Newswise What gets the leader of the NIH jazzed?

Speaking to a packed West Pavilion auditorium March 6, Francis Collins, M.D., Ph.D., director of the National Institutes of Health, shared his picks of 10 areas of particular excitement and promise in biomedical research. (Watch the full talk here.)

In nearly every area, UAB scientists are helping to lead the way as Collins himself noted in several cases. At the conclusion of his talk, Collins addedhis advice for young scientists. Here is Collins top 10 list, annotated with some of the UAB work ongoing in each area and ways that faculty, staff and students can get involved.

1. Single-cell sequencing

[see this section of the talk here]

I am so jazzed with what has become possible with the ability to study single cells and see what they are doing, Collins said. They have been out of our reach now we have reached in. Whether you are studying rheumatoid arthritis, diabetes or the brain, you have the chance to ask each cell what it is doing.

Single-cell sequencing and UAB:Collins noted that Robert Carter, M.D., the acting director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, was a longtime faculty member at UAB (serving as director of the Division of Clinical Immunology and Rheumatology). For the past several years, UAB researchers have been studying gene expression in subpopulations of immune cells inpatients with rheumatoid arthritis.

Join in:Researchers can take advantage of the single-cell sequencing core facility in UABsComprehensive Flow Cytometry Core, directed by John Mountz, M.D., Ph.D., Goodwin-Blackburn Research Chair in Immunology and professor in the Department of Medicine Division of Clinical Immunology and Rheumatology.

Learn more:Mountz and other heavy users of single-cell sequencing explain how the techniqueslet them travel back in time and morein this UAB Reporter story.

2. New ways to see the brain

[See this section of the talk here]

The NIHsBRAIN Initiativeis making this the era where we are going to figure out how the brain works all 86 billion neurons between your ears, Collins said. The linchpin of this advance will be the development of tools to identify new brain cell types and circuits that will improve diagnosis, treatment and prevention of autism, schizophrenia, Parkinsons and other neurological conditions, he said.

Brain tech and UAB:Collins highlighted thework of BRAIN Initiative granteeHarrison Walker, M.D., an associate professor in the Department of Neurology, whose lab has been developing a more sophisticated way to understand the benefits of deep brain stimulation for people with Parkinsons and maybe other conditions, Collins said.

Join in:UABs planned new doctoral program in neuroengineering would be the first of its kind in the country.

Learn more:Find out why neuroengineering is asmart career choicein this UAB Reporter story.

3. Induced pluripotent stem (iPS) cells

[See this section of the talk here]

Researchers can now take a blood cell or skin cell and, by adding four magic genes, Collins explained, induce the cells to become stem cells. These induced pluripotent stem (iPS) cells can then in turn be differentiated into any number of different cell types, including nerve cells, heart muscle cells or pancreatic beta cells. The NIH has invested in technology to put iPS-derived cells on specialized tissue chips. Youve got you on a chip, Collins explained. Some of us dream of a day where this might be the best way to figure out whether a drug intervention is going to work for you or youre going to be one of those people that has a bad consequence.

iPS cells at UAB:Collins displayed images of thecutting-edge cardiac tissue chipdeveloped by a UAB team led by Palaniappan Sethu, Ph.D., an associate professor in the Department of Biomedical Engineering and the Division of Cardiovascular Disease. The work allows the development of cardiomyocytes that can be used to study heart failure and other conditions, Collins said.

Join in:UABs biomedical engineering department, one of the leading recipients of NIH funding nationally, is a joint department of the School of Engineering and School of Medicine. Learn more about UABsundergraduate and graduate programs in biomedical engineering, and potential careers, here.

Learn more:See howthis novel bioprinterdeveloped by UAB biomedical researchers is speeding up tissue engineering in this story from UAB News.

4. Microbiome advances

[See this section of the talk here]

We have kind of ignored the fact that we have all these microbes living on us and in us until fairly recently, Collins said. But now it is clear that we are not an organism we are a superorganism formed with the trillions of microbes present in and on our bodies, he said. This microbiome plays a significant role not just in skin and intestinal diseases but much more broadly.

Microbiome at UAB:Collins explained that work led by Casey Morrow, Ph.D., and Casey Weaver, M.D., co-directors of theMicrobiome/Gnotobiotics Shared Facility, has revealed intriguing information abouthow antibiotics affect the gut microbiome. Their approach has potential implications for understanding, preserving and improving health, Collins said.

Join in:Several ongoing clinical trials at UAB are studying the microbiome, including a studymodifying diet to improve gut microbiotaand an investigation of the microbiomes ofpostmenopausal women looking for outcomes and response to estrogen therapy.

Learn more:This UAB News storyexplains the UAB researchthat Collins highlighted.

5. Influenza vaccines

[See this section of the talk here]

Another deadly influenza outbreak is likely in the future, Collins said. What we need is not an influenza vaccine that you have to redesign every year, but something that would actually block influenza viruses, he said. Is that even possible? It just might be.

Influenza research at UAB:Were probably at least a decade away from a universal influenza vaccine. But work ongoing at UAB in the NIH-fundedAntiviral Drug Discovery and Development Center(AD3C), led by Distinguished Professor Richard Whitley, M.D., is focused on such an influenza breakthrough.

Join in:For now, the most important thing you can do to stop the flu is to get a flu vaccination. Employees can schedule afree flu vaccination here.

Learn more:Why get the flu shot? What is it like? How can you disinfect your home after the flu? Get all the information atthis comprehensive sitefrom UAB News.

6. Addiction prevention and treatment of pain

[See this section of the talk here]

The NIH has a role to play in tackling the crisis of opioid addiction and deaths, Collins said. The NIHs Helping to End Addiction Long-term (HEAL) initiative is an all-hands-on-deck effort, he said, involving almost every NIH institute and center, with the goal of uncovering new targets for preventing addiction and improving pain treatment by developing non-addictive pain medicines.

Addiction prevention at UAB:A big part of this initiative involves education to help professionals and the public understand what to do, Collins said. The NIH Centers of Excellence in Pain Education (CoEPE), including one at UAB, are hubs for the development, evaluation and distribution of pain-management curriculum resources to enhance pain education for health care professionals.

Join in:Find out how to tell if you or a loved one has a substance or alcohol use problem, connect with classes and resources or schedule an individualized assessment and treatment through theUAB Medicine Addiction Recovery Program.

Learn more:Discover some of the many ways that UAB faculty and staff aremaking an impact on the opioid crisisin this story from UAB News.

7. Cancer Immunotherapy

[See this section of the talk here]

We are all pretty darn jazzed about whats happened in the past few years in terms of developing a new modality for treating cancer we had surgery, we had radiation, we had chemotherapy, but now weve got immunotherapy, Collins said.

Educating immune system cells to go after cancer in therapies such as CAR-T cell therapy is the hottest science in cancer, he said. I would argue this is a really exciting moment where the oncologists and the immunologists together are doing amazing things.

Immunotherapy at UAB:I had to say something about immunology since Im at UAB given that Max Cooper, whojust got the Lasker Awardfor [his] B and T cell discoveries, was here, Collins said. This is a place I would hope where lots of interesting ideas are going to continue to emerge.

Join in:The ONeal Comprehensive Cancer Center at UAB is participating in a number of clinical trials of immunotherapies.Search the latest trials at the Cancer Centerhere.

Learn more:Luciano Costa, M.D., Ph.D., medical director of clinical trials at the ONeal Cancer Center, discusses the promise ofCAR-T cell therapy in this UAB MedCast podcast.

Assistant Professor Ben Larimer, Ph.D., is pursuing a new kind of PET imaging test that could give clinicians afast, accurate picture of whether immunotherapy is workingfor a patient in this UAB Reporter article.

