Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program
“We are taking a multi-front approach in the battle against Duchenne
muscular dystrophy,” said
The experimental program explores the potential surrogate gene therapy approach to Duchenne muscular dystrophy. As a “surrogate gene therapy approach”, the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin, with the goal of producing 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 have this opportunity to help advance our Galgt2 gene
therapy program,” said Dr.
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.
Named to the Top 10 Honor Roll on
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 exclusive license agreement Sarepta has entered into with Nationwide Children’s Hospital for their Galgt2 gene therapy program, the potential for this therapeutic approach to treat all patients suffering from DMD regardless of their mutation, age and disease severity, the expected clinical progress of the program in 2017, the potential for the gene therapy to be used in several muscular dystrophies, the gene therapy’s mechanism of action and potential efficacy and Sarepta taking a multi-front approach in the battle against DMD.
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 exclusive license agreement and related agreements between the parties may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; the partnership between Sarepta and Nationwide Children’s hospital may not result in any viable treatments suitable for clinical research or commercialization due to a variety of reasons including that the results of additional 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 or may never become commercialized products due to other various reasons including any potential future inability of the parties to fulfill their commitments and obligations under the agreements; and even if the agreements result in commercialized products, the parties may not achieve any significant revenues from the sale of such products.
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
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.
Sarepta Therapeutics 2019 Corporate Presentation
Latest Annual Report( FY 2018 )