STAT+: Wagering on FDA changes, Regenxbio will submit Duchenne gene therapy for approval

Regenxbio announced it will submit its Duchenne muscular dystrophy gene therapy for accelerated approval, despite the FDA previously requesting an additional trial. The FDA appears to be reversing recent rejections of gene therapies, including a Hunter syndrome treatment reconsidered after key officials, Marty Makary and Vinay Prasad, left the agency.