8. Tapping the potential of precision medicine

[See this section of the talk here]

The All of Us Research Program from NIH aims to enroll a million Americans to move away from the one-size-fits-all approach to medicine and really understand individual differences, Collins said. The program, which launched in 2018 and is already one-third of the way to its enrollment goal, has a prevention rather than a disease treatment approach; it is collecting information on environmental exposures, health practices, diet, exercise and more, in addition to genetics, from those participants.

All of Us at UAB:UAB has been doing a fantastic job of enrolling participants, Collins noted. In fact, the Southern Network of the All of Us Research Program, led by UAB, has consistently been at the top in terms of nationwide enrollment, as School of Medicine Dean Selwyn Vickers, M.D., noted in introducing Collins.

Join in:Sign up forAll of Usat UAB today.

Learn more:UABs success in enrolling participants has led to anew pilot study aimed at increasing participant retention rates.

9. Rare diseases

[See this section of the talk here]

Rare Disease Day, on Feb. 29, brought together hundreds of rare disease research advocates at the NIH, Collins said. NIH needs to play a special role because many diseases are so rare that pharmaceutical companies will not focus on them, he said. We need to find answers that are scalable, so you dont have to come up with a strategy for all 6,500 rare diseases.

Rare diseases at UAB: The Undiagnosed Diseases Network, which includes aUAB siteled by Chief Genomics Officer Bruce Korf, M.D., Ph.D., is a national network that brings together experts in a wide range of conditions to help patients, Collins said.

Participants in theAlabama Genomic Health Initiative, also led by Korf, donate a small blood sample that is tested for the presence of specific genetic variants. Individuals with indications of genetic disease receive whole-genome sequencing. Collins noted that lessons from the AGHI helped guide development of the All of Us Research Program.

Collins also credited UABs Tim Townes, Ph.D., professor emeritus in the Department of Biochemistry and Molecular Genetics, for developing the most significantly accurate model of sickle cell disease in a mouse which has been a great service to the [research] community. UAB is now participating in anexciting clinical trial of a gene-editing technique to treat sickle cellalong with other new targeted therapies for the devastating blood disease.

Join in:In addition to UABs Undiagnosed Diseases Program (which requires a physician referral) and the AGHI, patients and providers can contact theUAB Precision Medicine Institute, led by Director Matt Might, Ph.D. The institute develops precisely targeted treatments based on a patients unique genetic makeup.

Learn more:Discover how UAB experts solved medical puzzles for patients by uncovering anever-before-described mutationandcracking a vomiting mysteryin these UAB News stories.

10. Diversity in the scientific workforce

[See this section of the talk here]

We know that science, like everything else, is more productive when teams are diverse than if they are all looking the same, Collins said. My number one priority as NIH director is to be sure we are doing everything we can to nurture and encourage the best and brightest to join this effort.

Research diversity at UAB:TheNeuroscience Roadmap Scholars Programat UAB, supported by an NIH R25 grant, is designed to enhance engagement and retention of under-represented graduate trainees in the neuroscience workforce. This is one of several UAB initiatives to increased under-represented groups and celebrate diversity. These include several programs from theMinority Health and Health Disparities Research Centerthat support minority students from the undergraduate level to postdocs; thePartnership Research Summer Training Program, which provides undergraduates and especially minority students with the opportunity to work in UAB cancer research labs; theDeans Excellence Award in Diversityin the School of Medicine; and the newly announcedUnderrepresented in Medicine Senior Scholarship Programfor fourth-year medical students.

Join in:The Roadmap program engages career coaches and peer-to-peer mentors to support scholars. To volunteer your expertise, contact Madison Bamman atmdbamman@uab.eduorvisit the program site.

Learn more:Farah Lubin, Ph.D., associate professor in the Department of Neurobiology and co-director of the Roadmap Scholars Program,shares the words and deeds that can save science careersin this Reporter story. In another story, Upender Manne, Ph.D., professor in the Department of Pathology and a senior scientist in the ONeal Comprehensive Cancer Center, explains how students in the Partnership Research Summer Training Program gethooked on cancer research.

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Looking to the future with Dr. Francis Collins - Newswise

IPS Cell Therapy Comments Off on Looking to the future with Dr. Francis Collins – Newswise | March 17th, 2020

New York, March 13, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Induced Pluripotent Stem Cells Market: Global Industry Analysis, Trends, Market Size, and Forecasts up to 2025" - https://www.reportlinker.com/p05874276/?utm_source=GNW 6% over the forecast period from 2019-2025. The study on induced pluripotent stem cells market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and RoW for the period of 2017 to 2025.

The report on induced pluripotent stem cells market is a comprehensive study and presentation of drivers, restraints, opportunities, demand factors, market size, forecasts, and trends in the global induced pluripotent stem cells market over the period of 2017 to 2025. Moreover, the report is a collective presentation of primary and secondary research findings.

Porters five forces model in the report provides insights into the competitive rivalry, supplier and buyer positions in the market and opportunities for the new entrants in the global induced pluripotent stem cells market over the period of 2017 to 2025. Further, IGR- Growth Matrix gave in the report brings an insight into the investment areas that existing or new market players can consider.

Report Findings1) Drivers Increased government fundings and rising industry focus on the development of novel therapies Rising interest in stem cell therapy2) Restraints High the cost associated with storage3) Opportunities Growing applications of iPS cells in several biopharmaceutical applications provides extensive potential to the key players in the market

Research Methodology

A) Primary ResearchOur primary research involves extensive interviews and analysis of the opinions provided by the primary respondents. The primary research starts with identifying and approaching the primary respondents, the primary respondents are approached include1. Key Opinion Leaders associated with Infinium Global Research2. Internal and External subject matter experts3. Professionals and participants from the industry

Our primary research respondents typically include1. Executives working with leading companies in the market under review2. Product/brand/marketing managers3. CXO level executives4. Regional/zonal/ country managers5. Vice President level executives.

B) Secondary ResearchSecondary research involves extensive exploring through the secondary sources of information available in both the public domain and paid sources. At Infinium Global Research, each research study is based on over 500 hours of secondary research accompanied by primary research. The information obtained through the secondary sources is validated through the crosscheck on various data sources.

The secondary sources of the data typically include1. Company reports and publications2. Government/institutional publications3. Trade and associations journals4. Databases such as WTO, OECD, World Bank, and among others.5. Websites and publications by research agencies

Segment CoveredThe global induced pluripotent stem cells market is segmented on the basis of derived cell type, application, and end user.

The Global Induced Pluripotent Stem Cells Market by Derived Cell Type Fibroblasts Amniotic Cells Hepatocytes Keratinocytes Others

The Global Induced Pluripotent Stem Cells Market by Application Drug Development Regenerative Medicine Toxicity Testing Academic Research

The Global Induced Pluripotent Stem Cells Market by End User Research Organizations Hospitals Biopharma Industries

Company Profiles Astellas Pharma Inc. Fate Therapeutics Inc. FUJIFILM Holdings Corporation Evotec SE Japan Tissue Engineering Co., Ltd ViaCyte, Inc. Vericel Corporation Bristol-Myers Squibb Company Aastrom Biosciences, Inc. Acelity Holdings, Inc.

What does this report deliver?1. Comprehensive analysis of the global as well as regional markets of the induced pluripotent stem cells market.2. Complete coverage of all the segments in the induced pluripotent stem cells market to analyze the trends, developments in the global market and forecast of market size up to 2025.3. Comprehensive analysis of the companies operating in the global induced pluripotent stem cells market. The company profile includes analysis of product portfolio, revenue, SWOT analysis and latest developments of the company.4. IGR- Growth Matrix presents an analysis of the product segments and geographies that market players should focus to invest, consolidate, expand and/or diversify.Read the full report: https://www.reportlinker.com/p05874276/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Global induced pluripotent stem cells market is expected to grow with a CAGR of 8.6% over the forecast period from 2019-2025 - GlobeNewswire

IPS Cell Therapy Comments Off on Global induced pluripotent stem cells market is expected to grow with a CAGR of 8.6% over the forecast period from 2019-2025 – GlobeNewswire | March 13th, 2020

First, by their nature, pluripotent stem cells can potentially be used to create any cell or tissue the body might need to counter a wide range of diseases, from diabetes to spinal cord injury, to childhood leukemia, to heart disease.

