Sarepta Therapeutics, Inc.

With 48-week data from a Phase II open-label trial in Duchenne muscular dystrophy (DMD) showing an advancement in clinical benefit beyond what a 24-week readout showed last year, Wave Life Sciences sa

The ultra-rare disease specialist Ultragenyx is approaching several important catalysts this year, including US Food and Drug Administration action on UX111 – the firm’s first gene therapy, which targ

It’s a big year for Regenxbio. The company has a potential commercial launch of its gene therapy program for MPS II (Hunter syndrome), a Biologics License Application for its Duchenne muscular dystrop

New Phase I/II data from its Duchenne gene therapy candidate, RGX-202, have boosted Regenxbio, just as new concerns emerged about the safety of Sarepta’s established gene therapy, Elevidys (delandistr