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Can Stem Cell Therapy Repair Damaged Knees?

In recent years, stem cell therapy has been hailed as a miracle cure for many conditions, from wrinkles to spinal repair. In animal studies, stem cell treatments have shown promise for various diseases, including heart disease, Parkinsons disease and muscular dystrophy.

Stem cell therapy could also potentially treat osteoarthritis (OA) of the knee. In OA, the cartilage covering the ends of the bones starts to deteriorate and wear away. As the bones lose this protective covering, they start to rub against one another. This leads to pain, swelling, and stiffness and, ultimately, loss of function and mobility.

Millions of people in the United States live with OA of the knee. Many manage their symptoms through exercise, weight loss, medical treatments, and lifestyle modification.

If symptoms become severe, total knee replacement is an option. Over 600,000 people a year undergo this operation in the United States alone. Yet stem cell therapy can be an alternative to surgery.

The human body is constantly manufacturing stem cells in the bone marrow. Based on certain conditions and signals in the body, stem cells are directed to where they are needed.

A stem cell is an immature, basic cell that has not yet developed to become, say, a skin cell or a muscle cell or a nerve cell. There are different types of stem cells that the body can use for different purposes.

There is evidence that stem cell treatments work by triggering damaged tissues in the body to repair themselves. This is often referred to as regenerative therapy.

However, research into stem cell treatment for OA of the knee is somewhat limited, and the results of studies are mixed.

The American College of Rheumatology and the Arthritis Foundation (ACR/AF) do not currently recommend stem cell treatment for OA of the knee, for the following reasons:

Currently, the Food & Drug Administration (FDA) considers stem cell treatment investigational. Until additional studies can demonstrate a clear benefit from stem cell injections, people who opt for this treatment must pay for them on their own and must understand that the treatment may not work.

That said, as researchers learn more about this type of treatment, it could one day become a viable option for the treatment of OA.

The cartilage covering the ends of the bones enables the bones to glide smoothly against one another with only slight friction. OA causes damage to the cartilage and leads to increased friction resulting in pain, inflammation, and ultimately, a loss of mobility and function.

In theory, stem cell therapy uses the bodys own healing mechanisms to help repair and slow the deterioration of body tissues, such as cartilage.

Stem cell therapy for knees aims to:

In simple terms, treatment involves:

Several studies have concluded that stem cell therapy improves arthritis symptoms of the knee. While overall results are promising, more research is needed to discover:

Stem cell treatment for knees is noninvasive, and studies suggest that side effects are minimal.

After the procedure, some people may experience temporary increased pain and swelling. However, the overwhelming majority of people who get stem cell injections have no adverse side effects.

The procedure uses stem cells that come from your own body. In theory, this dramatically reduces the risk of any serious side effects. However, there are various ways of harvesting and processing the stem cells, which likely affects the various success rates of the published studies.

Before receiving any treatment, its best to:

Despite conflicting evidence about whether stem cell injections work, many clinics offer them as an option for the treatment of arthritic knee pain.

Since stem cell treatment for arthritic knee pain is still considered investigational by the FDA, the treatment is not yet standardized and there is no limit to what doctors and clinics can charge.

The cost can be several thousands of dollars per knee and most insurance companies do not cover the treatment.

If OA is causing knee pain or affecting your mobility, the ACR/AF recommend the following options:

If these dont work or become ineffective, a total knee replacement surgery may be an option. Knee replacement surgery is a very common operation that can greatly improve mobility, decrease pain, and significantly improve quality of life.

Research into stem cell therapy for treatment of osteoarthritic knee pain is ongoing. Some research has shown promising results and it may one day become an accepted treatment option. For now, it remains costly and experts remain cautiously optimistic.

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Can Stem Cell Therapy Repair Damaged Knees?

COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle – BioPharma-Reporter.com

In mid-August, the US Food and Drug Administrations (FDA) Oncologic Drugs Advisory Committee voiced support for the efficacy of remestemcel-L in children with steroid-resistant graft-versus-host (GvHD) disease. The positive vote moved Mesoblast a step closer to winning approval for the mesenchymal stem cell therapy.

While seeking approval in that long-targeted indication, Mesoblast is also working to show the cell therapy is effective in patients with acute respiratory distress syndrome (ARDS) caused by infection with SARS-CoV-2.

Talking to investors on a fourth quarter results conference call late last week, Mesoblast CEO Silviu Itescu explained how the potential size of the COVID-19 market opportunity creates a substantial challenge.

We ... have to be prepared to substantially scale up manufacturing ... to be in a position next year to make sufficient quantity of product to start to meet some of this unmet need. We are able to implement proprietary xeno-free technologies and we certainly have plans to move into 3D bioreactors to allow us to have sufficient capability to meet this large unmet need, said Itescu.

Like many organizations targeting COVID-19, including groups such as AstraZeneca and Regeneron Pharmaceuticals that have large in-house operations, Mesoblast is planning to partner to gain the scale needed to manufacture the quantities of remestemcel-L it may need.

Mesoblast is currently running a phase 3 trial of remestemcel-L in ARDS. Itescu is assuming that the company will be entering into a strategic partnership for manufacturing commercialization to serve the ARDS indication.

The need for remestemcel-L in ARDS will depend on the progress of COVID-19 vaccines, which could significantly reduce the number of people suffering the complication of SARS-CoV-2 infection, and the strength of the phase 3 data.

Itescu explained the rationale for developing remestemcel-L in COVID-19 on the conference call. Like GvHD, a hyperactive immune response, known as a cytokine storm, is implicated in ARDS. In ARDS the cytokine storm manifests in severe inflammation of the lungs.

Remestemcel-L has shown anti-inflammatory effects during its development in GvHD. In addition, there is evidence the cell therapy migrates to the lungs after intravenous administration, suggesting it will accumulate in the part of the body where it is needed most in ARDS,

US physicians administered remestemcel-L to ventilator-dependent patients under a compassionate use program earlier this year. Nine of the 12 patients were taken off ventilator support, after 10 days in median, and later discharged from the hospital.

The evidence to support the use of remestemcel-L in COVID-19 led Mesoblast to start a 300-subject clinical trial. Mesoblast is assessing the effect of remestemcel-L on mortality after 30 days and is set to hold a series of interim analyses as increasing percentages of participants reach that point. If the data link remestemcel-L to improved survival, Mesoblast will seek expedited regulatory approval.

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COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle - BioPharma-Reporter.com

Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and Metagenomi, a gene editing company discovering breakthrough systems for curing genetic disease, today announced that Vor will evaluate the potential use of Metagenomis gene editing technology to develop engineered hematopoietic stem cell-based therapies for the treatment of blood cancers, such as acute myeloid leukemia.

Cancer patients deserve therapies with strong effects on cancer cells and minimal effects on all other cells, said Tirtha Chakraborty, Ph.D., Vors VP and Head of Research. Our new partnership with Metagenomi will help us achieve this goal by engineering hematopoietic stem cells using precise yet flexible gene editing thereby ensuring that targeted therapies can live up to their name."

The collaboration is non-exclusive and applies to pre-clinical research only. Further terms of the agreement are not being disclosed.

This partnership unites two transformative technologies our proprietary gene editing enzymes, and Vors platform for engineering hematopoietic stem cells such that they are inherently treatment-resistant, said Brian C. Thomas, Metagenomis CEO and co-founder. We are excited to be working together to bring both of these cutting-edge approaches into the clinic.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

About Metagenomi

Metagenomi is harnessing the vast information found in life on Earth to develop cures for genetic disease. Using proprietary data collected from around the world, Metagenomi has developed novel gene editing tools that enable next-generation gene and cell therapies.

Metagenomi is based out of Emeryville, California, and was founded by pioneers in the field of metagenomics, Jill Banfield and Brian C. Thomas. Metagenomi generates massive quantities of data from natural environments, producing complete genomes from organisms that are otherwise unknown. Metagenomi then unlocks the information captured in these genomes to develop game-changing in vivo and ex vivo therapeutics.

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Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies - Business Wire

Optimized Freezing Solutions for Clinical Application of Cell Therapy Products – Technology Networks

AMSBIO has announced new additions and certifications for its range of clinical grade, chemically defined cryopreservation excipient solutions STEM-CELLBANKER and HSC-BANKER.

STEM-CELLBANKER DMSO Free GMP grade is a new chemically defined freezing solution that does not contain DMSO as an anti-freezing agent. It was developed for customers who prefer not to use DMSO-containing cryopreservation solution due to the intended application of the samples. STEM-CELLBANKER DMSO Free GMP grade is manufactured in compliance with JPN, EU, US, and PIC/S GMP guidelines.

STEM-CELLBANKER is a chemically defined freezing media optimized for stem cells and iPS cells storage, as well as fragile primary cells. Published data supports its ability to cryopreserve organoids and tissues to allow the recovery of viable cells. STEM-CELLBANKER GMP grade is manufactured in compliance with JPN, EU, US, and PIC/S GMP guidelines. Free from animal derived components this popular cryopreservation medium contains only chemically defined USP, EP and JP grade ingredients. Available in both DMSO containing and DMSO-Free formulations, STEM-CELLBANKER is an optimal freezing solution for basic research and is finding widespread use in the clinical application of cell therapy products.

Manufactured to be completely free of serum and animal derived components, HSC-BANKER contains only European or US Pharmacopoeia graded ingredients making it suitable for storage of hematopoietic stem cells developed for cell therapy applications.

Recently the master files of HSC-BANKER were accepted by the Center for Biologics Evaluation and Research (CBER) within the US FDA (Food and Drug Administration). Master files are submissions to the FDA used to provide confidential, detailed information about facilities, processes, or articles used in the manufacturing, processing, packaging, and storing of human drug products. Beneficially they allow researchers to reference material without disclosing Master file contents to those parties.

HSC-BANKER is supplied ready-to-use and requires no special devices, such as a controlled rate freezer, in order to achieve consistently high viabilities following resuscitation from cryopreservation, even over extended long-term storage. HSC-BANKER significantly increases cell viability while maintaining cell pluripotency, normal karyotype and proliferation ability after freeze-thaw. Evaluated for endotoxins, pH, osmolarity and mycoplasma contaminants to ensure GMP equivalent quality. HSC-BANKER is part of the CELLBANKER range of cryopreservation media for cells, organoids and tissues.

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Optimized Freezing Solutions for Clinical Application of Cell Therapy Products - Technology Networks

New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem…

Impact of New Tissue Stem Cell Counting Algorithms

BOSTON (PRWEB) September 01, 2020

Stem cell biotechnology company, Asymmetrex, has been counting tissue stem cells like those used for bone marrow and cord blood transplantation therapies for a few years now. Recently, the company announced the issue of patents for its first-in-kind technology both in the U.S. and the U.K. However, until last Friday, August 28, Asymmetrex had not reported in the peer-reviewed academic literature how it achieves this feat that had been pursued by many distinguished labs for more than six decades.

Now in a report published in a special issue of OBM Transplantation, a peer-review journal for transplantation medicine research, Asymmetrex completes its introduction of the new technology to the fields of stem cell science and stem cell medicine. The report is the second invited article published in a special issue focused on the Isolation and Characterization of Adult Therapeutic Cells.

