Sarepta Therapeutics Introduces Online Resource Center on Exon Skipping for the Duchenne Muscular Dystrophy Community
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, announced a new online resource center, called Let's Skip Ahead, for families affected by Duchenne muscular dystrophy (DMD) and their healthcare providers. The new website, available at www.skipahead.com, includes information and educational resources intended to make the science of exon skipping simple, and provides an opportunity to sign up for updates about upcoming Sarepta clinical trials.
“Sarepta is deeply committed to pursuing exon-skipping treatments for patients with DMD who can potentially benefit from our technology, and this website will help families and physicians stay informed about our efforts," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We recognize that many in the Duchenne community are seeking to understand exon skipping and the promise this innovative and complex technology may hold for them. Resources on the Let's Skip Ahead website include information tools that can support discussions between patients, their families and the physicians who care for them.”
Let's Skip Ahead (www.skipahead.com) also offers an exon mapping tool that allows visitors to explore the potential link between specific genetic mutations in DMD and exon skipping. In addition, the website includes important information on genetic testing and the clinical trial process.
About Exon Skipping
Exon skipping is a potential therapeutic approach in DMD designed to produce functional dystrophin, a protein involved in muscle function that is lacking in patients with the disorder. Each potential therapy based on this technology is designed to skip a specific exon, and thereby correct for certain genetic mutations and restore the gene's ability to make a functional, though shorter, form of the dystrophin protein. Available data suggest that at least 65 percent of genetic mutations in DMD could potentially be addressed by exon skipping.
Eteplirsen is Sarepta's lead investigational exon-skipping drug candidate in clinical studies, and is designed to skip exon 51 in the dystrophin gene. Sarepta plans to initiate a confirmatory clinical trial of eteplirsen in DMD patients with amenable genotypes in the first quarter of 2014. Eteplirsen is not yet approved or licensed for use in any country.
In addition, Sarepta is conducting early-stage research, intended to support future clinical studies in patients, on drug candidates targeting exons 53, 50 and 45. Additional research to target other exons is also planned.