Second, pluripotent stem cells can potentially be customized to provide a perfect genetic match for any patient. This means that patients could receive transplants of tissue and cells without tissue matching and tissue rejection problems, and without the need to take powerful immune-suppressing drugs for the rest of their lives. Although this vision hasnt yet been achieved, researchers at Boston Childrens Hospital have successfully treated mouse models of human disease using this strategy and hope that the same can be done with patients.

Disease in a dish:Third, pluripotent stem cells make excellent laboratory models for studying how a disease unfolds, which helps scientists pinpoint and track the very earliest disease-causing events in cells. Immune deficiencies, Type 1 diabetes, muscular dystrophy, and myriad other disorders are rooted in fetal development. In the lab, researchers can recapture these early originsobserving where the first muscle cell comes from, or the first blood cell, and how this differs when the patient has a genetic disease. Using this information, doctors may be able to intervene and correct the genetic defect before the disease advances.

Unique applications:Each type of pluripotent stem cell has different characteristics that make it useful in different ways, and each has different lessons to teach.

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Why are Pluripotent Stem Cells Important? Boston ...

IPS Cell Therapy Comments Off on Why are Pluripotent Stem Cells Important? Boston … | March 12th, 2020

In 2020, the National Eye Institute is launching a clinical trial to test the safety of a patient-specific stem cell therapy to treat geographic atrophy, the advanced dry form of age-related macular degeneration (AMD). The protocol is the first of its kind in the United States to replace a patients eye tissue with tissue derived from induced pluripotent stem (iPS) cells engineered from a patients own blood.

If successful, this new approach to AMD treatment could prevent millions of Americans from going blind. AMD is a leading cause of vision loss in people age 65 and older. By 2050, the estimated number of people with AMD is expected to more than double from 2.07 million to 5.44 million.

The first symptoms of age-related macular degeneration are dark spots in ones central vision, which is used for daily activities such as reading, seeing faces and driving. But as the disease progresses, the spots grow larger and increase in number, which can lead to significant loss of the central vision.

There are two kinds of AMD: the neovascular, or wet, form and the geographic atrophy, or dry form. Remarkable progress has been made in the ability to prevent vision loss from the neovascular form. In particular, anti-VEGF therapy has been shown to preserve vision required for driving among about half of patients who take it for five years.

By contrast, no therapies exist for treating geographic atrophy. Should this NEI-led study, and future studies, confirm the safety and efficacy of iPS cell-derived RPE-replacement therapy, it would likely be the first therapy approved for the treatment of geographic atrophy.

To produce the therapy, we isolate cells from a patients blood and, in a lab, convert them into iPS cells. These iPS cells are theoretically capable of becoming any cell type of the body.

The iPS cells are then programmed to become retinal pigment epithelium (RPE). RPE cells are crucial for eye health because they nourish and support photoreceptors, the light-sensing cells in the retina. In geographic atrophy, RPE cells die, leading to the death of photoreceptors and blindness. The goal of the iPS cell-based therapy is to protect the health of the remaining photoreceptors by replacing dying RPE tissue with healthy iPS cell-derived RPE tissue.

We grow a single-cell layer of iPS cell-derived RPE on a biodegradable scaffold. That patch is then surgically placed next to the photoreceptors where, as we have seen in animal models, it integrates with cells of the retina and protects the photoreceptors from dying.

This years clinical trial is a phase I/IIa study, which means it will focus solely on assessing the safety and feasibility of this RPE replacement therapy. The dozen participants will have one eye treated. Importantly, everyone will already have substantial vision loss from very advanced disease, such that the therapy is not expected to be capable of significant vision restoration. Once safety is established, later study phases will involve individuals with earlier stage disease, for which we are hopeful that therapy will restore vision.

A safety concern with any stem cell-based therapy is its oncogenic potential: the ability for cells to multiply uncontrollably and form tumours. On this point, animal model studies are reassuring. When we genetically analysed the iPSC-derived RPE cells, we found no mutations linked to potential tumour growth.

Likewise, the risk of implant rejection is minimised by the fact that the therapy is derived from patient blood.

Several noteworthy innovations have occurred along the way to launching the trial. Artificial intelligence has been applied to ensure that iPS cell-derived RPE cells function similar to native RPE cells. In addition, Good Manufacturing Practices, have been developed to ensure quality control, which will be crucial for scaling up production of the therapy should it receive approval from the U.S. Food and Drug Administration. Furthermore, the iPS cell-derived RPE patch is being leveraged to develop more complex RPE/photoreceptor replacement therapies.

Potential breakthroughs in treatment cannot move forward without the support of patients willing to participate in clinical trial research. Patients who volunteer for trials such as this are the real heroes of this work because theyre doing it for altruistic reasons. The patients in this first trial are not likely to benefit, so they are doing it to help move the field forward for future patients.

Editor's Recommended Articles

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Eye health: Testing the safety of stem cell therapy for age-related macular degeneration - Open Access Government

IPS Cell Therapy Comments Off on Eye health: Testing the safety of stem cell therapy for age-related macular degeneration – Open Access Government | March 10th, 2020

AgeX Therapeutics, Inc. ("AgeX"; NYSE American: AGE), a biotechnology company focused on developing therapeutics for human aging and regeneration, announced a new paper co-authored by two AgeX scientists that could lead to new insights into the fundamental mechanisms of aging and why super-centenarians not only live the longest, but also experience extraordinary healthspans; an extension of the healthy years of life that compresses morbidity to a very short period near the end of life. The paper, "Induced pluripotency and spontaneous reversal of cellular aging in supercentenarian donor cells," is published online in the peer-reviewed scientific journal "Biochemical and Biophysical Research Communications" from Elsevier. The senior author is Dana Larocca, PhD, VP of Discovery Research at AgeX, and the first author is Jieun Lee, PhD, Scientist at AgeX.

"Clearly, we can learn a lot about aging and longevity from the longest of the long-lived, the supercentenarians, and we hope that this paper accelerates such research," commented Dr. Larocca. "Now that we have converted the cells of one of the longest-lived people in history, a deceased 114-year-old American woman, to a young pluripotent state, researchers can do so with cells from other supercentenarians. The goal is to understand specifically how these "extreme agers" manage to avoid the major chronic illnesses of aging better than any other age group including centenarians. We can essentially put their cells in a time machine and revert them to an earlier state, then study their biology to help unlock the mysteries of super-longevity. Scientists have long wondered, and now we know that we can indeed reset the developmental state and cellular age in the oldest of the old."

By way of comparison, the paper also describes undertaking a similar process with cells from two other donors: an eight-year-old with a rapid-aging syndrome commonly known as Progeria, and a 43-year-old, healthy disease-free control (HDC) subject. The paper notes that the supercentenarians cells reverted to induced pluripotent stem (iPS) cells at the same rate as the HDC subject and the Progeria patient. However, there may be some negative impact of extreme age on telomere resetting as this did not occur as frequently in the supercentenarian as in the other two donors.

The donated cells were from "the longevity collection," a cell bank established by the NIHs National Institute on Aging.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200228005122/en/

Contacts

Media Contact for AgeX:

Bill Douglass Gotham Communications, LLCbill@gothamcomm.com (646) 504-0890

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AgeX Therapeutics Researchers Publish Paper on the Age Reprogramming of Super-Centenarian Cells - Yahoo Finance

IPS Cell Therapy Comments Off on AgeX Therapeutics Researchers Publish Paper on the Age Reprogramming of Super-Centenarian Cells – Yahoo Finance | February 29th, 2020

CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells

T cells and B cells are 2 primary cell types in our adaptive immune system, the source of immunological memory protecting us from subsequent pathogen exposure. B cells secrete pathogen-specific antibodies, which neutralize pathogens directly, or tag them for attack by other immune cells. T cells destroy pathogenic cells directly as well as secrete cytokines to attract additional immune cells.

