Longeveron Announces Japanese Approval of Clinical Trial for Treatment of Aging Frailty With Longeveron’s Stem Cells | DNA RNA and Cells | News…

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The Phase 2 study will assess the safety and efficacy of Longeveron's stem cell treatment under Japan's accelerated regulatory pathway for regenerative medicine.

MIAMI, FL, USA I June 1, 2020 I Longeveron LLC announced today that Japan's Pharmaceutical and Medical Devices Agency (PMDA) (the Japanese agency akin to the United States' Food & Drug Administration) approved a Clinical Trial Notification (CTN) application (akin to an Investigational New Drug Application or "IND" in the US regulatory system), approving the initiation of a Phase 2 clinical trial evaluating the safety and efficacy of Longeveron's Mesenchymal Stem Cells (LMSCs) for the treatment of Aging Frailty in Japanese patients. This is another key milestone for Longeveron's Aging Frailty program, which includes two ongoing Phase 2 clinical trials in the U.S.

"We are extremely pleased to achieve this significant milestone," said Geoff Green, President of Longeveron."This study is designed to determine whether the transplant of donor-derived mesenchymal stem cells can improve healthspan in mild to moderately frail patients, thereby improving functionality and potentially lowering their risk of disability, and dependence on others for care."

Aging Frailty is a common, but reversible, life-threatening geriatric condition affecting millions of Japanese over the age of 65.Frail individuals are vulnerable to adverse health outcomes compared to their age-matched peers despite sharing similar comorbidities and demographics.Clinically, frailty manifests as a combination of symptoms that may include loss of muscle and decreased strength, slowed walking (sarcopenia), lower activity and energy levels, poor endurance, nutritional deficiencies, weight loss and fatigue.Collectively, these lead to overall decline in functionality, and increased risk of disability, dependency, and death.

"The biology of frailty is complex, and includes diminished stem cell activity, reduced ability to repair and regenerate tissue, and immunosenescence (deterioration of the immune system) and chronic systemic inflammation," said Dr. Anthony Oliva, Senior Scientist at Longeveron. "LMSCs have multiple mechanisms of action that can potentially address all of these issues, and thus make them extremely attractive as a therapeutic candidate for the unmet medical need of Aging Frailty."

The planned study is an investigator-initiated, randomized, double-blind, placebo-controlled design,and will be conducted at Juntendo University Hospital (Tokyo) and Japan's National Center for Geriatrics and Gerontology (NCGG) in Nagoya.The study's Principal Investigator, Dr. Hidenori Arai, President of the NCGG, commented that "Japan has one of the oldest and fastest aging societies in the world, with nearly 30% of Japan's citizens over the age of 65.Preventing and reversing functional decline associated with frailty is one of the nation's top priorities, and Longeveron's regenerative medicine approach is an exciting and innovative potential therapeutic option.With the disproportionate infection and mortality rate of older people with COVID-19 and Influenza infection, it is critically important to rapidly test treatments that may be effective."

In Japan, the "Pharmaceutical and Medical Device Act" and the "Act on the Safety of Regenerative Medicine" came into effect in 2014. Under this system, a "Time-limited Conditional Approval" option exists, which allows a manufacturer to conditionally sell regenerative medicine products while proceeding with its Phase 3 clinical trial.

Longeveron's Aging Frailty Research Program

Longeveron sponsors the most extensive and advanced Aging Frailty clinical research program in the world, with more than 200 patients treated with LMSCs worldwide.In the U.S., two clinical trials are currently ongoing:

About LMSCs

Longeveron Allogeneic Mesenchymal Stem Cells (LMSCs) is a regenerative medicine product sourced from the bone marrow of young healthy adult donors.LMSCs are culture expanded under the FDA's current good manufacturing practices (cGMP) to high standards, and maintained as individual "off-the-shelf" doses.

