Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today 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.

"We are thrilled with the addition of these experts to our team of advisors," said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. "The background and experience our SSAB members bring to Sarepta will be invaluable as we move our follow-on exon skipping therapy product candidates forward into later stage clinical development, but also as we work towards the goal of discovering complementary therapies to further improve the lives of patients with DMD and expanding our RNA technology for other genetic neuromuscular diseases. As we begin to build our SSAB, I am proud to have leaders the caliber of Drs. Kunkel, Davidson, and Chamberlain as founding members and look forward to working with them.”

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.

About Sarepta Therapeutics

Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of infectious diseases such as drug-resistant bacteria and other rare human diseases. For more information, please visit us at www.sarepta.com.

Forward-Looking Statements

This press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to the ability of the SSAB to advance Sarepta’s platform technology, identify new applications of Sarepta’s proprietary PMO chemistry and provide insight on novel technologies; and the value of SSAB member insights to and Sarepta’s plans to move follow-on exons forward in clinical development and to discover complementary therapies to further improve the lives of DMD patients and expanding our RNA technology for other diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to capitalize on our experience or that of the SSAB to advance candidates; we may not be able to develop, advance or complete research or clinical development for our pipeline of product candidates and the results of our ongoing research and development efforts may not be positive, demonstrate safety or efficacy and may not support the advancement of candidates; our other product candidates and/or Sarepta’s anti-sense based technology platform may not be successful and we may not discover or be able to obtain complimentary therapies; there may be delays in our projected regulatory timelines relating to conducting research and initiating new clinical trials for our product candidates, or making a product commercially available for various reasons including possible limitations of Company resources and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; manufacturing of candidates may not be successful and any or all of the Company’s drug candidates may fail in research or development or may not receive required regulatory approvals for commercialization; we may need and may not be able to obtain additional funds to conduct our planned research, development or commercialization efforts and execute our business plans; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K or Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. You should not place undue reliance on forward-looking statements. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except to the extent required by applicable law or SEC rules.

Internet Posting of Information

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