The new report describes Asymmetrexs discovery of mathematical formulas, call algorithms, that can be used to determine the number of stem cells in complex tissue cell preparations, like experimental samples or patient treatments. The stem cell counting algorithms are specific for different types of tissue stem cells. So, the algorithms defined for blood stem cells are distinct from the algorithms for liver stem cells, or lung stem cells. Once an algorithm is defined by the Asymmetrex technology, it can be used repeatedly as a simple, rapid, and inexpensive test to determine the quantity and dosage of its specific tissue stem cell type.

Asymmetrexs founder and director, James L. Sherley, M.D., Ph.D., anticipated the August publication of the new algorithms in a talk given earlier at the 6th Annual Perinatal Stem Cell Society Congress in March of this year. Then and now, he says that he believes, Now that the tissue stem cell counting algorithms are available, everything will change in stem cell science and medicine.

Prior to Asymmetrexs technology, there was no method for counting tissue stem cells in research, medicine, or for any other of their many uses. So, the impact of the stem cell counting algorithms in research and medicine is far-reaching. Such information is a game changer for accelerating progress in stem cell science and stem cell medicine, including improving treatments like gene therapy whose success depends on targeting tissue stem cells. There will also be tremendous gains in cell biomanufacturing, drug development, and environmental toxicology, all whose capabilities are currently limited by the lack of a facile means to quantify tissue stem cells.

To make the new counting technology readily accessible for evaluation by the greater academic, medical, and industrial stem cell communities, Asymmetrex provides free tissue stem cell counting on its company website.

About Asymmetrex

Asymmetrex, LLC is a Massachusetts life sciences company with a focus on developing technologies to advance stem cell medicine. The companys U.S. and U.K. patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of effective use of human adult tissue stem cells for regenerative medicine and drug development. Asymmetrex markets the first technology for determination of the dose and quality of tissue stem cell preparations (the AlphaSTEM Test) for use in stem cell transplantation therapies and pre-clinical drug evaluations. Asymmetrex is a member company of the Advanced Regenerative Manufacturing Institute BioFabUSA and the Massachusetts Biotechnology Council.

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New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem...

Global Cord Blood Banking Market 2020 with Analysis of 44 Industry Players – PRNewswire

DUBLIN, Sept. 2, 2020 /PRNewswire/ -- The "Global Cord Blood Banking Industry Report 2020" report has been added to ResearchAndMarkets.com's offering.

This report presents the number of cord blood units stored in inventory by the largest cord blood banks worldwide and the number of cord blood units (CBUs) released by registries across the world for hematopoietic stem cell (HSC) transplantation. Although cord blood is now used to treat more than 80 different diseases, this number could substantially expand if applications related to regenerative medicine start receiving approvals in major healthcare markets worldwide.

From the early 1900s through the mid-2000s, the global cord blood banking industry expanded rapidly, with companies opening for business in all major markets worldwide. From 2005 to 2010, the market reached saturation and stabilized.

Then, from 2010 to 2020, the market began to aggressively consolidate. This has created both serious threats and unique opportunities within the industry.

Serious threats to the industry include low rates of utilization for stored cord blood, expensive cord blood transplantation procedures, difficulty educating obstetricians about cellular therapies, and an increasing trend toward industry consolidation. There are also emerging opportunities for the industry, such as accelerated regulatory pathways for cell therapies in leading healthcare markets worldwide and expanding applications for cell-based therapies. In particular, MSCs from cord tissue (and other sources) are showing intriguing promise in the treatment and management of COVID-19.

Cord Blood Industry Trends

Within recent years, new themes have been impacting the industry, including the pairing of stem cell storage services with genetic and genomic testing services, as well as reproductive health services. Cord blood banks are diversifying into new types of stem cell storage, including umbilical cord tissue storage, placental blood and tissue, amniotic fluid and tissue, and dental pulp. Cord blood banks are also investigating means of becoming integrated therapeutic companies. With hundreds of companies offering cord blood banking services worldwide, maturation of the market means that each company is fighting harder for market share.

Growing numbers of investors are also entering the marketplace, with M&A activity accelerating in the U.S. and abroad. Holding companies are emerging as a global theme, allowing for increased operational efficiency and economy of scale. Cryoholdco has established itself as the market leader within Latin America. Created in 2015, Cryoholdco is a holding company that will control nearly 270,000 stem cell units by the end of 2020. It now owns a half dozen cord blood banks, as well as a dental stem cell storage company.

Globally, networks of cord blood banks have become commonplace, with Sanpower Group establishing its dominance in Asia. Although Sanpower has been quiet about its operations, it holds 4 licenses out of only 7 issued provincial-level cord blood bank licenses in China. It has reserved over 900,000 cord blood samples in China, and its reserves amount to over 1.2 million units when Cordlife' reserves within Southeast Asian countries are included. This positions Sanpower Group and it's subsidiary Nanjing Cenbest as the world's largest cord blood banking operator not only in China and Southeast Asia but in the world.

The number of cord blood banks in Europe has dropped by more than one-third over the past ten years, from approximately 150 to under 100. The industry leaders in this market segment include FamiCord Group, who has executed a dozen M&A transactions, and Vita34, who has executed approximately a half dozen. Stemlab, the largest cord blood bank in Portugal, also executed three acquisition deals prior to being acquired by FamiCord. FamiCord is now the leading stem cell bank in Europe and one of the largest worldwide.

Cord Blood Expansion Technologies

Because cord blood utilization is largely limited to use in pediatric patients, growing investment is flowing into ex vivo cord blood expansion technologies. If successful, this technology could greatly expand the market potential for cord blood, encouraging its use within new markets, such as regenerative medicine, aging, and augmented immunity.

Key strategies being explored for this purpose include:

Currently, Gamida Cell, Nohla Therapeutics, Excellthera, and Magenta Therapeutics have ex vivo cord blood expansion products proceeding through clinical trials. Growing numbers of investors have also entered the cord blood banking marketplace, led by groups such as GI Partners, ABS Capital Partners & HLM Management, KKR & Company, Bay City Capital, GTCR, LLC, and Excalibur.

Cord Blood Banking by Region

Within the United States, most of the market share is controlled by three major players: Cord Blood Registry (CBR), Cryo-Cell, and ViaCord. CBR has been traded twice, once in 2015 to AMAG Pharmaceuticals for $700 million and again in 2018 to GI Partners for $530 million. CBR also bought Natera's Evercord Cord Blood Banking business in September 2019. In total, CBR controls over 900,000 cord blood and tissue samples, making it one of the largest cord blood banks worldwide.

In China, the government controls the industry by authorizing only one cord blood bank to operate within each province, and official government support, authorization, and permits are required. Importantly, the Chinese government announced in late 2019 that it will be issuing new licenses for the first time, expanding from the current 7 licensed regions for cord blood banking to up to 19 regions, including Beijing.

In Italy and France, it is illegal to privately store one's cord blood, which has fully eliminated the potential for a private market to exist within the region. In Ecuador, the government created the first public cord blood bank and instituted laws such that private cord blood banks cannot approach women about private cord blood banking options during the first six months of pregnancy. This created a crisis for private banks, forcing most out of business.

Recently, India's Central Drugs Standard Control Organization (CDSCO) restricted commercial banking of stem cells from most biological materials, including cord tissue, placenta, and dental pulp stem cells - leaving only umbilical cord blood banking as permitted and licensed within the country.

While market factors vary by geography, it is crucial to have a global understanding of the industry, because research advances, clinical trial findings, and technology advances do not know international boundaries. The cord blood market is global in nature and understanding dynamics within your region is not sufficient for making strategic, informed, and profitable decisions.

Overall, the report provides the reader with the following details and answers the following questions:

1. Number of cord blood units cryopreserved in public and private cord blood banks globally2. Number of hematopoietic stem cell transplants (HSCTs) globally using cord blood cells3. Utilization of cord blood cells in clinical trials for developing regenerative medicines4. The decline of the utilization of cord blood cells in HSC transplantations since 20055. Emerging technologies to influence the financial sustainability of public cord blood banks6. The future scope for companion products from cord blood7. The changing landscape of cord blood cell banking market8. Extension of services by cord blood banks9. Types of cord blood banks10. The economic model of public cord blood banks11. Cost analysis for public cord blood banks12. The economic model of private cord blood banks13. Cost analysis for private cord blood banks14. Profit margins for private cord blood banks15. Pricing for processing and storage in private banks16. Rate per cord blood unit in the U.S. and Europe17. Indications for the use of cord blood-derived HSCs for transplantations18. Diseases targeted by cord blood-derived MSCs in regenerative medicine19. Cord blood processing technologies20. Number of clinical trials, number of published scientific papers and NIH funding for cord blood research21. Transplantation data from different cord blood registries

Key questions answered in this report are:

1. What are the strategies being considered for improving the financial stability of public cord blood banks?2. What are the companion products proposed to be developed from cord blood?3. How much is being spent on processing and storing a unit of cord blood?4. How much does a unit of cryopreserved cord blood unit fetch on release?5. Why do most public cord blood banks incur a loss?6. What is the net profit margin for a private cord blood bank?7. What are the prices for processing and storage of cord blood in private cord blood banks?8. What are the rates per cord blood units in the U.S. and Europe?9. What are the revenues from cord blood sales for major cord blood banks?10. Which are the different accreditation systems for cord blood banks?11. What are the comparative merits of the various cord blood processing technologies?12. What is to be done to increase the rate of utilization of cord blood cells in transplantations?13. Which TNC counts are preferred for transplantation?14. What is the number of registered clinical trials using cord blood and cord tissue?15. How many clinical trials are involved in studying the expansion of cord blood cells in the laboratory?16. How many matching and mismatching transplantations using cord blood units are performed on an annual basis?17. What is the share of cord blood cells used for transplantation from 2000 to 2020?18. What is the likelihood of finding a matching allogeneic cord blood unit by ethnicity?19. Which are the top ten countries for donating cord blood?20. What are the diseases targeted by cord blood-derived MSCs within clinical trials?

