REGENXBIO Announces Orphan Drug Designation Granted to RGX-202, a Novel Gene Therapy Candidate for the Treatment of Duchenne Muscular Dystrophy
ROCKVILLE, Md., Nov. 22, 2021 /PRNewswire/ --
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.
"This important designation is a milestone in the development of RGX-202 and highlights the need for potential new treatment options for patients with Duchenne," said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. "The novel microdystrophin transgene in RGX-202 includes coding regions that retain essential functional elements of naturally occurring dystrophin to potentially improve muscle strength and resistance in patients with Duchenne. We look forward to advancing this one-time gene therapy into the clinic."
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.
FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.
RGX-202 is designed to deliver a novel microdystrophin transgene which retains key elements of the dystrophin protein, including an extended coding region of the C-Terminal (CT) domain found in naturally-occurring dystrophin, as well as other fundamental improvements to the transgene. Presence of the CT domain has been shown to recruit several key proteins to the muscle cell membrane, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic mice. Additional design features, including codon optimization and reduction of CpG content, may potentially improve gene expression, increase translational efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of genes throughout skeletal and heart muscle using the NAV AAV8 vector, a vector used in numerous clinical trials, and a well-characterized muscle targeting promoter (Spc5-12).
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
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SOURCE REGENXBIO Inc.
Company Codes: NASDAQ-NMS:RGNX