REGENXBIO Gets FDA's Orphan Drug Designation For RGX-202

(RTTNews) - REGENXBIO Inc. (RGNX), a biotechnology company, said Monday that the U.S. Food and Drug Administration or FDA has granted Orphan Drug Designation for RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy or Duchenne. RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with C-terminal domain and a muscle specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne. RGX-202 uses REGENXBIO's proprietary NAV AAV8 vector.

Olivier Danos, Chief Scientific Officer of REGENXBIO, said, "The novel microdystrophin transgene in RGX-202 includes coding regions that retain essential functional elements of naturally occurring dystrophin to potentially improve muscle strength and resistance in patients with Duchenne. We look forward to advancing this one-time gene therapy into the clinic."

REGENXBIO expects to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021.