 (GLOBE NEWSWIRE) -- NexImmune, a clinical-stage biopharma company advancing a new generation of nanoparticle-based therapies for targeted immune system response, today announced thatJohn Trainer, Chief Financial Officer, will present a company overview at theJefferies Virtual Healthcare ConferenceonTuesday, June 2, 2020at3:30 p.m. Eastern time.

About NexImmunes Lead T Cell Therapy Programs

NexImmunes two lead T cell therapy programs, NEXI-001 and NEXI-002, are in Phase 1/2 clinical trials for the treatment of relapsed AML after allogeneic stem cell transplantation and multiple myeloma refractory to >3 prior lines of therapy, respectively. The Company expects initial data in the fourth quarter of 2020.The Companys pipeline also consists of four additional preclinical programs, including cell therapy and injectable product candidates for the treatment of oncology, autoimmune diseases, and infectious diseases.

About NexImmune

NexImmune is a Gaithersburg, MD-based clinical-stage biopharma company advancing a new generation of immunotherapies based on its proprietary artificial immune modulation (AIM) technology. The AIM platform is designed to generate a targeted T cell-mediated immune response and is initially being developed as a cell therapy for the treatment of hematologic cancers. AIM nano-particles (AIM-np) act as synthetic dendritic cells to deliver immune-specific signals to targeted T cells and can direct the activation or suppression of cell-mediated immunity. In cancer, AIM-expanded T cells have demonstrated best-in-class anti-tumor properties, including the ability to address key mechanisms of tumor escape and relapse through a unique combination of anti-tumor potency, multi-antigen target-specific killing, and long-term T cell persistence. The modular design of the AIM platform enables rapid expansion across multiple therapeutic areas (autoimmune diseases and infectious diseases), with both cell therapy and injectable products. For more information, visit http://www.neximmune.com.

Media Contact:Mike BeyerSam Brown Inc. Healthcare Communications312-961-2502mikebeyer@sambrown.com

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Global Stem Cell Therapy Market 2020 Research Report Insights and Analysis, Forecast to 2026 – 3rd Watch News

The Stem Cell Therapy market has witnessed growth from USD XX million to USD XX million from 2014 to 2019. With the CAGR of X.X%, this market is estimated to reach USD XX million in 2026.

The report mainly studies the size, recent trends and development status of the Stem Cell Therapy market, as well as investment opportunities, government policy, market dynamics (drivers, restraints, opportunities), supply chain and competitive landscape. Technological innovation and advancement will further optimize the performance of the product, making it more widely used in downstream applications. Moreover, Porters Five Forces Analysis (potential entrants, suppliers, substitutes, buyers, industry competitors) provides crucial information for knowing the Stem Cell Therapy market.

Download PDF Sample of Stem Cell Therapy Market report @ https://www.arcognizance.com/enquiry-sample/1030514

Major Players in the global Stem Cell Therapy market include:, Holostem Terapie Avanzate, Osiris Therapeutics, NuVasive, BIOTIME, Advanced Cell Technology, Caladrius, Pharmicell, JCR Pharmaceuticals, RTI Surgical, AlloSource, MEDIPOST, Anterogen, BrainStorm Cell Therapeutics

On the basis of types, the Stem Cell Therapy market is primarily split into:, Autologous, Allogeneic

On the basis of applications, the market covers:, Musculoskeletal disorders, Wounds and injuries, Cardiovascular diseases, Surgeries, Gastrointestinal diseases, Other applications

Brief about Stem Cell Therapy Market Report with [emailprotected] https://www.arcognizance.com/report/global-stem-cell-therapy-market-report-2019-competitive-landscape-trends-and-opportunities

Geographically, the report includes the research on production, consumption, revenue, market share and growth rate, and forecast (2014-2026) of the following regions:, United States, Europe (Germany, UK, France, Italy, Spain, Russia, Poland), China, Japan, India , Southeast Asia (Malaysia, Singapore, Philippines, Indonesia, Thailand, Vietnam), Central and South America (Brazil, Mexico, Colombia), Middle East and Africa (Saudi Arabia, United Arab Emirates, Turkey, Egypt, South Africa, Nigeria), Other Regions

Chapter 1 provides an overview of Stem Cell Therapy market, containing global revenue, global production, sales, and CAGR. The forecast and analysis of Stem Cell Therapy market by type, application, and region are also presented in this chapter.

