Archive for the ‘Cardiac Stem Cells’ Category

DUBLIN, Feb. 9, 2021 /PRNewswire/ -- The "Cell Therapy Market by Cell Type, Therapy Type, Therapeutic Area, and End User: Global Opportunity Analysis and Industry Forecast, 2020-2027" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market accounted for $7,754. 89 million in 2019, and is expected to reach $48,115. 40 million by 2027, registering a CAGR of 25. 6% from 2020 to 2027.

Cell therapy involves administration of somatic cell preparations for treatment of diseases or traumatic damages. Cell therapy aims to introduce new, healthy cells into a patient's body to replace diseased or missing ones.

This is attributed to the fact that specialized cells, such as brain cells, are difficult to obtain from human body. In addition, specialized cells typically have a limited ability to multiply, making it difficult to produce sufficient number of cells required for certain cell therapies. Some of these issues can be overcome through the use of stem cells. In addition, cells such as blood and bone marrow cells, mature, immature & solid tissue cells, adult stem cells, and embryonic stem cells are widely used in cell therapy procedures.

Moreover, transplanted cells including induced pluripotent stem cells (iPSCs), embryonic stem cells (ESCs), neural stem cells (NSCs), and mesenchymal stem cells (MSCs) are divided broadly into two main groups including autologous cells and non-autologous cells. Development of precision medicine and advancements in Advanced Therapies Medicinal Products (ATMPS) in context to their efficiency and manufacturing are expected to be the major drivers for the market. Furthermore, automation in adult stem cells and cord blood processing and storage are the key technological advancements that fuel growth of the market for cell therapy.

In addition, growth in aging patient population, The rise in cell therapy transplantations globally, and surge in disease awareness drive growth of the global cell therapy market. Furthermore, The rise in adoption of human cells over animal cells for cell therapeutics research, technological advancements in field of cell therapy, and increase in incidences of diseases such as cancer, cardiac abnormalities, and organ failure are the key factors that drive growth of the global market.

Moreover, implementation of stringent government regulations regarding the use of cell therapy is anticipated to restrict growth of the market. On the contrary, surge in number of regulations to promote stem cell therapy and increase in funds for research in developing countries are expected to offer lucrative opportunities to the market in the future.

The global cell therapy market is categorized on the basis of therapy type, therapeutic area, cell type, end user, and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutics, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others.

The global cell therapy market is categorized on the basis of therapy type, therapeutic, cell type, end user and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutic area, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others. On the basis of cell type, it is segregated into stem cell therapy and non-stem cell type. On the basis of end user, it is segregated into hospital & clinics and academic & research institutes. On the basis of region, the market is studied across North America, Europe, Asia-Pacific, and LAMEA.

Key Benefits

Key Topics Covered:

Chapter 1: Introduction1.1. Report Description1.2. Key Benefits for Stakeholders1.3. Key Market Segments1.4. Research Methodology1.4.1. Secondary Research1.4.2. Primary Research1.4.3. Analyst Tools & Models

Chapter 2: Executive Summary2.1. Key Findings of the Study2.2. Cxo Perspective

Chapter 3: Market Overview3.1. Market Definition and Scope3.2. Key Findings3.2.1. Top Player Positioning3.2.2. Top Investment Pockets3.2.3. Top Winning Strategies3.3. Porter's Five Forces Analysis3.4. Impact Analysis3.4.1. Drivers3.4.1.1. Technological Advancements in the Field of Cell Therapy3.4.1.2. The Rise in Number of Cell Therapy Clinical Studies3.4.1.3. The Rise in Adoption of Regenerative Medicine3.4.2. Restraint3.4.2.1. Developing Stage and Pricing3.4.3. Opportunity3.4.3.1. High Growth Potential in Emerging Markets3.5. Impact of Covid-19 on Cell Therapy Market

Chapter 4: Cell Therapy Market, by Cell Type4.1. Overview4.1.1. Market Size and Forecast4.2. Stem Cell4.2.1. Key Market Trends and Opportunities4.2.2. Market Size and Forecast, by Region4.2.3. Market Size and Forecast, by Type4.2.3.1. Bone Marrow, Market Size and Forecast4.2.3.2. Blood, Market Size and Forecast4.2.3.3. Umbilical Cord-Derived, Market Size and Forecast4.2.3.4. Adipose-Derived Stem Cell, Market Size and Forecast4.2.3.5. Others (Placenta, and Nonspecific Cells), Market Size and Forecast4.3. Non-Stem Cell4.3.1. Key Market Trends and Opportunities4.3.2. Market Size and Forecast, by Region

Chapter 5: Cell Therapy Market, by Therapy Type5.1. Overview5.1.1. Market Size and Forecast5.2. Autologous5.2.1. Key Market Trends and Opportunities5.2.2. Market Size and Forecast, by Region5.2.3. Market Analysis, by Country5.3. Allogeneic5.3.1. Key Market Trends and Opportunities5.3.2. Market Size and Forecast, by Region5.3.3. Market Analysis, by Country

Chapter 6: Cell Therapy Market, by Therapeutic Area6.1. Overview6.1.1. Market Size and Forecast6.2. Malignancies6.2.1. Market Size and Forecast, by Region6.2.2. Market Analysis, by Country6.3. Musculoskeletal Disorders6.3.1. Market Size and Forecast, by Region6.3.2. Market Analysis, by Country6.4. Autoimmune Disorders6.4.1. Market Size and Forecast, by Region6.4.2. Market Analysis, by Country6.5. Dermatology6.5.1. Market Size and Forecast, by Region6.5.2. Market Analysis, by Country6.6. Others6.6.1. Market Size and Forecast, by Region6.6.2. Market Analysis, by Country

Chapter 7: Cell Therapy Market, by End-user7.1. Overview7.1.1. Market Size and Forecast7.2. Hospitals & Clinics7.2.1. Key Market Trends and Opportunities7.2.2. Market Size and Forecast, by Region7.2.3. Market Analysis, by Country7.3. Academic & Research Institutes7.3.1. Key Market Trends and Opportunities7.3.2. Market Size and Forecast, by Region7.3.3. Market Analysis, by Country

Chapter 8: Cell Therapy Market, by Region8.1. Overview8.2. North America8.3. Europe8.4. Asia-Pacific8.5. LAMEA

Chapter 9: Company Profiles9.1. Allosource9.1.1. Company Overview9.1.2. Company Snapshot9.1.3. Operating Business Segments9.1.4. Product Portfolio9.1.5. Key Strategic Moves and Developments9.2. Cells for Cells9.2.1. Company Overview9.2.2. Company Snapshot9.2.3. Operating Business Segments9.2.4. Product Portfolio9.3. Holostem Terapie Avanzate Srl9.3.1. Company Overview9.3.2. Company Snapshot9.3.3. Operating Business Segments9.3.4. Product Portfolio9.4. Jcr Pharmaceuticals Co. Ltd.9.4.1. Company Overview9.4.2. Company Snapshot9.4.3. Operating Business Segments9.4.4. Product Portfolio9.4.5. Business Performance9.4.6. Key Strategic Moves and Developments9.5. Kolon Tissuegene, Inc.9.5.1. Company Overview9.5.2. Company Snapshot9.5.3. Operating Business Segments9.5.4. Product Portfolio9.5.5. Key Strategic Moves and Developments9.6. Medipost Co. Ltd.9.6.1. Company Overview9.6.2. Company Snapshot9.6.3. Operating Business Segments9.6.4. Product Portfolio9.6.5. Business Performance9.7. Mesoblast Ltd9.7.1. Company Overview9.7.2. Company Snapshot9.7.3. Operating Business Segments9.7.4. Product Portfolio9.7.5. Business Performance9.8. Nuvasive, Inc.9.8.1. Company Overview9.8.2. Company Snapshot9.8.3. Operating Business Segments9.8.4. Product Portfolio9.8.5. Business Performance9.9. Osiris Therapeutics, Inc.9.9.1. Company Overview9.9.2. Company Snapshot9.9.3. Operating Business Segments9.9.4. Product Portfolio9.10. Stemedica Cell Technologies, Inc.9.10.1. Company Overview9.10.2. Company Snapshot9.10.3. Operating Business Segments9.10.4. Product Portfolio

For more information about this report visit https://www.researchandmarkets.com/r/shw12n

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Worldwide Cell Therapy Industry to 2027 - Profiling Allosource, Medipost and Mesoblast Among Others - PRNewswire

The Daily Beast

Andrew Harnik/APSenate Majority Leader Chuck Schumer (D-NY) stood alongside high-profile progressives in Congress in front of a podium that said #CANCEL STUDENT DEBT, a favorite slogan of the activist class, to push the Biden administration on a key economic issue.The resolution, which Schumer first introduced last fall with Sen. Elizabeth Warren (D-MA), would cancel $50,000 in student loan debt for each borrower through executive action, a sum that goes far beyond what Biden has already pledged to nix while in office.In an outdoor briefing on Thursday, the Democratic leader said he has already had a receptive response from the White House.We have met with the president, we are pushing the president and his people, and we are very hopeful, Schumer said, sharing that he and Warren met with Biden and administration officials privately for 45 minutes to lay out a proposed executive action.Biden has promised to eliminate $10,000 in federal student loan debt for each student.Asked about the renewed push later in the afternoon, White House press secretary Jen Psaki reiterated Bidens support for his original proposal, suggesting that it was unlikely that anything more would be done through executive orders.On day one, the first day of his administration, he directed the Department of Education to extend the existing pause on student loan payments and interest for millions of Americans with federal student loans, Psaki said. That was a step he took through executive action, but he certainly supports efforts by members in Congress to take additional steps, and he would look forward to signing it.Schumer was joined by Warren and Squad Reps. Ayanna Pressley (D-MA) and Ilhan Omar (D-MN)the original co-sponsors of the companion House resolution from last Decemberas well as other House members pressing the issue.America does not suffer from scarcity, we suffer from greed, Omar said, linking burdensome debt to the differing chances of students who come from wealthy families versus those in middle and working class households.Progressives Cant Find Anyone in Biden's Cabinet to Be Mad AboutYetSchumers desire to publicly present a loan forgiveness alternative to what Biden has offered has been perceived by some on the left as a way to help stave off a possible primary challenge in his native New York. The senior Democrat is up for re-election in 2022 and Rep. Alexandria Ocasio-Cortez (D-NY) is thought to be contemplating a primary challenge for this Senate seat.Read more at The Daily Beast.Get our top stories in your inbox every day. Sign up now!Daily Beast Membership: Beast Inside goes deeper on the stories that matter to you. Learn more.

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Evotec and Medical Center Hamburg-Eppendorf Enter Partnership to Develop iPSC-Based Tissue Therapy for Heart Failure - Yahoo Finance UK

Excitement took wing in the scientific community in the early 1990s, when the first gene therapy trial showed significant success, only to crash at the end of the decade with a patients tragic death.

Twenty years later, the excitement is back and greater than before. Although safety remains a concern, investigators are breaking ground in cell and gene therapy, and many believe that ultimately, a string of cured cancers will follow.

In 2017, the excitement over these therapies returned in spades when the FDA signed off on a cell-therapy drug for the first time, approving the chimeric antigen receptor (CAR) T-cell treatment tisagenlecleucel (Kymriah; Novartis) for patients with B-cell precursor acute lymphoblastic leukemia. At last, scientists had devised a way to reprogram a persons own T cells to attack tumor cells.

Were entering a new frontier, said Scott Gottlieb, MD, then-FDA commissioner, in announcing the groundbreaking approval.

Gottlieb was not exaggerating. The growth in CAR T-cell research is exploding. Although only a handful of cell and gene therapies are on the market, the FDA predicted in 2019 that it will receive more than 200 investigational new drug applications per year for cell and gene therapies, and that by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.

We can absolutely cut the number of cancer deaths down so that one day in our lifetimes it can be a rare thing for people to die of cancer, said Patrick Hwu, MD, president and CEO of Moffitt Cancer Center in Florida and among gene therapys pioneers. It still may happen here and there, but itll be kind of like people dying of pneumonia. Its like, He died of pneumonia? Thats kind of weird. I think cancer can be the same way.

Essentially, you can kill any cancer cell that has an antigen that is recognized by the immune cell, Hwu said. The key to curing every single cancer, which is our goal, is to have receptors that can recognize the tumor but dont recognize the normal cells.

Community oncologists will need to be increasingly familiar about the various products, including their immediate and longer-term risks, Bo Wang, MD, and Deepu Madduri, MD, recently wrote in OncologyLive.1 It is key to understand the optimal time for referring these patients to an academic institution, as well as how to manage the requisite post CAR T-cell therapy in the community setting. Madduri is an assistant professor of medicine, hematology and medical oncology, as well as associate director of cellular therapy service, and director of clinical operations with the Center of Excellence for Multiple Myeloma at The Tisch Cancer Institute and the Icahn School of Medicine at Mount Sinai in New York, New York. Wang is a third-year clinical fellow in hematology/oncology at Mount Sinai.

Early referral to academic centers and hospitals equipped to deliver therapies is crucial for patients eligible for therapy. However, as advances continue in the field, community practices may be called upon to administer therapies in their clinic.

The Community Oncology Alliance (COA) envisions a broader role for the settings in which CAR T-cell therapies can be administered. When the Centers for Medicare & Medicaid Services (CMS) was considering coverage for CAR T-cell therapies in 2019, COA officials argued against limiting approvals to hospitals.