Chimeric antigen receptor (CAR) T cells are patient derived T cells genetically manipulated to express an artificial transmembrane receptor. The artificial receptor is engineered from modular parts to bind to a surface protein (also called an antigen) on malignant cells and activate the T cell via engineered T cell signaling switches on the CAR.

Current FDA-approved CAR-T cell therapies express CARs recognizing CD19, which is expressed on the surface of almost all B cells, making these therapies specific for B-cell malignancies. Following binding with a CD19-expressing cell, the CAR T cell is activated to proliferate, eliminate the CD19-expressing cell, and persist within the patient.

Tisagenlecleucel (Kymriah, Novartis) is approved to treat pediatric and young adult patients (up to age 25) with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (R/R ALL, ELIANA trial) and adult patients with R/R diffuse large B-cell lymphoma (R/R DLBCL, JULIET trial) after 2 or more lines of systemic therapy. Axicabtagene ciloleucel (Yescarta, Kite Pharma) is approved for adult patients with R/R large B-cell lymphoma (R/R DLBCL, ZUMA-1 trial, NCT02348216) after 2 or more lines of systemic therapy. There are approximately 700 new cases of pediatric and young adult R/R ALL annually in the United States. New cases of R/R DLBCL are approximately 7000 annually in the United States.

CARs can be engineered to recognize virtually any cell surface antigen and can be expressed in a variety of immune cells, suggesting that product development will result in many modular CAR units with vast application versatility. Many different antigen and cell type combinations are already currently in development to address several cancers, such as CAR T cells for pancreatic cancer (NCT03323944).

The first step in creating these personalized genemodified cell therapies is collecting patient lymphocytes via leukapheresis at a clinic or infusion center. Lymphocytes include T cells, B cells, and natural killer cells. The leukapheresis process lasts up to 4 hours and must be coordinated with the patients continuing care regimen to ensure sufficient T cells. The lymphocytes are cryopreserved and immediately shipped to a centralized manufacturing facility.

T cells are separated from the other cells in the leukapheresis product and genetically manipulated, typically using a lentiviral gene delivery method to carry DNA encoding the CAR protein, resulting in CAR T cells. The CAR T cells are cultured to a patient-specific appropriate dose. As this process is finishing, the manufacturer coordinates with the patients health care team to ensure the patient and team are prepared to infuse the CAR T cells. The manufacturing process from the apheresis process to the clinical CAR-T cell product varies widely from patient to patient, from 14 days up to a few months. The limiting step is typically reaching the appropriate CAR-T cell dose.

About a week before the scheduled CAR-T cell infusion, the patient receives multiple days of low-dose conditioning chemotherapy. This step serves to deplete lymphocytes before administration of the CAR T cells, improving the efficacy and persistence of therapy. The CAR T cells are then administered intravenously, and the patient is monitored for adverse events (AEs). The most common AEs with both currently approved products include cytokine release syndrome (CRS), neurological toxicity (NT), hypersensitivity reactions, serious infection, prolonged cytopenias, and hypogammaglobulinemia.3,4 Both products caution that therapy could cause hepatitis B viral reactivation.

The most severe reactions are CRS and NT, both of which can be life threatening. CRS, a common immune reaction following infusion of monoclonal antibodies and CAR T cells, is characterized by fever, nausea, chills, hypotension, tachycardia, asthenia, headache, rash, and dyspnea.5 Mild cases are easily managed, whereas severe cases require more aggressive and invasive therapy, such as mechanical ventilation and intravenous administration of tocilizumab. NT associated with CAR-T cell therapy is characterized by encephalopathy, headache, aphasia, delirium, insomnia, anxiety, tremor, dizziness, seizures, and peripheral neuropathy.

During the clinical trials of Kymriah and Yescarta, CRS and NT occurred in most patients with more than 10% experiencing severe CRS and more than 20% with severe NT.3,4 Initially, the 2 AEs appeared to be independent, but data are beginning to emerge suggesting a correlation. CRS might be a predictor of neurological events; however, neurological events do not predict CRS.6

Real-world evidence from patients treated with Kymriah, presented at the 2019 Society of Hematologic Oncology annual meeting, reported that slightly more than half experienced either of these conditions and less than 20% had severe cases.7 Two-year followup data regarding Yescarta reported severe CRS cases in 11% of patients and severe NT cases in 32%.8 Due to the high rate of occurrence and severity of CRS and NT, both Yescarta and Kymriah have restricted availability through Risk Evaluation and Mitigation Strategies.3,4 Both medications must be administered at a Risk Evaluation and Mitigation Strategiescertified health care facility with health care providers trained in the management and treatment of CRS and 2 doses of tocilizumab available for each patient before CART cell infusion.

Regardless of the potential for severe adverse events (AEs), the benefit of both Yescarta and Kymriah outweigh the risks, as they are highly effective singleadministration therapies. Despite the aggressive nature of the cancers treated with CAR T-cell therapy, meaningful clinical benefit can be achieved within 1 month. A summary of clinical trial primary response rates as well as 2-year data and published real-world data can be found in Table.

Despite differences between the 2 clinically available products, their safety and efficacy profiles in patients with R/R DLBCL are comparable. Differences between the therapies range from the molecular units comprising the CARs, manufacturing differences, lymphodepletion regimen, number of CAR T cells and volume infused, and whether the infusion and short-term patient monitoring occurs in an inpatient or outpatient setting.

Patient-specific genetically modified cell therapies can present many manufacturing challenges. One stark difference between the available therapies, relevant to the patient and clinician experience, has been the manufacturing time and failure rate. During ZUMA-1, of the 101 patients treated with Yescarta, the median time from leukapheresis to product delivery was 17 days (range, 14-51 days), and a 1% manufacture failure rate was reported.4 The ELIANA trial of Kymriah in patients with R/R ALL reported a 9% manufacture failure rate, whereas the JULIET R/R DLBCL trial reported a failure rate of 6.9%, and of the 106 patients receiving Kymriah, the median manufacture time was 113 days (range, 47-196 days).3

Commercial manufacture of Kymriah for DLBCL has struggled to meet specifications.11 While addressing the production issue, Novartis has initiated a safety study evaluating out-of-specification product (NCT04094311) and a managed-access program (NCT03601442).

Known causes of CAR-T cell therapy failure are T cell exhaustion and antigen escape. T cell exhaustion is characterized by a loss of responsive T cells due to changes in gene expression and can be prevented by immune checkpoint inhibitors PD-1, PD-L1, or CTLA- 4. Antigen escape describes a condition in which some cancer cells do not express the CAR-targeted antigen; therefore, they escape immune activation and survive within the patient. Engineering a secondary CAR to a different antigen, such as CD22 in the case of ALL, increases the likelihood of targeting all malignant cells. Solutions to both of these inhibitory mechanisms are currently under clinical trial investigation.12,13

The therapeutic success of CAR T cells ensures gene-modified immune cell therapy will be refined, optimized, and broadly applied until limits are reached. Many clinical groups are investigating biomarkers associated with severe AEs to provide an additional layer of precision care to the CAR-T cell therapy model.6,14 In addition, clinical trials are underway evaluating combination therapies to enhance the efficacy and improve the safety of CART cell therapy. Early-stage research is evaluating the possibility of off-the-shelf CAR-T cell therapy, not a patient-unique manufactured product, to reduce the time to treatment and achieve manufacturing efficiencies and consistencies.15,16 Gene-modified cell therapy, such as CAR T-cell therapy, is revolutionizing oncology, and this living drug model is breathing life into the hopes of patients with cancer and caregivers.