Chapter 2 is about the market landscape and major players. It provides competitive situation and market concentration status along with the basic information of these players.

Chapter 3 provides a full-scale analysis of major players in Stem Cell Therapy industry. The basic information, as well as the profiles, applications and specifications of products market performance along with Business Overview are offered.

Chapter 4 gives a worldwide view of Stem Cell Therapy market. It includes production, market share revenue, price, and the growth rate by type.

Chapter 5 focuses on the application of Stem Cell Therapy, by analyzing the consumption and its growth rate of each application.

Chapter 6 is about production, consumption, export, and import of Stem Cell Therapy in each region.

Chapter 7 pays attention to the production, revenue, price and gross margin of Stem Cell Therapy in markets of different regions. The analysis on production, revenue, price and gross margin of the global market is covered in this part.

Chapter 8 concentrates on manufacturing analysis, including key raw material analysis, cost structure analysis and process analysis, making up a comprehensive analysis of manufacturing cost.

Chapter 9 introduces the industrial chain of Stem Cell Therapy. Industrial chain analysis, raw material sources and downstream buyers are analyzed in this chapter.

Chapter 10 provides clear insights into market dynamics.

Chapter 11 prospects the whole Stem Cell Therapy market, including the global production and revenue forecast, regional forecast. It also foresees the Stem Cell Therapy market by type and application.

Chapter 12 concludes the research findings and refines all the highlights of the study.

Chapter 13 introduces the research methodology and sources of research data for your understanding.

Years considered for this report:, Historical Years: 2014-2018, Base Year: 2019, Estimated Year: 2019, Forecast Period: 2019-2026,

Some Point of Table of Content:

Chapter One: Stem Cell Therapy Market Overview

Chapter Two: Global Stem Cell Therapy Market Landscape by Player

Chapter Three: Players Profiles

Chapter Four: Global Stem Cell Therapy Production, Revenue (Value), Price Trend by Type

Chapter Five: Global Stem Cell Therapy Market Analysis by Application

Chapter Six: Global Stem Cell Therapy Production, Consumption, Export, Import by Region (2014-2019)

Chapter Seven: Global Stem Cell Therapy Production, Revenue (Value) by Region (2014-2019)

Chapter Eight: Stem Cell Therapy Manufacturing Analysis

Chapter Nine: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter Ten: Market Dynamics

Chapter Eleven: Global Stem Cell Therapy Market Forecast (2019-2026)

