Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program
“Given the complexities of Duchenne muscular dystrophy, we know that it
is going to require multiple treatment approaches,” said
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
“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
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.
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This press release contains statements that are forward-looking. 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 about the research and exclusive option agreement Sarepta has entered into with Nationwide Children’s Hospital for their microdystrophin gene therapy program, that DMD is going to require multiple treatment approaches, Sarepta supporting Nationwide’s gene therapy program, the goal to help all boys with DMD, Sarepta’s resources bolstering Nationwide’s ability to conduct an even more rigorous and robust trial, and the expected clinical progress of the program in 2017.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: the expected benefits and opportunities related to the agreements may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; in particular the agreements may not result in any viable treatments suitable for commercialization due to a variety of reasons including any inability of the parties to perform their commitments and obligations under the agreements, the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates and even if commercialization of any product under the agreements is achieved this may not result in any significant revenues to the parties.
Any of the foregoing risks could adversely affect Sarepta'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 2015 Annual Report on Form 10-K and
most recent Quarterly Report on Form 10-Q for the quarter ended
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