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CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells - Pharmacy Times

IPS Cell Therapy Comments Off on CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells – Pharmacy Times | February 25th, 2020

Dublin, Feb. 17, 2020 (GLOBE NEWSWIRE) -- The "Stem Cell Therapy Contract Manufacturing Market, 2019-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study on contract service providers engaged in the development and manufacturing of stem cell therapies. The study features in-depth analyses, highlighting the capabilities of various stem cell therapy CMOs

Advances in the fields of cell biology and regenerative medicine have led to the development of a variety of stem cell-based therapies for many cardiovascular, oncological, metabolic and musculoskeletal disorders. Driven by the revenues generated from stem cell therapies, the regenerative medicine market is anticipated to generate revenues worth USD 100 billion by 2030.

With a promising pipeline of over 200 stem cell therapy candidates, it has become essential for developers to scale up the production of such therapeutic interventions. Given that stem cell therapy manufacturing requires highly regulated, state-of-the-art technologies, it is difficult for stakeholders to establish in-house expertise for large-scale manufacturing of stem cell therapies.

As a result, stem cell therapy developers have begun outsourcing their manufacturing operations to contract manufacturing organizations (CMOs). Specifically, small and mid-sized players in this sector tend to outsource a substantial proportion of clinical and commercial-scale manufacturing processes to contract service providers. In addition, even big pharma players, with established in-house capabilities, are gradually entering into long-term business relationships with CMOs in order to optimize resource utilization and manage costs.

According to a recent Nice Insight CDMO survey, about 55% of 700 respondents claimed to have collaborated with a contract service provider for clinical and commercial-scale product development requirements. Considering the prevalent trends, we believe that the stem cell therapy manufacturing market is poised to grow at a steady pace, driven by a robust pipeline of therapy candidates and technological advances aimed at mitigating challenges posed by conventional methods of production. Amidst tough competition, the availability of cutting-edge tools and technologies has emerged as a differentiating factor and is likely to grant a competitive advantage to certain CMOs over other players in the industry.

One of the key objectives of the report was to estimate the future size of the market. Based on parameters, such as increase in number of clinical studies, target patient population, anticipated adoption of stem cell therapies and expected variation in manufacturing costs, we have provided an informed estimate of the likely evolution of the market in the mid to long term, for the period 2019-2030.

Amongst other elements, the report includes:

In order to provide a detailed future outlook, our projections have been segmented on the basis of:

Key Topics Covered

1. Preface

2. Executive Summary

3. Introduction

4. Market Overview

5. Regulatory Landscape

6. Stem Cell Therapy Contract Manufacturers in North America

7. Stem Cell Therapy Contract Manufacturers in Europe and Asia-Pacific

8. Partnerships and Collaboration

9. Contract Manufacturing Opportunity Assessment

10. Capacity Analysis

11. Demand Analysis

12. Market Forecast

13. Key Performance Indicators

14. Concluding Remark

15. Executive Insights

16. Appendix 1: Tabulated Data

17. Appendix 2: List of Companies and Organizations

For more information about this report visit https://www.researchandmarkets.com/r/rktm8d

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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Stem Cell Therapy Contract Manufacturing Industry, 2019-2030 - Availability of Cutting-Edge Tools & Technologies has Emerged as a Differentiating...

IPS Cell Therapy Comments Off on Stem Cell Therapy Contract Manufacturing Industry, 2019-2030 – Availability of Cutting-Edge Tools & Technologies has Emerged as a Differentiating… | February 17th, 2020

The comprehensive report published by Persistence Market Research offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the Stem Cell-Derived Cells Market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the Stem Cell-Derived Cells Market during the forecast period 2019 2029.

As per the findings of the presented study, the Stem Cell-Derived Cells Market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the Stem Cell-Derived Cells in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/28780

The report segregates the Stem Cell-Derived Cells Market into different segments to provide a detailed understanding of the various aspects of the market. The competitive analysis of the Stem Cell-Derived Cells Market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the Stem Cell-Derived Cells Market.

Key findings of the report:

The report aims to eliminate the following doubts related to the Stem Cell-Derived Cells Market:

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key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on:

Regional analysis includes

Report Highlights:

In order to get a strategic overview of the market,Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/28780

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Tags: Stem CStem Cell-Derived Cells MarketStem Cell-Derived Cells Market DynamicsStem Cell-Derived Cells Market GrowthStem Cell-Derived Cells Market KeyplayersStem Cell-Derived Cells Market Trends

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Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -...

IPS Cell Therapy Comments Off on Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -… | February 17th, 2020

News Release

Monday, December 16, 2019

NEI-led study to test safety of treatment for a form of age-related macular degeneration that currently lacks treatment.

Researchers at the National Eye Institute (NEI) are launching a clinical trial to test the safety of a novel patient-specific stem cell-based therapy to treat geographic atrophy, the advanced dry form of age-related macular degeneration (AMD), a leading cause of vision loss among people age 65 and older. The geographic atrophy form of AMD currently has no treatment.

The protocol, which prevented blindness in animal models, is the first clinical trial in the U.S. to use replacement tissues from patient-derived induced pluripotent stem cells (iPSC), said Kapil Bharti, Ph.D., a senior investigator and head of the NEI Ocular and Stem Cell Translational Research Section. The NEI is part of the National Institutes of Health.

The therapy involves taking a patients blood cells and, in a lab, converting them into iPS cells, which have the potential to form any type of cell in the body. The iPS cells are programmed to become retinal pigment epithelial (RPE) cells, the type of cell that dies early in the geographic atrophy stage of macular degeneration. RPE cells nurture photoreceptors, the light-sensing cells in the retina. In geographic atrophy, once RPE cells die, photoreceptors eventually also die, resulting in blindness. The therapy is an attempt to shore up the health of remaining photoreceptors by replacing dying RPE with iPSC-derived RPE.

Before they are transplanted, the iPSC-derived RPE are grown in sheets one cell thick, replicating their natural structure within the eye. This monolayer of iPSC-derived RPE is grown on a biodegradable scaffold designed to promote the integration of the cells within the retina. Surgeons position the patch between the RPE and the photoreceptors using a surgical tool designed specifically for that purpose.

Under the phase I/IIa clinical trial protocol 12 patients with advanced-stage geographic atrophy will receive the iPSC-derived RPE implant in one of their eyes and be closely monitored for a period of at least one year to confirm safety.

A concern with any stem cell-based therapy is its oncogenic potential: the ability for cells to multiply uncontrollably and form tumors. In animal models, the researchers genetically analyzed the iPSC-derived RPE cells and found no mutations linked to potential tumor growth.

Furthermore, the use of an individuals autologous (own) blood cells is expected to minimize the risk of the body rejecting the implant.

Should early safety be confirmed, later study phases will include more patients to assess the efficacy of the implant to prevent blindness and restore vision in patients with geographic atrophy.

A Food and Drug Administration (FDA) requirement for moving forward with the clinical trial was the establishment of good manufacturing practice (GMP) protocols to ensure that the iPSC-derived RPE are a clinical-grade product. GMP protocols are key for making the therapy reproducible and for scaling up production should the therapy receive FDA approval.

The preclinical research for the trial was supported by the NEI Intramural Research Program and by an NIH Common Fund Therapeutic Challenge Award. The trial is being conducted at the NIH Clinical Center in Bethesda, MD.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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NIH launches first U.S. clinical trial of patient-derived ...

IPS Cell Therapy Comments Off on NIH launches first U.S. clinical trial of patient-derived … | February 4th, 2020

I get asked many questions about stem cell therapies, but one of the most common over the years has been about the stem cell treatment cost. For instance, a reporter might ask, How much does a stem cell treatment for MS cost? and a patient might ask me, How much is a fair cost for a stem cell therapy for arthritis? Or, patients will voluntarily tell me what they paid or mention it in the comments. We hear various numbers thrown around about costs so I decided to do a poll on this. I even did an early update on the results of this poll, voicing my skepticism that the costs paid were worth it.

But the poll has gotten well over 500 responses now so I thought I would revisit it and what it might mean.