Key Topics Covered

1. REPORT OVERVIEW1.1 Statement of the Report1.2 Executive Summary1.3 Introduction1.3.1 Cord Blood: An Alternative Source for HPSCs1.3.2 Utilization of Cord Blood Cells in Clinical Trials1.3.3 The Struggle of Cord Blood Banks1.3.4 Emerging Technologies to Influence Financial Sustainability of Banks1.3.4.1 Other Opportunities to Improve Financial Stability1.3.4.2 Scope for Companion Products1.3.5 Changing Landscape of Cord Blood Cell Banking Market1.3.6 Extension of Services by Cord Blood Banks

2. CORD BLOOD & CORD BLOOD BANKING: AN OVERVIEW2.1 Cord Blood Banking (Stem Cell Banking)2.1.1 Public Cord Blood Banks2.1.1.1 Economic Model of Public Cord Blood Banks2.1.1.2 Cost Analysis for Public Banks2.1.1.3 Relationship between Costs and Release Rates2.1.2 Private Cord Blood Banks2.1.2.1 Cost Analysis for Private Cord Blood Banks2.1.2.2 Economic Model of Private Banks2.1.3 Hybrid Cord Blood Banks2.2 Globally Known Cord Blood Banks2.2.1 Comparing Cord Blood Banks2.2.2 Cord Blood Banks in the U.S.2.2.3 Proportion of Public, Private and Hybrid Banks2.3 Percent Share of Parents of Newborns Storing Cord Blood by Country/Region2.4 Pricing for Processing and Storage in Commercial Banks2.4.1 Rate per Cord Blood Unit in the U.S. and Europe2.5 Cord Blood Revenues for Major Cord Blood Banks

3. CORD BLOOD BANK ACCREDITATIONS3.1 American Association of Blood Banks (AABB)3.2 Foundation for the Accreditation of Cellular Therapy (FACT)3.3 FDA Registration3.4 FDA Biologics License Application (BLA) License3.5 Investigational New Drug (IND) for Cord Blood3.6 Human Tissue Authority (HTA)3.7 Therapeutic Goods Act (TGA) in Australia3.8 International NetCord Foundation3.9 AABB Accredited Cord Blood Facilities3.10 FACT Accreditation for Cord Blood Banks

4. APPLICATIONS OF CORD BLOOD CELLS4.1 Hematopoietic Stem Cell Transplantations with Cord Blood Cells4.2 Cord Cells in Regenerative Medicine

5. CORD BLOOD PROCESSING TECHNOLOGIES5.1 The Process of Separation5.1.1 PrepaCyte-CB5.1.2 Advantages of PrepaCyte-CB5.1.3 Treatment Outcomes with PrepaCyte-CB5.1.4 Hetastarch (HES)5.1.5 AutoXpress (AXP)5.1.6 SEPAX5.1.7 Plasma Depletion Method (MaxCell Process)5.1.8 Density Gradient Method5.2 Comparative Merits of Different Processing Methods5.2.1 Early Stage HSC Recovery by Technologies5.2.2 Mid Stage HSC (CD34+/CD133+) Recovery from Cord Blood5.2.3 Late Stage Recovery of HSCs from Cord Blood5.3 HSC (CD45+) Recovery5.4 Days to Neutrophil Engraftment by Technology5.5 Anticoagulants used in Cord Blood Processing5.5.1 Type of Anticoagulant and Cell Recovery Volume5.5.2 Percent Cell Recovery by Sample Size5.5.3 TNC Viability by Time Taken for Transport and Type of Anticoagulant5.6 Cryopreservation of Cord Blood Cells5.7 Bioprocessing of Umbilical Cord Tissue (UCT)5.8 A Proposal to Improve the Utilization Rate of Banked Cord Blood

6. CORD BLOOD CLINICAL TRIALS, SCIENTIFIC PUBLICATIONS & NIH FUNDING6.1 Cord Blood Cells for Research6.2 Cord Blood Cells for Clinical Trials6.2.1 Number of Clinical Trials involving Cord Blood Cells6.2.2 Number of Clinical Trials using Cord Blood Cells by Geography6.2.3 Number of Clinical Trials by Study Type6.2.4 Number of Clinical Trials by Study Phase6.2.5 Number of Clinical Trials by Funder Type6.2.6 Clinical Trials Addressing Indications in Children6.2.7 Select Three Clinical Trials Involving Children6.2.7.1 Sensorineural Hearing Loss (NCT02038972)6.2.7.2 Autism Spectrum (NCT02847182)6.2.7.3 Cerebral Palsy (NCT01147653)6.2.8 Clinical Trials for Neurological Diseases using Cord Blood and Cord Tissue6.2.9 UCB for Diabetes6.2.10 UCB in Cardiovascular Clinical Trials6.2.11 Cord Blood Cells for Auto-Immune Diseases in Clinical Trials6.2.12 Cord Tissue Cells for Orthopedic Disorders in Clinical Trials6.2.13 Cord Blood Cells for Other Indications in Clinical Trials6.3 Major Diseases Addressed by Cord Blood Cells in Clinical Trials6.4 Clinical Trials using Cord Tissue-Derived MSCs6.5 Ongoing Clinical Trials using Cord Tissue6.5.1 Cord Tissue-Based Clinical Trials by Geography6.5.2 Cord Tissue-Based Clinical Trials by Phase6.5.3 Cord Tissue-Based Clinical Trials by Sponsor Types6.5.4 Companies Sponsoring in Trials using Cord Tissue-Derived MSCs6.6 Wharton's Jelly-Derived MSCs in Clinical Trials6.6.1 Wharton's Jelly-Based Clinical Trials by Phase6.6.2 Companies Sponsoring Wharton's Jelly-Based Clinical Trials6.7 Clinical Trials Involving Cord Blood Expansion Studies6.7.1 Safe and Feasible Expansion Protocols6.7.2 List of Clinical Trials involved in the Expansion of Cord Blood HSCs6.7.3 Expansion Technologies6.8 Scientific Publications on Cord Blood6.9 Scientific Publications on Cord Tissue6.10 Scientific Publications on Wharton's Jelly-Derived MSCs6.11 Published Scientific Papers on Cord Blood Cell Expansion6.12 NIH Funding for Cord Blood Research

7. PARENT'S AWARENESS AND ATTITUDE TOWARDS CORD BLOOD BANKING7.1 Undecided Expectant Parents7.2 The Familiar Cord Blood Banks Known by the Expectant Parents7.3 Factors Influencing the Choice of a Cord Blood Bank

8. CORD BLOOD: AS A TRANSPLANTATION MEDICINE8.1 Comparisons of Cord Blood to other Allograft Sources8.1.1 Major Indications for HCTs in the U.S.8.1.2 Trend in Allogeneic HCT in the U.S. by Recipient Age8.1.3 Trends in Autologous HCT in the U.S. by Recipient Age8.2 HCTs by Cell Source in Adult Patients8.2.1 Transplants by Cell Source in Pediatric Patients8.3 Allogeneic HCTs by Cell Source8.3.1 Unrelated Donor Allogeneic HCTs in Patients &lessThan;18 Years8.4 Likelihood of Finding an Unrelated Cord Blood Unit by Ethnicity8.4.1 Likelihood of Finding an Unrelated Cord Blood Unit for Patients &lessThan;20 Years8.5 Odds of using a Baby's Cord Blood8.6 Cord Blood Utilization Trends8.7 Number of Cord Blood Donors Worldwide8.7.1 Number of CBUs Stored Worldwide8.7.2 Cord Blood Donors by Geography8.7.2.1 Cord Blood Units Stored in Different Geographies8.7.2.2 Number of Donors by HLA Typing8.7.3 Searches Made by Transplant Patients for Donors/CBUs8.7.4 Types of CBU Shipments (Single/Double/Multi)8.7.5 TNC Count of CBUs Shipped for Children and Adult Patients8.7.6 Shipment of Multiple CBUs8.7.7 Percent Supply of CBUs for National and International Patients8.7.8 Decreasing Number of CBU Utilization8.8 Top Ten Countries in Cord Blood Donation8.8.1 HLA Typed CBUs by Continent8.8.2 Percentage TNC of Banked CBUs8.8.3 Total Number of CBUs, HLA-Typed Units by Country8.9 Cord Blood Export/Import by the E.U. Member States8.9.1 Number of Donors and CBUs in Europe8.9.2 Number of Exports/Imports of CBUs in E.U.8.10 Global Exchange of Cord Blood Units

9. CORD BLOOD CELLS AS THERAPEUTIC CELL PRODUCTS IN CELL THERAPY9.1 MSCs from Cord Blood and Cord Tissue9.1.1 Potential Neurological Applications of Cord Blood-Derived Cells9.1.2 Cord Tissue-Derived MSCs for Therapeutic use9.1.2.1 Indications Targeted by UCT-MSCs in Clinical Trials9.2 Current Consumption of Cord Blood Units by Clinical Trials9.3 Select Cord Blood Stem Cell Treatments in Clinical Trials9.3.1 Acquired Hearing Loss (NCT02038972)9.3.2 Autism (NCT02847182)9.3.3 Cerebral Palsy (NCT03087110)9.3.4 Hypoplastic Left Heart Syndrome (NCT01856049)9.3.5 Type 1 Diabetes (NCT00989547)9.3.6 Psoriasis (NCT03765957)9.3.7 Parkinson's Disease (NCT03550183)9.3.8 Signs of Aging (NCT04174898)9.3.9 Stroke (NCT02433509)9.3.10 Traumatic Brain Injury (NCT01451528)

10. MARKET ANALYSIS10.1 Public vs. Private Cord Blood Banking Market10.2 Cord Blood Banking Market by Indication

11. PROFILES OF SELECT CORD BLOOD BANKS11.1 AllCells11.1.1 Whole Blood11.1.2 Leukopak11.1.3 Mobilized Leukopak11.1.4 Bone Marrow11.1.5 Cord Blood11.2 AlphaCord LLC11.2.1 NextGen Collection System11.3 Americord Registry, Inc.11.3.1 Cord Blood 2.011.3.2 Cord Tissue11.3.3 Placental Tissue 2.011.4 Be The Match11.4.1 Hub of Transplant Network11.4.2 Partners of Be The Match11.4.3 Allogeneic Cell Sources in Be The Match Registry11.4.4 Likelihood of a Matched Donor on Be The Match by Ethnic Background11.5 Biocell Center Corporation11.5.1 Chorionic villi after Delivery11.5.2 Amniotic Fluid and Chorionic Villi during Pregnancy11.6 BioEden Group, Inc.11.6.1 Differences between Tooth Cells and Umbilical Cord Cells11.7 Biovault Family11.7.1 Personalized Cord Blood Processing11.8 Cell Care11.9 Cells4Life Group, LLP11.9.1 Cells4Life's pricing11.9.2 TotiCyte Technology11.9.3 Cord Blood Releases11.10 Cell-Save11.11 Center for International Blood and Marrow Transplant Research (CIBMTR)11.11.1 Global Collaboration11.11.2 Scientific Working Committees11.11.3 Medicare Clinical Trials and Studies11.11.4 Cellular Therapy11.12 Crio-Cell International, Inc.11.12.1 Advanced Collection Kit11.12.2 Prepacyte-CB11.12.3 Crio-Cell International's Pricing11.12.4 Revenue for Crio-Cell International11.13 Cord Blood Center Group11.13.1 Cord Blood Units Released11.14 Cordlife Group, Ltd.11.14.1 Cordlife's Cord Blood Release Track Record11.15 Core23 Biobank11.16 Cord Blood Registry (CBR)11.17 CordVida11.18 Crioestaminal11.18.1 Cord Blood Transplantation in Portugal11.19 Cryo-Cell International, Inc.11.19.1 Processing Method11.19.2 Financial Results of the Company11.20 CryoHoldco11.21 Cryoviva Biotech Pvt. Ltd11.22 European Society for Blood and Bone Marrow Transplantation (EBMT)11.22.1 EBMT Transplant Activity11.23 FamiCord Group11.24 GeneCell International11.25 Global Cord Blood Corporation11.25.1 The Company's Business11.26 HealthBaby Hong Kong11.26.1 BioArchive System Service Plan11.26.2 MVE Liquid Nitrogen System11.27 HEMAFUND11.28 Insception Lifebank11.29 LifebankUSA11.29.1 Placental Banking11.30 LifeCell International Pvt. Ltd.11.31 MiracleCord, Inc.11.32 Maze Cord Blood Laboratories11.33 New England Cord Blood Bank, Inc.11.34 New York Cord Blood Center (NYBC)11.34.1 Products11.34.2 Laboratory Services11.35 PacifiCord11.35.1 FDA-Approved Sterile Collection Bags11.35.2 AXP Processing System11.35.3 BioArchive System11.36 ReeLabs Pvt. Ltd.11.37 Smart Cells International, Ltd.11.38 Stem Cell Cryobank11.39 StemCyte, Inc.11.39.1 StemCyte Sponsored Clinical Trials11.39.1.1 Spinal Cord Injury Phase II11.39.1.2 Other Trials11.40 Transcell Biolife11.40.1 ScellCare11.40.2 ToothScell11.41 ViaCord11.42 Vita 34 AG11.43 World Marrow Donor Association (WMDA)11.43.1 Search & Match Service11.44 Worldwide Network for Blood & Marrow Transplantation (WBMT)