About Longeveron LLC

Longeveron (www.longeveron.com) is a regenerative medicine therapy company founded in 2014. Longeveron's mission is to provide biological solutions for aging-related diseases and life-threatening conditions, and is dedicated to developing safe and effective cell-based therapeutics for unmet medical needs such as Aging Frailty, the Metabolic Syndrome, Alzheimer's Disease, Acute Respiratory Distress Syndrome (ARDS) from COVID-19 infection, and congenital heart defects in children (hypoplastic left heart syndrome).

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CSL Behring and Seattle Children’s Research Institute to Advance Gene Therapy Treatments for Primary Immunodeficiency Diseases – P&T Community

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

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

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

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

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

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

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

About Seattle Children's

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

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

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

About CSL Behring

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

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Exceptional stem cell science on tap for ISSCR 2020 Virtual June 23-27, 2020 – 7thSpace Interactive

Exceptional stem cell science on tap for ISSCR 2020 Virtual June 23-27, 2020

Skokie, IL - The International Society for Stem Cell Research has transformed its annual scientific meeting into a virtual experience, bringing the global stem cell community together to share knowledge, collaborate, and network. Attendee registration and complementary media registration are open!

The ISSCR 2020 Virtual Meeting, cosponsored by the Harvard Stem Cell Institute (HSCI), will deliver a comprehensive scientific education program that includes plenaries featuring world-renowned scientists in the field. Following are some highlights of the program:

New Session: ISSCR Response to COVID-19 Featuring Opening Address by Dr. Anthony Fauci, Director, National Institute for Allergy and Infectious Diseases, USA

The COVID 19 pandemic has mobilized the global scientific and clinical communities from across disciplines. This collective effort is essential to understand the biology of the novel coronavirus infection process, the resulting clinical pathology and develop potential treatments, including a vaccine. In this session, the ISSCR brings together leading scientists to discuss the latest research and clinical findings in this rapidly moving field. Speakers to be announced soon!

Clinical Innovation and Gene Editing Sponsored by BlueRock Therapeutics

Patient Advocate Address: Matthew Might, University of Alabama at Birmingham, USA

John McNeish Lecture: Brian Wainger, Harvard University, USA Katherine High, Spark Therapeutics, USA Michel Sadelain, Memorial Sloan-Kettering Cancer Center, USA

Keynote Address: Sekar Kathiresan, Verve Therapeutics, USA

Which new cell therapies will progress to the clinic this year? Learn about the cutting-edge advances propelling regenerative medicine forward. Leading scientists will share their latest data on the use of stem cells to treat diseases from ALS to heart disease, and from blindness to cancer. This session will explore the stem cell engineering, gene editing, and precision medicine approaches that are advancing stem cell therapies.

Presidential Symposium Sponsored by: Fate Therapeutics

Ernest McCulloch Lecture: Fiona Watt, King's College London, UK Steven Finkbeiner, Gladstone Institutes, USA Eric Olson, University of Texas Southwestern, USA Aviv Regev, Broad Institute, USA

Each year the current ISSCR president assembles speakers who highlight some of the most exciting work in stem cell biology. In this year's Presidential Symposium, Deepak Srivastava brings together researchers that span many key topics in the field. Attendees will explore foundational topics such as self-renewal, lineage commitment, transcriptional regulation, and tissue maintenance as well as multi-disciplinary topics such as how biological circuits function and evolve or how neuronal activity leads to memory.

Machine Learning and Computational Approaches Sponsored by: T-CiRA Joint Program

ISSCR Dr. Susan Lim Outstanding Young Investigator Award Lecture: Allon Klein, Harvard Medical School, USA Trey Ideker, University of California, San Diego School of Medicine, USA Ajamete Kaykas, insitro, USA Hiroaki Kitano, Okinawa Institute of Science and Technology, Japan Emma Lundberg, KTH Royal Institute of Technology, Sweden

Machine learning and computational approaches are revolutionizing all fields of biology. These constantly evolving technologies provide new windows into stem cell population dynamics, heterogeneity, and clonality. Researchers also use computational modeling to improve the translation of stem cell science, including methods to investigate disease states, predict drug targeting, and develop the proper conditions for stem cell expansion and differentiation. Discover the newest techniques and approaches are applied to stem cell science and gain insights that you can adapt to your research.