You can see a screenshot of the images. Its fair to say, as much as Internet polls arent considered particularly accurate, that this one largely fits with what is reported out in the field.

(On a side note, I wish there was such a thing as going out into the field for stem cell scientists as Ive always been a bit jealous of scientists who really do go out in the field. What do we do, go out in the wild and catch wild or feral stem cells in the bush?)

Patients self-reported most often paying between $2,500 and $7,500 for their stem cell therapy so if we take the average of those we get that $5,000 figure that is what I hear most often from others. Yes, not necessarily very rigorous, but the result makes good sense. Not far behind though were responses in the $7,500-20,000 range.

About 1 in 10 respondents reported paying $20,000 or more, including some beyond $100,000. Thats a whopping stem cell treatment cost, especially for something most often unproven and unapproved by the FDA.

If we consider these responses, the average cost may be more like $7,500-$10,000.

Notably, about 1/16 respondents indicated their stem cells were free. Im not sure what that means in terms of how that came to be.

Interestingly, most respondents who also went on to answer a 2nd poll in that post about where they got the treatment indicate it was at a stem cell clinic (scroll down in that Oct. 2017 post and youll see the 2nd poll). This 2nd poll has about 200 responses.

So today buying a simple stem cell treatment, most often unproven and non-FDA approved, is often not so different in cost than buying a 10-year old used car, while less often it is similar to buy various new cars including at the high end of stem cell therapy cost, some very expensive new cars. This cost and the risks involved are why I have suggested to patients in the past to be assertive when considering a stem cell treatment, ask questions, dont just accept too good to be true kinds of answers, etc. In short, be at least (or ideally much more) rigorous about unproven stem cell treatments as you are about buying a car.

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Update on stem cell treatment cost for 2018 from ongoing ...

IPS Cell Therapy Comments Off on Update on stem cell treatment cost for 2018 from ongoing … | February 4th, 2020

A team based at Osaka University has conducted the first ever transplant of cardiac muscle cells generated from iPS cells, around the globe, in a clinical trial initiated physician.

A professor in the universitys cardiovascular surgery unit, Yoshiki Sawa, together with his colleagues at the university, in a clinical procedure to verify the efficiency and security of the therapy with the help of induced pluripotent stem cells, intend to transplant heart muscle cell sheets over the time of 3 years into 10 individuals undergoing severe heart malfunction a result of ischemic cardiomyopathy.

The team conducted, the present month, an operation on an individual, in an attempt to take a first step into the project. This operation was successful. The individual has now been moved to a general ward.

It is predicted that the cells on the degradable sheets which attach to the hearts surface will grow and eliminate a protein which has the power to regenerate blood vessels and advance cardiac function. Already, the iPS cells have been stored after being taken from the blood cells of healthy donors.

Every sheet that goes on the hearts surface is 4 to 5 centimeters in width, and 0.1 millimeter in thickness.

The team from Osaka University will be observing the patient throughout the year.

At a news conference, Yoshiki Sawa expressed his hopes of the transplant becoming the medical technology that succeeds in saving as many individuals as it can, since he has come across many lives that he was unable to save.

On Monday, the universitys researchers stated how they chose to carry out a clinical trial in a clinical researchs stead so they could gain timely approval from the health ministry for clinical applications.

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Team based at Osaka University carries out world's first transplant of heart cells generated from iPS cells - Medical Herald

IPS Cell Therapy Comments Off on Team based at Osaka University carries out world’s first transplant of heart cells generated from iPS cells – Medical Herald | January 31st, 2020

A team based at Osaka University stated how it had succeeded in carrying out the first transplant of cardiac muscle cells, around the globe, developed from iPS cells in a clinical trial which as physician-initiated.

A professor in Osaka Universitys cardiovascular surgery unit, Yoshiki Sawa, along with his colleagues at the university, intend to transplant heart muscle cell sheets into 10 individuals experiencing severe heart malfunction as a result of ischemic cardiomyopathy, in a clinical trial, to validate the safety and the effectiveness of the therapy with the use of induced pluripotent stem cells.

On the surface of the hearts of the partaking individuals, the cells on the degradable sheets are attached. It is predicted that these cells will develop to release a protein that can allow for the regeneration of blood vessels as well as the improvement of the cardiac function.

Already, the iPS cells have been taken, and then stored, from the blood cells donated by healthy individuals

On Monday, the researchers stated how they chose to carry out a clinical trial in a clinical researchs stead as they had hoped to attain, as early as possible, authorization from the health ministry for clinical applications.

There are severe evaluating risks involved in the clinical trial. These may include the possibility of cancer as well as the efficacy of transplanting many million cells per patient, which may consist of tumor cells.

In Japan, this will be marked as the second clinical trial based on iPS. The first clinical trial of such kind was carried out on patients suffering from eye-linked ailments. This was done so by the Riken research institute.

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Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe - Medical Herald

IPS Cell Therapy Comments Off on Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe – Medical Herald | January 30th, 2020

OSAKA An Osaka University team said it has carried out the worlds first transplant of cardiac muscle cells created from iPS cells in a physician-initiated clinical trial.

In the clinical project to verify the safety and efficacy of the therapy using induced pluripotent stem cells, Yoshiki Sawa, a professor in the universitys cardiovascular surgery unit, and colleagues aim to transplant heart muscle cell sheets into 10 patients suffering from serious heart malfunction caused by ischemic cardiomyopathy.

The cells on the degradable sheets attached to the surface of the patients hearts are expected to grow to secrete a protein that can regenerate blood vessels and improve cardiac function. The iPS cells have already been derived from healthy donors blood cells and stored.

The researchers said Monday they decided to conduct a clinical trial instead of a clinical study in hopes of obtaining approval from the health ministry for clinical applications as soon as possible.

The trial involves stringently evaluating risks, particularly cancer possibilities, and the efficacy of transplanting some 100 million cells per patient that may include tumor cells.

This is the second iPS cell-based clinical trial in Japan. The first was conducted on eye disease patients by the Riken research institute.

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Osaka University transplants iPS cell-based heart cells in world's first clinical trial - The Japan Times

IPS Cell Therapy Comments Off on Osaka University transplants iPS cell-based heart cells in world’s first clinical trial – The Japan Times | January 27th, 2020

KYOTO An expert panel of the health ministry on Friday approved a clinical research program proposed by a Kyoto University team to transplant cartilage made from induced pluripotent stem (iPS) cells to damaged knee joints.

Professor Noriyuki Tsumaki and other members of the team are planning to create cartilage with a diameter of 2 to 3 millimeters using iPS cells stored at the universitys Center for iPS Cell Research and Application (CiRA).

The team aims to carry out the first transplant this year. After a clinical trial by Asahi Kasei Corp., which supports the project, it hopes to put the technology into practical use in 2029.

Four people between the ages of 20 and 70 will undergo transplant operations using iPS cell-derived cartilage for their damaged knee joints, with the area of damage ranging from 1 centimeter to 5 centimeters. The team does not plan to seek additional patients for the program.

The team will monitor the four patients for one year after the operations to keep an eye out for possible development of tumors. If the operations succeed, the transplanted material will fuse with existing cartilage.

There are many patients experiencing inconvenience due to damaged cartilage, Tsumaki told a news conference at the Kyoto University Hospital on Friday. Well work hard so that we can offer therapy methods.

The team will also aim to apply the therapy to patients with osteoarthritis.

In 2014, Riken, a Japanese government-affiliated research institute, transplanted retina cells made from iPS cells as a treatment for an incurable eye disease, in the worlds first transplant of iPS-derived cells.

Later, similar transplant operations were conducted by Kyoto University for Parkinsons disease and by Osaka University for corneal disease.

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Kyoto University team gets OK from ministry for plan to transplant iPS-derived cartilage into knee joints - The Japan Times

IPS Cell Therapy Comments Off on Kyoto University team gets OK from ministry for plan to transplant iPS-derived cartilage into knee joints – The Japan Times | January 25th, 2020

KYOTO A panel of experts from the Ministry of Health approved a clinical research program proposed by a team from the University of Kyoto on Friday for the transplantation of cartilage from induced pluripotent stem cells [iPS] into damaged knee joints.

