Potential one-time gene therapy for the treatment of Duchenne, includes a novel, optimized microdystrophin transgene and REGENXBIO's proprietary NAV® AAV8 vector
Commercial-scale cGMP material to be used in clinical development
Company on track to submit IND by end of 2021
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.
https://finance.yahoo.com/news/regenxbio-announces-orphan-drug-designation-120000054.html
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