Press Release

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 1, 2015-- Sarepta Therapeutics (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced a four year collaborative research agreement to establish the Sarepta Translational Laboratory with Murdoch University, Perth, Western Australia. The Laboratory, led by Murdoch University professors Steve Wilton, Ph.D. and Sue Fletcher, Ph.D., will explore the applicability of the Company’s phosphorodiamidate morpholino oligomer (PMO) technology for disease targets beyond Duchenne muscular dystrophy (DMD), such as cystic fibrosis and spinal muscular atrophy.

The agreement provides the Murdoch University researchers with access to Sarepta’s PMO platform technology, as well as additional funding for researchers and materials. The initial project for the collaboration is anticipated to target multiple sclerosis. As part of the collaborative agreement, Sarepta will have exclusive rights to license technology and/or products resulting from the research projects.

“We’re very excited about this opportunity to support important early stage research and, through that, generate data on the applicability of our PMO technology for additional disease targets,” said Edward Kaye, Sarepta’s interim chief executive officer and chief medical officer. “We are hopeful that through this collaboration our technology may lead to important breakthrough treatments for patients.”

Murdoch University’s Acting Vice Chancellor Professor Andrew Taggart noted that the agreement with Sarepta allows the University’s laboratory to become a pipeline of drug development, and the Sarepta association will facilitate getting the drugs from the laboratory to clinical trials.

“Murdoch’s translational research endeavors to bring positive change through new discoveries,” said Professor Taggart. “Our partnership with Sarepta is yet another example of our successes in international health research as it will see our accomplished researchers use innovative technologies to devise treatments for children suffering from debilitating genetic diseases.”

The partnership will allow Murdoch researchers from the Centre for Comparative Genomics, Professors Steve Wilton and Sue Fletcher, to build on their research which won them an Australian Museum Eureka Prize in 2013. The researchers were recognized for developing a new drug that works by ‘skipping over’ the faulty part of the gene message in children suffering from Duchenne muscular dystrophy.

“We are delighted, excited, and proud to now have a more formal and focused academic and industry partnership, combining Sarepta’s morpholino technology and translational experience with our expertise and to develop treatments for many other human diseases,” said Professor Wilton.

About Sarepta Therapeutics

Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of infectious diseases, such as drug-resistant bacteria and other rare human diseases. For more information, please visit us at www.sarepta.com.

Forward-Looking Statements

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 four year collaborative research agreement Sarepta has entered into with Murdoch University, plans to develop the Sarepta Translational Laboratory with Dr. Wilton and Dr. Fletcher to explore the applicability of Sarepta’s PMO technology for disease targets beyond DMD, the initial targets under the collaboration, the future rights and obligations of the parties under the agreement, Sarepta’s plans to support this important early stage research and generate data on the applicability of its PMO technology for additional disease targets, Sarepta’s hope that through this collaboration its technology may lead to important breakthrough treatments for patients, the University’s ability under the agreement to become a pipeline of drug development and facilitate getting drugs from the laboratory to clinical trials, Murdoch’s translational research ability to bring positive change through new discoveries and have its researchers use innovative technologies and Sarepts’s technology to devise treatments for children suffering from debilitating genetic diseases and other human diseases.

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 collaboration and agreement may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in new product research and development; the research and development done at the Sarepta Translational laboratory may not result in any viable investigational treatments suitable for clinical research; even if any investigational treatments do make it to the clinical stage, they may fail to demonstrate safety or efficacy, may not receive required regulatory approvals, or may never become commercialized products various reasons,

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 2014 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q for the quarter ended June 30, 2015 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Sarepta. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

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Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.
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