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Global Cord Blood Banking Market 2020 with Analysis of 44 Industry Players - PRNewswire

Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respiratory Distress Syndrome and COVID-19 – PRNewswire

GAITHERSBURG, Md., Sept. 1, 2020 /PRNewswire/ --Cartesian Therapeutics, a fully integrated, clinical-stage biopharmaceutical company developing cell and gene therapies for cancer, autoimmune diseases and respiratory diseases, today announced that it has initiated a Phase 1/2 clinical trial of its lead RNA-engineered mesenchymal stem cell (MSC) therapy, Descartes-30, in patients with moderate-to-severe acute respiratory distress syndrome (ARDS), including that caused by COVID-19. Based upon the company's research and analysis, this program is understood to be the first RNA-engineered cell therapy to enter clinical development for ARDS and COVID-19. It is also the first cell therapy to specifically degrade NETs, webs of extracellular DNA and histones that entrap inflammatory cells, block alveoli and vessels, and drive the pathogenesis of ARDS and COVID-19.

"Patients with ARDS, especially those with COVID-19 ARDS, generate copious amounts of NETs that physically obstruct alveoli and vessels, which leads to respiratory distress, immune-mediated thrombosis and a vicious cycle of inflammation," said Bruce Levy, MD, Chief of Pulmonary and Critical Care Medicine at Brigham and Women's Hospital and Parker B. Francis Professor at Harvard Medical School, and a clinical investigator in the Descartes-30 trial. "We would therefore expect that degrading NETs would improve oxygenation as well as resolve thrombi and quell inflammation in these patients. If successful, Descartes-30 would be a highly differentiated game-changer within our limited toolkit in managing this exceedingly difficult condition."

Descartes-30 is an off-the-shelf (allogeneic) MSC product engineered with Cartesian's RNA ArmorySM cell therapy platform. By expressing a unique combination of DNases that work synergistically, Descartes-30 can eliminate large, macroscopic amounts of NETs within minutes. MSCs are inherently immunomodulatory and naturally travel to the lungs, where they are expected to provide continuous, local delivery of DNases to NET-laden lung tissue.

"We engineered Descartes-30 without genomic modification, and therefore the production of DNases is expected to be time-limited to match the acute nature of ARDS," said Metin Kurtoglu, MD, PhD, Chief Medical Officer at Cartesian. "Given thatDescartes-30will produce DNases locally and transiently, we anticipate that it will have a favorable benefit-to-risk profile. We also anticipate that these properties will enable Descartes-30 to treat a wide array of NET-related autoimmune and cardiovascular diseases."

About the Phase 1/2a Clinical Trial

The "Phase 1/2a Study of Descartes-30 in Acute Respiratory Distress Syndrome" (NCT04524962) is enrolling patients with ARDS at multiple critical care units in the United States. Patients with ARDS due to COVID-19 are given enrollment priority. This first-in-human study aims to determine the safety and preliminary efficacy of Descartes-30 in patients with moderate to severe ARDS. The study, which is estimated to begin treatment in September, aims to enroll approximately 20 patients prior to initiation of a larger study. For more information visit http://www.cartesiantherapeutics.com/Descartes-30-ARDS.

About ARDS and NETs

ARDS is a severe inflammatory lung disease with a mortality of over 40%. Inflammation leads to injury of lung tissue and leakage of blood and plasma into air spaces, resulting in low oxygen levels and often requiring mechanical ventilation. Inflammation in the lung may lead to inflammation elsewhere, causing shock and injury or dysfunction in the kidneys, heart, and muscles. Some causes of ARDS include COVID-19, severe pneumonia (including influenza), sepsis, trauma, and smoke inhalation.

NETs are inflammatory webs of DNA and proteins produced by neutrophils. NETs are commonly found in ARDS and are thought to exacerbate the disease by physically occluding air spaces and vessels, leading to reduced oxygenation and increased risk of immune thrombi. NETs are implicated in a variety of conditions beyond ARDS, including autoimmune and cardiovascular diseases.

About the RNA ArmorySM

The RNA ArmorySM is Cartesian's proprietary RNA-based cell engineering platform that activates and arms cells with carefully selected, mRNA-based therapeutics. Unmodified donor cells enter the RNA ArmorySMin the millions; a battle-ready cell army leaves the RNA ArmorySMin the tens of billions. Each cell is equipped with a combination of therapeutics rationally chosen to have a synergistic effect on the disease. In the body, the cells deliver a precision-targeted treatment regimen directly to the site of disease. The cells express therapeutics with a defined half-life, enhancing their safety profile and making repeat dosing and outpatient administration possible. The platform is agnostic to cell type: we choose the best cell for the job, whether autologous or off-the shelf. For more information visithttps://www.cartesiantherapeutics.com/rna-armory/.

About Cartesian Therapeutics

Founded in 2016,Cartesianis a fully integrated, clinical-stage biopharmaceutical company developing potent yet safer cell and gene therapies designed to benefit the broadest range of patients with cancer, autoimmune and respiratory diseases. Cartesianhas three products in clinical development under four open investigational new drug application (INDs) with the U.S. Food & Drug Administration (FDA). All investigational therapies are manufactured at Cartesian's wholly owned, state-of-the-art cGMP manufacturing facility in Gaithersburg, MD.Cartesian's commanding IP position benefits in part from a broad, exclusive patent license from the National Cancer Institute. For more information visithttps://www.cartesiantherapeutics.com/clinical-trials/.

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Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respiratory Distress Syndrome and COVID-19 - PRNewswire

New Comprehensive Report on Stem Cell Therapy Market to Witness an Outstanding Growth during 2020 2025 with Top Players Like Chiesi Pharmaceuticals,…

The Stem Cell TherapyIndustry study now available at Grand View Report, is a detailed sketch of the business sphere in terms of current and future trends driving the profit matrix. The report also indicates a pointwise outline of market share, market size, industry partakers, and regional landscape along with statistics, diagrams, & charts elucidating various noteworthy parameters of the industry landscape.

The Stem Cell Therapy Market research report offers an exhaustive analysis of this business space. The key trends that define the Stem Cell TherapyIndustry market during the analysis timeframe are mentioned in the report, alongside other factors such as regional scope and regulatory outlook. Also, the document elaborates on the impact of current industry trends on key market driving factors as well as top challenges.

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New Comprehensive Report on Stem Cell Therapy Market to Witness an Outstanding Growth during 2020 2025 with Top Players Like Chiesi Pharmaceuticals,...

CAR T-Cell Optimization Starts in Production, Extends to Therapy – Genetic Engineering & Biotechnology News

Just as heat-seeking missiles race toward the infrared signatures of their targets, chimeric antigen receptor (CAR) T cells home in on cancer-associated or -specific antigens. Once the antigens are engaged, CAR T cells let fly with cytotoxic flak, granules containing perforin and granzymes, while activating supplementary tumor-killing mechanisms such as stromal sensitization and macrophage polarization. It is to be hoped that by the time the cytotoxic smoke clears, the cancer will have been destroyed.

The development of CAR T cells has revolutionized adoptive cellular therapies against cancer. CARs are genetically engineered to combine antigen- or tumor-specific-binding with T-cell activating domains. T cells, obtained from the patient (autologous cells) or from a healthy donor (allogeneic cells), are typically transduced with an engineered vector, expanded, and infused back into the patient for tumor eradication.

In the 10 years since its inception, the CAR T-cell field has progressed rapidly. Two CAR T-cell products have been approved for clinical use, and many more products are undergoing clinical trials or are in development. Although the field initially focused on B-cell malignancies, it is now advancing on solid tumors.

Despite its initial success, the CAR T-cell field must find ways to generate products that are potent, affordable, and available. To achieve enduring success, the CAR T-cell field is undertaking a range of initiatives. These include the engineering of bridging proteins for multiantigen targeting; the creation of nonviral allogeneic off-the-shelf products; the organization of vein-to-vein networks; and the development of precisely tuned therapies, that is, precisely timed and dosed therapies.

Cellular therapy is a living drug, declares Steve Shamah, PhD, senior vice president, Obsidian Therapeutics. As with any drug, damage can occur if the therapy is not carefully regulated. Our company focuses on creating controllable cell therapies by engineering CAR T cells or tumor-infiltrating lymphocytes to produce regulatable cytokines and proteins that can enhance functional activity, especially against solid tumors.

For example, the company is developing a platform that armors CAR T cells with immunomodulatory factors such as interleukin-15 (IL-15) or CD40 ligand. Shamah explains, These factors can enhance functional activity by driving T-cell expansion, conferring resistance to immunosuppression, improving antigen presentation, and inducing antigen spread. However, both factors can also produce systemic toxicity. Our technology modulates the level and timing of their activity in a fully controlled, dose-dependent manner using an FDA-approved small-molecule drug.

The Obsidian platform, cytoDRiVE, adds a drug-responsive domain (DRD) onto a therapeutic protein of interest. DRD tags are misfolded or inherently unstable in the cell. However, they can be reversibly stabilized by the binding of approved small-molecule drugs. When the drug is absent, the DRD-tagged protein is turned off. When the drug is present, the DRD-tagged protein is turned on. When DRD tags are in place, the concentration of the small-molecule drug serves as a biological rheostat for controlling the dosing of the therapeutic protein.

Preclinical in vivo mouse studies assessed anti-CD19 CAR T cells that were engineered to express an IL-15-DRD that responded to the FDA-approved drug acetazolamide. In these studies, tumor regression was demonstrated.

Controlling the precise timing and expression level of these immunomodulatory factors in CAR T cells could significantly enhance safety and therapeutic efficacy, concludes Shamah.

Obsidian is currently focusing on the oncology space, but the company is also exploring other areas such as autoimmunity and even the regulation of transcription factors to enable controllable in vivoCRISPR-Cas9 gene editing.

Despite the remarkable success of CAR T-cell therapies, relapses can occur within six months for up to 50% of patients treated with anti-CD19 CAR T-cell therapy.Failures can occur due to loss of CD19 expression or to continued tumor proliferation. Aleta Biotherapeutics has developed a novel technology to reactivate CAR T cells in relapsed patients.

