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Additional Positive Interim Data from Phase I/II Trial of REGENXBIO'S RGX-111 for the Treatment of Severe MPS I Presented at WORLDSymposium™
RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative
About this update from Regenxbio Inc.
[{"type":"text","content":"RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative disease programsRGX-111, a potential one-time gene therapy for MPS I, continues to be well-tolerated across two dose levels, with no drug-related serious adverse eventsNew biomarker and neurodevelopmental data continue to indicate encouraging CNS profile in patients dosed with RGX-111 Company is on track to manufacture commercial-scale cGMP material to support the continued development of RGX-111 ROCKVILLE, Md., Feb. 24, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive interim data from the Phase I/II trial of RGX-111 for the treatment of severe Mucopolysaccharidosis Type I (MPS I). Data from the Phase I/II trial and single-patient Investigational New Drug (IND) application of RGX-111 were presented by Ray Wang, M.D., Campbell Foundation Director of the Multidisciplinary Lysosomal Program, Division of Metabolic Disorders, CHOC Children's Hospital, Department of Pediatrics, University of California, Irvine, at the 19th Annual WORLDSymposium™.\n\"RGX-111 is our second-most advanced clinical candidate in our neurodegenerative disease pipeline and is part of our '5x'25' strategy to have five gene therapies either on the market or in late-stage development by 2025. We are encouraged to see that this potential one-time gene therapy using our NAV AAV9 vector continues to demonstrate compelling evidence of CNS biomarker activity,\" said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. \"In connecting with MPS I families, we understand the need for new treatment options that can impact daily living, and we're pleased to see that most patients in this trial demonstrated continued skill acquisition across multiple neurodevelopmental assessments.\"\n\"There is a great need for new treatment options that provide lasting expression of the IDUA enzyme and the reduction of glycosaminoglycans in the central nervous system,\" said Dr. Wang. \"I am encouraged by the emerging clinical profile of RGX-111 based on this data, including the important neurodevelopment gains in cognition, language, fine motor skills and personal and social skills for daily living.\"\nRGX-111 is an investigational one-time gene therapy designed to deliver the gene that encod...