It is important to understand that there are state-of-the-art community oncology practices that have significant experience and capabilities in administering highly complex treatments, COA officials wrote in a letter to CMS. For example, stem cell transplants, which are similar in complexity to CAR T therapy, are performed successfully in the community oncology practice setting.2

Broader use of gene therapies depends on several factors, including navigating the logistics of gene therapies, addressing the high costs, and managing toxicities.3

Autologous CAR T-cell therapies involve a manufacturing process that requires coordination between the treating facility and the processing facility. Following leukapheresis, patients may require maintenance therapy to control disease progression during the manufacturing time, which can take 3 to 5 weeks.

In terms of cost, gene and cell therapies can cost from $375,000 to $475,000 per dose and they may face coverage restrictions from payers. Approvals could take weeks to obtain.3,4

Because of cytokine release syndrome and neurotoxicities associated with CAR T-cell therapy, the FDA mandates risk evaluation and mitigation strategy training for centers.

Further, providers may find that real-world experiences with gene therapies are different from those seen in the clinical trial setting, according to Ankit J. Kansagra, MD.

In a presentation at the 2020 American Society of Clinical Oncology Virtual Education Program, Kansagra, an assistant professor of medicine and Eugene P. Frenkel, MD, Scholar in Clinical Medicine at Harold C. Simmons Comprehensive Cancer Center in Dallas, Texas, said that in practice patients may be older and have more aggressive disease, with double- and triple-hit lymphomas.4

Specifically, Kansagra noted that medications such as steroids and/or tocilizumab (Actemra) to prevent or treat cytokine release syndrome or other toxicities were more frequently used in the real-world setting than what had been seen in clinical trials.

As it stands now, only a fraction of eligible patients are receiving CAR T-cell therapies, Kansagra said. Potentially, 9750 patients a year may be eligible for CAR T-cell therapies in approved and upcoming hematologic indications. From 2016 to 2019, a total of 2058 patients received CAR T-cell infusion.4

Next steps for transplanting these novel therapies to clinical practice will require changes in key areas, Kansagra said, such as supply chain management, patient support, and financial systems (Figure).4

Figure. Next Steps for Effective Delivery of Gene and Cell Therapies4

Meanwhile, multiple myeloma experts advise providers to be ready for change. As commercially available myeloma CAR T-cell therapies are approved, it will be even more important for community oncologists to better understand these therapies so they can offer them to their patients, Wang and Madduri wrote.1

Cell therapy involves cultivating or modifying immune cells outside the body before injecting them into the patient. Cells may be autologous (self-provided) or allogeneic (donor-provided); they include hematopoietic stem cells and adult and embryonic stem cells. Gene therapy modifies or manipulates cell expression. There is considerable overlap between the 2 disciplines.

Juliette Hordeaux, PhD, senior director of translational research for the University of Pennsylvanias gene therapy program, is cautious about the FDAs predictions, saying shed be thrilled with 5 cell and/or gene therapy approvals annually.

For monogenic diseases, there are only a certain number of mutations, and then well plateau until we reach a stage where we can go after more common diseases, Hordeaux said.

Safety has been the main brake around adeno-associated virus vector [AAV] gene therapy, added Hordeaux, whose hospitals program has the institutional memory of both Jesse Gelsingers tragic death during a 1999 gene therapy trial as well as breakthroughs by 2015 Giants of Cancer Care winner in immuno-oncology Carl H. June, MD, and others in CAR T-cell therapy. Sometimes there are unexpected toxicity [events] in trials.I think figuring out ways to make gene therapy safer is going to be the next goal for the field before we can even envision many more drugs approved.

In total, 3 CAR T-cell therapies are now on the market, all targeting the CD19 antigen. Tisagenlecleucel was the first. Gilead Sciences received approval in October 2017 for axicabtagene ciloleucel (axi-cel; Yescarta), a CAR T-cell therapy for adults with large B-cell non-Hodgkin lymphoma. Kite Pharma, a subsidiary of Gilead, received an accelerated approval in July 2020 for brexucabtagene autoleucel (Tecartus) for adults with relapsed/ refractory mantle cell lymphoma.

Another CD19-directed therapy under FDA review for relapsed/refractory large B-cell lymphoma, is lisocabtagene maraleucel (liso-cel; JCAR017; Bristol Myers Squibb). Idecabtagene vicleucel (ide-cel; bb2121; Bristol Myers Squibb) is under priority FDA review, with a decision expected by March 31, 2021. The biologics license application for ide-cel seeks approval for the B-cell maturation antigendirected CAR therapy to treat adult patients with multiple myeloma who have received at least 3 prior therapies.5

The number of clinical trials evaluating CAR T-cell therapies has risen sharply since 2015, when investigators counted a total of 78 studies registered on the ClinicalTrials. gov website. In June 2020, the site listed 671 trials, including 357 registered in China, 256 in the United States, and 58 in other countries.6 Natural killer (NK) cells are the research focus of Dean A. Lee, MD, PhD, a physician in the Division of Hematology and Oncology at Nationwide Childrens Hospital in Columbus, Ohio. He developed a method for consistent, robust expansion of highly active clinical-grade NK cells that enables repeated delivery of large cell doses for improved efficacy. This finding led to several first-in-human clinical trials evaluating adoptive immunotherapy with expanded NK cells under an FDA investigational new drug application. Lee is developing both genetic and nongenetic methods to improve tumor targeting and tissue homing of NK cells. His efforts are geared toward pediatric sarcomas.

The biggest emphasis over the past 20 to 25 years has been cell therapy for cancer, talking about trying to transfer a specific part of the immune system for cells, said Lee, who is also director of the Cellular Therapy and Cancer Immunology Program at Nationwide Childrens Hospital, at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital, and at the Richard J. Solove Research Institute.

However, Lee said, NKs have wider potential. This is kind of a natural swing back. Now that we know we can grow them, we can reengineer them against infectious disease targets and use them in that [space], he said.

Lee is part of a coronavirus disease 2019 (COVID-19) clinical trial, partnering with Kiadis, for off-the-shelf K-NK cells using Kiadis proprietary platforms. Such treatment would be a postexposure preemptive therapy for treating COVID-19. Lee said the pivot toward treating COVID19 with cell therapy was because some of the very early reports on immune responses to coronavirus, both original [SARS-CoV-2] and the new [mutation], seem to implicate that those who did poorly [overall] had poorly functioning NK cells.

The revolutionary gene editing tool CRISPR is making its initial impact in clinical trials outside the cancer area. Its developers, Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, won the Nobel Prize in Chemistry 2020.

For patients with sickle cell disease (SCD), CRISPR was used to reengineer bone marrow cells to produce fetal hemoglobin, with the hope that the protein would turn deformed red blood cells into healthy ones. National Public Radio (NPR) did a story on one patient who, so far, thanks to CRISPR, has been liberated from the attacks of SCD that typically have sent her to the hospital, as well from the need for blood transfusions.7

Its a miracle, you know? the patient, Victoria Gray of Forest, Mississippi, told NPR.

She was among 10 patients with SCD or transfusion-dependent beta-thalassemia treated with promising results, as reported by the New England Journal of Medicine.8

Stephen Gottschalk, MD, chair of the department of bone marrow transplantation and cellular therapy at St Jude Childrens Research Hospital, said, Theres a lot of activity to really explore these therapies with diseases that are much more common than cancer.

Animal models use T cells to reverse cardiac fibrosis, for instance, Gottschalk said. Using T cells to reverse pathologies associated with senescence, such as conditions associated with inflammatory clots, are also being studied.

CAR T, I think, will become part of the standard of care, Gottschalk said. The question is how to best get that accomplished. To address the tribulations of some autologous products, a lot of groups are working with off-the-shelf products to get around some of the manufacturing bottlenecks. I believe those issues will be solved in the long run.

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Harnessing the Potential of Cell and Gene Therapy - OncLive

Newswise PHILADELPHIAScientists in the Perelman School of Medicine at the University of Pennsylvania have uncovered the molecular causes of a congenital form of dilated cardiomyopathy (DCM), an often-fatal heart disorder.

This inherited form of DCM which affects at least several thousand people in the United States at any one time and often causes sudden death or progressive heart failure is one of multiple congenital disorders known to be caused by inherited mutations in a gene called LMNA. The LMNA gene is active in most cell types, and researchers have not understood why LMNA mutations affect particular organs such as the heart while sparing most other organs and tissues.

In the study, published this week in Cell Stem Cell, the Penn Medicine scientists used stem cell techniques to grow human heart muscle cells containing DCM-causing mutations in LMNA. They found that these mutations severely disrupt the structural organization of DNA in the nucleus of heart muscle cells but not two other cell types studied leading to the abnormal activation of non-heart muscle genes.

Were now beginning to understand why patients with LMNA mutations have tissue-restricted disorders such as DCM even though the gene is expressed in most cell types, said study co-senior author Rajan Jain, MD, an assistant professor of Cardiovascular Medicine and Cell and Developmental Biology at the Perelman School of Medicine.

Further work along these lines should enable us to predict how LMNA mutations will manifest in individual patients, and ultimately we may be able to intervene with drugs to correct the genome disorganization that these mutations cause, said study co-senior author Kiran Musunuru, MD, PhD, a professor of Cardiovascular Medicine and Genetics, and Director of the Genetic and Epigenetic Origins of Disease Program at Penn Medicine.

Inherited LMNA mutations have long puzzled researchers. The LMNA gene encodes proteins that form a lacy structure on the inner wall of the cell nucleus, where chromosomes full of coiled DNA are housed. This lacy structure, known as the nuclear lamina, touches some parts of the genome, and these lamina-genome interactions help regulate gene activity, for example in the process of cell division. The puzzle is that the nuclear lamina is found in most cell types, yet the disruption of this important and near-ubiquitous cellular component by LMNA mutations causes only a handful of relatively specific clinical disorders, including a form of DCM, two forms of muscular dystrophy, and a form of progeria a syndrome that resembles rapid aging.

To better understand how LMNA mutations can cause DCM, Jain, Musunuru, and their colleagues took cells from a healthy human donor, and used the CRISPR gene-editing technique to create known DCM-causing LMNA mutations in each cell. They then used stem cell methods to turn these cells into heart muscle cells cardiomyocytes and, for comparison, liver and fat cells. Their goal was to discover what was happening in the mutation-containing cardiomyocytes that wasnt happening in the other cell types.

The researchers found that in the LMNA-mutant cardiomyocytes but hardly at all in the other two cell types the nuclear lamina had an altered appearance and did not connect to the genome in the usual way. This disruption of lamina-genome interactions led to a failure of normal gene regulation: many genes that should be switched off in heart muscle cells were active. The researchers examined cells taken from DCM patients with LMNA mutations and found similar abnormalities in gene activity.

A distinctive pattern of gene activity essentially defines what biologists call the identity of a cell. Thus the DCM-causing LMNA mutations had begun to alter the identity of cardiomyocytes, giving them features of other cell types.

The LMNA-mutant cardiomyocytes also had another defect seen in patients with LMNA-linked DCM: the heart muscle cells had lost much of the mechanical elasticity that normally allows them to contract and stretch as needed. The same deficiency was not seen in the LMNA-mutant liver and fat cells.

Research is ongoing to understand whether changes in elasticity in the heart cells with LMNA mutations occurs prior to changes in genome organization, or whether the genome interactions at the lamina help ensure proper elasticity. Their experiments did suggest an explanation for the differences between the lamina-genome connections being badly disrupted in LMNA-mutant cardiomyocytes but not so much in LMNA-mutant liver and fat cells: Every cell type uses a distinct pattern of chemical marks on its genome, called epigenetic marks, to program its patterns of gene activity, and this pattern in cardiomyocytes apparently results in lamina-genome interactions that are especially vulnerable to disruption in the presence of certain LMNA mutations.

The findings reveal the likely importance of the nuclear lamina in regulating cell identity and the physical organization of the genome, Jain said. This also opens up new avenues of research that could one day lead to the successful treatment or prevention of LMNA-mutations and related disorders.

Other co-authors of the study were co-first authors Parisha Shah and Wenjian Lv; and Joshua Rhoades, Andrey Poleshko, Deepti Abbey, Matthew Caporizzo, Ricardo Linares-Saldana, Julie Heffler, Nazish Sayed, Dilip Thomas, Qiaohong Wang, Liam Stanton, Kenneth Bedi, Michael Morley, Thomas Cappola, Anjali Owens, Kenneth Margulies, David Frank, Joseph Wu, Daniel Rader, Wenli Yang, and Benjamin Prosser.

Funding was provided by the Burroughs Wellcome Career Award for Medical Scientists, Gilead Research Scholars Award, Pennsylvania Department of Health, American Heart Association/Allen Initiative, the National Institutes of Health (DP2 HL147123, R35 HL145203, R01 HL149891, F31 HL147416, NSF15-48571, R01 GM137425), the Penn Institute of Regenerative Medicine, and the Winkelman Family Fund for Cardiac Innovation.

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Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.6 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $494 million awarded in the 2019 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 43,900 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2019, Penn Medicine provided more than $583 million to benefit our community.

