Amarna Therapeutics Announces Formation of Scientific Advisory Board to Support Advancement of Breakthrough Nimvec™ AM510 Gene Therapy for Type 1 Diabetes
Members: Professor Colin Dayan, Professor Desmond Schatz, Professor Didac Mauricio, Dr. Luisa Calamori, Dr. Kei Kishimoto, Professor Roberto Marrone, Dr. Sylvain Yu.
Leiden, Netherlands, 17 June 2024 – Amarna TherapeuticsAmarna (“Amarna” or the “Company”), a privately held biotechnology company focused on developing innovative gene therapies for a range of rare and common genetic diseases, including Type 1 Diabetes Mellitus (T1DM), is pleased to announce the formation of a new Scientific Advisory Board (SAB).
The newly formed SAB is comprised of seven renowned international scientific opinion leaders with extensive experience and knowledge in the areas of preclinical and clinical development, gene therapy, immunology, endocrinology and regulatory issues.
SAB will leverage its extensive expertise to ensure Amarna’s groundbreaking Nimvec™ AM510 program meets the highest scientific and regulatory standards, underscoring the company’s commitment to pioneering therapies for T1DM and improving patient outcomes. SAB’s insights will be critical in navigating the complexities of clinical trials and regulatory approval, ultimately accelerating the delivery of this groundbreaking therapy to market.
“The establishment of this Scientific Advisory Board marks a major development step for Amalna as it continues to evolve into a clinical-stage gene therapy development company.” Amarna CEO Henk Striefkerk said: “We are proud and excited to bring together leading clinicians and scientists from around the world who have been involved for decades in the clinical development of new treatments for genetic diseases. Their combined expertise will be instrumental in the development of Amalna’s lead drug candidate, Nimbec™ AM510We are committed to unlocking its full potential to transform the treatment of type 1 diabetes and other common debilitating genetic diseases.”
The Scientific Advisory Board (SAB) members are:
Collin Dayan, MA, MBBS, FRCP, PhDis a Clinical Professor of Diabetes and Metabolism at the Cardiff University Medical School, Cardiff (UK), a Senior Clinical Investigator at the University of Oxford and Director of the Cardiff Collaborative Research Office. His main research interests are translational research in the immunopathology of type 1 diabetes and clinical trials of peptide immunotherapy and nanoparticles in type 1 diabetes.
Desmond Schatz, MDHe has served as Professor of Pediatrics, Medical Director of the Diabetes Institute and Medical Director of the Clinical Research Center at the University of Florida, and was Scientific and Medical President of the American Diabetes Association in 2016. Dr. Schatz has been involved in type 1 diabetes research since the mid-1980s and has published over 400 papers, mostly on the prediction, natural history, genetics, immunopathology, prevention, and management of children and adolescents with type 1 diabetes.
Didac Mauricio, MDHe is Head of the Department of Endocrinology and Nutrition at Hospital Santa Creu i Sant Pau in Barcelona and Professor at the Faculty of Medicine at the University of Vic and Central University of Catalonia (UVic/UCC). He leads the Diabetes Research Group at his current institution and was recently appointed Scientific Director of the Institute of Diabetes and Related Metabolic Diseases (CIBERDEM) at the Carlos III Health Institute (Ministry of Science, Technology and Innovation), Spain.
Luisa Calamori, M.D., M.S., Ph.D.Dr. Calamori is a physician in the Endocrinology and Metabolism Institute at the Cleveland Clinic Foundation and the Department of Cardiovascular and Metabolic Sciences at the Lerner Research Institute in Cleveland, Ohio, and an Adjunct Associate Professor at the University of Minnesota, Minneapolis, MN, USA. Dr. Calamori is a physician-scientist with over 25 years of research experience and his primary research interests include the relationship between renal structure and function in type 1 diabetes, early molecular and structural predictors of diabetic kidney disease (DKD) in patients with type 1 diabetes, and clinical trials investigating repurposed and novel drugs for the prevention and treatment of DKD in patients with types 1 and 2 diabetes.
