Research Advisory Committee
Paul August, PhD
Chief Scientific Officer, ReviR Therapeutics
Dr. August is a scientific and business leader with extensive drug discovery experience within multiple therapeutic areas. He played a pivotal role in the advancement of the DTX CMT1A program through his advocacy efforts at the CMTRF. With an 18-year tenure at Sanofi, he demonstrated exceptional leadership of the Early to Candidate Unit and pioneered innovative rare disease research programs. He is a Co-founder of AcuraStem, a leader in the development of therapeutics for neurodegenerative diseases such as ALS, FTD and CMT. In his prior role at Agios Pharmaceuticals, he led the Genetically Defined Disease preclinical research efforts. Currently, Dr. August’s expertise is focused on Neuroscience and Oncology at ReviR Therapeutics. Primarily he is focused on the development of therapeutics to treat and prevent Huntington’s Disease.
Toby Ferguson, MD, PhD
Chief Medical Officer, Voyager Therapeutics
Dr. Ferguson is a neuromuscular neurologist and neuroscientist focused on developing treatments for neuromuscular diseases. He is currently the Chief Medical Officer for Voyager Therapeutics, a biotechnology company developing gene therapies for neurological diseases. Before Voyager, he was Head of the Neuromuscular Development Unit at Biogen, leading the neuromuscular clinical development group driving preclinical strategy within neurodegenerative and neuromuscular diseases. His group also worked closely with the scientific, biomarker and commercial teams at Biogen, Ionis and other external collaborators to identify novel disease targets and to develop the needed tools for efficient clinical development. Dr. Ferguson has advanced multiple programs into the clinic for ALS, SMA, Myotonic Dystrophy and Parkinson’s disease. Prior to Biogen, he had a clinical neuromuscular neurology practice and a lab focused on peripheral axon injury and regeneration at Shriners Research Center and Temple University in Philadelphia. Dr. Ferguson trained in neurology and neuromuscular neurology at the University of Pennsylvania and earned an MD and PhD (Neuroscience) from the University of Florida.
Kleopas Kleopa, MD, PhD, FAAN, FEAN
Professor and Senior Neurologist, Cyprus Institute of Neurology and Genetics
Dr. Kleopa is a Professor and Senior Neurologist at the Cyprus Institute of Neurology and Genetics and the head of the Neuroscience Department and Coordinator of the Center for Neuromuscular Disorders, delivering cutting edge diagnostic and therapeutic services to patients with neuromuscular and rare neurological disorders. His research focuses on understanding neurological disease mechanisms and the development of innovative cell-targeted gene therapies for inherited neuropathies and leukodystrophies. Results of his research are currently being developed for clinical translation in collaboration with industry. Dr. Kleopa is an elected Board Member of the International Charcot-Marie-Tooth and Related Disorders Consortium. He also serves as a Member of the Scientific Advisory Boards of the USA CMT Association and the Gilbert Foundation Neurofibromatosis-1 Gene Therapy Initiative. Dr. Kleopa earned his MD and PhD at the University of Wuerzburg, Germany.
William Motley, MD, PhD
Senior Director of Clinical Development and Program Lead, Rapport Therapeutics
Dr. Motley is currently Senior Director of Clinical Development and Program Lead at Rapport Therapeutics, a precision neuroscience company, and worked as a consultant to the company’s founding team at Boston-based Third Rock Ventures. Previously he served as Director of Translational Medicine for Flare Therapeutics and was a Senior Associate at Third Rock Ventures where he was a member of Flare’s founding team and worked on diverse aspects of company creation. Dr. Motley also serves as a member of the Medical and Scientific Advisory Board of Palvella Therapeutics, a company focused on bringing therapies to patients with rare dermatologic diseases with no approved therapies. A member of CMTRF’s Board of Directors since 2023, he holds an MD from the University of Pennsylvania, a Doctorate in Neurogenetics from the University of Oxford (UK), and a BA in Biochemistry from Middlebury College. He completed a neurology residency at the Johns Hopkins University School of Medicine. His doctoral and post-doctoral research experiences focused on identifying new causes of CMT and characterizing pathological mechanisms of the disease.
