Juggling Gene Therapies: Sarepta's Focus Grows, With Many Balls In The Air

Sarepta Therapeutics Inc.'s micro-dystrophin gene therapy program for Duchenne muscular dystrophy (DMD) is getting a lot of attention from investors as a near-term potential cure for the progressive and fatal disease. But while Sarepta is hyper-focused on executing on that program as quickly as possible, the company also is branching out in new directions with the aim of being a leading gene therapy specialist.

In 2018, Sarepta signed multiple partnerships that gave the company access to new gene therapy technologies, broadened its manufacturing footprint, and established a new gene therapy business unit led by Louise Rodino-Klapac, a gene therapy pioneer who previously led gene therapy research for muscular dystrophies at