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Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program

On the heels of PPMD’s announcement last week of a $2.2 million grant to Nationwide Children’s Hospital’s gene therapy study being led by Dr. Jerry Mendell and Dr. Louise Rodino-Klapac, Sarepta Therapeutics today announced their commitment to the trial through a separate research agreement with Nationwide Children’s, including an exclusive option to license the program.

We are thrilled that PPMD's grant, with support from additional Duchenne foundations and families, provided incentive for Sarepta to help expand and accelerate this opportunity! The additional resources will bolster Nationwide’s ability to conduct an even more rigorous and robust trial.

Read Sarepta's Press Release: 

Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 10, 2017-- 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 ProjectMuscular Dystrophy.

Read the full press release.

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