Sarepta Therapeutics’ positive data on its lead muscular dystrophy drug eteplirsen puts the company in a strong position at the partnering table and sets the stage for a neck and neck race with GSK/Prosensa to bring the first treatment for the rare disease to market.

The RNA drug-discovery company, formerly AVI BioPharma, is reinventing itself as an orphan disease drug specialist called Sarepta Therapeutics. With the release of positive Phase IIb data on its lead candidate for Duchenne muscular dystrophy, the transformation has gotten a big boost.

The company is optimistic about a significant portion of eligible patients opting for its newly approved long-acting HIV therapy over daily, oral antiviral therapy.