Chapter Twelve: Research Findings and Conclusion

Chapter Thirteen: Appendix continued

List of tablesList of Tables and FiguresFigure Stem Cell Therapy Product PictureTable Global Stem Cell Therapy Production and CAGR (%) Comparison by TypeTable Profile of AutologousTable Profile of AllogeneicTable Stem Cell Therapy Consumption (Sales) Comparison by Application (2014-2026)Table Profile of Musculoskeletal disordersTable Profile of Wounds and injuriesTable Profile of Cardiovascular diseasesTable Profile of SurgeriesTable Profile of Gastrointestinal diseasesTable Profile of Other applicationsFigure Global Stem Cell Therapy Market Size (Value) and CAGR (%) (2014-2026)Figure United States Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Europe Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Germany Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure UK Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure France Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Italy Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Spain Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Russia Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Poland Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure China Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Japan Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure India Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Southeast Asia Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Malaysia Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Singapore Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Philippines Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Indonesia Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Thailand Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Vietnam Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Central and South America Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Brazil Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Mexico Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Colombia Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Middle East and Africa Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Saudi Arabia Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure United Arab Emirates Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Turkey Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Egypt Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure South Africa Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Nigeria Stem Cell Therapy Revenue and Growth Rate (2014-2026)Figure Global Stem Cell Therapy Production Status and Outlook (2014-2026)Table Global Stem Cell Therapy Production by Player (2014-2019)Table Global Stem Cell Therapy Production Share by Player (2014-2019)Figure Global Stem Cell Therapy Production Share by Player in 2018Table Stem Cell Therapy Revenue by Player (2014-2019)Table Stem Cell Therapy Revenue Market Share by Player (2014-2019)Table Stem Cell Therapy Price by Player (2014-2019)Table Stem Cell Therapy Manufacturing Base Distribution and Sales Area by PlayerTable Stem Cell Therapy Product Type by PlayerTable Mergers & Acquisitions, Expansion PlansTable Holostem Terapie Avanzate ProfileTable Holostem Terapie Avanzate Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table Osiris Therapeutics ProfileTable Osiris Therapeutics Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table NuVasive ProfileTable NuVasive Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table BIOTIME ProfileTable BIOTIME Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table Advanced Cell Technology ProfileTable Advanced Cell Technology Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table Caladrius ProfileTable Caladrius Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table Pharmicell ProfileTable Pharmicell Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table JCR Pharmaceuticals ProfileTable JCR Pharmaceuticals Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table RTI Surgical ProfileTable RTI Surgical Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table AlloSource ProfileTable AlloSource Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table MEDIPOST ProfileTable MEDIPOST Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table Anterogen ProfileTable Anterogen Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table BrainStorm Cell Therapeutics ProfileTable BrainStorm Cell Therapeutics Stem Cell Therapy Production, Revenue, Price and Gross Margin (2014-2019)Table Global Stem Cell Therapy Production by Type (2014-2019)Table Global Stem Cell Therapy Production Market Share by Type (2014-2019)Figure Global Stem Cell Therapy Production Market Share by Type in 2018Table Global Stem Cell Therapy Revenue by Type (2014-2019)Table Global Stem Cell Therapy Revenue Market Share by Type (2014-2019)Figure Global Stem Cell Therapy Revenue Market Share by Type in 2018Table Stem Cell Therapy Price by Type (2014-2019)Figure Global Stem Cell Therapy Production Growth Rate of Autologous (2014-2019)Figure Global Stem Cell Therapy Production Growth Rate of Allogeneic (2014-2019)Table Global Stem Cell Therapy Consumption by Application (2014-2019)Table Global Stem Cell Therapy Consumption Market Share by Application (2014-2019)Table Global Stem Cell Therapy Consumption of Musculoskeletal disorders (2014-2019)Table Global Stem Cell Therapy Consumption of Wounds and injuries (2014-2019)Table Global Stem Cell Therapy Consumption of Cardiovascular diseases (2014-2019)Table Global Stem Cell Therapy Consumption of Surgeries (2014-2019)Table Global Stem Cell Therapy Consumption of Gastrointestinal diseases (2014-2019)Table Global Stem Cell Therapy Consumption of Other applications (2014-2019)Table Global Stem Cell Therapy Consumption by Region (2014-2019)Table Global Stem Cell Therapy Consumption Market Share by Region (2014-2019)Table United States Stem Cell Therapy Production, Consumption, Export, Import (2014-2019)Table Europe Stem Cell Therapy Production, Consumption, Export, Import (2014-2019)Table China Stem Cell Therapy Production, Consumption, Export, Import (2014-2019)Table Japan Stem Cell Therapy Production, Consumption, Export, Import (2014-2019)Table India Stem Cell Therapy Production, Consumption, Export, Import (2014-2019)Table Southeast Asia Stem Cell Therapy Production, Consumption, Export, Import (2014-2019)Table Central and South America Stem Cell Therapy Production, Consumption, Export, Import (2014-2019)continued

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NOTE: Our report does take into account the impact of coronavirus pandemic and dedicates qualitative as well as quantitative sections of information within the report that emphasizes the impact of COVID-19.

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Cell Expansion Market Worth $39.7 Billion by 2027 l CAGR 9.4%: Grand View Research, Inc. – PRNewswire

SAN FRANCISCO, June 3, 2020 /PRNewswire/ --The global cell expansion marketsize is expected to reach USD 39.7 billion by 2027 registering a CAGR of 9.4%, according to a new report by Grand View Research, Inc. Cell expansion techniques are increasingly employed for the development of cellular and gene therapies from a single cord blood collection. These techniques can also be used for the expansion of stored Stem Cells (SCs) for the development of cancer therapies. Therefore, significant developments in cord blood SCs expansion technologies are expected to boost market growth.