Our approach utilizes antigen-bridging proteins to coat tumors with CD19, says Paul Rennert, PhD, Aletas president and CSO. [The tumors are then] recognized by the patients anti-CD19 CAR T cells, essentially creating a cytotoxic synapse that results in tumor cell death.

To thwart anti-CD19 CAR T-cell therapy relapses, the company developed a bridging protein using the extracellular domain of CD19 and an anti-CD20 antibody domain. CD20 is an antigen present on the majority of B-cell malignancies. Rennert explains that these injected bridging proteins will coat the patients tumor cells with CD19, creating a target to activate or reactivate a patients anti-CD19 CAR T cells.

To show proof-of-principle, the company performed in vivo studies using a half-life-extended form of the bridging protein injected into mice carrying CD20-positive tumor cells and anti-CD19 CAR T cells. Rennert emphasizes, Our studies demonstrated this strategy can be used to reactivate CD19 CAR T cells to prevent and reverse relapses.

Other programs in development include a bridging protein for injection to improve outcomes in multiple myeloma patients treated with CAR T cells, and bridging protein programs for HER2-positive breast cancer patients with central nervous system metastases. The company is preparing investigational new drug applications for its technology and plans to start Phase I trials in 2021.

Assessing whether engineered CAR T-cell and T-cell receptor (TCR) therapies have successfully attacked and penetrated solid tumors (and not normal cells) can be like finding the proverbial needle in the haystack. Traditional methods using immunohistochemistry are useful for immune profiling, but they cannot differentiate engineered versus endogenous cells, points out Christopher Bunker, PhD, senior director of business development, Advanced Cell Diagnostics, a Bio-Techne brand. We developed a means to easily detect and track engineered therapeutic cells and delineate their pharmacokinetics within the tumor microenvironment of intact tumor biopsies, as well as their on-target/off-tumor activity.

Enter RNAscope, an RNA in situ hybridization technology that can enable single-cell spatial transcriptomics. RNAscope, Bunker asserts, is the only off-the-shelf method that can specifically detect engineered CAR T cells and TCR T cells in solid tumor patient biopsies.

Most cell therapies employ lentivirus transduction. Because CAR or TCR transgenes have unique sequences in the viral untranslated regions, these can be used as tags for identification of engineered cell therapies with RNAscope probes. The technology utilizes pools of paired oligos that can be thought of as a ZZ pair, where the paired 3 ends hybridize to ~50 bases of target mRNA, and where the paired 5 ends hybridize to a signal amplification module, which is built through sequential hybridization steps. The signal amplification of paired oligos results in an assay able to detect individual transcripts that appear as visible and quantifiable dots.

Its a little like planting and lighting Christmas trees, quips Bunker. The ZZ pairs plant trees along the mRNA with branches that are decorated either with fluorophores or chromogens. Although the primary technology currently features four colors, the company has developed a HiPlex (12-plex) assay and foresees even higher-plex assays with different detection methods.

We envision assays based on our core technologies that enable spatial analysis of perhaps a hundred transcripts in combination with tens of proteins, Bunker projects. In the context of cell therapy development, these will enable a more comprehensive understanding of tumor biology and immune cell profiles to determine the most effective treatment strategy for a patient, as well as for monitoring efficacy of solid tumor cellular therapies.

Companies developing CAR T-cell products are also eyeing a future involving GMP production. Thus, a critical early question is how to choose the best T-cell medium for manufacturing processes. To test the suitability of a CAR T-cell growing medium, companies must assess factors such as cell viability, cell expansion, cytokine profiles, and cell purity. A medium suitable for a CAR T-cell manufacturing process also needs to support rapid activation and CAR transduction. Additionally, the selected medium needs to be compatible with a variety of donors.

There are many available choices for T-cell culture media ranging from do-it-yourself recipes to commercially available one-size-fits-all complete formulations. CellGenix has developed a novel T-cell medium that avoids the use of human serum. Sebastian Warth, PhD, a senior scientist at CellGenix, explains, To achieve consistent results, human serum requires extensive testing prior to its use for production of cellular products due to lot-to-lot inconsistencies. Since human serum is a limited resource and might not be available in large quantities, it is unfavorable for commercial-scale manufacturing. Furthermore, the human origin of serum poses a certain risk of containing adventitious agents and is, therefore, a risk to the safety of the T-cell therapy product.

The companys TCM GMP-Prototype medium provides a serum-free and xeno-free product for early-onset T-cell expansion. According to Warth, key advantages include promotion of sustained viability, support for expansion of CD4+ and CD8+ T cells, promotion of a central memory and early differentiated memory T-cell phenotype, and maintenance of a high proportion of cytokine-producing cells including polyfunctional cells. Further, it was optimized for and verified with CAR T cells.

While autologous CAR T-cell therapies have proven highly successful, they also require a long and expensive manufacturing process. The dream of being able to utilize off-the-shelf allogeneic T cells is on the horizon.

Devon J. Shedlock, PhD, senior vice president, research and development,Poseida Therapeutics, reports, With our technology, we are able to genetically modify cells to create a fully allogenic, or off-the-shelf, product that does not require additional immunosuppression treatment like earlier generation approaches. We also have developed technology to allow us to make hundreds of doses from a single manufacturing run from healthy donors, thereby dropping the cost substantially.

According to Shedlock, the technology consists of three key aspects: 1) the piggyback DNA Modification System, 2) the Cas-CLOVER site-specific gene editing system, and 3) the Booster Molecule.

The PiggyBac DNA Modification System is a nonviral technology for stably integrating genes into DNA. One key feature is that piggyBac preferentially inserts into less mature T cells, enabling the production of therapies that have a high proportion of stem cell memory T cells, or Tscm cells.

Viral technologies are virtually excluded from Tscm cells, Shedlock states. However, Tscm cells are the ideal cell type for cell-based therapies because they have the ability to engraft and potentially last a lifetime, can produce wave after wave of more differentiated cells to attack the tumor, and are much more tolerable with low levels of adverse events compared to other CAR T-cell products.

The companys Cas-CLOVER gene editing technology is a hybrid gene editing technology used to edit the T cells to make allogeneic products. Cas-CLOVER works well in resting T cells, which is important in preserving Tscm cells in a fully allogeneic CAR T-cell product, Shedlock elaborates. It also is a very precise and clean system. This is a particularly important safety issue for allogeneic products that may be given to many patients.

The Booster Molecule is added during manufacture and is temporarily expressed on the cell surface to allow cell stimulation. Normally when allogeneic CAR T-cell products are created, the T-cell receptor must be eliminated to avoid the graft-versus-host reaction, which is a major safety issue. Importantly, this booster stimulation occurs while preserving the Tscm phenotype.

Poseida Therapeutics expects to launch a clinical trial for its multiple myeloma allogeneic product late this year or early next year. The company will also begin clinical trials later in 2021 on its pan-solid tumor allogeneic program.

Creation of partnerships can help drive development of CAR T-cell therapeutics from concept through clinical trials. Advanced therapies require advanced supply chain and data management, advises Minh Hong, PhD, head of autologous cell therapy, Lonza Pharma & Biotech. Prior biopharmaceutical models of mass production and distributionand the systems that support themare not effective for personalized therapeutics. As manufacturing demand increases for autologous cell therapies, there is an overarching need to both industrialize and simplify the entire supply chain ecosystem.

Hong says the overall project needs to be considered from a more comprehensive perspective: Due to the criticality of the starting material, everything from cell sourcing, patient coordination and scheduling for collection/infusion, transportation logistics, and manufacturing logistics needs to be coordinated, ensuring the highest standards, regulatory compliance, and safety throughout the process.

To meet these needs, Lonza is building a network of partners to develop a fully integrated vein-to-vein solution, that is, a system that includes all touch points involved in patient scheduling and sample collection, through material shipping logistics, manufacturing, and eventually the infusion of the cell therapy back into the patient. The partner network, Hong indicates, will help participants define smart workflows and execute an integration strategy. Hong sums up the networks therapeutic implications as follows: We believe these partnerships will decrease time to clinical program setup.

Lonza has more than a 20-year history of providing clinical and commercial manufacturing. Hong asserts, Our company brings to the table our process development and manufacturing experience along with proprietary solutions including a manufacturing execution system solution, MODA-ESTM, for electronic batch records and manufacturing traceability. In addition, we have announced partnerships with Vineti for a supply chain orchestration system and Cryoport to aid in shipping and logistics.

Lonza is also looking beyond CAR T-cell therapies. We would not limit our solutions and partnerships to autologous cell therapies, Hong declares. We can envision solutions for our in vivo viral vector manufacturing clients as well as our traditional allogeneic cell therapy clients.

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CAR T-Cell Optimization Starts in Production, Extends to Therapy - Genetic Engineering & Biotechnology News

Ruxolitinib May Be Another Option for Children With Steroid Refractory aGVHD – AJMC.com Managed Markets Network

Ruxolitinib may be a promising second-line treatment option for pediatric patients with steroid refractory advanced graft-versus-host disease (aGVHD), according to a new study. Based on the findings, researchers suggest their results be validated in a prospective large-scale pharmacokinetic and efficacy trial.

The small retrospective study of 29 patients recruited from 15 pediatric transplantation centers demonstrated that after 28 days, 6 patients achieved a complete response (CR). By 41 days, 19 (65.5%) patients achieved a CR. Another 2 patients had a partial response (PR). Among the 16 patients who were under the age of 6 years, there was a 75% CR and 6.3% PR. All patients who achieved a response discontinued corticosteroid treatment.

Prior to ruxolitinib, patients received an average of 2 immunosuppressive agents, with a range of 1-6.

The prognosis of patients with aGVHD depends mainly on the response of steroid treatment, which is known to be effective in approximately 50% of cases. Indeed, the mortality rate of steroid-refractory or recurrent aGVHD remains as high as 50% to 70%, wrote the researchers. There is currently no standardized second-line strategy for steroid resistant aGVHD.

While several immunosuppressive therapies are used in the second line of treatment, they carry variable response rates. For example, Monoclonal anti-TNF have demonstrated response rates between 30% and 60%. But, relapse at discontinuation was high at approximately 80%.

Results of the current study led the researchers to argue that ruxolitinib may be a possible treatment for these patients, including in cases of gastrointestinal involvement or severe GVHD.

A total of 8 patients experienced treatment failure, and 23 patients were alive after a median follow-up of 685 days after their hematopoietic stem cell transplantation and a median 480 days after initiating ruxolitinib treatment.

CR/PR was a significant factor of survival with a rate of 90% versus 50% in case of treatment failure (P = 0.02), wrote the researchers. Nevertheless, we could not find any association of baseline characteristics and CR/PR to ruxolitinib. Neither the number of immunosuppressive agents before using ruxolitinib nor involved organs was significant.

The researchers were also unable to demonstrate a relationship between ruxolitinib dose and rate of efficacy nor the speed of efficacy. Throughout the study, the median dose of ruxolitinib was 12.6 mg/m2/day.

The study also showed a favorable safety profile of ruxolitinib, with no observed severe hematologic adverse events and with all cytopenias requiring a dose modification resolving.