Professor Noriyuki Tsumaki and other members of the team are planning to produce 2 to 3 millimeter diameter cartilage using iPS cells, which will be stored at the Universitys Center for iPS Cell Research and Application [CiRA].

The team plans to perform the first transplant this year. According to a clinical study by Asahi Kasei Corp., which supports the project, the technology should be put into practice in 2029.

Four people between the ages of 20 and 70 are transplanted with iPS cell cartilage for their damaged knee joints, with the damage range between 1 cm and 5 cm. The team does not plan to seek additional patients for the program.

Immunosuppressors are not used in the transplant because cartilage usually does not show an immune response.

The team will monitor the four patients for possible tumor development for a year after the operation. If the operations are successful, the transplanted material melts into the existing cartilage.

There are many patients who experience discomfort from cartilage damage, said Tsumaki at a press conference at Kyoto University Hospital on Friday. We will work hard to offer therapy methods.

The team will also try to apply the therapy to patients with osteoarthritis.

In 2014, Riken, a research institute affiliated with the Japanese government, transplanted retina cells made from iPS cells to treat an incurable eye disease in the worlds first transplant of iPS-derived cells.

Similar transplants were later performed by Kyoto University for Parkinsons and Osaka University for corneal diseases.

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The Kyoto University team's plan to transplant iPS cartilage into knee joints is OK - gotech daily

IPS Cell Therapy Comments Off on The Kyoto University team’s plan to transplant iPS cartilage into knee joints is OK – gotech daily | January 25th, 2020

The Indian Psychiatric Society (IPS) the professional body that represents psychiatrists in India, strongly condemned the use of stem cell therapy in psychiatric disorders, particularly autism, until such a time that research evidence substantiated its effectiveness.

IPS, in its position statement on stem cell therapy on January 17, said that till now, there is no scientifically validated and scrutinized research evidence that proves that stem cells are helpful in any psychiatric disorders including autism.

Autism is a complex neurodevelopmental disorder with no known single cause.

The advisory from the IPS comes at a time when stem cell therapy clinics that claim to have developed stem cell therapies to treat complex psychiatric problems such as autism, cerebral palsy (movement disorder), muscular dystrophy (weakness of muscles), mental retardation, spinal cord injury and brain stroke have mushroomed across the country.

These stem cell therapy centres extract stem cells from the bone marrow of each child and then inject it into the childs spinal canal. The whole procedure takes place under general anaesthesia.

These clinics use aggressive marketing techniques and false claims to lure parents of children who are suffering from disease like autism.

The Indian Council of Medical Research (ICMR) has already published guidelines that cover the various diseases that are applicable for stem cell treatment. No psychiatric disorders, including autism, are listed there under this advisory.

Stem cells are special human cells that have the ability to develop into many different cell types, from muscle cells to brain cells. In some cases, they also have the potential to repair damaged tissues, and provide a cure for various diseases. But the clinical evidence at this point is low.

Psychiatric disorders including autism are combined derangements of both neurodevelopmental and neurodegenerative trajectories of brain and are polygenetic in origin. So they actually are symptomatic manifestations of a variety of different pathogenetic processes about which scientific evidence is as yet inconclusive, IPS said.

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Psychiatric body condemns use of stem cell therapies to treat psychiatric disorders - Moneycontrol.com

IPS Cell Therapy Comments Off on Psychiatric body condemns use of stem cell therapies to treat psychiatric disorders – Moneycontrol.com | January 20th, 2020

SAN DIEGO--(BUSINESS WIRE)--Allele Biotechnology and Pharmaceuticals, Inc. (President and CEO: Jiwu Wang, Ph.D., Allele), a San Diego-based private company, and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., Astellas), through its Massachusetts-based subsidiary Astellas Institute for Regenerative Medicine (AIRM), entered into a licensing agreement to expand Astellas access to Alleles induced pluripotent stem cell (iPSC) technologies for various cell therapy programs.

Astellas, one of the largest pharmaceutical companies in Japan and already a leader in the development of cell-based therapeutics, has further dedicated to development of the field through its commitment to state-of-the-art iPS cell generation, modification, and manufacturing. iPSC lines can differentiate into all somatic tissue types, enabling a wide variety of therapeutic applications. The field of iPSC-derived cells has seen dramatic growth in clinical trials recently--the majority of the ~12 clinical trials around the world were initiated within the last 18 months and many more are upcoming.

Allele has been developing its core strength in reprogramming somatic cells into iPSCs with granted patents and the first commercial cGMP system it developed over the past 10 years. Allele also engages in more than a dozen different human tissue derivation activities through its own R&D efforts for internal programs and partnerships. To realize the unparalleled potential of iPSC, Alleles researchers and cGMP team are committed to setting up and validating cell assays for product quality control, genome analysis pipelines, closed-system automation for reprogramming, and machine learning in iPSC-related fields.

Under the terms of the new license agreement, Astellas will pay Allele upfront and milestones, product-based royalties, and potentially manufacture fees.

About AlleleAllele Biotechnology and Pharmaceuticals was founded in 1999. In 2015, the company completed an 18,000 square foot state-of-the-art facility in San Diego for the production of GMP-grade human iPSC lines. The facility also supports the production of tissue-specific cells differentiated from these iPSCs, including pancreatic beta cells, neural progenitor cells, and cardiomyocytes.

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Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines - Business Wire

IPS Cell Therapy Comments Off on Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines – Business Wire | January 19th, 2020

Otology sponges are cotton balls used after otology surgery. They are placed in the ear to hold the skin and eardrum in place after otology surgery. After otology surgery, the ear canal is packed with antibiotic ointment and otology sponges. Myringotomy with the insertion of tympanostomy tubes is the most common ontology surgical procedure in the U.S., and approximately 2 million procedures conducted each year. The field of otology has witnessed remarkable advancements in the management of complex ailments, such as hearing disorders, through the ongoing progress of sophisticated intricate and microscopic surgeries.

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Most common causes of surgeries are the retraction of the tympanic membrane, chronic otitis media and collapsed eustachian tube. Otology sponges are sterile devices used post-surgery for 6 weeks or for a month. Otology surgeries are mostly performed in outpatient systems and they do not require overnight stay. Since patients can go outdoor immediately after surgery, the chances of wound infection increase.

To prevent infection, these sponges are placed in ear canal with the lubrication of antibiotics. Sometimes, an incision is made behind the year to operate the internal canal. In this situation, sterile dressings along with antibiotic lubricants are placed over the stiches to prevent microbial infection. Owing to the shape of the ear, there is very less pace to operate inside it, owing to which otology surgeries are performed with the help of microscopes for greater accuracy and success. Increase in the number of ENT specialists, coupled with the availability of technologically sound surgical methods, is boosting the number of otology surgeries.

Otology Sponges Market: Drivers and Restraints

An increase in the number of otology surgeries due to the availability of advanced surgical methods is expected to drive the market. Advanced methods of otology surgeries have spread significantly in the developing world, which is also contributing to the growth of the market. Ease of use due to flexibility and the compressed configuration of these sponges is also driving the market.

Otology sponges are sterile and available in different sizes, hence, they are effective in preventing ear canal infections. Increase in awareness about the availability of otology surgical treatment is contributing to market growth. The effectiveness of otology sponges in preventing ear canal infections and holding the shape of the eardrum is driving the market. However, low awareness among the general public about their usage is restraining market growth.

Otology Sponges Market: Segmentation

The global otology sponges market can be segmented on the basis of material, end user type and geography.