Embryogenesis and Development Sponsored by: Semma Therapeutics

Benoit Bruneau, Gladstone Institutes, USA Elaine Fuchs, HHMI, Rockefeller University, USA Ken Zaret, University of Pennsylvania School of Medicine, USA Kathy Niakan, The Francis Crick Institute, UK

Stem cell scientists constantly unravel and expose the mysterious embryo development process. Leading innovators will dissect the earliest decisions in the mammalian embryo, decode the genetic regulation that leads to specific cell and tissue identity, and uncover what happens when these developmental programs go awry. These presentations will explore developmental principles that are fundamental to all parts of stem cell biology.

Stem Cells and Aging

ISSCR Tobias Award Lecture: Margaret Goodell, Baylor College of Medicine, USA Guanghui Liu, Institute of Biophysics, CAS, China Emi Nishimura, Tokyo Medical and Dental University, Japan Michael Rudnicki, Ottawa Hospital Research Institute, Canada Beth Stevens, Boston Children's Hospital, USA

Stem cell function often is dramatically affected during aging. Understanding these processes may help scientists determine how to prevent degeneration. Through the presentations, the decline in stem cell frequency and function that often accompanies the aging process will be explored. Attendees will gain new knowledge about principles unique to specific tissues, or common between all, and how misregulation of stem cells plays a key role in aging and disease.

Dissecting Organogenesis Sponsored by: Semma Therapeutics

ISSCR Momentum Award Lecture: Mitinori Saitou, Kyoto University, Japan Miki Ebisuya, EMBL Barcelona, Spain Madeline Lancaster, MRC Laboratory of Molecular Biology, UK Hans Snoeck, Columbia University Medical Center, USA

Novel techniques allow scientists to examine mechanisms of morphogenesis and differentiation during in vivo and in vitro organogenesis. Attendees will discover how researchers apply interdisciplinary approaches from epigenetic reprogramming to synthetic biology to organoids in order to dissect the cellular and molecular mechanisms of organogenesis. Additionally, attendees will hear about new translational studies that are bringing this research to the clinic and comparative studies that explain what makes humans unique.

Reprogramming and Regeneration Sponsored by FujiFilm

ISSCR Achievement Award Lecture: Fred H. Gage, Salk Institute for Biological Studies, USA

Anne McLaren Lecture: Alta Charo, University of Wisconsin USA Botond Roska, Friedrich Miescher Institute, Switzerland Li Qian, University of North Carolina, Chapel Hill, USA Shinya Yamanaka, Center for iPS Cell Research & Application, Japan

Reprogramming and regeneration are essential focal points in the study of stem cell biology. Leading researchers will illuminate the current state of these topic areas and how they are advancing stem cell therapies from bench to bedside. Attendees will hear about how reprogrammed cells are helping us understand disease and screen for new drugs, new insights into the mechanisms of regeneration, and the ethical issues surrounding it all.

Not only will ISSCR 2020 host outstanding plenaries, but also concurrent programming sessions organized around four core themes covering areas of the field. The meeting will host workshops from industry leaders on clinical translation, and biotech entrepreneurs discussing new ventures and investment. Special sessions cover public policy, women in science, science and ethics, stem cell technology, and preclinical development of investigational stem cell products. See speakers and sessions.

The ISSCR 2020 Virtual Meeting will feature live Q&A, networking hubs, a digital poster hall, and a vibrant, virtual exhibit floor. Attendees can access on-demand viewing of all sessions 24 hours-a-day for 30 days. All networking and professional development workshops and special sessions are now included with registration. Learn more about the high-caliber speakers and explore the workshops and special sessions that are incorporated into the virtual meeting this year.

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Registration is open. Complementary registration is available for media; apply for credentials. To connect for interviews, contact Kym Kilbourne at kkilbourne@isscr.org or media@isscr.org.

About the International Society for Stem Cell Research

With nearly 4,000 members from more than 60 countries, the International Society for Stem Cell Research is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health. Additional information about stem cell science is available at A Closer Look at Stem Cells, an initiative of the Society to inform the public about stem cell research and its potential to improve human health.

