RGX-202 is an investigational gene therapy utilizing a novel microdystrophin construct for the treatment of Duchenne Muscular Dystrophy (Duchenne). Duchenne is a severe, progressive, degenerative muscle disease caused by mutations in the DMD gene, which encodes dystrophin, a protein involved in muscle...
Regenxbio Inc. announced the US Food and Drug Administration (FDA) granted Orphan Drug Designation for RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with a unique...
(Nasdaq: RGNX) today announced that it will host a webcast to discuss the AFFINITY DUCHENNE® pivotal program and new clinical data, including the first functional data from the ongoing Phase I/II study of RGX-202, the company's next-generation gene therapy for the treatment...