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Marks a milestone in the development of RGX-202 and highlights the need for potential new treatment options for patients with Duchenne.
November 22, 2021
By: Charlie Sternberg
Associate Editor
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for Regenxbio’s 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. ...
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