Key suggestions from the report:

Read 170 page research report with ToC on "Cell Expansion Market Size, Share & Trends Analysis Report By Product (Instruments, Consumables), By Cell Type (Mammalian, Animal), By Application, By End Use, And Segment Forecasts, 2020 - 2027" at: https://www.grandviewresearch.com/industry-analysis/cell-expansion-market

Companies have made heavy investments for the expansion of tissue-engineered products and the development of biologics. For instance, in March 2019, Merck KGaA invested USD 168 million for the expansion of its biologics manufacturing facility in Switzerland. Such initiatives are expected to boost the demand for solutions required for biologic development, thereby leading to market growth.

Bioreactors are fundamental tools in this market. Extensive research studies related to the applications of bioreactor engineering approaches have led to the incorporation of novel culture technologies. Moreover, the combined use of automated bioreactors with the microcarrier technology leads to an efficient expansion and enrichment of the cancer SCs. As a result, these approaches have gained immense traction in this market.

Grand View Research has segmented the global cell expansion market on the basis of product, cell type, application, end use, and region:

Find more research reports on Biotechnology Industry, by Grand View Research:

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About Grand View Research

Grand View Research, U.S.-based market research and consulting company, provides syndicated as well as customized research reports and consulting services. Registered in California and headquartered in San Francisco, the company comprises over 425 analysts and consultants, adding more than 1200 market research reports to its vast database each year. These reports offer in-depth analysis on 46 industries across 25 major countries worldwide. With the help of an interactive market intelligence platform, Grand View Research helps Fortune 500 companies and renowned academic institutes understand the global and regional business environment and gauge the opportunities that lie ahead.

Contact:Sherry JamesCorporate Sales Specialist, USAGrand View Research, Inc.Phone: 1-415-349-0058Toll Free: 1-888-202-9519Email: [emailprotected] Web: https://www.grandviewresearch.com Follow Us: LinkedIn | Twitter

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Cell Expansion Market Worth $39.7 Billion by 2027 l CAGR 9.4%: Grand View Research, Inc. - PRNewswire

CSL Behring and Seattle Children’s Research Institute to Advance Gene Therapy Treatments for Primary Immunodeficiency Diseases | DNA RNA and Cells |…

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 03 June 2020 09:39Hits: 93

Initially, the alliance will develop treatment options for patients with two rare, life-threatening primary immunodeficiency diseases -- Wiskott-Aldrich Syndrome (WAS) and X-linked Agammaglobulinemia (XLA)

SEATTLE, WA and KING of PRUSSIA, PA, USA I June 2, 2020 I Seattle Children's Research Institute, one of the top pediatric research institutions in the world, and global biotechnology leader CSL Behring announced a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases.

Initially, the alliance will focus on the development of treatment options for patients with two rare, life-threatening primary immunodeficiency diseases -- Wiskott-Aldrich Syndrome and X-linked Agammaglobulinemia. These are two of more than 400 identified primary immunodeficiency diseases in which a part of the body's immune system is missing or functions improperly.

"CSL Behring will collaborate with Seattle Children's experts to apply our novel gene therapy technology to their research pipeline, with an aim to address unmet needs for people living with certain rare primary immunodeficiency diseases," said Bill Mezzanotte, MD, Executive Vice President, Head of Research and Development for CSL Behring. "Expanding our gene therapy portfolio into an area of immunology well known to CSL exemplifies how we are strategically growing our capabilities in this strategic scientific platform and are collaborating with world class institutions to access innovation with the potential to vastly improve patients' lives."