This story has been published on: 2020-06-02. To contact the author, please use the contact details within the article.

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New Leaders at ASTCT and CIBMTR Provide Guidelines and Measures for Combating COVID-19 and Protecting Transplant Patients – Business Wire

CHICAGO--(BUSINESS WIRE)--The American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood and Marrow Transplant Research (CIBMTR) outlined today their guidelines and measures for combating the spread of COVID-19, while providing protection to transplant patients. The guidelines and measures include new practices for data collection, sharing of information, and administrative relief for facilities impacted by COVID-19 precautions.

The ASTCT and CIBMTR work hand-in-hand to collect and share data used to improve both quality of transplant care and survivability. Among the new measures to help combat COVID-19 is the introduction of the CIBMTR Respiratory Virus Post-Infusion Data Form (2149). This form collects detailed data regarding the diagnosis, treatment, and outcome of COVID-19 infections.

We urge all participating facilities to make the best effort to secure consent and enrollment of patients in our databases, said the CIBMTRs Chief Scientific Director and Professor of Medicine at the Medical College of Wisconsin, Mary Horowitz, MD, MS. The data we collect is invaluable in the fight against COVID-19 and in promoting patient health. Weve already used CIBMTR data to examine several issues related to COVID-19 that are important for patient care during the pandemic.

CIBMTR reporting does not require any patient contact beyond what is necessary for clinical care, and the consent form is usually administered by clinicians at the time the patient signs the general consent for treatment.

To help caregivers protect their patients, the ASTCT and CIBMTR are sharing data on COVID-19 and providing regularly updated guidelines addressing the management, treatment, and outcomes of patients. They are also sharing knowledge through informational webinars, online meetings, and other methods as circumstances allow. Through this research and education, the ASTCT and CIBMTR are informing patient care and management; including the biologic, economic, and logistical aspects of care.

The best and fastest way for us to fight COVID-19 is through innovative research and care delivery, said Pavan Reddy, MD, ASTCT President and Chief of Hematology and Oncology at the University of Michigan. Understanding the disease in transplant patients offers unique clinical and biological insights that can lead to greater understanding of the disease in the general population.

In addition to collecting data and sharing knowledge, the ASTCT and CIBMTR are easing administrative requirements to provide impacted clinics with additional flexibility. This includes postponing on-site data audits and the relaxation of deadlines for forms submission.

The ASTCT and CIBMTR are committed to addressing challenges facing transplant patients, and the COVID-19 pandemic was a considerable topic of discussion during the organizations combined annual event, Transplantation & Cellular Therapy Meetings (TCT Meetings), held in February 2020. During the meetings, each organization transitioned leadership as they forged ahead in the fight against COVID-19. New leaders for each organization are as follows:

ASTCT:

CIBMTR:

About the American Society for Transplantation and Cellular Therapy

The American Society for Transplantation and Cellular Therapy (ASTCT), with headquarters in Chicago, is a professional society of more than 2,200 healthcare professionals and scientists from over 45 countries who are dedicated to improving the application and success of blood and marrow transplantation and related cellular therapies. ASTCT strives to be the leading organization promoting research, education, and clinical practice to deliver the best, comprehensive patient care.

About the CIBMTR

The CIBMTR (Center for International Blood and Marrow Transplant Research) is a research collaboration between the National Marrow Donor Program (NMDP)/Be The Match in Minneapolis and the Medical College of Wisconsin (MCW) in Milwaukee. The CIBMTR collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide to increase survival and to enrich quality of life for patients. The CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of transplant centers, and a unique and extensive clinical outcomes database.

For more information about the CIBMTR, please visit http://www.cibmtr.org, or follow the CIBMTR on Facebook, LinkedIn, or Twitter at @CIBMTR.

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New Leaders at ASTCT and CIBMTR Provide Guidelines and Measures for Combating COVID-19 and Protecting Transplant Patients - Business Wire