Date: 2015-08-12
Type of information: Nomination
Compound:
Company: Sarepta Therapeutics (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: nomination
Action mechanism:
Disease:
Details: * On August 12, 2015, Sarepta Therapeutics announced the formation of a Strategic & Scientific Advisory Board (SSAB) with three key appointments: Louis Kunkel, PhD, Beverly Davidson, PhD, and Jeffrey Chamberlain, PhD.
The SSAB will work closely with Sarepta’s management team to advance the Company’s platform technology in Duchenne muscular dystrophy (DMD), identify multiple new applications for Sarepta’s proprietary PMO chemistry in the neuromuscular rare disease and infectious disease areas, as well as provide insight on novel technologies to complement the Company’s pipeline.
The initial appointments to Sarepta’s Strategic & Scientific Advisory Board include:
Louis M. Kunkel, PhD, Past Chief of the Division of Genetics and currently a member of the Division of Genetics and Genomics, Boston Children’s Hospital. Professor of Pediatrics and Genetics, Harvard Medical School; Co-Director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center of Worchester MA; HHMI Investigator, 1987-2010; and Member of the National Academy of Sciences and the American Academy of Arts and Sciences. Dr. Kunkel is an internationally recognized geneticist with decades of experience and scientific success in the understanding of the basis for muscular dystrophies. Dr. Kunkel is responsible for the identification of the gene and encoded protein, dystrophin. He has received numerous awards and honors for scientific leadership and achievement and currently serves as the committee chair for the Muscular Dystrophy Association (MDA), a committee he has been a member of for over 15 years.
Beverly Davidson, PhD, Director of the Raymond G Perelman Center for Cellular and Molecular Therapeutics and Arthur V. Meigs Chair in Pediatrics, Children’s Hospital of Philadelphia; Professor in the Department of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. Dr. Davidson’s research focuses on the pathogenesis and therapy of hereditary neurogenetic diseases and the role of noncoding RNAs in neural development. She is an internationally known expert in the study of inherited neurological diseases and the development of molecular therapies. Dr. Davidson has been named a fellow by the American Association for the Advancement of Science, received the NIH Mathilde Solowey Award, was elected to the Advisory Council for the American Society of Gene and Cell Therapy and NINDS council, and was past chair of the Medical Sciences Section for the American Association for the Advancement of Science.
Jeffrey Chamberlain, PhD, Professor in the Departments of Neurology, Medicine, and Biochemistry; McCaw Endowed Chair in Muscular Dystrophy at the University of Washington; Director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center of Seattle. Dr. Chamberlain’s research is primarily focused on two major goals: to develop a better understanding of the molecular basis of the pathophysiology of muscle disorders, and to develop gene and cell therapies that will correct and treat the muscular dystrophies. Dr. Chamberlain is a close collaborator with a number of prominent institutions, including Children’s Hospital of Seattle, Fred Hutchinson Research Center, University of Rochester, and Harborview Medical Center. Dr. Chamberlain also serves on the advisory boards for CureDuchenne, Akashi Therapeutics, and Solid Ventures.
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