REGENXBIO, co-founded by James M. Wilson, MD, PhD, the Rose H. Weiss Orphan Disease Center Director’s Professor in Penn Medicine, announced that the FDA has accepted their Biologics License Application for its gene therapy candidate for Hunter syndrome, potentially the first one-time treatment for this disorder. Read more here.
REGENXBIO also released promising data on its gene therapy candidate for Duchenne muscular dystrophy (DMD). Read more here.