BioHealth Capital Region

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

By Cat Thoreson · · 1 min read


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. 



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.

Read the full article at: regenxbio.gcs-web.com

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Cat Thoreson

Cat Thoreson

Chief Operating Officer at BioBuzz

A gritty, resourceful operator with a clear line of sight from strategy to execution — she excels at building high-performing teams and driving scalable growth.

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