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Sarepta Therapeutics Enters Into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin 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 research agreement with Nationwide Children’s Hospital on their microdystrophin gene therapy program. Jerry Mendell, MD and Louise Rodino-Klapac, PhD, are the lead principal investigators of the program.
 
“Given the complexities of Duchenne muscular dystrophy, we know it is going to it require multiple treatment approaches,” said Edward Kaye, Sarepta’s chief executive officer. “With that goal in mind, we are excited to support the trial for the Nationwide Children's gene therapy program with the goal to help all boys with DMD.”
 
The initial trial, expected to go into Phase 1/2a trial in late 2017, will be conducted at Nationwide Children’s. Parent Project Muscular Dystrophy (PPMD) has committed 2.2 million dollars to the trial, with support from additional Duchenne foundations and families. Sarepta will also provide support to the trial through a separate research agreement with Nationwide Children’s, and will have an exclusive option to license the program. PPMD's grant provided incentive for Sarepta to help expand and accelerate this opportunity.
 
“We are thrilled Sarepta has entered into this research agreement with Nationwide Children's Hospital. The additional resources will bolster Nationwide Children's ability to conduct an even more rigorous and robust trial,” said Pat Furlong, founding president and chief executive officer of Parent Project 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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