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.
Research focus: Exon Skipping.
Status: Accelerated approval of EXONDYS 51 (eteplirsen) by FDA. Other candidates for exon skipping in various stages of clinical development.
“CureDuchenne helped us very early on in in the development of the program. They gave us a jump start on clinical trial development. This is a very long journey. I would look at eteplirsen as the first step in a long process of trying to get better and better drugs for this community.” — Ed Kaye, M.D., CEO and Chief Medical Officer of Sarepta.