Dr. Kei Kishimoto, is an accomplished immunologist and drug developer with over 30 years of experience in the biopharmaceutical field. Most recently, he served as Chief Scientific Officer at Selecta Biosciences, where he led the development of immune tolerance technologies that have proven effective in Phase 3 clinical trials. Prior to joining Selecta, he was Vice President of Research at Momenta Pharmaceuticals and Senior Director of Inflammation at Millennium Pharmaceuticals. His areas of expertise include immunology, immune tolerance, autoimmune diseases, vaccines, immunogenicity and gene therapy.
Kay Roberto MaroneDr. MD, PhD, is Professor and Hospital Physician of Clinical Immunology and Diabetology at the Cochin Hospital of the Paris-Cité University and Director of Research at the INSERM U1016 Cochin Research Institute in Paris, France and the Indiana Biological Sciences Institute (IBRI) in Indianapolis, USA. His research ranges from preclinical studies with human samples and mouse models to clinical trials. His research focuses on understanding the mechanisms of autoimmune T cell and beta cell vulnerability and type 1 diabetes, and is working on developing T cell-based biomarkers and therapies aimed at blunting T cell aggressiveness and enhancing beta cell resilience.
Sylvain Yu, MDis a renowned researcher specializing in the immune mechanisms that drive type 1 diabetes mellitus (T1DM) and the development of treatment strategies. She is the Director of Research at the Institut Cochin (INSERM) in Paris, France, where she focuses on T-cell resistance, biomarkers, and therapeutics in type 1 diabetes. Additionally, she co-leads a research team at the Indiana Bioscience Institute (IBRI) investigating the mechanisms that fail to keep autoreactive T lymphocytes harmless, making beta cells more visible and vulnerable to autoimmune attack.
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About Type 1 Diabetes
Type 1 diabetes is a debilitating disease affecting millions of patients worldwide, with incidence rates increasing every year, but life expectancy remains lower than the general population, despite advances in treatment. Diabetes is an autoimmune disease in which autoreactive T lymphocytes selectively attack and destroy insulin-producing beta cells in the pancreas, leaving patients unable to maintain glucose homeostasis. Proinsulin (PI) is the main autoantigen involved in autoimmune beta cell destruction. Currently, type 1 diabetes cannot be cured, and patients’ glucose homeostasis can be more or less maintained by daily insulin injections. Although diabetes is considered a manageable disease today, secondary complications of current treatments are substantial, leading to significant morbidity and mortality. With Nimvec™ AM510, we intend to restore immune tolerance to proinsulin and cure patients.
About Nimvec™ AM510
The development of AM510 is based on our own NimvecTM The platform has demonstrated great promise in preclinical studies. Unlike other gene therapies that induce strong immune responses, limiting the potential for repeated dosing and efficacy, NimvecTM does not induce such a response; instead, it moderates the immune system and induces tolerance, making it an ideal vehicle for our therapeutic approach. Our preclinical data with Nimvec™ AM510 shows a protective effect in delaying the onset of hyperglycemia and preventing the development of T1DM in relevant animal models.
About Amarna
Amarna Therapeutics develops Nimvec, a breakthrough non-immunogenic viral platformTM Any transgene can be introduced into humans. The platform has shown extraordinary potential in preclinical studies to provide treatments, potential cures, and disease prevention worldwide. Amarna is advancing a pipeline of transformative gene therapies for a range of rare and common diseases, including single gene indications, autoimmune diseases, and chronic inflammation. The lead program, Nimvec™ AM510, is being developed for the treatment of patients with type 1 diabetes. Follow-up programs include multiple sclerosis, age-related macular degeneration, and hemophilia.
For more information, please visit www.amarnatherapeutics.com
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For further information, please contact:
Amarna Therapeutics
Henk Striefkerk, CEO
Email: info@amarnatherapeutics.com
Lifespring Life Science Communications, Amsterdam
Leon Merens
Phone: +31 6 538 16 427
Email: lmelens@lifespring.nl
