On Monday, Sarepta Therapeutics announced positive results from a small study of SRP-9003, its gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). This is a type of muscular dystrophy that affects both males and females. To date, the company’s successes have been in Duchenne’s muscular dystrophy (DMD), which primary affects boys.
The success has caused analysts and investors to speculate that the company is on the right track with gene therapies, which the company’s chief executive officer, Doug Ingram, saying the company would spend $600 million on expanding. Sarepta currently has 27 possible gene-augmenting therapies in development.