CAMBRIDGE, Mass., March 30, 2017 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, today announced the addition of Kenneth Fischbeck, M.D., and Matthew Wood, M.D., Ph.D., to the Company’s Strategic and Scientific Advisory Board. Dr. Fischbeck and Dr. Wood join Dr. Beverly Davidson and Dr. Louis Kunkel, who were appointed to the board at the time of its formation in 2015.
“We are honored and excited to have Dr. Fischbeck and Dr. Wood join our advisory board,” said Edward Kaye, Sarepta’s chief executive officer. “Following the approval of our exon 51 skipping drug, EXONDYS 51™ (eteplirsen) Injection, our board members helped in guiding our strategy to move additional exon skipping therapies into later stage clinical development, rapidly advance our PPMO platform into the clinic, explore complimentary therapies to further improve the lives of patients with DMD, and expand our partnerships and research collaborations. The addition of Dr. Fischbeck and Dr. Wood adds to our board’s diversity of experience with Duchenne and neurological diseases and provides Sarepta with unique counsel for our research and development efforts.”
Kenneth Fischbeck, M.D., received A.B. and A.M. degrees from Harvard University and a M.D. degree from Johns Hopkins. After a medical internship at Case Western Reserve University and a neurology residency at the University of California in San Francisco, he did postdoctoral research on muscular dystrophy at the University of Pennsylvania. In 1982, he joined the faculty in the Neurology Department at the University of Pennsylvania Medical School. In 1998, he joined the National Institute of Neurological Disorders and Stroke (NINDS) as Chief of the Neurogenetics Branch. He received the Cotzias Award from the American Academy of Neurology and the Jacoby Award from the American Neurological Association, and he was elected to the Institute of Medicine. His research group is identifying the causes and studying the mechanisms of hereditary neurological and neuromuscular diseases with the goal of developing effective treatment for these disorders.
Matthew Wood, M.D., Ph.D., is a Professor of Neuroscience and Deputy Head of the Medical Sciences Division at the University of Oxford and directs the Laboratory of RNA biology and Neuromuscular Disease. Dr. Wood is a leading pioneer in the field of oligonucleotide therapies and co-leads the International MDEX Consortium, a major international translational medicine collaboration to develop oligonucleotide treatments for DMD and related neuromuscular conditions, with Dr. Francesco Muntoni of University College London. Sarepta’s EXONDYS 51 was the first oligonucleotide worked on by this Consortium. Dr. Wood is an executive member of the global alliance TREAT-NMD. Dr. Wood serves as a Director of several organizations, including the Oxford MDUK Centre for Translational Neuromuscular Science, the University of Oxford’s technology transfer organization, Oxford University Innovation, and Evox Therapeutics, a company he founded based on his work with exosomes. He currently serves as the Deputy Head of the Medical Sciences Division of the University of Oxford, and holds a Director role on the Board of MedCity.
About EXONDYS 51™
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51 has not been established.
