Larimar Therapeutics Announces Formation of Scientific Advisory Board

Larimar Therapeutics announced the formation of its Scientific Advisory Board (SAB). Larimar’s SAB is comprised of distinguished research scientists, professors and industry experts recognized as key opinion leaders in the fields of rare disease, pediatrics and mitochondrial disease.

Nancy M. Ruiz, MD, FACP, FIDSA, Chief Medical Officer of Larimar Therapeutics Said: “Larimar is privileged to have this group of prestigious, multidisciplinary advisors who are committed to advancing the research and development of CTI-1601 for Friedreich’s ataxia, Their scientific perspectives will be invaluable to determine our strategic scientific pathway and support the development of other potential treatments for complex rare diseases to help fill unmet medical needs in this space.”

Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar Therapeutics Said: “Formalizing the SAB adds to our recent accomplishments, which include resuming our Phase 1 clinical trial of CTI-1601 for Friedreich’s ataxia and receiving a positive opinion on orphan drug designation for CTI-1601 from the European Medicines Agency’s Committee for Orphan Medicinal Products, This progress helps position Larimar for success as we continue to execute our strategy of developing treatments for complex rare diseases.”

The members of Larimar’s SAB are as follows:

Russell (Rusty) Clayton, DO – Dr. Clayton will serve as the Scientific Advisory Board Chair for Larimar Therapeutics. He brings more than 15 years of executive experience in pharmaceutical, biologics and medical device development and commercialization as a consultant in clinical development, medical affairs and regulatory affairs. Prior to becoming a consultant, Dr. Clayton was the chief medical officer at Alcresta Therapeutics, a medical device company. Prior to Alcresta Therapeutics, he was the senior vice president of research and development at Discovery Labs, a pharmaceutical and medical device company, where he led the scientific and regulatory efforts leading to the marketing authorization of Discovery’s first product. Dr. Clayton is a board-certified pediatric pulmonologist who practiced at St. Christopher’s Hospital for Children and the Children’s Hospital of Philadelphia prior to beginning his career in the pharmaceutical, biologics, and medical device industry. He received his DO from the Philadelphia College of Osteopathic Medicine.

Marni J. Falk, MD – Dr. Falk is Executive Director of the Mitochondrial Medicine Frontier Program at The Children’s Hospital of Philadelphia (CHOP) and Professor in the Division of Human Genetics, Department of Pediatrics at University of Pennsylvania Perelman School of Medicine. She also serves as a principal investigator of a National Institutes of Health, pharma and philanthropic-funded translational laboratory group at CHOP that investigates the causes and global metabolic consequences of mitochondrial disease and directs multiple clinical treatment trials in mitochondrial disease patients. Dr. Falk received her BS in biology and MD from the George Washington University School of Medicine. In addition, she completed dual specialty training in the Pediatrics and Clinical Genetics residency program at Case Western Reserve University.

Giovanni Manfredi, MD, PhD – Dr. Manfredi is the Finbar and Marianne Kenny Professor in Clinical and Research Neurology at Weill Cornell Medicine. He is also a Professor of Neuroscience and directs the graduate program in Neuroscience at Weill Cornell Medicine. Dr. Manfredi’s lab studies alterations of mitochondrial metabolism in neurodegenerative diseases, particularly amyotrophic lateral sclerosis and primary inherited mitochondrial encephalomyopathies. Dr. Manfredi has authored more than 100 publications focused in areas including neurodegenerative and mitochondrial diseases. Dr. Manfredi received his MD and PhD in anatomy and cell biology from Catholic University of the Sacred Heart in Rome, where he also completed a residency in neurology.

Mark Payne, MD – Dr. Payne is a renowned scientist and practicing cardiovascular physician who brings a long-standing scientific focus on protein targeting to mitochondria and a dedication to treating cardiomyopathies of childhood, including Friedreich’s ataxia. He is the inventor of the original therapy for frataxin protein replacement in Friedreich’s ataxia and co-founded Chondrial Therapeutics, which became Larimar Therapeutics, Inc. He holds multiple patents on mitochondrial biology and repair. He is a tenured professor of pediatrics at Indiana University School of Medicine where he directs multiple NIH-funded training, clinical, and research programs as a principal investigator. Dr. Payne received his BS in natural sciences from Washington & Lee University, and his MD from the University of Texas at Houston. He performed his postdoctoral clinical and research training at Washington University in St. Louis. He is a Fellow of the American College of Cardiology and the American Academy of Pediatrics. 

Marshall Summar, MD – Dr. Summar serves as Chief of the Division of Genetics and Metabolism, Director of the Rare Disease Institute and is the Margaret O'Malley Chair of Genetic Medicine at Children’s National Hospital. In addition to guiding clinical research and treatment, he developed and launched the world’s first Rare Disease Institute (RDI) at Children’s. The RDI is the first Clinical Center of Excellence designated by the National Organization for Rare Diseases (NORD) and focuses on building best clinical practices and diagnostic pathways for patients. With NORD and the FDA, Dr. Summar has worked to develop a patient-driven natural history platform employed by over 35 rare disease advocacy organizations. He received his BS in molecular biology from Vanderbilt University and his MD from University of Tennessee Center for Health Sciences.

Source: Larimar Therapeutics, Inc

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