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Stem Cell Study Illuminates the Cause of a Devastating Inherited Heart Disorder - Newswise

KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of an expanded label for KEYTRUDA, Mercks anti-PD-1 therapy. The opinion is recommending KEYTRUDA as monotherapy for the treatment of adult and pediatric patients aged 3 years and older with relapsed or refractory classical Hodgkin lymphoma (cHL) who have failed autologous stem cell transplant (ASCT) or following at least two prior therapies when ASCT is not a treatment option.

This recommendation is based on results from the pivotal Phase 3 KEYNOTE-204 trial, in which KEYTRUDA monotherapy demonstrated a significant improvement in progression-free survival (PFS) compared with brentuximab vedotin (BV), a commonly used treatment. KEYTRUDA reduced the risk of disease progression or death by 35% (HR=0.65 [95% CI, 0.48-0.88]; p=0.00271) and showed a median PFS of 13.2 months versus 8.3 months for patients treated with BV. The recommendation is also based on supportive data from an updated analysis of the KEYNOTE-087 trial, which supported the European Commissions (EC) approval of KEYTRUDA for the treatment of adult patients with relapsed or refractory cHL who have failed ASCT and BV or who are transplant ineligible and have failed BV. The CHMPs recommendation will now be reviewed by the EC for marketing authorization in the European Union (EU), and a final decision is expected in the first quarter of 2021. If approved, this will be the first pediatric indication for KEYTRUDA in the EU.

This positive opinion reinforces the importance of KEYTRUDA for certain adult and pediatric patients with relapsed or refractory classical Hodgkin lymphoma in the European Union, said Dr. Vicki Goodman, vice president, clinical research, Merck Research Laboratories. We look forward to the decision by the European Commission and will continue to expand our clinical development program in blood cancers with KEYTRUDA and our recently acquired investigational therapies to help address the unmet needs of patients.

Merck is studying KEYTRUDA across hematologic malignancies through a broad clinical program, including multiple registrational trials in cHL and primary mediastinal large B-cell lymphoma and more than 60 investigator-initiated studies across 15 tumors. In addition to KEYTRUDA, Merck is evaluating two clinical-stage assets for the treatment of patients with hematologic malignancies: MK-1026 (formerly ARQ 531), a Brutons tyrosine kinase inhibitor, and VLS-101, an antibody-drug conjugate targeting ROR1.

About KEYNOTE-204

KEYNOTE-204 (ClinicalTrials.gov, NCT02684292) is a randomized, open-label, Phase 3 trial evaluating KEYTRUDA monotherapy compared with BV for the treatment of patients with relapsed or refractory cHL. The primary endpoints are PFS and overall survival (OS), and the secondary endpoints include objective response rate (ORR), complete remission rate (CRR) and safety. The study enrolled 304 patients, aged 18 years and older, who were randomized to receive either:

About Hodgkin Lymphoma

Hodgkin lymphoma is a type of lymphoma that develops in the white blood cells called lymphocytes, which are part of the immune system. Hodgkin lymphoma can start almost anywhere most often in lymph nodes in the upper part of the body, with the most common sites being in the chest, neck or under the arms. Worldwide, there were approximately 83,000 new cases of Hodgkin lymphoma diagnosed, and more than 23,000 people died from the disease in 2020. In the EU, there were nearly 20,000 new cases of Hodgkin lymphoma diagnosed, and nearly 4,000 people died from the disease in 2020. Classical Hodgkin lymphoma accounts for more than nine in 10 cases of Hodgkin lymphoma in developed countries.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,300 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications in the U.S.

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS 10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Triple-Negative Breast Cancer

KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the programmed death receptor-1 (PD-1) or the programmed death ligand 1 (PD-L1), blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of antiPD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% of these patients interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA as a Single Agent

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA with Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen, which was at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT 3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT 3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT 3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism.

Type 1 Diabetes Mellitus (DM), Which Can Present With Diabetic Ketoacidosis

Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold KEYTRUDA depending on severity. Type 1 DM occurred in 0.2% (6/2799) of patients receiving KEYTRUDA. It led to permanent discontinuation in <0.1% (1) and withholding of KEYTRUDA in <0.1% (1). All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Immune-Mediated Nephritis With Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.1%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 89% (8/9) of patients. Nephritis led to permanent discontinuation of KEYTRUDA in 0.1% (3) and withholding in 0.1% (3) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Nephritis resolved in 56% of the 9 patients.

Immune-Mediated Dermatologic Adverse Reactions

KEYTRUDA can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome, drug rash with eosinophilia and systemic symptoms, and toxic epidermal necrolysis, has occurred with antiPD-1/PD-L1 treatments. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes. Withhold or permanently discontinue KEYTRUDA depending on severity. Immune-mediated dermatologic adverse reactions occurred in 1.4% (38/2799) of patients receiving KEYTRUDA, including Grade 3 (1%) and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 40% (15/38) of patients. These reactions led to permanent discontinuation in 0.1% (2) and withholding of KEYTRUDA in 0.6% (16) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 6% had recurrence. The reactions resolved in 79% of the 38 patients.

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received KEYTRUDA or were reported with the use of other antiPD-1/PD-L1 treatments. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis; Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barr syndrome, nerve paresis, autoimmune neuropathy; Ocular: Uveitis, iritis and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: Pancreatitis, to include increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: Myositis/polymyositis rhabdomyolysis (and associated sequelae, including renal failure), arthritis (1.5%), polymyalgia rheumatica; Endocrine: Hypoparathyroidism; Hematologic/Immune: Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% of 2799 patients receiving KEYTRUDA. Monitor for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion for Grade 1 or Grade 2 reactions. For Grade 3 or Grade 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Fatal and other serious complications can occur in patients who receive allogeneic HSCT before or after antiPD-1/PD-L1 treatment. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute and chronic GVHD, hepatic veno-occlusive disease after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between antiPD-1/PD-L1 treatment and allogeneic HSCT. Follow patients closely for evidence of these complications and intervene promptly. Consider the benefit vs risks of using antiPD-1/PD-L1 treatments prior to or after an allogeneic HSCT.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with an antiPD-1/PD-L1 treatment in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

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Merck Receives Positive EU CHMP Opinion for Expanded Approval of KEYTRUDA (pembrolizumab) in Certain Patients With Relapsed or Refractory Classical...

DUBLIN, Jan. 4, 2021 /PRNewswire/ -- The "Biopreservation Market by Type, Application, End-user, and Geography - Global Forecast to 2026" report has been added to ResearchAndMarkets.com's offering.

Biopreservation is a process that assists in the conservation of biospecimens such as DNA, saliva, and plasma. This process of biopreservation generally increases the durability, shelf life, and purity of the biosamples. The types of equipment in this process include freezers, liquid nitrogen, consumables, and also media & laboratory information management systems.

This process is also used to preserve food and extend its shelf life, specifically by using lactic acid bacteria. Growth in healthcare spending is assumed for better access to quality healthcare and advanced technology products such as biopreservation facilities, thereby widening the growth expectations. Moreover, the bio-banks, hospitals, and gene banks, which are major end-users for this market, are stimulating the key providers to establish technologically advanced biopreservation products to improve patient outcomes. The Biopreservation Market is projected to grow at a rate of 9.2% CAGR by 2026.

The biopreservation market has been analyzed by utilizing the optimum combination of secondary sources and in-house methodology, along with an irreplaceable blend of primary insights. The real-time assessment of the market is an integral part of our market sizing and forecasting methodology. Our industry experts and panel of primary participants have helped in compiling relevant aspects with realistic parametric estimations for a comprehensive study. The participation share of different categories of primary participants is given below:

In the market for biopreservation, the application of biopreservation consists of therapeutic applications, research applications, clinical trials, and other applications. The biopreservation is primarily applied in therapeutics due to the advancements in regenerative medicine & customized medicine, an increase in the shift of cord blood banking, and the rising incidence of chronic diseases.

The end-users of the biopreservation market include biobanks, gene banks, hospitals, and other end users. The biobanks segment is expected to have a major share in the market. The major share of this segment is attributed to the increasing preference for the preservation of stem cells and the rising numbers of sperm and egg banks.

Further, according to the regional market of biopreservation, the North American region is recorded for the colossal share in the market. This is due to the continuous drug developments and the arrival of advanced therapies in the domain of biomedical research. Additionally, the increasing requirement of expensive and improved treatment for patients' chronic diseases is the key factor.

The rising incidence of chronic diseases, including cardiac, renal diseases, diabetes, and obesity, is the crucial factor that will propel the biopreservation market growth in the prevailing period. Government initiatives to encourage stem cell therapies to treat the disease, which will again propel market growth. Conversely, the strict regulations for producing biopreservation products and the evolution of room temperature storage procedures may limit the biopreservation market growth.

Merck KGaA, Avantor, Inc., Bio-Techne Corporation, BioLife Solutions, Inc., Thermo Fisher Scientific Inc, ThermoGenesis Holdings, Inc., Worthington Industries, Inc., Chart Industries, Inc, So-Low Environmental Equipment Co., Inc., Princeton BioCision, LLC, Shanghai Genext Medical Technology Co. Ltd, Exact Sciences Corporation, Helmer Scientific, Inc., CryoTech, Inc., Arctiko, Nippon Genetics Europe, PHC Holdings Corporation, STEMCELL Technologies, Inc., AMS Biotechnology, and OPS Diagnostics. These are the few companies list of the biopreservation market.

Since the rapid increase in the number of research and developments gives the way of potentials for market growth, the biopreservation of biological samples has become a crucial segment. This helps the researchers to access the data of the number of people by the preserved biological samples.

This research presents a thorough analysis of market share, the present trends, and forthcoming evaluations to explain the approaching investment pockets.

This research provides market insights from 2020 to 2026, which is predicted to allow the shareholders to capitalize on the forthcoming opportunities.

This report further offers comprehensive insights into the region, which helps to understand the geographical market and assist in strategic business planning and ascertain future opportunities.

Key Topics Covered:

1. Executive Summary

2. Industry Outlook2.1. Industry Overview2.2. Industry Trends

3. Market Snapshot3.1. Market Definition3.2. Market Outlook3.2.1. PEST Analysis3.2.2. Porter Five Forces3.3. Related Markets

4. Market characteristics4.1. Market Evolution4.2. Market Trends and Impact4.3. Advantages/Disadvantages of Market4.4. Regulatory Impact4.5. Market Offerings4.6. Market Segmentation4.7. Market Dynamics4.7.1. Drivers4.7.2. Restraints4.7.3. Opportunities4.8. DRO - Impact Analysis

5. Type: Market Size & Analysis5.1. Overview5.2. Biopreservation Media5.2.1. Nutrient Media5.2.2. Sera5.2.3. Growth Factors & Supplements5.3. Biospecimen Equipment5.3.1. Temperature Control Systems5.4. Freezers5.5. Cryogenic Storage Systems5.6. Thawing Equipment5.7. Refrigerators5.7.1. Accessories5.7.2. Alarms & Monitoring systems5.7.3. Incubators5.7.4. Centrifuges5.7.5. Other Equipment

6. Application: Market Size & Analysis6.1. Overview6.2. Therapeutic Applications6.3. Research Applications6.4. Clinical Trials6.5. Other Applications

7. End User: Market Size & Analysis7.1. Overview7.2. Biobanks7.3. Gene Banks7.4. Hospitals7.5. Other End Users

8. Geography: Market Size & Analysis8.1. Overview8.2. North America8.3. Europe8.4. Asia Pacific8.5. Rest of the World

9. Competitive Landscape9.1. Competitor Comparison Analysis9.2. Market Developments9.2.1. Mergers and Acquisitions, Legal, Awards, Partnerships9.2.2. Product Launches and execution

10. Vendor Profiles10.1. Merck KGaA10.1.1. Overview10.1.2. Financials10.1.3. Products & Services10.1.4. Recent Developments10.1.5. Business Strategy10.2. Avantor, Inc10.2.1. Overview10.2.2. Financials10.2.3. Products & Services10.2.4. Recent Developments10.2.5. Business Strategy10.3. Bio-Techne Corporation10.3.1. Overview10.3.2. Financials10.3.3. Products & Services10.3.4. Recent Developments10.3.5. Business Strategy10.4. BioLife Solutions, Inc10.4.1. Overview10.4.2. Financials10.4.3. Products & Services10.4.4. Recent Developments10.4.5. Business Strategy10.5. Thermo Fisher Scientific Inc10.5.1. Overview10.5.2. Financials10.5.3. Products & Services10.5.4. Recent Developments10.5.5. Business Strategy10.6. ThermoGenesis Holdings, Inc10.6.1. Overview10.6.2. Financials10.6.3. Products & Services10.6.4. Recent Developments10.6.5. Business Strategy10.7. Worthington Industries, Inc10.7.1. Overview10.7.2. Financials10.7.3. Products & Services10.7.4. Recent Developments10.7.5. Business Strategy10.8. Chart Industries, Inc10.8.1. Overview10.8.2. Financials10.8.3. Products & Services10.8.4. Recent Developments10.8.5. Business Strategy10.9. So-Low Environmental Equipment Co.,Inc10.9.1. Overview10.9.2. Financials10.9.3. Products & Services10.9.4. Recent Developments10.9.5. Business Strategy10.10. Princeton BioCision, LLC10.10.1. Overview10.10.2. Financials10.10.3. Products & Services10.10.4. Recent Developments10.10.5. Business Strategy

11. Companies to Watch11.1. Shanghai Genext Medical Technology Co. Ltd11.1.1. Overview11.1.2. Products & Services11.1.3. Business Strategy11.2. Exact Sciences Corporation11.2.1. Overview11.2.2. Products & Services11.2.3. Business Strategy11.3. Helmer Scientific, Inc11.3.1. Overview11.3.2. Products & Services11.3.3. Business Strategy11.4. CryoTech, Inc11.4.1. Overview11.4.2. Products & Services11.4.3. Business Strategy11.5. Arctiko11.5.1. Overview11.5.2. Products & Services11.5.3. Business Strategy11.6. Nippon Genetics Europe11.6.1. Overview11.6.2. Products & Services11.6.3. Business Strategy11.7. PHC Holdings Corporation11.7.1. Overview11.7.2. Products & Services11.7.3. Business Strategy11.8. STEMCELL Technologies, Inc11.8.1. Overview11.8.2. Products & Services11.8.3. Business Strategy11.9. AMS Biotechnology11.9.1. Overview11.9.2. Products & Services11.9.3. Business Strategy11.10. OPS Diagnostics11.10.1. Overview11.10.2. Products & Services11.10.3. Business Strategy

12. Analyst Opinion

13. Annexure13.1. Report Scope13.2. Market Definitions13.3. Research Methodology13.3.1. Data Collation and In-house Estimation13.3.2. Market Triangulation13.3.3. Forecasting13.4. Report Assumptions13.5. Declarations13.6. Stakeholders13.7. Abbreviations

For more information about this report visit https://www.researchandmarkets.com/r/711zgr

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Worldwide Industry for Biopreservation to 2026 - Key Drivers, Restraints and Opportunities - Yahoo Finance

The vape flavorings so popular with kids and young adults are cardiotoxic and disrupt the hearts normal electrical activity, a University of South Florida Health preclinical study finds.