"Stem cell gene therapies that correct the genetic abnormality driving a child's disease will transform the therapeutic options for children with Wiskott-Aldrich Syndrome, X-Linked Agammaglobulinemia and other immunodeficiency diseases,"said David J. Rawlings, MD, director of the Center for Immunity and Immunotherapies and division chief of immunology at Seattle Children's, and a professor of pediatrics and immunology at the University of Washington School of Medicine."The collaboration with CSL Behring supports our longstanding research programs for pediatric immunodeficiency diseases and will accelerate this research toward clinical trials, helping get these innovations to the children who need them."

CSL Behring researchers, working with researchers from Seattle Children's Research Institute, will investigate applying the proprietary platform technologies, Select+ and Cytegrity, to several pre-clinical gene therapy programs. These technologies, which have broad applications in ex vivo stem cell gene therapy, are designed to address some of the major challenges associated with the commercialization of stem cell therapy, including the ability to manufacture consistent, high-quality products, and to improve engraftment, efficacy and tolerability.

Wiskott-Aldrich Syndrome (WAS) has an estimated incidence between one and 10 cases per million males worldwide, according to the National Institutes of Health. WAS patients' immune systems function abnormally, making them susceptible to infections. They also experience eczema, autoimmunity and difficulty forming blood clots, leaving them vulnerable to life threatening bleeding complications. Today the only knowncurefor WAS is a stem cell transplant, if a suitable donor can be found.

X-linked Agammaglobulinemia (XLA) is another rare primary immunodeficiency in which patients have low levels of immunoglobulins (also known as antibodies), which are key proteins made by the immune system to help fight infections. Like WAS, XLA affects males almost exclusively, although females can be genetic carriers of the condition. While no cure exists for XLA, the goal of treatment is to boost the immune system by replacing missing antibodies and preventing or aggressively treating infections that occur, according to the Immune Deficiency Foundation.

About Seattle Children's

Seattle Children's mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children's Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top children's hospitals in the country by U.S. News & World Report, Seattle Children's serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho the largest region of any children's hospital in the country. As one of the nation's top five pediatric research centers, Seattle Children's Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Children's Hospital and Research Foundation works with the Seattle Children's Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. Join Seattle Children's bold initiative It Starts With Yes: The Campaign for Seattle Children's to transform children's health for generations to come.

For more information, visit seattlechildrens.org or follow us on Twitter, Facebook, Instagram or on our On the Pulse blog.

About CSL Behring

CSL Behring is a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 26,000 people, and delivers its life-saving therapies to people in more than 70 countries. For more information, visit http://www.cslbehring.com and for inspiring stories about the promise of biotechnology, visit Vita http://www.cslbehring.com/Vita.

SOURCE: CSL Behring

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CSL Behring and Seattle Children's Research Institute to Advance Gene Therapy Treatments for Primary Immunodeficiency Diseases | DNA RNA and Cells |...

Toronto centre solving cell manufacturing challenges to benefit patients and global industry CCRM and Cytiva, formerly part of GE Healthcare Life…

TORONTO and MARLBOROUGH, Mass., June 02, 2020 (GLOBE NEWSWIRE) -- With Health Canada and the Food and Drug Administration beginning to approve and reimburse cell and gene therapies in significant numbers, the demand for cell and viral vector manufacturing will continue to grow. Consequently, the industrialization challenges associated with the variability of cell and gene therapies, and with manufacturing them on a commercial scale, must be overcome. CCRM and Cytiva, formerly part of GE Healthcare Life Sciences, have renewed their Collaboration Agreement for continued operation of the Centre for Advanced Therapeutic Cell Technologies (CATCT), which was created to accelerate the development and adoption of cell manufacturing technologies for novel regenerative medicine-based therapies.

Together, CCRM and Cytiva have established a commercialization hub where great minds, state-of-the-art equipment and a spirit of innovation meet, says Michael May, President and CEO of CCRM. Continuing to partner in the operation of CATCT will enable us to move the cell and gene therapy industry closer to fulfilling its promise of creating cures, and enabling treatments to get to patients.

By creating an innovative platform and approach to tackle the issues facing commercialization of living therapies, we are supporting the viability of the regenerative medicine industry, says Catarina Flyborg, Vice President, Cell & Gene Therapy, Cytiva. In CATCT, we are creating the technologies, processes and equipment that will enable our customers, and the broader industry, to achieve its goals and help patients.