Viral replication (including cytomegalovirus, Epstein-Barr virus, and adenovirus) was observed in 41.4% of cases indicating that viral replications need to be closely monitored, wrote the researchers. This rate should be interpreted with caution because a comparable frequency of viral replications was reported in children treated with other immunosuppressive drugs, including infliximab, alemtuzumab, and daclizumab.

Reference

Laisne L, Neven B, Dalle J, et al. Ruxolitinib in children with steroid-refractory acute graft-versus-host disease: a retrospective multicenter study of the pediatric group of SFGM-TC. Pediatr Blood Cancer. Published online July 2, 2020. doi: 10.1002/pbc.28233.

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Ruxolitinib May Be Another Option for Children With Steroid Refractory aGVHD - AJMC.com Managed Markets Network

Growing Focus on R&D Likely to Accelerate the Growth of the Stem Cell Therapy Market – The News Brok

New Study on the Global Stem Cell Therapy Market by PMR

Persistence Market Research recently published a market study that sheds light on the growth prospects of the global Stem Cell Therapy market during the forecast period (20XX-20XX). In addition, the report also includes a detailed analysis of the impact of the novel COVID-19 pandemic on the future prospects of the Stem Cell Therapy market. The report provides a thorough evaluation of the latest trends, market drivers, opportunities, and challenges within the global Stem Cell Therapy market to assist our clients arrive at beneficial business decisions.

As per the report, the global Stem Cell Therapy market is expected to grow at a CAGR of ~XX% during the stipulated timeframe owing to a range of factors including, favorable government policies, and growing awareness related to the Stem Cell Therapy , surge in research and development and more.

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Competitive Outlook

The competitive outlook section provides valuable information related to the different companies operating in the current Stem Cell Therapy market landscape. The market share, product portfolio, pricing strategy, sales and distribution channels of each company is discussed in the report.

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Prominent players covered in the report are:

Regional Assessment

The presented market study touches upon the market scenario in different regions and provides a deep understanding of the influence of micro and macro-economic factors on the prospects of the market in each region.

Some of the major companies operating in the global stem cell therapy market are Mesoblast Ltd., Celgene Corporation, Aastrom Biosciences, Inc. and StemCells, Inc.

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The market report addresses the following queries related to the Stem Cell Therapy market:

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Growing Focus on R&D Likely to Accelerate the Growth of the Stem Cell Therapy Market - The News Brok

FDA Approves First Maintenance Therapy for AML – Medscape

The US Food and Drug Administration (FDA) has approved an oral form of azacitidine (Onureg) for use as maintenance therapy for patients with acute myeloid leukemia (AML) who have achieved a first complete remission.

The approval extends to patients who have achieved complete remission with incomplete blood count recovery following intensive induction chemotherapy and who are unable to complete intensive curative therapy.

The approval was based on data from the QUAZAR AML-001 trial, which showed that oral azacitadine significantly improved overall survival when compared to placebo.

"It's not too hard to get these patients into remission," Harry P. Erba, MD, PhD, director of the Leukemia Program at the Duke Cancer Institute, Durham, North Carolina, told Medscape Medical News last year, when these results were first presented at the 2019 annual meeting of the American Society of Hematology. "The problem comes in keeping them in remission."

Despite various attempts, there has been no success over the past 30 years in defining maintenance treatment for these patients, Andrew H. Wei, MBBS, PhD, from the Alfred Hospital in Melbourne, Australia, said.

"Oral azacitidine represents a new therapeutic standard for patients with AML in remission," he said.

Azacitidine is a hypomethylating agent that incorporates into DNA and RNA. It has long been used as an injectable therapy for the treatment of myelodysplastic syndromes.

The approval of the new oral formulation for the new indication of AML "is the culmination of over a decade of research and 13 preclinical and clinical trials," said Giovanni Caforio, M.D., chairman and chief executive officer of Bristol-Myers Squibb, in a statement.

The QUAZAR AML-001 trial was a phase 3, international study involving 472 patients with AML who were within achieving a first complete remission or remission with incomplete blood recovery. All patients has received intensive induction chemotherapy with or without consolidation treatment, per investigator preference prior to study entry, and were not candidates for hematopoietic stem cell transplant at the time of screening.

Patients were randomly assigned to receive either oral azacitidine 200 mg daily on days 1 to 14 of a repeat 28-day cycle (n = 278) or matching placebo (n = 274). Treatment was continued indefinitely until blast count was more than 15% or patients experienced unacceptable toxicity or underwent transplant.

At a median follow-up of over 41.2 months, the median overall survival was significantly longer for patients who received oral azacytidine, at 24.7 monthsvs 14.8 months for those who received placebo (P < .0009; hazard ratio [HR], 0.69).

Relapse-free survival was also significantly prolonged to 10.2 months for patients who received oral azacitidinevs 4.8 months for those who received placebo (HR, 0.65; P < .0001).

Serious adverse reactions occurred in 15% of patients who received azacytidine. Events that occurred in 2% of patients include pneumonia (8%) and febrile neutropenia (7%). There was one fatal event.

The most common adverse reactions were nausea (65%, 24%), vomiting (60%, 10%), diarrhea (50%, 21%), fatigue/asthenia (44%, 25%), constipation (39%, 24%), pneumonia (27%, 17%), abdominal pain (22%, 13%) arthralgia (14%, 10%), decreased appetite (13%, 6%), febrile neutropenia (12%, 8%), dizziness (11%, 9%) and pain in extremity (11%, 5%). Permanent discontinuation because of an adverse reaction occurred in 8% of patients.

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FDA Approves First Maintenance Therapy for AML - Medscape

Oldest male polar bear on record humanely euthanized at Point Defiance Zoo – MyNorthwest.com

Boris the polar bear swimming. (Photo courtesy of Point Defiance Zoo & Aquarium)

Boris, a polar bear believed to be the oldest male of his species, was humanely euthanized at Point Defiance Zoo & Aquarium on Tuesday following a significant decline in his health.

Woodland Park Zoos oldest animal dies one day shy of 52

The 34-year-old polar bear was a circus bear before he found his home in Tacoma.

Point Defiance says he was loved and revered by the community, gaining international recognition seven years ago when photographs of his dental procedure circulated around the world.

Staff made the decision to euthanize Boris due to deteriorating health issues that were affecting his quality of life, according to head veterinarian Dr. Karen Wolf. Recent exams showed significant arthritis, several fused vertebrae in his neck, and skin issues, Wolf said. He also had a history of gastrointestinal problems, dental and liver disease.

We cared for Boris as long as possible with a combination of groundbreaking medical treatment and daily TLC, Wolf said. But he had increasing difficulty getting up, had recently fallen, and his quality of life had declined dramatically. We did not want him to suffer. His loss will be felt deeply around the Zoo.

Boris was a medical pioneer of sorts when stem cells grown from his fat tissue were used in an attempt to treat his arthritis. He was believed to be the first polar bear to receive the protocol.

A news release from Point Defiance says Boris participated in his medical care and would stick his paw through a specially built sleeve in his bedroom so veterinary staff could get the voluntary blood samples to monitor his health.

Keepers also gave Boris his favorite foods and daily enrichments, like a den full of fresh wood wool shavings to roll in. He moved slowly on land, but enjoyed splashing in the deep saltwater pool and diving onto toys provided by keepers. He would also occasionally play-wrestle with 24-year-old polar bear Blizzard.

This is a very sad day for us, said Alan Varsik, director of Zoological and Environmental Education for Metro Parks Tacoma, in a news release. Boris held a special place in the hearts of our staff and our community.

Boris moved to Point Defiance in 2002 when he was a scrawny, malnourished, and mistreated animal seized by the U.S. Fish & Wildlife Service from a traveling circus. Boris and five other bears were provided homes, nutrition, and expert care at zoos around the nation, and Boris was the last surviving member of the group.

We were pleased we could give him a home when he was rescued from the circus, Varsik said. But we are even happier that Boris became a beloved ambassador for his species, inspiring our guests to take action that can help polar bears in the wild.

Point Defiance says the median life expectancy for male polar bears in human care is 23.4 years, which Boris surpassed at 34 years. In the wild, they typically live 15-18 years.

According to data from zoos in North America and around the world, Point Defiance says Boris was the oldest male of his species on record.

Boris stem-cell therapy treatment will add to the knowledge of how to help polar bears and large animals with arthritis, Wolf says, and an upcoming necropsy (the animal equivalent of an autopsy) will help with future polar bear conservation.

We want to take samples so that even after death, Boris can help us all learn more about his species and how to care for them, Wolf explained.

Zoo guests can sign a giant card for Boris caregivers in memory of him in the main plaza Sept. 2-4. Read more about Boris, and find out how to donate to polar bears in Boris memory online here.

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Oldest male polar bear on record humanely euthanized at Point Defiance Zoo - MyNorthwest.com

Elixirgen Therapeutics planning to begin Phase I/II Clinical Trials of its COVID-19 Vaccine Candidate EXG-5003 at Fujita Health University -…

BALTIMORE, Sept. 1, 2020 /PRNewswire/ -- Elixirgen Therapeutics, Inc., a Baltimore-based biotechnology company focused on the discovery, development and commercialization of therapies for genetic diseases and vaccines, announced that Fujita Health University has received acontract from the Japan Agency for Medical Research and Development (AMED) to initiate Phase I/II clinical trials of the company's COVID-19 vaccine candidate, EXG-5003. Clinical trials are expected to begin at Fujita Health University Hospital in Aichi, Japan in Q1 2021.

EXG-5003 is a temperature-sensitive self-replicating RNA vaccine expressing the receptor binding domain of the SARS-CoV-2 spike protein. EXG-5003 was optimized for intradermal injection withpotential dose-sparing and safety benefits.

About Elixirgen Therapeutics, Inc.

Elixirgen Therapeutics, Inc. is a Baltimore-based biotechnology company, which is focused on curing humanity's ailments through innovations in gene and cell therapy, including stem cell therapy. Elixirgen Therapeutics, Inc. is now applying its RNA technology to the development of a COVID-19 vaccine.For more information visit http://www.ElixirgenTherapeutics.com

About Fujita Health University

Fujita Health University plays a major role in treating COVID-19 patients and conducting its clinical trials in Japan. For more information visit http://www.fujita-hu.ac.jp/en/

Forward-Looking Statements

This press release may contain "forward-looking" statements, including statements regarding the potential to develop a COVID-19 vaccine and our planned clinical relationship with Fujita Health University. Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in vaccine research and development. Any forward-looking statements in this press release speak only as of the date of this press release, and Elixirgen Therapeutics undertakes no obligation to update or revise the statementsin the future, even if new information becomes available.

ContactMedia RelationsElixirgen Therapeutics, Inc.(443) 869-5420[emailprotected]

SOURCE Elixirgen Therapeutics

https://elixirgentherapeutics.com/

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Elixirgen Therapeutics planning to begin Phase I/II Clinical Trials of its COVID-19 Vaccine Candidate EXG-5003 at Fujita Health University -...

Hill Highlights the Potential of Selinexor as a Less Intensive Option for DLBCL – OncLive

Selinexor (Xpovio) may serve as a favorable therapeutic option for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for intensive chemotherapy or CAR T-cell therapy, according to Brian T. Hill, MD, PhD, who added that the agent is now being explored in combination as well as in earlier lines of treatment.