The appealing array of fruit and candy flavors that entice millions of young people take up vaping can harm their hearts, a preclinical study by University of South Florida Health (USF Health) researchers found.

Mounting studies indicate that the nicotine and other chemicals delivered by vaping, while generally less toxic than conventional cigarettes, can damage the lungs and heart. But so far there has been no clear understanding about what happens when the vaporized flavoring molecules in flavored vaping products, after being inhaled, enter the bloodstream and reach the heart, said the studys principal investigator Sami Noujaim, PhD, an associate professor of molecular pharmacology and physiology at the USF Health Morsani College of Medicine.

In their study published on November 20, 2020, in the American Journal of Physiology- Heart and Circulatory Physiology, Dr. Noujaim and colleagues report on a series of experiments assessing the toxicity of vape flavorings in cardiac cells and in young mice.

The flavored electronic nicotine delivery systems widely popular among teens and young adults are not harm-free, Dr. Noujaim said. Altogether, our findings in the cells and mice indicate that vaping does interfere with the normal functioning of the heart and can potentially lead to cardiac rhythm disturbances.

Dr. Noujaims laboratory is among the first beginning to investigate the potential cardiotoxic effects of the many flavoring chemicals added to the e-liquids in electronic nicotine delivery systems, or ENDS. He recently received a five-year, $2.2-million grant from the NIHs National Institute of Environmental Health Sciences to carry out this laboratory research. Commonly called e-cigarettes, ENDS include different products such as vape pens, mods, and pods.

Sami Noujaim, PhD, associate professor of molecular pharmacology and physiology at the University of South Florida Health (USF Health) Morsani College of Medicine, has begun investigating preclinically the potential cardiotoxic effects of many flavoring chemicals added to the e-liquids in electronic nicotine delivery systems. Credit: Photo courtesy of USF Health

Vaping involves inhaling an aerosol created by heating an e-liquid containing nicotine, solvents such as propylene glycol and vegetable glycerin, and flavorings. The vaping devices battery-powered heat converts this e-liquid into a smoke-like aerosolized mixture (e-vapor). Manufacturers tout e-cigarettes as a tool to help quit smoking, but evidence of their effectiveness for smoking cessation is limited, and they are not FDA approved for this use. E-cigarettes contain the same highly addictive nicotine found in tobacco products, yet many teens and young adults assume they are safe.

Among the USF Health study key findings:

Whether the mouse findings will translate to people is unknown. Dr. Noujaim emphasizes that more preclinical and human studies are needed to further determine the safety profile of flavored ENDS and their long-term health effects.

A partial government ban on flavored e-cigarettes aimed at stopping young people from vaping focused on enforcement against flavored e-cigarettes with pre-filled cartridges, like those produced by industry leader JUUL. However, teens quickly switched to newer disposable e-cigarettes still sold in a staggering assortment of youth-appealing fruity and dessert-like flavors.

Our research matters because regulation of the vaping industry is a work in progress, Dr. Noujaim said. The FDA needs input from the scientific community about all the possible risks of vaping in order to effectively regulate electronic nicotine delivery systems and protect the publics health. At USF Health, in particular, we will continue to examine how vaping may adversely affect cardiac health.

In 2020, 3.6 million U.S. youths still used e-cigarettes, and among current users, more than eight in 10 reported using flavored varieties, according to the Centers for Disease Control and Prevention.

Reference: In Vitro and In Vivo Cardiac Toxicity of Flavored Electronic Nicotine Delivery Systems by Obada Abou-Assali, Mengmeng Chang, Bojjibabu Chidipi, Jose L. Martinez-de-Juan, Michelle Reiser, Manasa Kanithi, Ravi Soni, Thomas Vincent McDonald, Bengt Herweg, Javier Saiz, Laurent Calcul and Sami F. Noujaim, 20 November 2020, American Journal of Physiology-Heart and Circulatory Physiology.DOI: 10.1152/ajpheart.00283.2020

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Vape Flavorings Are Cardiotoxic and Can Damage the Heart - SciTechDaily

Dublin, Dec. 21, 2020 (GLOBE NEWSWIRE) -- The "Biopreservation Market by Type, Application, End-user, and Geography - Global Forecast to 2026" report has been added to ResearchAndMarkets.com's offering.

Biopreservation is a process that assists in the conservation of biospecimens such as DNA, saliva, and plasma. This process of biopreservation generally increases the durability, shelf life, and purity of the biosamples. The types of equipment in this process include freezers, liquid nitrogen, consumables, and also media & laboratory information management systems.

This process is also used to preserve food and extend its shelf life, specifically by using lactic acid bacteria. Growth in healthcare spending is assumed for better access to quality healthcare and advanced technology products such as biopreservation facilities, thereby widening the growth expectations. Moreover, the bio-banks, hospitals, and gene banks, which are major end-users for this market, are stimulating the key providers to establish technologically advanced biopreservation products to improve patient outcomes. The Biopreservation Market is projected to grow at a rate of 9.2% CAGR by 2026.

The biopreservation market has been analyzed by utilizing the optimum combination of secondary sources and in-house methodology, along with an irreplaceable blend of primary insights. The real-time assessment of the market is an integral part of our market sizing and forecasting methodology. Our industry experts and panel of primary participants have helped in compiling relevant aspects with realistic parametric estimations for a comprehensive study. The participation share of different categories of primary participants is given below:

In the market for biopreservation, the application of biopreservation consists of therapeutic applications, research applications, clinical trials, and other applications. The biopreservation is primarily applied in therapeutics due to the advancements in regenerative medicine & customized medicine, an increase in the shift of cord blood banking, and the rising incidence of chronic diseases.

The end-users of the biopreservation market include biobanks, gene banks, hospitals, and other end users. The biobanks segment is expected to have a major share in the market. The major share of this segment is attributed to the increasing preference for the preservation of stem cells and the rising numbers of sperm and egg banks.

Further, according to the regional market of biopreservation, the North American region is recorded for the colossal share in the market. This is due to the continuous drug developments and the arrival of advanced therapies in the domain of biomedical research. Additionally, the increasing requirement of expensive and improved treatment for patients' chronic diseases is the key factor.

The rising incidence of chronic diseases, including cardiac, renal diseases, diabetes, and obesity, is the crucial factor that will propel the biopreservation market growth in the prevailing period. Government initiatives to encourage stem cell therapies to treat the disease, which will again propel market growth. Conversely, the strict regulations for producing biopreservation products and the evolution of room temperature storage procedures may limit the biopreservation market growth.

Merck KGaA, Avantor, Inc., Bio-Techne Corporation, BioLife Solutions, Inc., Thermo Fisher Scientific Inc, ThermoGenesis Holdings, Inc., Worthington Industries, Inc., Chart Industries, Inc, So-Low Environmental Equipment Co., Inc., Princeton BioCision, LLC, Shanghai Genext Medical Technology Co. Ltd, Exact Sciences Corporation, Helmer Scientific, Inc., CryoTech, Inc., Arctiko, Nippon Genetics Europe, PHC Holdings Corporation, STEMCELL Technologies, Inc., AMS Biotechnology, and OPS Diagnostics. These are the few companies list of the biopreservation market.

Since the rapid increase in the number of research and developments gives the way of potentials for market growth, the biopreservation of biological samples has become a crucial segment. This helps the researchers to access the data of the number of people by the preserved biological samples.

This research presents a thorough analysis of market share, the present trends, and forthcoming evaluations to explain the approaching investment pockets.

This research provides market insights from 2020 to 2026, which is predicted to allow the shareholders to capitalize on the forthcoming opportunities.

This report further offers comprehensive insights into the region, which helps to understand the geographical market and assist in strategic business planning and ascertain future opportunities.

Key Topics Covered:

1. Executive Summary

2. Industry Outlook2.1. Industry Overview2.2. Industry Trends

3. Market Snapshot3.1. Market Definition3.2. Market Outlook3.2.1. PEST Analysis3.2.2. Porter Five Forces3.3. Related Markets

4. Market characteristics4.1. Market Evolution4.2. Market Trends and Impact4.3. Advantages/Disadvantages of Market4.4. Regulatory Impact4.5. Market Offerings4.6. Market Segmentation4.7. Market Dynamics4.7.1. Drivers4.7.2. Restraints4.7.3. Opportunities4.8. DRO - Impact Analysis

5. Type: Market Size & Analysis5.1. Overview5.2. Biopreservation Media5.2.1. Nutrient Media5.2.2. Sera5.2.3. Growth Factors & Supplements5.3. Biospecimen Equipment5.3.1. Temperature Control Systems5.4. Freezers5.5. Cryogenic Storage Systems5.6. Thawing Equipment5.7. Refrigerators5.7.1. Accessories5.7.2. Alarms & Monitoring systems5.7.3. Incubators5.7.4. Centrifuges5.7.5. Other Equipment

6. Application: Market Size & Analysis6.1. Overview6.2. Therapeutic Applications6.3. Research Applications6.4. Clinical Trials6.5. Other Applications

7. End User: Market Size & Analysis7.1. Overview7.2. Biobanks7.3. Gene Banks7.4. Hospitals7.5. Other End Users

8. Geography: Market Size & Analysis8.1. Overview8.2. North America8.3. Europe8.4. Asia Pacific8.5. Rest of the World

9. Competitive Landscape9.1. Competitor Comparison Analysis9.2. Market Developments9.2.1. Mergers and Acquisitions, Legal, Awards, Partnerships9.2.2. Product Launches and execution

10. Vendor Profiles10.1. Merck KGaA10.1.1. Overview10.1.2. Financials10.1.3. Products & Services10.1.4. Recent Developments10.1.5. Business Strategy10.2. Avantor, Inc10.2.1. Overview10.2.2. Financials10.2.3. Products & Services10.2.4. Recent Developments10.2.5. Business Strategy10.3. Bio-Techne Corporation10.3.1. Overview10.3.2. Financials10.3.3. Products & Services10.3.4. Recent Developments10.3.5. Business Strategy10.4. BioLife Solutions, Inc10.4.1. Overview10.4.2. Financials10.4.3. Products & Services10.4.4. Recent Developments10.4.5. Business Strategy10.5. Thermo Fisher Scientific Inc10.5.1. Overview10.5.2. Financials10.5.3. Products & Services10.5.4. Recent Developments10.5.5. Business Strategy10.6. ThermoGenesis Holdings, Inc10.6.1. Overview10.6.2. Financials10.6.3. Products & Services10.6.4. Recent Developments10.6.5. Business Strategy10.7. Worthington Industries, Inc10.7.1. Overview10.7.2. Financials10.7.3. Products & Services10.7.4. Recent Developments10.7.5. Business Strategy10.8. Chart Industries, Inc10.8.1. Overview10.8.2. Financials10.8.3. Products & Services10.8.4. Recent Developments10.8.5. Business Strategy10.9. So-Low Environmental Equipment Co.,Inc10.9.1. Overview10.9.2. Financials10.9.3. Products & Services10.9.4. Recent Developments10.9.5. Business Strategy10.10. Princeton BioCision, LLC10.10.1. Overview10.10.2. Financials10.10.3. Products & Services10.10.4. Recent Developments10.10.5. Business Strategy

11. Companies to Watch11.1. Shanghai Genext Medical Technology Co. Ltd11.1.1. Overview11.1.2. Products & Services11.1.3. Business Strategy11.2. Exact Sciences Corporation11.2.1. Overview11.2.2. Products & Services11.2.3. Business Strategy11.3. Helmer Scientific, Inc11.3.1. Overview11.3.2. Products & Services11.3.3. Business Strategy11.4. CryoTech, Inc11.4.1. Overview11.4.2. Products & Services11.4.3. Business Strategy11.5. Arctiko11.5.1. Overview11.5.2. Products & Services11.5.3. Business Strategy11.6. Nippon Genetics Europe11.6.1. Overview11.6.2. Products & Services11.6.3. Business Strategy11.7. PHC Holdings Corporation11.7.1. Overview11.7.2. Products & Services11.7.3. Business Strategy11.8. STEMCELL Technologies, Inc11.8.1. Overview11.8.2. Products & Services11.8.3. Business Strategy11.9. AMS Biotechnology11.9.1. Overview11.9.2. Products & Services11.9.3. Business Strategy11.10. OPS Diagnostics11.10.1. Overview11.10.2. Products & Services11.10.3. Business Strategy

12. Analyst Opinion

13. Annexure13.1. Report Scope13.2. Market Definitions13.3. Research Methodology13.3.1. Data Collation and In-house Estimation13.3.2. Market Triangulation13.3.3. Forecasting13.4. Report Assumptions13.5. Declarations13.6. Stakeholders13.7. Abbreviations

For more information about this report visit https://www.researchandmarkets.com/r/pl06wm

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Outlook on the Biopreservation Global Market to 2026 - Profiling Avantor, BioLife Solutions and ThermoGenesis Among Others - GlobeNewswire

Blocking an enzyme linked with inflammation makes it possible for stem cells to repair damaged heart tissue, new research from UC Davis Health scientists shows.

