Sarepta is known for several RNA-based medicines that it’s brought to market for Duchenne muscular dystrophy, each of which help patients produce a shortened form of the muscle-protecting protein they would otherwise lack. But the drugs only produce small amounts of the protein, called dystrophin, and it’s still unclear how much they benefit patients.
Confirmatory stories meant to prove their efficacy are ongoing. In the meantime, Sarepta has been working on a newer technology it claims will produce more potent medicines. The company has six in development and SRP-5051, currently in Phase 2 testing, is the most advanced.