In June 2020, the FDA approved selinexor for the treatment of patients with relapsed/refractory DLBCL, not otherwise specified, including DLBCL arising from follicular lymphoma, following at least 2 lines of systemic therapy.

The regulatory decision was based on data from the phase 2b SADAL trial(NCT02227251),in which the agent elicited a 29% overall response rate (ORR; 95% CI, 22-38) in a total of 129 patients with DLBCL after 2 to 5 systemic regimens; this included a complete response rate of 13%. Notably, 38% of patients who achieved a partial response or CR had response durations of at least 6 months; 15% had response durations that persisted for at least 12 months. These responses were encouraging, noted Hill, especially for such a heavily pretreated population.

Selinexor is an oral agent that can potentially be taken for prolonged periods of time with adequate supportive care and monitoring for adverse effects (AEs); that's new to the field and new to this disease, said Hill. All of the effective therapies we've had previously have been exclusively intravenous or rely on intravenous therapy. Particularly for older patients who are not candidates for intensive therapy such as autologous stem cell transplant or CAR T-cell therapy, [selinexor] may represent a viable treatment strategy.

In an interview withOncLive,Hill, director of the Lymphoid Malignancies Program and a staff physician at the Cleveland Clinic Taussig Cancer Institute, shed light on XPO1 as a target in DLBCL, the emergence of selinexor in the treatment landscape, and exciting agents in the pipeline.

OncLive: Could you start off by describing the challenges faced in managing heavily pretreated patients with DLBCL?

Hill: After [progression on] frontline therapy, patients with DLBCL are at high risk for [additional] treatment failure and poor survival. We had autologous stem cell transplant for appropriate candidates and now we have CAR T-cell therapy, as well. However, beyond those 2 relatively intense modalities of treatment, historically, we've had very few tools to treat patients who are in a deep relapsed or refractory state.

How has treatment evolved in recent years?

The major change in the treatment of [patients with] relapsed DLBCL was introduced a couple of years ago with the FDA approval of CAR T-cell therapy. Before this modality, we really did not have any good [methods for] achieving durable remission for patients who had relapsed after autologous stem cell transplant, or for those who could never achieve sufficient disease control or have enough chemosensitivity to make it to transplant.

We're now in this postCAR T era, but the reality is that the rate of durable remission in the best of circumstances, even with CAR T-cell therapy, [ranges from approximately] 40% to 50% [of patients who go on to achieve] durable remission. That means that even with CAR T cells, over half of patients are still going to progress on their treatment. There's still an unmet need for effective therapies that can keep patients going beyond that.

Could you speak to XPO1 as a target in this disease?

XPO1 is a nuclear export protein, which shuttles various transcription factors and other regulatory proteins in and out of the cell nucleus. By inhibiting XPO1 with the selective nuclear export inhibitor selinexor, their cell undergoes apoptosis through a variety of mechanisms. This is a novel target we haven't had before in oncology.

Selinexor was recently approved by the FDA. Could you speak to thefindings from the phase 2b SADAL trialthat led to its approval?

Selinexor was previously FDA approved for use in heavily pretreated multiple myeloma and [the agent] recently gained approval for relapsed/refractory DLBCL based on findings from the SADAL trial. This [trial was done in] a heavily pretreated patient population, many of whom received multiple lines of previous therapy, including autologous stem cell transplant. In these patients, selinexor was given orally twice a week at a couple of different doses, either 100 mg or 60 mg. The toxicity seen at the high dose was significant in terms of cytopenia and gastrointestinal [AEs]. However, those AEs [were reduced when the drug was given] at the dose of 60 mg twice a week. The ORR was [around] 30%, which for a heavily pretreated patient population is very reasonable.

Is selinexor under examination in any other clinical trials?

As is frequently the case with new drug approvals, selinexor was shown to have activity in an extensively pretreated patient population. The natural progression of [research and] development is going to be to move the agent into earlier lines of treatment.

Right now, the drug is being [evaluated in] various second- and third-line platinum-based chemotherapy combinations that still have activity in DLBCL; the potential that this may be additive [in terms of] efficacy without additional toxicity is being examined, as well.

Beyond this agent, are any efforts examining XPO1 inhibition?

This target is now being explored in a wide variety of malignancies. In addition to multiple myeloma and DLBCL, [XPO1 inhibitors] now being combined with other pathway inhibitors, both for hematologic malignancies and for solid tumors.

Are any other notable agents coming down the pike that you wanted to highlight?

Just within the past year, we've had 3 drugs approved in the relapsed/refractory DLBCL setting. We have the antibody-drug conjugate polatuzumab vedotin(Polivy); the monoclonal antibody [targeted] against CD19, tafasitamab-cxix (Monjuvi), which is used in combination with lenalidomide(Revlimid); and selinexor.

[These agents are] welcome additions; [its important] to have more than 1 option [for] this patient population because it's unlikely that any of these [agents] are going to be curative. However, if were able to extend the patient's wellbeing and livelihood for a period of time following progression on curative intent therapy, [were] still clinically benefitting them.

FDA approves selinexor for relapsed/refractory diffuse large B-cell lymphoma. News release. FDA. June 22, 2020. Accessed August 30, 2020. bit.ly/37VnEXd.

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Hill Highlights the Potential of Selinexor as a Less Intensive Option for DLBCL - OncLive

Global Rheumatoid Arthritis Stem Cell Therapy Market Dynamics, Forecast, Analysis and Supply Demand 2018 to 2028 – Scientect

The globalRheumatoid Arthritis Stem Cell Therapymarketstudy presents an all in all compilation of the historical, current and future outlook of the market as well as the factors responsible for such a growth. With SWOT analysis, the business study highlights the strengths, weaknesses, opportunities and threats of each Rheumatoid Arthritis Stem Cell Therapy market player in a comprehensive way. Further, the Rheumatoid Arthritis Stem Cell Therapy market report emphasizes the adoption pattern of the Rheumatoid Arthritis Stem Cell Therapy across various industries.Request Sample Reporthttps://www.factmr.com/connectus/sample?flag=S&rep_id=1001The Rheumatoid Arthritis Stem Cell Therapy market report highlights the following players:The global market for rheumatoid arthritis stem cell therapy is highly fragmented. Examples of some of the key players operating in the global rheumatoid arthritis stem cell therapy market include Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc, International Stem Cell Corporation, TiGenix and others.

The Rheumatoid Arthritis Stem Cell Therapy market report examines the operating pattern of each player new product launches, partnerships, and acquisitions has been examined in detail.Important regions covered in the Rheumatoid Arthritis Stem Cell Therapy market report include:

North America (U.S., Canada)Latin America (Mexico, Brazil)Western Europe (Germany, Italy, U.K., Spain, France, Nordic countries, BENELUX)Eastern Europe (Russia, Poland, Rest Of Eastern Europe)Asia Pacific Excluding Japan (China, India, Australia & New Zealand)JapanMiddle East and Africa (GCC, S. Africa, Rest Of MEA)

The Rheumatoid Arthritis Stem Cell Therapy market report takes into consideration the following segments by treatment type:

Allogeneic Mesenchymal stem cellsBone marrow TransplantAdipose Tissue Stem Cells

The Rheumatoid Arthritis Stem Cell Therapy market report contain the following distribution channel:

HospitalsAmbulatory Surgical CentersSpecialty ClinicsHave Any Query? Ask our Industry Experts-https://www.factmr.com/connectus/sample?flag=AE&rep_id=1001

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The Rheumatoid Arthritis Stem Cell Therapy market report offers a plethora of insights which include:

Changing consumption pattern among individuals globally.Historical and future progress of the global Rheumatoid Arthritis Stem Cell Therapy market.Region-wise and country-wise segmentation of the Rheumatoid Arthritis Stem Cell Therapy market to understand the revenue, and growth lookout in these areas.Accurate Year-on-Year growth of the global Rheumatoid Arthritis Stem Cell Therapy market.Important trends, including proprietary technologies, ecological conservation, and globalization affecting the global Rheumatoid Arthritis Stem Cell Therapy market.

The Rheumatoid Arthritis Stem Cell Therapy market report answers important questions which include:

Which regulatory authorities have granted approval to the application of Rheumatoid Arthritis Stem Cell Therapy in Health industry?How will the global Rheumatoid Arthritis Stem Cell Therapy market grow over the forecast period?Which end use industry is set to become the leading consumer of Rheumatoid Arthritis Stem Cell Therapy by 2028?What manufacturing techniques are involved in the production of the Rheumatoid Arthritis Stem Cell Therapy?Which regions are the Rheumatoid Arthritis Stem Cell Therapy market players targeting to channelize their production portfolio?Get Full Access of the Report @https://www.factmr.com/report/1001/rheumatoid-arthritis-stem-cell-therapy-market

Pertinent aspects this study on the Rheumatoid Arthritis Stem Cell Therapy market tries to answer exhaustively are:

What is the forecast size (revenue/volumes) of the most lucrative regional market? What is the share of the dominant product/technology segment in the Rheumatoid Arthritis Stem Cell Therapy market? What regions are likely to witness sizable investments in research and development funding? What are Covid 19 implication on Rheumatoid Arthritis Stem Cell Therapy market and learn how businesses can respond, manage and mitigate the risks? Which countries will be the next destination for industry leaders in order to tap new revenue streams? Which new regulations might cause disruption in industry sentiments in near future? Which is the share of the dominant end user? Which region is expected to rise at the most dominant growth rate? Which technologies will have massive impact of new avenues in the Rheumatoid Arthritis Stem Cell Therapy market? Which key end-use industry trends are expected to shape the growth prospects of the Rheumatoid Arthritis Stem Cell Therapy market? What factors will promote new entrants in the Rheumatoid Arthritis Stem Cell Therapy market? What is the degree of fragmentation in the Rheumatoid Arthritis Stem Cell Therapy market, and will it increase in coming years?Why Choose Fact.MR?

Fact.MR follows a multi- disciplinary approach to extract information about various industries. Our analysts perform thorough primary and secondary research to gather data associated with the market. With modern industrial and digitalization tools, we provide avant-garde business ideas to our clients. We address clients living in across parts of the world with our 24/7 service availability.

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Global Rheumatoid Arthritis Stem Cell Therapy Market Dynamics, Forecast, Analysis and Supply Demand 2018 to 2028 - Scientect

Growing at an annualized rate of over 20%, the cell therapy manufacturing market is estimated to reach close to USD 10 Billion by 2030, claims Roots…

The approval of KYMRIAH, YESCARTA, Alofisel and Zyntelgo has increased the interest of pharma stakeholders in cell therapies; further, owing to the technical challenges in this field, outsourcing manufacturing operations has become a necessity

Roots Analysis has announced the addition of Cell Therapy Manufacturing Market (3rd Edition), 2019 2030 report to its list of offerings.

Owing to various reasons, the demand for cell therapies is anticipated to increase over the coming years. Therefore, both therapy developers and contract service providers may need to strengthen their capabilities and expand available capacity. In this context, automation is expected to be a key enabler within the cell therapy manufacturing and contract services industry.