Researchers Phung Thai (left) and Padmini Sirish were part of a research team seeking stem cell solutions to heart failure care.

The enzyme soluble epoxide hydrolase, or sEH is a known factor in lung and joint disease. Now, it is a focus of heart-disease researchers as well.

The authors expect their work will lead to a new and powerful class of compounds that overcome the cell death and muscle thickening associated with heart failure a common outcome of a heart attack or long-term cardiovascular disease.

The study, conducted in mice, is published in Stem Cells Translational Medicine. The work was led by cardiologist Nipavan Chiamvimonvat.

The science of using stem cell treatments for heart disease has been full of promise but little progress, Chiamvimonvat said. The inflammation that accompanies heart disease is simply not conducive to stem cell survival.

Prior studies show that stem cells transplanted to the heart experience significant attrition in a very short period of time.

We think weve found a way to quiet that inflammatory environment, giving stem cells a chance to survive and do the healing work we know they can do, said lead author and cardiovascular medicine researcher Padmini Sirish.

Heart failure occurs when the heart no longer pumps blood efficiently, reducing oxygen throughout the body. Survival is around 45-60% five years after diagnosis. It affects approximately 5.7 million people in the U.S., with annual costs of nearly $30 billion. By 2030, it could affect as many as 9 million people at a cost of nearly $80 billion.

Chiamvimonvat often treats patients with heart failure and has been frustrated by the lack of effective medications for the disease, especially when it progresses to later stages. The best current therapies for end-stage heart failure are surgical heart transplants or mechanical heart pumps.

This research was led by cardiologist Nipavan Chiamvimonvat.

She expects her outcome will lead to a two-part treatment for end-stage heart failure that combines an sEH-blocking compound with stem cell transplantation.

Chiamvimonvat and her team tested that theory in mice using cardiac muscle cells known as cardiomyocytes, which were derived from human-induced pluripotent stem cells (hiPSCs). A hiPSC is a cell taken from any human tissue (usually skin or blood) and genetically modified to behave like an embryonic stem cell. They have the ability to form all cell types.

The specific sEH inhibitor used in the study TPPU was selected based on the work of co-author and cancer researcher Bruce Hammock, whose lab has provided detailed studies of nearly a dozen of the enzyme inhibitors.

The researchers studied six groups of mice with induced heart attacks. A group treated with a combination of the inhibitor and hiPSCs had the best outcomes in terms of increased engraftment and survival of transplanted stem cells. That group also had less heart muscle thickening and improved cardiac function.

Taken together, our data suggests that conditioning hiPSC cardiomyocytes with sEH inhibitors may help the cells to better survive the harsh conditions in the muscle damaged by a heart attack, Hammock said.

Chiamvimonvat and her team will next test the process in a larger research animal model to provide more insights into the beneficial role of TPPU. She also wants to test the process with additional heart diseases, including atrial fibrillation. Her ultimate goal, in collaboration with Hammock, is to launch human clinical trials to test the safety of the treatment.

It is my dream as a clinician and scientist to take the problems I see in the clinic to the lab for solutions that benefit our patients, Chiamvimonvat said. It is only possible because of the incredible strength of our team and the extraordinarily collaborative nature of research at UC Davis.

Additional co-authors were Phung Thai, Jun Yang, Xiao-Dong Zhang, Lu Ren, Ning Li, Valeriy Timofeyev, Kin Sing Lee, Carol Nader, Douglas Rowland, Sergey Yechikov, Svetlana Ganaga, J. Nilas Young and Deborah Lieu, all from UC Davis.

Their work was funded by the American Heart Association, Harold S. Geneen Charitable Trust. Rosenfeld Heart Foundation, U.S. Department of Veterans Affairs and the National Institutes of Health (grants T32HL86350, F32HL149288, K99R00ES024806, R35ES030443, P42ES04699, IR35 ES0443-1, P01AG051443, R01DC015135, R56HL138392, R01HL085727, R01HL085844, R01HL137228 and S10RR033106).

The study, titled Suppression of Inflammation and Fibrosis using Soluble Epoxide Hydrolase Inhibitors Enhances Cardiac Stem Cell-Based Therapy, is available online.

More information about UC Davis Health, including its cardiovascular medicine and stem cell programs, is at health.ucdavis.edu.

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UC Davis researchers find a way to help stem cells work ...

Avery Therapeutics is projected to be one of the first companies in the US to seek approval for a clinical trial using iPSC-derived technology for heart failure. The goal of this collaboration is to develop a new off-the-shelf treatment to improve the quality of life of patients suffering from heart failure, a debilitating disease that affects tens of millions of people worldwide.

The iPSCs are manufactured at I Peace's state-of-the-art GMP facility in Kyoto, Japan, under comprehensive validation programs of the facility, equipment, and processes including donor recruiting, screening, blood draw, iPSC generation, storage, and distribution. I Peace has obtained a US-based independent institutional review board (IRB) approval for its process of donor sourcing for commercial-use iPSCs. The facility is designed to be PMDA and USFDA compliant.

As Avery Therapeutics expects to expand the application of its regenerative medicine technology to various types of heart diseases and beyond, iPSCs are the key enabling technology for quality and future scalability. This agreement provides a solid foundation to improve the welfare of those suffering from diseases through advancement of tissue-engineered therapeutics.

"We are thrilled to announce this collaboration with I Peace. It is a big step forward in the development of novel cell-based therapeutics for unmet medical needs. Through this collaboration, I Peace brings deep iPSC development and manufacturing expertise to enable Avery's proprietary MyCardia cell delivery platform technology. Together we hope to positively impact millions of patients worldwide in the near future," Said Jordan Lancaster, PhD, Avery Therapeutics' CEO.

This agreement reflects an innovative collaboration involving multiple locations internationally and marks a significant milestone for both I Peace, Inc. and Avery Therapeutics to pursue one of the first US clinical trials using iPSC technology in the area of heart diseases. Koji Tanabe, PhD, founder and CEO of I Peace stated: "By combining I Peace's proprietary clinical grade iPSC technology and Avery's tissue engineering technology, we can bring the regenerative medicine dream closer to reality. We are very excited by Avery's technology and look forward to continue working together."

About I Peace, Inc

I Peace, Inc. is a global supplier of clinical and research grade iPSCs. It was founded in 2015 in Palo Alto, California, USA by Dr. Tanabe, who earned his doctorate at Kyoto University under Nobel laureate Dr. Shinya Yamanaka. I Peace's mission is to alleviate the suffering of diseased patients and help healthy people maintain a high quality of life by making cell therapy accessible to all. I Peace's state-of-the-art GMP facility and proprietary manufacturing platform enables the fully-automated mass production of discrete iPSCs from multiple donors in a single room. Increasing the available number of clinical-grade iPSC lines allows I Peace customers to take differentiation propensity into account to select the most appropriate iPSC line for their clinical research at significantly reduced cost. I Peace aims to create iPSCs for every individual that become their stem cell for life.

Founder, CEO: Koji TanabeSince: 2015Head Quarter: Palo Alto, CaliforniaJapan subsidiary: I Peace, Ltd. (Kyoto, Japan)Cell Manufacturing Facility: Kyoto, JapanWeb: https://www.ipeace.com

About Avery Therapeutics

Avery Therapeutics is a company developing advanced therapies for patients suffering from cardiovascular diseases. Avery's lead candidate is an allogeneic tissue engineered cardiac graft, MyCardia in development for treatment of chronic heart failure. Using Avery's proprietary manufacturing process MyCardia can be manufactured at scale, cryopreserved, and shipped ready to use. Avery is leveraging its proprietary tissue platform to pursue other cardiovascular indications. For more information visit: AveryThera.com. Follow Avery Therapeutics on LinkedInand Twitter.Since: 2016Headquarter: Tucson, AZWebsite: https://www.AveryThera.com

SOURCE I Peace, Inc.

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I Peace, Inc. and Avery Therapeutics announce collaboration to bring iPSC derived cell therapy for heart failure to the clinic - PRNewswire

TAMPA, Fla. While health officials and lawmakers continue trying to steer young people away from vaping, the wide variety of enticing flavors added to these products make that a tough task. Although most of the worry over vaping comes from the risk of addiction, lung damage, and threat of switching to conventional cigarettes, a new study finds the flavoring chemicals these products use may be just as harmful as anything else. Researchers from the University of South Florida Health say vaporized flavoring molecules are toxic to the heart and damage the organs ability to beat correctly.

While other studies find that vaping is generally less harmful than smoking traditional tobacco products, the nicotine and other chemicals in e-cigarettes still damages the heart and lungs. Until now however, researchers say the impact of flavoring additives inhaled into the bloodstream remained unclear.

The flavored electronic nicotine delivery systems widely popular among teens and young adults are not harm-free, says principal investigator Dr. Sami Noujaim in a university release. Altogether, our findings in the cells and mice indicate that vaping does interfere with the normal functioning of the heart and can potentially lead to cardiac rhythm disturbances.

Dr. Noujaims study is one of the first to investigate the cardiotoxic effects of flavoring chemicals added to the e-liquids in electronic nicotine delivery systems (ENDS). ENDS include a variety of different vaping products like vape pens, mods, and pods.

Researchers define vaping as inhaling aerosols (tiny droplets) which e-cigarettes create by heating liquid nicotine and solvents like propylene glycol and vegetable glycerin. A vaping devices battery-powered heater converts this liquid into a smoke-like mix, or vapor.

The study tested how three popular e-liquid flavors fruit, cinnamon, and vanilla custard affect cardiac muscle cells (HL-1) of mice. After being exposed to e-vapor in a lab dish, the results reveal all three flavors are toxic to HL-1 cells.

The USF team also examined what happens to cardiac cells grown from human stem cells that are exposed to three types of e-vapors. The first substance containing only solvents interfered with the cells electrical activity and beating rate. The second substance, containing both nicotine and solvents, proved to be even more toxic to the heart cells.

The third substance however, containing nicotine, solvents, and vanilla custard flavoring, caused the most damage to the heart and its ability to spontaneously beat correctly. Researchers also determined that vanilla custard flavoring is the most toxic of the varieties tested.

This experiment told us that the flavoring chemicals added to vaping devices can increase harm beyond what the nicotine alone can do, Dr. Noujaim says.

The study also tested flavored vapings impact on live mice. Researchers implanted each subject with a tiny electrocardiogram device before exposing them to 60 puffs of vanilla-flavored e-vapor five days a week for 10 weeks.

Study authors looked at how this exposure impacted heart rate variability (HRV), which is the change in time intervals between successive heartbeats. The results show that HRV decreased in vaping mice compared to those only exposed to puffs of clean air.

The USF team finds vaping interferes with normal HRV by disrupting the autonomic nervous system and its control over heart rate. Mice exposed to flavored vaping are also more prone to a dangerous heart rhythm problem called ventricular tachycardia.

Researchers say they still have to confirm these results in humans. Dr. Noujaim urges policymakers to continue looking at the growing evidence that vaping is not a particularly safer alternative to smoking.

Our research matters because regulation of the vaping industry is a work in progress, Dr. Noujaim explains. The FDA needs input from the scientific community about all the possible risks of vaping in order to effectively regulate electronic nicotine delivery systems and protect the publics health. At USF Health, in particular, we will continue to examine how vaping may adversely affect cardiac health.

The study appears in the American Journal of Physiology- Heart and Circulatory Physiology.

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Flavors added to vaping devices damage the heart, vanilla custard the most toxic of all - Study Finds

The Covid-19 can damage the heart both directly and indirectly, and lead to complications ranging from inflammation of the heart (myocarditis), injury to heart cells (necrosis), heart rhythm disorders (arrhythmias), heart attack, and muscle dysfunction that can lead to acute or protracted heart failure, experts said.

Covid-19 is a vascular disease that injures heart cells and muscle. It also leads to the formation of blood clots, both in the microvasculature and large vessels, which can block blood supply to the heart, brain and lungs and lead to stroke, heart attack and respiratory failure, said Dr Ravi R Kasliwal, chairman of clinical and preventive cardiology department at Medanta -The Medicity Hospital.