Established in 2016, CATCT is a partnership between CCRM and Cytiva, with initial funding from the Federal Economic Development Agency for Southern Ontario (FedDev Ontario). Its staff of 40 works in a 10,000 ft (~930 m) process development facility, located in the MaRS Discovery District, next to Torontos world-leading hospitals and the University of Toronto.

The global regenerative medicine market was valued at US$23.8 billion (2018), and it is anticipated to grow to US$151 billion by 2026 with an annual growth rate of 26.1 per cent.i Operating CATCT allows CCRM and Cytiva to address the manufacturing bottlenecks that would otherwise have the potential to impede the industrys growth.

CATCTs key areas of expertise are:

The work conducted in CATCT can be categorized as follows: the first is fee-for-service development projects that advance customers therapeutic technologies towards industrialization; second, the teams New Product Introductions (NPIs) efforts provide core biological expertise in Cytivas product development process; finally, internal technology development builds additional capabilities and innovative solutions for cell and gene therapies.

A recent success stemming from the work being done in CATCT is the involvement of CCRM and Cytiva in a consortium led by iVexSol Canada, with conditional funding from Next Generation Manufacturing Canada (NGen), to build an advanced manufacturing platform for lentiviral vectors. As core partners in this consortium, which was announced in August 2019, CCRM will provide supporting manufacturing infrastructure and downstream processing capabilities, and Cytiva will share expertise of manufacturing processes, and access to and use of specialized tools and technology.

The new collaboration agreement between CCRM and Cytiva has a three-year term and it became effective on October 15, 2019. The funding will be a combination of in-kind contributions, milestone payments, reinvested fee-for-service revenue and any successful grant opportunities. FedDevs funding of CATCT was for a three-year term and ended in December 2018.

About CCRM CCRM, a Canadian not-for-profit organization funded by the Government of Canada, the Province of Ontario, and leading academic and industry partners, supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding, and infrastructure. CCRM is the commercialization partner of the Ontario Institute for Regenerative Medicine and the University of Torontos Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About CytivaCytiva is a 3.3 billion USD global life sciences leader with nearly 7,000 associates operating in 40 countries dedicated to advancing and accelerating therapeutics. As a trusted partner to customers that range in scale and scope, Cytiva brings speed, efficiency and capacity to research and manufacturing workflows, enabling the development, manufacture and delivery of transformative medicines to patients. Visit http://www.cytiva.com for more.

For more information, please contact:

Stacey JohnsonDirector, Communications and Marketing, CCRM416-946-8869stacey.johnson@ccrm.ca

Colleen ConnollySenior Communications Manager, Cytiva774-245-3893Colleen.Connolly@cytiva.com

ihttps://www.fortunebusinessinsights.com/industry-reports/regenerative-medicine-market-100970

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Toronto centre solving cell manufacturing challenges to benefit patients and global industry CCRM and Cytiva, formerly part of GE Healthcare Life...

Professor Wolf Reik appointed acting director of the Babraham Institute – Cambridge Independent

Professor Wolf Reik has been appointed acting director of the Babraham Institute.

It follows the death of Professor Michael Wakelam, who died from suspected a Covid-19 infection on March 31.

Prof Reik has been the institutes associate director since 2004 and has headed up its epigenetics research programme since 2008.

Prof Peter Rigby, chair of the institutes board of trustees, said: Prof Reik is a world-class scientist, internationally renowned for his work in epigenetics, who has been at the Institute for over 30 years.

The Biotechnology and Biological Sciences Research Council (BBSRC), which funds the institute, approved of the move, he said.

The BBSRC fully support the board's appointment, which will ensure the institute continues to be strongly led, building on the excellent work of Prof Michael Wakelam. I know that Wolf will provide much needed leadership and stability during the uncertain times that we all face, said Prof Rigby.

Prof Reik added: I am really honoured by this appointment; I look forward to working with everyone at the Institute, the campus and with BBSRC.

