Sarepta Therapeutics Appoints Louise Rodino-Klapac, Ph.D. to Lead Newly Created Gene Therapy Business Unit

June 4, 2018 at 8:30 AM EDT

-- Dr. Rodino-Klapac, a National Institutes of Health Fellow, is a pioneer in the advancement of gene therapy to treat human disease, whose discoveries have advanced six gene therapy programs from bench to human clinical development --

-- Served as a principal investigator and co-inventor of the micro-dystrophin technology for the gene therapy Duchenne muscular dystrophy (DMD) clinical trial underway by Sarepta and Nationwide Children’s Hospital --

-- Co-founded Myonexus Therapeutics, Inc. and is inventor of its Limb-girdle muscular dystrophy (LGMD) portfolio --

CAMBRIDGE, Mass., June 04, 2018 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, today announced the appointment of Louise Rodino-Klapac, Ph.D. to lead its newly created gene therapy business unit as vice president, gene therapy, overseeing the development of the Company’s gene therapy and gene editing programs and serving as a member of the Company’s executive team.

Until her appointment with Sarepta, Dr. Rodino-Klapac was head of the laboratory for gene therapy research for Muscular Dystrophies at Nationwide Children’s Hospital and is renowned for her work in molecular genetics and gene therapy. Her ground-breaking work has involved 11 investigational new drug applications for gene therapy, research resulting in numerous field-advancing peer-reviewed publications, and the advancement of six gene therapy programs from bench to human clinical development.  

She was an Associate Professor, Department of Pediatrics, The Ohio State University College of Medicine and Principal Investigator, Center for Gene Therapy for The Research Institute at Nationwide Children's Hospital and a faculty member of the Biomedical Sciences Graduate Program and Molecular, Cellular, and Developmental Biology Graduate Programs, The Ohio State University College of Medicine.

Dr. Rodino-Klapac will retain her position as acting chief scientific officer of Myonexus Therapeutics, Inc. In early May 2018, Sarepta announced a collaboration with Myonexus to develop potentially transformative gene therapies to treat five distinct forms of LGMD.

“I could not be prouder to welcome Dr. Rodino-Klapac, a celebrated genetic medicine luminary, to Sarepta and to our mission to improve the lives of those living with neuromuscular conditions. Her appointment to lead our gene therapy division perfectly exemplifies our strategy of quickly becoming among the most meaningful global genetic medicine companies by retaining and nurturing the field’s best and brightest gene therapy and neuromuscular scientists,” said Doug Ingram, Sarepta’s president and chief executive officer. “It would be challenging to find a scientist that has matched Dr. Rodino-Klapac’s record of advancing our understanding of gene therapy and its application to neuromuscular conditions while accelerating bench-side research into human clinical trials.” 

“Making an impact on the lives of patients affected by muscular dystrophies has been central to my professional work. Sarepta’s RNA platforms and multiple gene therapy programs provide significant opportunity to make a profound difference for patients with neuromuscular diseases,” said Dr. Rodino-Klapac. “I’m excited to join the Sarepta team leading its gene therapy development efforts, and share the organization’s urgency to advance its robust pipeline of potential therapies.”

Dr. Rodino-Klapac is the recipient of numerous awards, including the Forty Under 40 Award by Columbus Business First, and the Department of Pediatrics Outstanding Junior Faculty Award for Innovation. She’s the author of numerous publications, among them papers published in The New England Journal of Medicine, Annals of Neurology, and Pediatric Neurology.

Dr. Rodino-Klapac also served as the Ruth L. Kirschstein F32 Post-doctoral Fellow, National Institutes of Health, The Research Institute at Nationwide Children's Hospital; and was a Post-doctoral Researcher, Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital. Dr. Rodino-Klapac received her bachelor’s degree in biology from Kings College, and a Ph.D. in molecular genetics from The Ohio State University.

About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates. For more information, please visit: www.sarepta.com.

Forward-Looking Statements

This press release contains "forward-looking statements." 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," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sarepta’s mission to improve the lives of those living with neuromuscular conditions; Dr. Rodino-Klapac’s appointment to lead Sarepta’s gene therapy division, perfectly exemplifying Sarepta’s strategy of quickly becoming among the most meaningful global genetic medicine companies by retaining and nurturing the field’s best and brightest gene therapy and neuromuscular scientists; and Sarepta’s RNA platforms and multiple gene therapy programs providing significant opportunity to make a profound difference for patients with neuromuscular diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: Sarepta’s gene therapy programs may not result in any viable treatments suitable for clinical research or commercialization due to a variety of reasons, including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates or may never become commercialized products due to other various reasons including possible limitations of Company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, 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; and even if Sarepta’s gene therapy programs result in new commercialized products, Sarepta may not achieve any significant revenues from the sale of such products; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2017 and most recent 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. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2017 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.         

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We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com.We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

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Source: Sarepta Therapeutics, Inc.