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Sarepta Therapeutics Enters Into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program

Columbus, OH - 1/10/2017

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, today announced it has entered a license agreement with Nationwide Children’s Hospital, for their Galgt2 gene therapy program developed by researcher Paul Martin, PhD
 
“We are taking a multi-front approach in the battle against Duchenne muscular dystrophy,” said Edward Kaye, Sarepta’s chief executive officer. “We find this therapeutic approach to be of particular interest as it has the potential to treat all patients suffering from the disease regardless their mutation, and look forward to developing the Galgt2 program with Nationwide.”
 
The experimental program, which is expected to enter into a clinical trial in 2017, explores the potential surrogate gene therapy approach to Duchenne Muscular Dystrophy and is being pursued as a treatment for DMD. As a “surrogate gene therapy approach”, the gene therapy looks to induced genes that make proteins that can basically do the same job as dystrophin, with the goal of a muscle cell that can function normally even when dystrophin is absent. It has the potential to be used broadly in several muscular dystrophies.  
 
“We are pleased to havethis opportunity to help advance our Galgt2 gene therapy program,” said Kevin Flanigan, MD, the Principal Investigator leading the clinical trial “Our goal is to have this program in the clinic during 2017 and begin to evaluate a therapy that has the potential to treat patients of all ages and disease severity.”
 
To learn more about Galgt2 gene therapy, please visit the Nationwide Children’s website for a 2013 podcast which discussed the program: http://www.nationwidechildrens.org/dr-paul-martin-discusses-galgt2-gene-therapy-for-muscular-dystrophy.
 
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of 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 us at www.sarepta.com.
 
Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our web site at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
 
About The Research Institute at Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report’s 2016-17 list of “America’s Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric healthcare systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational and health services research at Nationwide Children’s. The Research Institute encompasses three research facilities totaling 525,000 square feet dedicated to research. More information is available at NationwideChildrens.org/Research.
 
Source: Sarepta Therapeutics, Inc.
 
Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan, 617-274-4052
iestepan@sarepta.com
or
W2O Group
Brian Reid, 212-257-6725
breid@w2ogroup.com

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Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000