REGENXBIO Announces Orphan Drug Designation Granted to RGX-202, a Novel Gene Therapy Candidate for the Treatment of Duchenne Muscular Dystrophy

REGENXBIO Inc. (Nasdaq: RGNX) today announced the U.S. 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 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. 

“This important designation is a milestone in the development of RGX-202 and highlights the need for potential new treatment options for patients with Duchenne,” said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. “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. Commercial-scale cGMP material has already been produced at 1,000 liter capacity using REGENXBIO’s suspension cell culture manufacturing process, and the Company’s internal cGMP facility is expected to allow for production up to 2,000 liters for the clinical development of RGX-202.

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