Regenxbio announced that the Phase 1/2 AFFINITY Duchenne trial of RGX-202 for the treatment of Duchenne muscular dystrophy is now active and recruiting patients. RGX-202 is designed to deliver a transgene for a novel microdystrophin protein that includes the functional elements of the C-Terminal domain found in naturally occurring dystrophin. RGX-202 uses Regenxbio’s proprietary NAV AAV8 vector. The trial is an open-label dose evaluation and dose expansion clinical trial to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous dose of RGX-202 in patients with Duchenne.
Published first on TheFly
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