CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, today announced it has entered a research and option agreement with Nationwide Children’s Hospital on their microdystrophin gene therapy program. Dr. Jerry Mendell, M.D. and Dr. Louise Rodino-Klapac, Ph.D., are the lead principal investigators of the program.
“Given the complexities of Duchenne muscular dystrophy, we know that it is going to require multiple treatment approaches,” said Edward Kaye, Sarepta’s chief executive officer. “With that goal in mind, we are excited to support clinical development for Nationwide’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 has committed to the trial through a separate research agreement with Nationwide Children’s, and has 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. The additional resources will bolster Nationwide’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.
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.