Also Read: Few Covid-19 deaths in Indias old-age homes, survey finds

A US study using MRI found cardiac abnormalities in 78 of 100 patients who had recently recovered from Covid-19, including 12 of 18 asymptomatic patients. Sixty patients had ongoing myocardial inflammation consistent with myocarditis, found the study, which was published in the Journal of American Medical Association Cardiology in July.

Even people with mild disease or no symptoms can develop life-threatening cardiovascular complications. Whats worrying is that this holds true for healthy adults with no pre-existing risk factors, which raise their risk of complications, said Dr Kasliwal, who recommends that everyone who has recovered from Covid-19 be screened for heart damage

Cardiac trouble

Extensive cardiac involvement is what differentiates Sars-CoV-2, the virus that causes Covid-19, from the six other coronaviruses that cause infection in humans, writes cardiologist Dr Eric J Topol, founder, director and professor of molecular medicine at the Scripps Research Translational Institute in La Jolla, California, in the journal Science.

The four human coronaviruses that cause cold-like symptoms have not been associated with heart abnormalities, though there have been isolated reports linking the Middle East Respiratory Syndrome (MERS) caused by MERS-CoV) with myocarditis, and cardiac disease with the Severe Acute Respiratory Syndrome (SARS) caused by Sars-CoV.

Also Read| Extraordinary uncertainties: Harvard prof on Covid-19, impact on mental health

Sars-CoV-2 is structurally different from Sars-CoV. The virus targets the angiotensin-converting enzyme 2 (Ace2) receptor throughout the body, facilitating cell entry by way of its spike protein, along with the cooperation of proteases. The heart is one of the many organs with high expression of Ace2. The affinity of Sars-CoV-2 to Ace2 is significantly greater than that of SARS, according to Dr Topol.

Topol notes the ease with which Sars-CoV-2 infects heart cells derived from induced pluripotent stem cells (iPSCs) in vitro, leading to a distinctive pattern of heart muscle cell fragmentation evident in autopsy reports. Besides directly infecting heart muscle cells, Sars-CoV-2 also enters and infects the endothelial cells that line the blood vessels to the heart and multiple vascular beds, leading to a secondary immune response. This causes blood pressure dysregulation, and activation of a proinflammatory response leading to a cytokine storm, which is a potentially fatal systemic inflammatory syndrome associated with Covid-19.

Persisting problems

Studies have found that injury to heart cells reflected in blood concentrations of a cardiac muscle-specific enzyme called troponin affects at least one in five hospitalised patients and more than half of those with pre-existing heart conditions, which raises the risk of death. Patients with higher troponin amounts also have high markers of inflammation (including C-reactive protein, interleukin-6, ferritin, lactate dehydrogenase), high neutrophil count, and heart dysfunction, all of which heighten immune response.

The heightened systemic inflammatory responses and diminished blood supply because of clotting, endotheliitis (blood vessel inflammation), sepsis, or hypoxemia (oxygen deprivation) because of acute lung infection leads to indirect cardiac damage, said Dr Kasliwal.

The cardiovascular damage associated with Sars-CoV-2 infection can persist beyond recovery. Since the virus affects the heart as much as the respiratory tract, further research is needed to understand why some people are more vulnerable to heart damage than others.

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Covid-19 can have impact on heart too, say experts - Hindustan Times

Over two million babies, children, and adults in the United States are living with congenital heart disease--a range of birth defects affecting the heart's structure or function. Now, researchers at Gladstone Institutes and UC San Francisco (UCSF) have made inroads into understanding how a broad network of genes and proteins go awry in a subset of congenital heart diseases.

"We now have a better understanding of what genes are improperly deployed in some cases of congenital heart disease," says Benoit Bruneau, PhD, director of the Gladstone Institute of Cardiovascular Disease and a senior author of the new study. "Eventually, this might help us get a handle on how to modulate genetic networks to prevent or treat the disease."

Congenital heart disease encompasses a wide variety of heart defects, ranging from mild structural problems that cause no symptoms to severe malformations that disrupt or block the normal flow of blood through the heart. A handful of genetic mutations have been implicated in contributing to congenital heart disease; the first to be identified was in a gene known as TBX5. The TBX5 protein is a transcription factor--it controls the expression of dozens of others genes, giving it far-reaching effects.

Bruneau has spent the last 20 years studying the effect of TBX5 mutations on developing heart cells, mostly conducting research in mice. In the new study published inDevelopmental Cell, he and his colleagues turned instead to human cells, using novel approaches to follow what happens in individual cells when TBX5 is mutated.

"This is really the first time we've been able to study this genetic mutation in a human context," says Bruneau, who is also a professor in the Department of Pediatrics at UCSF. "The mouse heart is a good proxy for the human heart, but it's not exactly the same, so it's important to be able to carry out these experiments in human cells."

The scientists began with human induced pluripotent stem cells (iPS cells), which have been reprogrammed to an embryonic-like state, giving them--like embryonic stem cells--the ability to become nearly every cell type in the body.

Then, Bruneau's group used CRISPR-Cas9 gene-editing technology to mutate TBX5 in the cells and began coaxing the iPS cells to become heart cells. As the cells became more like heart cells, the researchers used a method called single-cell RNA sequencing to track how the TBX5 mutation changed which genes were switched on and off in tens of thousands of individual cells.

The experiment revealed many genes that were expressed at higher or lower levels in cells with mutated TBX5. Importantly, not all cells responded to the TBX5 mutation in the same way; some had drastic changes in gene expression while other were less affected. This diversity, the researchers say, reflects the fact that the heart is composed of many different cell types.

"It makes sense that some are more affected than others, but this is the first experimental data in human cells to show that diversity," says Bruneau.

Bruneau's team then collaborated with computational researchers to analyze how the impacted genes and proteins were related to each other. The new data let them sketch out a complex and interconnected network of molecules that work together during heart development.

"We've not only provided a list of genes that are implicated in congenital heart disease, but we've offered context in terms of how those genes are connected," says Irfan Kathiriya, MD, PhD, a pediatric cardiac anesthesiologist at UCSF Benioff Children's Hospital, an associate professor in the Department of Anesthesia and Perioperative Care at UCSF, a visiting scientist at Gladstone, and the first author of the study.

Several genes fell into known pathways already associated with heart development or congenital heart disease. Some genes were among those directly regulated by TBX5's function as a transcription factor, while others were affected in a less direct way, the study revealed. In addition, many of the altered genes were relevant to heart function in patients with congenital heart disease as they control the rhythm and relaxation of the heart, and defects in these genes are often found together with the structural defects.

The new paper doesn't point toward any individual drug target that can reverse a congenital heart disease after birth, but a better understanding of the network involved in healthy heart formation, as well as congenital heart disease may lead to ways to prevent the defects, the researchers say. In the same way that folate taken by pregnant women is known to help prevent neural tube defects, there may be a compound that can help ensure that the network of genes and proteins related to congenital heart disease stays balanced during embryonic development.

"Our new data reveal that the genes are really all part of one network--complex but singular--which needs to stay balanced during heart development," says Bruneau. "That means if we can figure out a balancing factor that keeps this network functioning, we might be able to help prevent congenital heart defects."

Reference: Kathiriya IS, Rao KS, Iacono G, et al. Modeling Human TBX5 Haploinsufficiency Predicts Regulatory Networks for Congenital Heart Disease. Developmental Cell. 2020. doi:10.1016/j.devcel.2020.11.020.

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

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Network of Genes Involved in Congenital Heart Disease Identified - Technology Networks

The agreement will accelerate the development and availability of new medical and pharmaceutical therapies to improve patients lives

Hamamatsu Photonics UK Ltd and Medical Technologies Innovation Facility (MTIF) are pleased to announce they have entered into a partnership agreement enabling customers the ability to view and utilize Hamamatsus Functional Drug Screening System (FDSS) CELL. This is the first FDSS/CELL to be made available in the UK in this way.

This new collaboration aims to leverage the photonics expertise, novel proprietary technology and applications of Hamamatsu, with the significant medical technology research and development capabilities of MTIF.

This is a high-end specialist piece of equipment utilised in the development of innovative medicines around the world. We are very excited to be able to provide customers with this capability, that complements our own research using this technically superb equipment. Says Professor John Hunt, Head of Strategic Research at MTIF and within Nottingham Trent University.

This partnership provides companies with a unique opportunity to use cutting edge high through-put technology to screen compounds for pharmacological activity. These capabilities are usually unavailable to all but the largest organisations. This collaboration allows organisations of every size the opportunity to accelerate their drug discovery programme. Says Professor Mike Hannay, Managing Director of the Medical Technologies Innovation Facility (MTIF) .

Hamamatsu has a long history in developing cutting edge scientific equipment for the life science market; our FDSS/CELL enables scientists, such as those working at MTIF, to make breakthroughs in the field of drug discovery and compound research. We are really excited about this new partnership between Hamamatsu and the team at MTIF helping to make such advanced instrumentation available to hundreds of potential users throughout the UK research community. Tim Stokes, Managing Director of Hamamatsu Photonics UK Ltd.

The FDSS/CELL is a compact, easy to use screening system that enables monitoring of GPCRs and ion channels for drug discovery and life science research. Screening various compounds at high throughput (96 / 384 well assays) is enabled by fluorescence or luminescence measurements using a highly sensitive Hamamatsu camera, which captures cell dynamics under the same conditions with no time lag between wells. It is also capable of recording changes in electrical potential in iPSC-derived neuronal and cardiac stem cells to gain a better understanding of toxic compound effects.

Through this new technical collaboration, HPUK and MTIF will organically integrate their respective advanced technologies and development capabilities to showcase this novel laboratory screening technology onsite at MTIF in Nottingham, UK.

Hamamatsu Photonics and MTIF aim to benefit the UK life science sector by accelerating the availability of new medical and pharmaceutical therapies. By aligning capabilities and ambitions, the parties will deliver benefit to clients by helping them to successfully navigate the complexities of discovering drug and cell therapy candidates.

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Industry News: Hamamatsu Photonics UK Ltd and the Medical Technologies Innovation Facility enter into a partnership agreement - SelectScience

Hopes for new treatments for osteoporosis and cartilage degeneration have been raised after scientists identified a gene that plays a key role in the ageing of bone, tendon, ligament and cartilage.

The researchers hope that they can use their findings to slow down treat age-related diseases connected to the skeletal system by creating treatments that slow down the ageing process behind them.

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Our findings are novel and significant in finding a critical answer to how skeletal tissues lose their capability to maintain their properties and functions when we age, said Wan-Ju Li, of the University of Wisconsin-Madison.

We can also develop new pharmacological therapies to treat age-associated diseases based on our findings [although] it will take a few years before we can see the application happens, he said.

The study is published in the journal Stem Cells. The journals editor-in-chief, Jan Nolta, of the University of California at Davis, said the discovery is a very important accomplishment.

Researchers said it is possible that the same mechanism that has been identified for the skeletal system may also be present in neural stem cells and cardic stem cells, where it may play a role in causing diseases associated with those areas of the body.

We dont know if the molecule and mechanism we have identified in the paper also play the same role in other stem cells, such as neural stem cells and cardiac stem cells, in causing Parkinsons disease and heart diseases, respectively, since we havent tested it with these cells, Dr Lin said.

But I am sure that other scientists in the fields of aging and brain and heart will follow our study to answer these questions in the future, Dr Lin said.

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Myocardial Infarction Drug used to treat Heart Attack. Medicines and chemical substances that can cause myocardial infarction. Treatment ranges from lifestyle changes and cardiac rehabilitation to medication, stents, and bypass surgery.

Myocardial Infarction Drug Market is anticipated to grow at a CAGR of +6% during the forecast period 2020-2028.

A Global Myocardial Infarction Drug Market analysis and forecast is released based on a wide study of the market. Statistics about the approaching market trends as well as the current scenario of the market is a vital implement for existence and development in the constantly developing industry.

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Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Myocardial Infarction Drug Market which would mention How the Covid-19 is affecting the Myocardial Infarction Drug Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Myocardial Infarction Drug Players to Combat Covid-19 Impact.

The Top Key Players of the global Myocardial Infarction Drug Market:

BioCardia, Inc., Laboratoires Pierre Fabre SA, Human Stem Cells Institute, CSL Limited, Capricor Therapeutics, Inc., Hemostemix Ltd, Compugen Ltd., Celyad SA, FibroGen, Inc., Lees Pharmaceutical Holdings Limited, Juventas Therapeutics, Inc., Cynata Therapeutics Limited, CellProthera, Biscayne Pharmaceuticals, Inc., HUYA Bioscience International, LLC, LegoChem Biosciences, Inc, Immune Pharmaceuticals Inc.

Segmentation by Product type:

Segmentation by Application:

Market Segmentation by Region:

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The Global Myocardial Infarction Drug Market has demonstrated an increasing need to alter the policies that are being currently used by the players so as to exhibit commercial capacities of the manufacturers, distributors, and vendors. This helps the key players in developing a firm strategy that is flexible enough to keep up with future events in the market space.

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In the United States alone, one person dies every 36 seconds from cardiovascular disease. Globally, it is also the leading cause of death, claiming over 17 million lives each year. In cases of severe illness, heart transplants have shown great promise in increasing the life expectancy of patients with heart disease. About 75% of heart transplant recipients survive for 5 more years and about 56% survive for 10 more years. However, the average wait times for heart transplants are long, often exceeding 6 months, and some patients simply cannot afford to wait that long.

