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REGENXBIO Announces FDA Clearance of IND for Clinical Trial of RGX-202, a Novel Gene Therapy Candidate for Duchenne Muscular Dystrophy
ROCKVILLE, Md., Jan. 6, 2022 /PRNewswire/ -- Potential one-time gene therapy for the treatment of Duchenne includes a novel, optimized microdystrophin
About this update from Regenxbio Inc.
[{"type":"text","content":"ROCKVILLE, Md., Jan. 6, 2022 /PRNewswire/ -- \nPotential one-time gene therapy for the treatment of Duchenne includes a novel, optimized microdystrophin transgene and REGENXBIO's proprietary NAV® AAV8 vector Innovative trial design, including comprehensive immunosuppressive regimen, to evaluate safety and optimal dose cGMP process material made at commercial-scale to be used throughout clinical development of RGX-202 REGENXBIO expects to initiate trial in the first half of 2022REGENXBIO Inc. (Nasdaq: RGNX) today announced the clearance of its Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA) to evaluate RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne) in a first-in-human clinical trial. RGX-202 is designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 uses REGENXBIO's proprietary NAV® AAV8 vector. REGENXBIO plans to initiate the trial in the first half of 2022.\n\"We are excited to advance RGX-202, our investigational gene therapy for patients with Duchenne, into the clinic. Additional therapeutic options are still needed for the treatment of Duchenne, and our trial design follows compelling evidence from preclinical studies which demonstrated that one-time treatment with RGX-202 can express meaningful levels of a novel, functional microdystrophin protein in muscle, and showed significant improvements in muscle force and function in animal models,\" said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. \"This innovative AAV gene therapy candidate for Duchenne was developed in-house at REGENXBIO through a highly collaborative process between our expert research and manufacturing teams and we believe that RGX-202 can potentially address unmet needs for patients with Duchenne.\"\nAFFINITY DUCHENNETM Trial Design The Phase I/II trial, named AFFINITY DUCHENNE, is a multicenter, open-label dose escalation and dose expansion clinical study to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in patients with Duchenne. In the dose escalation phase of the trial, six ambulatory, pediatric patients (ages 4 to 11 years old) with Duchenne are expected to en...