After Michaels sad death, my primary aim is to bring us back to our labs in a safe and considerate fashion, and to jointly tackle the opportunities and challenges for the science of the Institute going forward strongly into the future.

The study of epigenetics explores the set of instructions that alter how our genome behaves - by regulating gene expression - without changing our underlying DNA code.

Prof Reik explores the role of epigenetics in establishing cell fate and identity during mammalian development and also the process of epigenetic reprogramming.

From the earliest steps in human development, to how stem cells maintain their pluripotency - that is, their ability to change into different cells - Prof Reiks lab is interested in some fundamental questions.

It also explores how the identity of cells is established during the process of differentiation, through which they change into all the different types of cells in our bodies.

Recently, the lab has been studying how the epigenome degrades with age - and whether there are ways of reversing this decay.

New technologies for single cell multi-omics sequencing, which allows unprecedented insights into cell fate changes during development or ageing, have been developed by the lab.

Prof Reik has an interest in collaboration both inside and outside the institute and leads a Wellcome-funded consortium studying cell fate decisions during mouse gastrulation and organ development.

He obtained his MD in 1985 from the University of Hamburg, where he undertook thesis work with Rudolf Jaenisch before completing postdoctoral work with Azim Surani at the Institute of Animal Physiology, which is now the Babraham Institute. During this spell, he became a fellow of the Lister Institute of Preventive Medicine which, in 1987, provided funding for him to start his own independent research group.

He is honorary professor of epigenetics and affiliate faculty at the Stem Cell Institute at the University of Cambridge and associate faculty at the Wellcome Sanger Institute. A member of EMBO and the Academia Europaea, a fellow of the Academy of Medical Sciences and the Royal Society, he has also been a member of funding committees such as UKRI-Medical Research Council, Cancer Research UK and Wellcome Trust.

Read more

How Wolf Reik is unravelling life's other set of instructions at the Babraham Institute

How to build a human: Babraham Institute to unlock secrets of early human development

How Babraham Institute's study of nematode worms can help us understand human ageing

Babraham Institute director Professor Michael Wakelam dies after suspected coronavirus infection

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Professor Wolf Reik appointed acting director of the Babraham Institute - Cambridge Independent

30,000-cell Study Maps the Development of Sound Sensing in the Mouse Inner Ear – Technology Networks

A team of researchers has generated a developmental map of a key sound-sensing structure in the mouse inner ear. Scientists at the National Institute on Deafness and Other Communication Disorders (NIDCD), part of the National Institutes of Health, and their collaborators analyzed data from 30,000 cells from mouse cochlea, the snail-shaped structure of the inner ear. The results provide insights into the genetic programs that drive the formation of cells important for detecting sounds. The study also sheds light specifically on the underlying cause of hearing loss linked to Ehlers-Danlos syndrome and Loeys-Dietz syndrome.

The study data is shared on a unique platform open to any researcher, creating an unprecedented resource that could catalyze future research on hearing loss. Led by Matthew W. Kelley, Ph.D., chief of the Section on Developmental Neuroscience at the NIDCD, the study appeared online in Nature Communications(link is external). The research team includes investigators at the University of Maryland School of Medicine, Baltimore; Decibel Therapeutics, Boston; and Kings College London.

Unlike many other types of cells in the body, the sensory cells that enable us to hear do not have the capacity to regenerate when they become damaged or diseased, said NIDCD Director Debara L. Tucci, M.D., who is also an otolaryngology-head and neck surgeon. By clarifying our understanding of how these cells are formed in the developing inner ear, this work is an important asset for scientists working on stem cell-based therapeutics that may treat or reverse some forms of inner ear hearing loss.

In mammals, the primary transducers of sound are hair cells, which are spread across a thin ribbon of tissue (the organ of Corti) that runs the length of the coiled cochlea. There are two kinds of hair cells, inner hair cells and outer hair cells, and they are structurally and functionally sustained by several types of supporting cells. During development, a pool of nearly identical progenitor cells gives rise to these different cell types, but the factors that guide the transformation of progenitors into hair cells are not fully understood.