Based on material type, the otology sponges market is segmented as:

Based on end use, the otology sponges market is segmented as:

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Otology Sponges Market: Overview

The global otology sponges market is expected to grow steadily owing to an increase in the number of otology surgeries. Advanced technological intervention for otology surgeries is also boosting the otology sponges market. By material type, the otology sponges market is expected to be dominated by latex-free otology sponges. By end users, the otology sponges market is expected to be dominated by ENT clinics owing to an increase in the number of outpatient surgeries. The widespread availability of otology sponges in different sizes makes them easy to use and one can wear them comfortably. Moreover, the number of otology surgical procedures has increased in developing countries as well, which is boosting the market in these countries.

Otology Sponges Market: Regional Outlook

The global otology sponges market is majorly dominated by North America owing to a significant number of otology surgical procedures in the region. Europe is the second most lucrative market owing to the availability of advanced otology surgical methods. Asia Pacific is expected to emerge as one of the most lucrative otology sponges markets owing to an increase in awareness about otology surgical treatments. Emerging economies, such as China and India, are potential markets for otology sponges because of their large population base. Latin America is also a lucrative market owing to the higher adoption of otology sponges. However, the Middle East and Africa is the least lucrative otology sponges market due to lack of awareness and the low availability of advanced otology surgical methods.

Otology Sponges Market: Key Players

Some of the global key players operating in otology sponges market areDeRoyal Industries, Inc.; Boston Medical Products, Inc.; Summit Medical, Inc.; American Surgical Company LLC; Medtronic and Olympus Corporation.

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends, macro-economic indicators and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments and geographies.

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Cell Therapy And Tissue Engineering Market Size 2020 by Top Leading Companies- BioCardia Betalin Therapeutics, MEDIPOST Co., MaxCyte BioReliance...

IPS Cell Therapy Comments Off on Cell Therapy And Tissue Engineering Market Size 2020 by Top Leading Companies- BioCardia Betalin Therapeutics, MEDIPOST Co., MaxCyte BioReliance… | January 16th, 2020

SAN DIEGO--(BUSINESS WIRE)-- Allele Biotechnology and Pharmaceuticals, Inc. (President and CEO: Jiwu Wang, Ph.D., Allele), a San Diego-based private company, and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., Astellas), through its Massachusetts-based subsidiary Astellas Institute for Regenerative Medicine (AIRM), entered into a licensing agreement to expand Astellas access to Alleles induced pluripotent stem cell (iPSC) technologies for various cell therapy programs.

Astellas, one of the largest pharmaceutical companies in Japan and already a leader in the development of cell-based therapeutics, has further dedicated to development of the field through its commitment to state-of-the-art iPS cell generation, modification, and manufacturing. iPSC lines can differentiate into all somatic tissue types, enabling a wide variety of therapeutic applications. The field of iPSC-derived cells has seen dramatic growth in clinical trials recently--the majority of the ~12 clinical trials around the world were initiated within the last 18 months and many more are upcoming.

Allele has been developing its core strength in reprogramming somatic cells into iPSCs with granted patents and the first commercial cGMP system it developed over the past 10 years. Allele also engages in more than a dozen different human tissue derivation activities through its own R&D efforts for internal programs and partnerships. To realize the unparalleled potential of iPSC, Alleles researchers and cGMP team are committed to setting up and validating cell assays for product quality control, genome analysis pipelines, closed-system automation for reprogramming, and machine learning in iPSC-related fields.

Under the terms of the new license agreement, Astellas will pay Allele upfront and milestones, product-based royalties, and potentially manufacture fees.

About Allele Allele Biotechnology and Pharmaceuticals was founded in 1999. In 2015, the company completed an 18,000 square foot state-of-the-art facility in San Diego for the production of GMP-grade human iPSC lines. The facility also supports the production of tissue-specific cells differentiated from these iPSCs, including pancreatic beta cells, neural progenitor cells, and cardiomyocytes.

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Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines - BioSpace

IPS Cell Therapy Comments Off on Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines – BioSpace | January 16th, 2020

Every year, BioSpace analyzes the biotech industry, looking for the hot new biotech startups to watch. We then produce the NextGen Bio Class of, twenty companies ranked based on several categories, including Finance, Collaborations, Pipeline, and Innovation. The companies were typically launched no more than 18 months before the list was created.

We thought it would be insightful to look back at our previous lists to see where some of those companies are today. Heres a look at the top three companies from the Top 20 Life Science Startups to Watch in 2018.

#1. BlueRock Therapeutics. Founded in 2016, BlueRock was #1 on our list of companies to watch in 2018. With facilities in Ontario, Canada; Cambridge, Massachusetts; and New York, New York, BlueRock launched in December 2016 with a $225 million Series A financing led by Bayer AG and Versant Ventures. The company focuses on cell therapies to regenerate heart muscle in patients who have had a heart attack or chronic heart failure, as well as therapies for patients with Parkinsons disease.

In October 2017, BlueRock and Seattle-based Universal Cells entered into a collaboration and license deal to create induced pluripotent stem (iPS) cell lines that can be used in the manufacture of allogeneic cellular therapies. Shortly afterwards, the company established its corporate headquarters in Cambridge, and in April 2018, established a research-and-development hub in New York City, as well as formalizing a sponsored research collaboration with the Center for Stem Cell Biology at Memorial Sloan Kettering (MSK) Cancer Center. The collaboration focuses on translating Ketterings expertise in creating multiple types of authentic neural cells from stem cells to address diseases of the central and peripheral nervous system. BlueRock also received $1 million from the State of New York and Empire State Development under its economic development initiatives program.

In April 2019, BlueRock partnered with Editas Medicine (which was on BioSpaces NextGen Bio Class of 2015 list) to combine their genome editing and cell therapy technologies to focus on novel engineered cell medicines. Part of the deal was to collaborate on creating novel, allogeneic pluripotent cell lines using a combination of Editas CRISPR genome editing technology and BlueRocks iPSC platform.

And finally, in August 2019, Bayer AG acquired BlueRock for the remaining stake in the company for about $240 million in cash and an additional $360 million in pre-defined development milestones.

#2. Prelude Fertility. Prelude Fertility is a bit of an outlier from the typical BioSpace NextGen company, because it isnt quite a biopharma company. It is a life sciences company whose business model is aimed at in vitro fertilization and egg freezing. It was founded with a $200 million investment by entrepreneur Martin Varsavsky. The investment was in the largest in vitro fertilization clinic in the Southeast, Reproductive Biology Associates of Atlanta, and its affiliate, My Egg Bank, the largest frozen donor egg bank in the U.S.

Since then it has expanded in various parts of the country, including adding San Francisco-based Pacific Fertility Center (PFC) to its network in September 25, 2017; partnering with Houston Fertility Institute and acquiring Vivere Health; partnering with the Advanced Fertility Center of Chicago; and in October 2018, partnered with NYU Langone Health.

In March 2019, Prelude merged with Inception Fertility to establish the Prelude Network as the fastest-growing network of fertility clinics and largest provider of comprehensive fertility services in the U.S. Inception is acting as the parent company, with the Prelude Network, both having board representatives from the previous organizations.

#3. Relay Therapeutics. Ranking #3 on our list for 2018, Relay Therapeutics launched in September 2016 with a $57 million Series A financing led by Third Rock Ventures with participation form D.E. Shaw Research. On December 14, 2017, it closed on a Series B round worth $63 million, led by BVF Partners, with new investors GV (formerly Google Ventures), Casdin Capital, EcoR1 Capital and Section 32.

The company focuses on the relationship between protein motion and function. It merges computational power with structural biology, biophysics, chemistry and biology. In December 2018, the company completed a $400 million Series C financing. It was led by the SoftBank Vision fund and included additional new investors, Foresite Capital, Perceptive Advisors and Tavistock Group. Existing investors also participated.

The company announced at the time it planned to use the funds to accelerate the implementation of its long-term strategy, expanding its discovery efforts, advancing existing programs into the clinic and improving its platform.

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Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 - BioSpace

IPS Cell Therapy Comments Off on Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 – BioSpace | January 7th, 2020