Pepgen aims to overtake Sarepta

Sarepta has built a $9.5bn business off the back of exon skippers for Duchenne muscular dystrophy with questionable efficacy. The company is trying to up its game with its next-generation project, but a new challenger reckons it can go one better with an enhanced exon skipper of its own.

That group, Pepgen, today reported phase 1 healthy volunteer data with PGN-EDO51 that it says support its case. The small player, one of 2022’s few IPOs, still has much to prove, but if it can meet its goals for phase 2 Sarepta could be looking over its shoulder.