Grace Pavlath, PhD
Vice President, Translational Development, Solid Biosciences
Dr. Pavlath was Chief Scientific Officer for the CMT Research Foundation from July 2019 to May 2020 managing funded research projects while curating more projects and acting as the steward for all scientific efforts and funding allocation. Currently, she is Vice President, Translational Development for Solid Biosciences and before that was Vice President, Research. She was Vice President, Research at Aavanti Bio just prior to its merger with Solid Biosciences. Dr. Pavlath served as Chief Research Officer for the Muscular Dystrophy Association (MDA) from 2014-2019. Prior to joining MDA, Dr. Pavlath led an internationally known laboratory as a tenured professor of Pharmacology at Emory University establishing herself as a leading researcher in cellular and molecular mechanisms involved in muscle regeneration and muscular dystrophy. She earned her doctorate in Pharmacology at Stanford University and then completed postdoctoral training at Stanford University and the University of Arizona.
Bill Skach, MD
Strategic Advisor for Research and Drug Discovery, Cystic Fibrosis Foundation
Dr. William Skach currently serves as a Strategic Advisor for Research and Drug Discovery at the Cystic Fibrosis Foundation and as an outside advisor to CMTRF. Dr. Skach received his bachelor’s degree in biochemistry and biophysics from Oregon State University in 1979 and his MD from Harvard Medical School in 1983, followed by clinical training in internal medicine, hematology/oncology and postdoctoral research training in cell biology and membrane protein biogenesis at the University of California, San Francisco. In 1994 he joined the faculty at the University of Pennsylvania Institute for Human Gene Therapy as Assistant Professor in the Departments of Medicine and Molecular and Cellular Engineering where he established his independent research laboratory and continued to treat cancer patients. He returned to Oregon in 1998 as Associate Professor of Molecular Medicine, and subsequently Biochemistry and Molecular Biology, at the Oregon Health & Science University where his research focused on basic principles of membrane protein folding, the molecular pathogenesis of cystic fibrosis, and small molecule correction of mutant CFTR proteins. In 2014 he moved to Bethesda, Maryland, to join the Cystic Fibrosis Foundation, ultimately becoming Executive Vice President and Chief Scientific Officer. At the Cystic Fibrosis Foundation, he was responsible for building a robust venture philanthropy portfolio, funding academic research labs around the world and working with scientists, numerous biotech and pharma partners, and the Cystic Fibrosis lay community to develop new life-saving drugs and ultimately a cure for people with Cystic Fibrosis. He also maintained a small research laboratory studying fundamental aspects of CFTR folding and trafficking. Dr. Skach stepped down as CSO in 2022.
Arthur Suckow, PhD
Former Co-Founder and CEO, DTx Pharma
Dr. Suckow is an innovative executive leader and scientist with 15 years of experience leading drug development programs across multiple therapeutic areas leveraging small molecules, biologics and oligonucleotides. Most recently, he founded DTx Pharma and served as its Chief Executive Officer until it was acquired for $1B by Novartis. In 6 years, he helped this San Diego-based RNA therapeutics company develop a pipeline of therapies for rare diseases, including CMT1A, and raise more than $120M through grants and leading healthcare investors. Prior to DTx, he served in roles of increasing responsibility at Johnson & Johnson, MedImmune/AstraZeneca and Regulus Therapeutics.
Dr. Suckow received his undergraduate degree in Biological Sciences from University of Delaware and his PhD in Biomedical Sciences/Pharmacology from University of California, San Diego. In addition to being on the board of CMTRF, he currently serves as a strategic and/or scientific adviser to Transcera, Actio Biosciences, Palm Therapeutics and numerous institutional investors. He has received numerous awards throughout his career including a Beckman Fellowship, a NSF graduate research fellowship, and a BIOCOM catalyst award.
Charlotte Sumner, MD
Professor of Neurology and Neuroscience, Johns Hopkins University School of Medicine
Dr. Sumner co-directs the Johns Hopkins Muscular Dystrophy Association Care Center, the Spinal Muscular Atrophy, and the Charcot-Marie-Tooth clinics, which deliver multi-disciplinary clinical care, engage in international natural history studies, and provide cutting-edge therapeutics. Her practice is notable for its focus on individuals with inherited neuromuscular disorders of peripheral nerves and motor neurons, including spinal muscular atrophy and CMT. Her research focuses on developing treatments for degenerative disorders of motor neurons and peripheral nerves. Notably, Dr. Sumner was a previous member of the SAB, and her recommendations played a pivotal role in steering the foundation towards addressing delivery challenges.
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