To learn more about how the cochlea forms, Kelleys team took advantage of a method called single-cell RNA sequencing. This powerful technique enables researchers to analyze the gene activity patterns of single cells. Scientists can learn a lot about a cell from its pattern of active genes because genes encode proteins, which define a cells function. Cells gene activity patterns change during development or in response to the environment.

There are only a few thousand hair cells in the cochlea, and they are arrayed close together in a complex mosaic, an arrangement that makes the cells hard to isolate and characterize, said Kelley. Single-cell RNA sequencing has provided us with a valuable tool to track individual cells behaviors as they take their places in the intricate structure of the developing cochlea.

Building on their earlier work on 301 cells, Kelleys team set out to examine the gene activity profiles of 30,000 cells from mouse cochleae collected at four time points, beginning with the 14th day of embryonic development and ending with the seventh postnatal day. Collectively, the data represents a vast catalog of information that researchers can use to explore cochlear development and to study the genes that underlie inherited forms of hearing impairment.

Kelleys team focused on one such gene, Tgfbr1, which has been linked to two conditions associated with hearing loss, Ehlers-Danlos syndrome and Loeys-Dietz syndrome. The data showed that Tgfbr1 is active in outer hair cell precursors as early as the 14th day of embryonic development, suggesting that the gene is important for initiating the formation of these cells.

To explore Tgfbr1s role, the researchers blocked the Tgfbr1 proteins activity in cochleae from 14.5-day-old mouse embryos. When they examined the cochleae five days later, they saw fewer outer hair cells compared to the embryonic mouse cochleae that had not been treated with the Tgfbr1 blocker. This finding suggests that hearing loss in people with Tgfbr1 mutations could stem from impaired outer hair cell formation during development.

The study revealed additional insights into the early stages of cochlear development. The developmental pathways of inner and outer hair cells diverge early on; researchers observed distinct gene activity patterns at the earliest time point in the study, the 14th day of embryonic development. This suggests that the precursors from which these cells derive are not as uniform as previously believed. Additional research on cells collected at earlier stages is needed to characterize the initial steps in the formation of hair cells.

In the future, scientists may be able to use the data to steer stem cells toward the hair cell lineage, helping to produce the specialized cells they need to test cell replacement approaches for reversing some forms of hearing loss. The studys results also represent a valuable resource for research on the hearing mechanism and how it goes awry in congenital forms of hearing loss.

The authors have made their data available through the gEAR portal(link is external) (gene Expression Analysis Resource), a web-based platform for sharing, visualizing, and analyzing large multiomic datasets. The portal is maintained by Ronna Hertzano, M.D., Ph.D., and her team in the Department of Otorhinolaryngology and the Institute for Genome Sciences (IGS)(link is external) at the University of Maryland School of Medicine.

Single-cell RNA sequencing data are highly complex and typically require significant skill to access, said Hertzano. By disseminating this study data via the gEAR, we are creating an encyclopedia of the genes expressed in the developing inner ear, transforming the knowledge base of our field and making this robust information open and understandable to biologists and other researchers.

This news release describes a basic research finding. Basic research increases our understanding of human behavior and biology, which is foundational to advancing new and better ways to prevent, diagnose, and treat disease. Science is an unpredictable and incremental process; each research advance builds on past discoveries, often in unexpected ways. Most clinical advances would not be possible without the knowledge gained through basic research.

Reference: Kolla, L., Kelly, M. C., Mann, Z. F., Anaya-Rocha, A., Ellis, K., Lemons, A., Palermo, A. T., So, K. S., Mays, J. C., Orvis, J., Burns, J. C., Hertzano, R., Driver, E. C., & Kelley, M. W. (2020). Characterization of the development of the mouse cochlear epithelium at the single cell level. Nature Communications, 11(1), 116. https://doi.org/10.1038/s41467-020-16113-y

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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30,000-cell Study Maps the Development of Sound Sensing in the Mouse Inner Ear - Technology Networks

Stem Cell Therapy Market Analysis and Demand 2017 2025 – Cole of Duty

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Analysis and Demand 2017 2025 - Cole of Duty