To order this 500+ page report, which features 160+ figures and 250+ tables, please visit this link

More than 160 organizations claim to be engaged in cell therapy manufacturingThe market landscape is dominated by industry players, representing more than 60% of the total number of stakeholders. Amongst these, over 55 are large or mid-sized firms (having more than 50 employees).

100+ players focused on T-cell and stem cell therapiesMost of these players are focused on manufacturing T-cell therapies, including CART, TCR or TILs. It is worth highlighting that more than 35 organizations claim to have necessary capabilities for the manufacturing of both types of therapies.

Presently, 70+ companies have commercial scale capacityAs majority of the cell therapy products are in clinical trials, the demand is high at this scale. However, it is worth noting that several players (~50%) have already developed commercial scale capacity for cell therapies.

Europe is currently considered a current hub for cell therapy productionMore than 220 manufacturing facilities have been established by various players, worldwide; of these, 35% are in Europe, followed by those based in North America. Other emerging regions include Australia, China, Japan, Singapore, South Korea and Israel.

50+ facility expansions reported between 2015-2019More than 85% of the expansions are related to setting up of new facilities across different regions. Maximum expansion activity was observed in the US and in certain countries within the Asia Pacific regions.

20+ companies offer automated solutions to cell therapy developersPlayers that claim to offer consultancy services related to automation include (in alphabetical order) Berkeley Lights, Cesca Therapeutics, Ferrologix, FluDesign Sonics, GE Healthcare and Terumo BCT. Further, we identified players, namely (in alphabetical order) Fraunhofer Institute for Manufacturing Engineering and Automation IPA, Invetech, KMC Systems, Mayo Clinic Center for Regenerative Medicine and RoosterBio, that offer consultancy solutions related to automation.

Partnership activity has grown at an annualized rate of 16%, between 2014 and 2018More than 200 agreements have been inked in the last 5 years; majority of these were focused on the supply of cell-based therapy products for clinical trials. Other popular types of collaboration models include manufacturing process development agreements (16%), services agreements (12%) and acquisitions (10%).

By 2030, developed geographies will capture over 60% of the market shareAsia Pacific is anticipated to capture the major share (~36%) of the market by 2030. It is also important to highlight that financial resources, technical expertise and established infrastructure is likely to drive cell therapy manufacturing market in Europe, which is estimated to grow at a CAGR of ~26%.

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The USD 10+ billion (by 2030) financial opportunity within the cell therapy manufacturing market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom the manufacturing of cell therapies is largely being outsourced due to exorbitant costs associated with the setting-up of in-house expertise. The report includes detailed transcripts of discussions held with the following experts:

The research covers profiles of key players (industry and non-industry) that offer manufacturing services for cell-based therapies, featuring a company overview, information on manufacturing facilities, and recent collaborations.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html or email [emailprotected]

You may also be interested in the following titles:

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]nalysis.com

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Growing at an annualized rate of over 20%, the cell therapy manufacturing market is estimated to reach close to USD 10 Billion by 2030, claims Roots...

Strategic Analysis to Understand the Competitive Outlook of Cell Therapy Manufacturing Market – The News Brok

Prophecy Market Insights Cell Therapy Manufacturing market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges. The research report study offers keen competitive landscape analysis including key development trends, accurate quantitative and in-depth commentary insights, market dynamics, and key regional development status forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.

Get Free Sample Copy of This Report @ https://www.prophecymarketinsights.com/market_insight/Insight/request-sample/21

An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Cell Therapy Manufacturing market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.

Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Segmentation Overview:

Cell Therapy ManufacturingMarket Key Companies:

harmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

The Cell Therapy Manufacturing research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Cell Therapy Manufacturing market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

Download PDF Brochure for report overview @ https://www.prophecymarketinsights.com/market_insight/Insight/request-pdf/21

The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. This report is a must-read for investors, entrepreneurs, consultants, researchers, business strategists, and all those who have any kind of stake or are planning to foray into the Cell Therapy Manufacturing industry in any manner.

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Strategic Analysis to Understand the Competitive Outlook of Cell Therapy Manufacturing Market - The News Brok

Tevogen Bio Announces Partnership With Preeminent Scientist Professor Neal Flomenberg, MD, to Investigate Proprietary T-Cell Therapy for Treatment of…

METUCHEN, N.J., Aug. 10, 2020 /PRNewswire/ --Tevogen Bio announces a joint partnership with renowned bone-marrow transplant expertNeal Flomenberg, M.D., Professor and Chair of the Department of Medical Oncology at Thomas Jefferson University, with the intent to evaluate Tevogen' s proprietary antigen-specific T cell technology as a potential treatment for COVID-19 and influenza-A patients.

This collaboration aims to harness Tevogen's proprietary immunotherapy platform and Dr. Flomenberg's expertise and research prowess to investigate potential treatments for viral infections.

Dr. Flomenberg has been at the forefront of immunogenetics and immunology for more than four decades. "Tevogen's technology resonated with me as there have been several groups who have used T cells to treat patients after bone-marrow transplants. The idea of utilizing T cell therapies to potentially treat COVID-19 and other viruses is truly remarkable," Flomenberg said. "I'm enthusiastic about moving forward with an investigation of Tevogen's technologies."

Tevogen CEO Ryan Saadi, M.D., M.P.H., is leading the new biotech's efforts. "Our work has been to pioneer T cell therapies that can be abundantly and efficiently reproduced to develop an affordable and scalable cellular treatment for the biggest global health threats, including COVID-19, influenza, and a variety of cancers. We are very excited about Dr. Flomenberg's contribution to our efforts and hope to initiate our investigational study soon."

In addition to developing its potential therapies, Tevogen is committed to organizational and manufacturing efficiency. This should allow it to engage in affordable innovation to the benefit of all patients.

About Tevogen Bio

Tevogen Bio was formed after decades of research by its contributors to concentrate and leverage their expertise, spanning multiple sectors of the health care industry, to help address some of the most common and deadly illnesses known today. The company's mission is to provide curative and preventative treatments that are affordable and scalablein order to positively impact global public health.

About Dr. Neal Flomenberg

Dr. Neal Flomenberg is the Chairman of Medical Oncology at Jefferson University in Philadelphia and also heads the Hematologic Malignancies, Blood and Marrow Transplantation (BMT) Program. Throughout his more than four decades of practice, he has maintained a longstanding interest in the immunogenetics and immunology of stem cell transplantation, with the goal of making transplantation safer and more widely available. Dr. Flomenberg developed an approach to bone-marrow transplants that uses half-matched relatives as donors, a breakthrough that assures that the majority of blood and bone-marrow cancer patients can benefit from this potentially curative treatment.

Media Contacts:

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Tevogen Bio Announces Partnership With Preeminent Scientist Professor Neal Flomenberg, MD, to Investigate Proprietary T-Cell Therapy for Treatment of...

In Depth Analysis and Survey of COVID-19 Pandemic Impact on Global Canine Stem Cell Therapy Coronavirus Impact Editon of Key Players VETSTEM…

Rising number of corona virus cases has impacted numerous lives and led to numerous fatalities, and has affected the overall economic structure globally. The Canine Stem Cell Therapy has analyzed and published the latest report on the global Canine Stem Cell Therapy market. Change in the market has affected the global platform. Along with the Canine Stem Cell Therapy market, numerous other markets are also facing similar situations. This has led to the downfall of numerous businesses, because of the widespread increase of the number of cases across the globe.href=mailto:nicolas.shaw@cognitivemarketresearch.com>nicolas.shaw@cognitivemarketresearch.com or call us on +1-312-376-8303.

Request Free Sample Copy of Canine Stem Cell Therapy Market Research Report@ https://cognitivemarketresearch.com/medical-devicesconsumables/canine-stem-cell-therapy-market-report

The major players in the Canine Stem Cell Therapy market are VETSTEM BIOPHARMA, Cell Therapy Sciences, Regeneus, Aratana Therapeutics, Medivet Biologics, Okyanos, Vetbiologics, VetMatrix, Magellan Stem Cells, ANIMAL CELL THERAPIES, Stemcellvet . Some of the players have adopted new strategies to sustain their position in the Canine Stem Cell Therapy market. A detailed research study is done on the each of the segments, and is provided in Canine Stem Cell Therapy market report. Based on the performance of the Canine Stem Cell Therapy market in various regions, a detailed study of the Canine Stem Cell Therapy market is also analyzed and covered in the study.

Report Scope:Some of the key types analyzed in this report are as follows: Allogeneic Stem Cells, Autologous Stem cells

Some of the key applications as follow: Veterinary Hospitals, Veterinary Clinics, Veterinary Research Institutes

Following are the major key players: VETSTEM BIOPHARMA, Cell Therapy Sciences, Regeneus, Aratana Therapeutics, Medivet Biologics, Okyanos, Vetbiologics, VetMatrix, Magellan Stem Cells, ANIMAL CELL THERAPIES, Stemcellvet

An in-depth analysis of the Canine Stem Cell Therapy market is covered and included in the research study. The study covers an updated and a detailed analysis of the Canine Stem Cell Therapy market. It also provides the statistical information of the Canine Stem Cell Therapy market. The study of the report consists of the detailed definition of the market or the overview of the Canine Stem Cell Therapy market. Furthermore, it also provides detailed information for the target audience dealing with or operating in this market is explained in the next section of the report.

Read Detailed Index of full Research Study @: https://cognitivemarketresearch.com/medical-devicesconsumables/canine-stem-cell-therapy-market-report#download_report

The report also provides detailed information on the research methodologies, which are used for the analysis of the Canine Stem Cell Therapy market. The methods are covered in detail in this section of the report. For the analysis of the market, several tools are used for the extraction of the market numbers. Among the several tools, primary and secondary research studies were also incorporated for the research study. These were further analyzed and validated by the market experts, to increase precision and make the data more reliable.

Moreover, the report also highlights and provides a detailed analysis of the drivers, restrains, opportunities, and challenges of the Canine Stem Cell Therapy market. This section of Canine Stem Cell Therapy market also covers the updated information, in accordance with the present situation of the market.

According to the estimation and the analysis of the market, the Canine Stem Cell Therapy market is likely to have some major changes in the estimated forecasts period. Moreover, these changes can be attributed to the changes due to economic and trading conditions across the globe. Moreover, several market players operating in the Canine Stem Cell Therapy market will have to strategically change their business strategies in order to survive in the market.

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Reasons for Buying this Canine Stem Cell Therapy Report1. Canine Stem Cell Therapy market advertise report helps with understanding the Basic product segments alongside likewise their potential future.2. This global Canine Stem Cell Therapy report offers pin-point evaluation for changing competitive dynamics.3. The Canine Stem Cell Therapy market supplies pin point analysis of changing competition dynamics and keeps you in front of competitors4. Original images and illustrated a SWOT evaluation of large segments supplied by the Canine Stem Cell Therapy market.5. This report supplies a forward-looking perspective on different driving factors or controlling Canine Stem Cell Therapy market gain.6. This report assists to make wise business choices using whole insights of the Canine Stem Cell Therapy and also from creating a comprehensive evaluation of market sections.Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

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In Depth Analysis and Survey of COVID-19 Pandemic Impact on Global Canine Stem Cell Therapy Coronavirus Impact Editon of Key Players VETSTEM...


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