Therefore, scientists tend to refer to other modes of treatment which rely on managing chronic symptoms, such as hypertension (high blood pressure), diabetes mellitus, obesity, and high cholesterol. This approach, however, does not address the root cause of the problem, which is impaired heart functioning. Since heart cells do not have a mechanism to replace damaged tissue, scientists have become increasingly excited about the possibility of repairing or replacing damaged heart tissue using stem cells (unique cells that have the ability to divide for an extended period of time and differentiate into specialized cells, such as cardiac cells or nerve cells).

Regenerative medicine has been a topic of excitement among researchers for decades. In 1999, Anthony Atala, director of the Wake Forest Institute for Regenerative Medicine, was the first to implant lab-grown organs into several patients between 4 and 19 years old. In his method, he obtained bladder cells from the children and coaxed those cells into dividing on a scaffold (a structure that mimics the normal organ). The engineered bladders functioned normally and no ill effects were reported. Pretty much I was able to live a normal life after, said Luke, one of Atalas patients.

More recently, Yoshiki Sawa, a professor of cardiovascular surgery at the University of Osakas medical school, and his team of Japanese researchers successfully transplanted lab-grown cardiac muscles into a human patient. The researchers first extracted adult stem cells from the patients blood or skin and genetically reprogrammed them into induced pluripotent stem (iPS) cells. They were then coaxed into 0.1-millimeter-thick sheets of cardiac tissue and grafted onto the diseased human hearts. According to Sawa, the cells do not seem to integrate into the heart tissue but rather release growth factors (proteins) that help regenerate blood vessels in the damaged muscle tissue and improve cardiac function. The team has conducted an operation on a patient in January 2020, marking the worlds first transplant of cardiac muscle cells.

The United States is also home to major breakthroughs in regenerative medicine. For decades, scientists have utilized embryonic stem cells to engineer heart muscle cells that are able to maintain synchronous breathing in a dish for hours. Despite this major feat, the creation of a working heart called for a more sophisticated technique. Doris Taylor, director of regenerative medicine research at the Texas Heart Institute (THI), has grown in her lab over 100 ghost hearts using protein scaffolds. She creates these scaffolds by first obtaining an animal heart and then decellularizing it by pumping a detergent through its blood vessels to strip away lipids, DNA, soluble proteins, sugars and almost all the other cellular material from the heart, leaving only a pale mesh of collagen, laminins, and the extracellular matrix. This heart does not necessarily have to be a human heart. She often finds pig hearts to be promising tissue because of their considerable safety and unlimited supply. She then recellularizes the heart by injecting it with millions of stem cells and attaching it to artificial lungs and a blood pump. Although her technique has only been used so far for growing animal hearts, she believes that it will eventually be used to create human heart transplants, thus, revolutionizing cardiovascular surgery and putting an end to organ shortage and anti-rejection drugs.

These groundbreaking results in regenerative medicine altogether have taken years of painstaking research to achieve. Taylor believes that her research is exceptionally close to building a working, human-sized heart, and Sawa says that his technique of grafting healthy cardiac muscle sheets onto the patients diseased heart tissue has already helped one of his patients move out of intensive care in just a few days. As the researchers gain more knowledge and get closer to the solution, however, they encounter more challenging obstacles. Sawa, for instance, has found that grafted cells do not always beat in synchrony. Researchers are also split on how these grafts work. On the other hand, investigating the best way to deliver cells still remains a challenge in Taylors research.

Stem cell research in tissue engineering could save millions of lives around the world; therefore, Taylor believes that a coordinated approach among the researchers, clinicians, industry, regulatory bodies and, finally, society should be invigorated to catapult the field forward. For instance, the Twenty-first Century Cures Act can help advance her work by facilitating cooperation among experts and regulatory bodies, providing for accelerated approvals for therapeutic tools in regenerative medicine, and improving the regulation of biologics products. She also maintains that tissue engineering efforts remain poorly funded and believes that more resources must be allocated before her studies can come to life. There is a lot of dependence on societal benevolence, she said. In an interview with RedMedNet, she also said that intense collaboration on a national and an international level is crucial and should be a priority, even though it could be challenging due to scheduling issues and differences in time zones.

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Breakthroughs in Stem Cell Based Treatment of Heart Disease - The Connecticut College Voice

Research may lead to identifying novel therapies for cardiac patients

(New York, NY November 19, 2020) Human placental stem cells may have the potential to regenerate heart tissue after a heart attack, according to Mount Sinai researchers who have received a $2.9 million grant from the National Institutes of Health to study them. Their findings could lead to new therapies for repairing the heart and other organs.

Hina W. Chaudhry, MD,Director of Cardiovascular Regenerative Medicine at the Icahn School of Medicine at Mount Sinai, is the Principal Investigator for this four-year award.

This is very exciting. These cells may represent the ideal cell type for heart repair, which has been very challenging because clinical trials of other cell types did not find much benefit, says Dr. Chaudhry. Weve never before seen a stem cell type that can be harvested from an adult organthe placentaand has the ability to travel through the circulation and not be attacked by the immune system.

Dr. Chaudhry and a team of investigators previously discovered thatmouse placental stem cells can help the hearts of mice recover from injury that could otherwise lead to heart failure. They identified a specific type of placental stem cells, called Cdx2 cells, as the most effective in making heart cells regenerate. They discovered this by inducing heart attacks in groups of male mice and then injecting the placental Cdx2 cells isolated from females into their bloodstream. Imaging showed that the mice with Cdx2 stem cell treatments had significant improvement in cardiac function and regeneration of healthy tissue in the heart. The mice without this stem cell therapy went into heart failure and their hearts had no evidence of regeneration.

This team also found that the mouse Cdx2 cells have all the proteins of embryonic stem cells, which are known to generate all organs of the body, but also additional proteins, giving them the ability to travel directly to the injury site, which is something embryonic stem cells cannot do, and the Cdx2 cells appear to avoid the host immune response.

The new grant allows the researchers to build upon this discovery by isolating human Cdx2 cells from human placentas and studying their ability to grow heart cells. They also plan to expand into other organs and tissues in the future.

This was a serendipitous discovery based on clinical observations of patients with peripartum cardiomyopathy. We surmised that stem cells originating from the placenta may be assisting in repair of the mothers heart and designed studies to identify the cell types involved. We then showed that they work very well in male mice also when isolated from female placentas and now we hope to design a human cell therapy strategy for heart regeneration with this grant. Given that these cells maintain all the stem properties of embryonic stem cells, we are hopeful to utilize them for other types of organ repair as well, adds Dr. Chaudhry.

The grant is being used in collaboration with the Departments of Obstetrics and Gynecology and Pathology at Cedars-Sinai Medical Center in Los Angeles.

About the Mount Sinai Health System

The Mount Sinai Health System is New York City's largest academic medical system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai is a national and international source of unrivaled education, translational research and discovery, and collaborative clinical leadership ensuring that we deliver the highest quality carefrom prevention to treatment of the most serious and complex human diseases. The Health System includes more than 7,200 physicians and features a robust and continually expanding network of multispecialty services, including more than 400 ambulatory practice locations throughout the five boroughs of New York City, Westchester, and Long Island.Mount Sinai Heart at The Mount Sinai Hospital is within the nations No. 6-ranked heart center, and The Mount Sinai Hospital is ranked No. 14on U.S. News & World Report's "Honor Roll" of the Top 20 Best Hospitals in the country and the Icahn School of Medicine as one of the Top 20 Best Medical Schools in country. Mount Sinai Health System hospitals are consistently ranked regionally by specialty and our physicians in the top 1% of all physicians nationally by U.S. News & World Report.

For more information, visithttps://www.mountsinai.orgor find Mount Sinai on Facebook, Twitter and YouTube.

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Mount Sinai Cardiologist Awarded $2.9 Million NIH Grant to Advance Work with Stem Cells and Heart Repair after Heart Attack - Cath Lab Digest

The Autologous Stem Cell Based Therapies Market was valued at US$ XX million in 2019 and is projected to reach US$ XX million by 2025, at a CAGR of XX percentage during the forecast period. In this study, 2019 has been considered as the base and 2020 to 2025 as the forecast period to estimate the market size for Autologous Stem Cell Based Therapies Market

Deep analysis about market status (2016-2019), competition pattern, advantages and disadvantages of products, industry development trends (2019-2025), regional industrial layout characteristics and macroeconomic policies, industrial policy has also been included. From raw materials to downstream buyers of this industry have been analysed scientifically. This report will help you to establish comprehensive overview of the Autologous Stem Cell Based Therapies Market

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The Autologous Stem Cell Based Therapies Market is analysed based on product types, major applications and key players

Key product type:Embryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Key applications:Neurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

Key players or companies covered are:RegeneusMesoblastPluristem Therapeutics IncU.S. STEM CELL, INC.Brainstorm Cell TherapeuticsTigenixMed cell Europe

The report provides analysis & data at a regional level (North America, Europe, Asia Pacific, Middle East & Africa , Rest of the world) & Country level (13 key countries The U.S, Canada, Germany, France, UK, Italy, China, Japan, India, Middle East, Africa, South America)

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Key questions answered in the report:1. What is the current size of the Autologous Stem Cell Based Therapies Market, at a global, regional & country level?2. How is the market segmented, who are the key end user segments?3. What are the key drivers, challenges & trends that is likely to impact businesses in the Autologous Stem Cell Based Therapies Market?4. What is the likely market forecast & how will be Autologous Stem Cell Based Therapies Market impacted?5. What is the competitive landscape, who are the key players?6. What are some of the recent M&A, PE / VC deals that have happened in the Autologous Stem Cell Based Therapies Market?

The report also analysis the impact of COVID 19 based on a scenario-based modelling. This provides a clear view of how has COVID impacted the growth cycle & when is the likely recovery of the industry is expected to pre-covid levels.

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Global 3D Cardiac Mapping Systems Market: Overview

Cardiac mapping is a special type of technique which helps in gathering and displaying the information from cardiac electrograms. Such technique is mainly used in the diagnosis of heart rhythms. Therefore, cardiac mapping technique has gained immense popularity in case of arrhythmia. The cardiac mapping procedure involves the percutaneous insertion of catheter into the heart chamber and recording the cardiac electrograms sequentially. Such procedure helps in correlating the cardiac anatomy with the electrograms. The latest 3D cardiac mapping systems provide the three dimensional model of hearts chamber, which further helps in tracking the exact location of the catheter. Such advantages are majorly driving the global 3D cardiac mapping systems market.

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From the perspective of technology, the global 3D cardiac mapping systems market is segmented into basket catheter mapping, electroanatomical mapping, and real-time positional management (Cardiac pathways) EP system. Among these segments, electroanatomical mapping segment accounts for the maximum share in the global 3D cardiac mapping systems market. This mapping are extensively used in several healthcare industry due to its potential in increasing the safety, accuracy, and efficiency of catheter. A research report by TMR Research (TMR) thoroughly explains the new growth opportunities in the global 3D cardiac mapping systems market. Additionally, the report also provides a comprehensive analysis of the markets competitive landscape.

Global 3D Cardiac Mapping Systems Market: Notable Developments

Some of the recent developments are contouring the shape of the global 3D cardiac mapping systems market in a big way:

Key players operating in the global 3D cardiac mapping systems market include BioScience Webster, Boston Scientific Corporation, and Abbott.

Global 3D Cardiac Mapping Systems Market: Key Growth Drivers

Rising Number of Patients with Cardiac Disorders and Arrhythmia Fillips Market

The global 3D cardiac mapping systems market has grown steadily over the years, owing to the convenience it provides to the patients with heart problem. Growing number of people with cardiovascular diseases and rising cases of arrhythmia are the major factors fueling growth in the global 3D cardiac mapping systems market. Along with this, increasing pressure for reducing diagnosis errors and rapidly rising healthcare expenditure are also responsible for boosting the global 3D cardiac mapping systems market. However, above all such factors, the global 3D cardiac mapping systems market is majorly fueled by the accuracy and patient safety provided through real-time monitoring. Such 3D cardiac mapping systems are mainly designed to improve the resolution. This system also helps in gaining prompt of cardiac activation maps. All such advantages are also providing impetus to the growth of the global 3D cardiac mapping systems market.

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Furthermore, rising ageing population who are prone to heart-attack and several chronic heart disorders and increasing diagnosis rate of cardiac illness are the factors stoking demand in the global 3D cardiac mapping systems market. Moreover, this 3D cardiac mapping helps in reducing the diagnosis time. Such factor is also contributing to the growth of the global 3D cardiac mapping systems market.

Global 3D Cardiac Mapping Systems Market: Regional Outlook

On the regional front, North America is leading the global 3D cardiac mapping systems market as the region has seen rapid growth in healthcare industry. Along with this, increasing prevalence of heart attacks, rising healthcare expenditure, and burgeoning population is also responsible for fueling growth in the 3D cardiac mapping systems market in this region.

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16 November 2020

Organoids can be used to study early stages of heart development in mouse embryos, a new study shows.

Researchers from the cole Polytechnique Fdrale de Lausanne, Switzerland, have reported that they were able to produce a mouse heart organoid from embryonic stem cells, which displayed essential features of an early developing heart. They suggested that this reveals a novel application of organoids for studying early embryonic stages of development.

'One of the advantages of embryonic organoids is that, through the co-development of multiple tissues, they preserve crucial interactions that are necessary for embryonic organogenesis,' said Dr Giuliana Rossi, lead author of the study. 'The emerging cardiac cells are thus exposed to a context similar to the one that they encounter in the embryo.'

In their study, published in Cell Stem Cell, the team exposed mouse embryonic stem cells to a mix of three factors involved in promoting heart growth. One week later, the stem cells self-organised into so-called gastruloids:organoids with an embryo-like organisation, which displayed signs of early heart development. The cell aggregatesnot only expressed several genes known to regulate cardiovascular development, but also generated a structure resembling a vascular network. Furthermore, the researchers found an 'anterior cardiac crescent-like domain' in the gastruloids, which even produced a beating heart tissue. Similar to the muscle cells of the embryonic heart, this area was also sensitive to calcium ions.

Organoids have been mostly the focus of research into the generation of adult tissues and organs for pharmaceutical and medical research. In their new publication, Professor Matthias Ltolf and his team suggested that they can also provide a system to study early embryonic stages of the developing heart and other organs, as they preserve important tissue-tissue interactions.

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Organoids mimic the early development of the heart in mouse embryos - BioNews

Once upon a time, growing organs in the lab were science fiction. But now, methods such as stem cell biology and tissue engineering have turned that fiction into reality with the advent of organoids.

Organoids are tiny lab-grown tissues and organs that are anatomically correct and physiologically functional.

Recently, the lab of Matthias Ltolf at the School of Life Sciences at EPFL has successfully produced a mouse heart organoid in its early embryonic stages. Scientists grew organoids from mouse embryonic stem cells, which, under the right conditions, can self-organize into structures that mimic aspects of the architecture, cellular composition, and function of tissues found in real organs.

Placed in cell-culture under specific conditions, the embryonic stem cells from a three-dimensional aggregate called a gastruloid, which can follow the mouse embryos developmental phases.

This studys idea was that the mouse gastruloid could be utilized to mimic the beginning phases of heart development in the embryo. This is a new use of organoids, which are commonly developed to mimic adult tissues and organs.

Also, there are three features of mouse gastruloids that make them a suitable template for mimicking embryonic development: they establish a body plan like real embryos. They show similar gene expression patterns. And when it comes to the heart, which is the first organ to form and function in the embryo, the mouse gastruloid also preserves important tissue-tissue interactions necessary to grow one.

Equipped with this, the scientists exposed mouse embryonic stem cells to a cocktail of three factors known to promote heart growth. Following 168 hours, the subsequent gastruloids gave early heart development indications: they expressed several genes that regulate cardiovascular development in the embryo. They even generated what resembled a vascular network.

Importantly, scientists found that the gastruloids developed what they call an anterior cardiac crescent-like domain. This structure produced a beating heart tissue, similar to the embryonic heart. As the muscle cells of the embryonic heart, the beating compartment was also sensitive to calcium ions.

Giuliana Rossi, a post-doctoral researcher from Ltolfs laboratory, said,Opening up an entirely new dimension to organoids, the breakthrough work shows they can also be used to mimic embryonic stages of development. One of the advantages of embryonic organoids is that, through the co-development of multiple tissues, they preserve crucial interactions that are necessary for embryonic organogenesis.

The emerging cardiac cells are thus exposed to a context similar to the one that they encounter in the embryo.

The study was conducted in collaboration with Viventis Microscopy, EPFL Bioimaging and Optics Platform, Institut de Biologie du Dveloppement de Marseille, Johns Hopkins University School of Medicine, EPFL Institute of Chemical Sciences and Engineering.

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Mimicking the early development of the heart - Tech Explorist

DUBLIN--(BUSINESS WIRE)--The "Cell Therapy and Gene Therapy Markets" report has been added to ResearchAndMarkets.com's offering.

This is an exciting and interesting time in the cell and gene therapy industry. The science is moving ahead as industry industrializes and standardizes the manufacturing and commercialization of products. Cell and gene therapy products are transforming the treatment of cancers and genetic diseases, as well as expanding into other areas of medicine including autoimmune diseases, cardiovascular diseases, musculoskeletal disease, dermatological diseases, and many others.

Cell Therapy and Gene Therapy Markets presents the market in segments that provide an overview of disease epidemiology, market estimates and forecasts, and competitive summary of leading providers:

The report examines developments in cell and gene therapy markets by condition/disorder, including principal products, trends in research and development, market breakdown of cell and gene therapies, regional market summary, and competitor summary.

The following conditions/disorders are covered:

Dermatology, including:

Oncology, including:

Ophthalmic Conditions, including:

Other Conditions, including:

The report comments on the current COVID-19 cell and gene therapy pipeline. There are a number of companies that are responding to the call to develop a therapeutic or vaccine for the coronavirus, including:

The leading influencers in the market are those which have become first-to-market participants in the cell and gene therapy segment, have new developments which may disrupt current market conditions, and/or have an extensive pipeline sure to impact the market in the long-term forecast:

Because gene therapies are currently not available in any wide capacity, there is little precedent upon which to base forecasts. Dollar figures represent the estimated global market for 2019 and the expected market for 2020 based on first-quarter company reports and are expressed in current dollars. Forecasts are provided through 2025 and an extended forecast for 2030. The size of each market segment refers to manufacturers' revenues.

For more information about this report visit https://www.researchandmarkets.com/r/ek1qqb

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Cell Therapy and Gene Therapy Markets, 2019-2020 & Forecast to 2025 and 2030 - ResearchAndMarkets.com - Business Wire

Another day, another win for Mercks blockbuster Keytruda.

The FDA has granted accelerated approval for the cash cow combined with chemotherapy in triple negative breast cancer, giving the drug the green light in its 18th different cancer. Mondays new indication comes for patients with PD-L1-expressing tumors with a Combined Positive Score of at least 10.

Merck noted that due to the nature of the accelerated approval, the thumbs up is contingent upon confirmatory trials.

Data for the approval first came back in February, when the Keynote-355 trial demonstrated Keytruda plus chemo significantly improved progression-free survival compared to chemo by itself. The study showed that, in the target population with a CPS of at least 10, the combination reduced the risk of disease progression or death by 35% with a median PFS of 9.7 months, against 5.6 months in the placebo arm.

On safety, the February data showed 2.5% of all patients in the drug arm saw fatal adverse events, including cardiac arrest and septic shock, with serious side effects appearing in 30% of patients. Keytruda was discontinued due to adverse events in 11% of patients.

Frontline triple negative breast cancer is a particularly difficult indication to treat, as the growth of the cancer is not fueled by the hormones estrogen and progesterone, or by the HER2 protein. Its one of the rare fields in which Roches PD-L1 Tecentriq has enjoyed a head start over Keytruda and Opdivo, the leaders in the checkpoint race, as Tecentriq is approved in combination with Abraxane for this indication.

Back in May 2019, Merck conceded a failure in the arena after a Phase III study flopped on overall survival. But a few months later, the pharma turned things around after discovering a neoadjuvant regimen of Keytruda and chemo followed by Keytruda monotherapy after surgery induced a higher pathological complete response rate.

Though execs presented that as a positive, some analysts didnt paint as sunny a picture. This past February, when the Keynote-355 topline data was first published, SVB Leerinks Daina Graybosch pointed out that because only patients with a CPS of at least 10 appeared to benefit, instead of a score of at least 1, it wont be able to treat as broad a population as Tecentriq. Roche, she noted, also has about a two-year head start.

A Merck spokesperson also had this to say about the CPS and IC percentages:

In TNBC, we measure PD-L1 with a combined positive score (CPS). The CPS includes staining for tumor cells, as well as tumor-infiltrating immune cells and it is not a percentage. We believe CPS 10 is roughly equivalent to how Roche scores PD-L1+ patients (IC>=1% based on the SP142 assay) on tumor-infiltrating immune cells (IC). The prevalence of the PD-L1 positive population in TNBC whether by CPS of greater than or equal to 10 or IC of 1% is both about 40%.

Keytruda is already one of the best-selling drugs in the world, having notched roughly $3.9 billion in the first half of 2020 alone. Some have predicted the drug may overtake AbbVies Humira as the top seller within the next few years, with the most optimistic estimate pegged for $22.2 billion in sales by 2025.

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UPDATED: Merck's Keytruda nets another approval, this time in triple negative breast cancer. Can it catch up to Tecentriq? - Endpoints News

Can hearts repair themselves via their own stem cells?

Sometimes what we scientists all know to be true turns out later on to be wrong and there are clear instancesof this in the stem cell field.

For example for decades the dogma was that the adult mammalianbrain did not have stem cells, but now most researchers believethat the adult brain does have stem cells, although for humans this is still being debated.

What we perceive as factual can change over time.

Yamanaka disproved the entrenched notion that differentiated cells were permanently locked into that differentiated state with his revolutionary findings on induced pluripotent stem cells. The new reality, which seemed revolutionary in some ways in 2006-2007, now is established fact.

So what about the idea of the human heart have resident populations of stem cells that can fix problems, perhaps as severe as damage from heart attacks? One of the first stem cell talks I ever saw way back around IPS cells were discovered was by a guy who assumed the factual answer to this question was Yes!

What about now in 2020? I have an ongoing Twitter poll on this question as of Nov. 5, 2020 so check it out below.

Today the cardiac regenerative field finds itself at an interesting crossroads.

A few say yes there are cardiac stem cells and that they can mediate repair. However, most heart researchers that Ive talked to in recent years feel just as strongly that there are no such cells. Some also have added that even if there are at least a handful of such cells or they arise due to damage, they cant do anything meaningful about serious heart damage.

I asked cardiac stem cell expert, Deepak Srivastava for his thoughts on this in a previous post and foundhis answercompelling. Because that was a fairly long time ago, I got an update to the same kind of question just now from two leading cardiac regenerative medicine and stem cell researchers.

Associate Professor of Medicine and Director of Cardiovascular Regenerative Medicine at Mt. Sinai, Hina Chaudhry had this to say:

One thing is certain: As both a clinical cardiologist who has cared for patients with heart attacks and a stem cell biologist, I can tell you that no scientific data supports an endogenous stem cell population in the adult heart and that no adult with a transmural myocardial infarction ever loses the resulting scar throughout their lifetime.

Professor Chuck Murry of the UW sent this:

Our current best evidence suggests that there are no stem cells in the adult heart that can give rise to new cardiomyocytes. This has been studied intensivelyfrom the bottom up, by tracing candidate stem cells and following their differentiated progeny, and from the top down by marking pre-existing cardiomyocytes and looking for their dilution as unmarked stem cells enter the pool. Both have shown the same thing: if this happens at all, its frequency is on the order of 10e-4 per year, which by any measure is next to nothing. There is slow turnover of cardiomyocytes in the adult mammalian heart, at ~1% per year, and this can be accounted for entirely by replication of pre-existing cardiomyocytes. One has to wonder, why has Nature done this? Why would such a vital organ have no stem cells for replenishment, along with such a low rate of endogenous replication?

I believe that Drs. Murry and Chaudhry are right. Chucks last question there is one for long discussions and is similar to discussions Ive had about the few stem cells/potential for endogenous repair in the adult human brain.

Still, you can find a diversity of papers now in 2020 in PubMed with Heart Regeneration or Cardiac Regeneration or Cardiac stem cells in their titles. However, many of the papers relate to stem cell infusions rather than invoking endogenous resident cells.

If not in humans, what about other mammals? There are glimpses of interesting possible stem cell activity in the mammalian heart, even if not in humans

A November 2014Cell Stem Cell paper from the lab of Juan Carlos Izpisua Belmonte, entitled InVivo Activation of a Conserved MicroRNA Program Induces Mammalian Heart Regeneration, argues for endogenous mammalian heart regeneration in part via dedifferentiation of cells into stem-like cells. This raises the interesting notion that while the mammalian heart does not normally have many (or any?) resident stem cells, damage can change some other cells into stem cell-like cells.

One of the biggest advocates of endogenous cardiac stem cells and repair, Piero Anversa formerly of Harvard and Brigham and Womens Hospital, has become one of the most controversial as well. His papers have come under fire and some have been retracted. Anversa was the subject of a Harvard investigation and was suing Harvard for how it has conducted the investigation and other matters related to his work.

In my view, his situation has raised even more skepticism about the idea of endogenous heart stem cells in people.

Even if the endogenous stem cell-like activity in the heart is absent or not enough to mediate clinically significant repair in humans, by deciphering the molecular basis of this kind of activity in other animals could the field still open the door to powerful new treatments for heart disease? For instance, if some adult mammalian hearts naturally replace 1 in 200 cells per year, perhaps cardiac researchers can find a way to boost that by an order of magnitude with a drug and have a meaningful impact for human patients.

Or if dedifferentiation of non-stem cells in the heart into stem cell-like cells can be induced by damage, could a drug therapy trigger that same effect even if damage occurred long ago or in the context of relatively minor damage?

Many researchers are focusing more on using injections of stem cells into the heart to repair damage. The types of stem cells being used for research attempts at heart repair are very diverse, including both placental cells and indirect use of IPS cells in Japan via a recently approved trial there.

Even the area of stem cell transplants into the heart generates its share of debate and well have to see in the long run how the clinical trial data turn out. I hope there can be positive impact in the future given the overwhelming number of people with heart damage.

Related

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Can Hearts Repair Themselves Via Stem Cells - The Niche