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REGENXBIO Reports Update on Advancement of Programs for CLN2 Disease
RGX-181 and RGX-381 are potential one-time AAV Therapeutics for the treatment of the CNS and ocular manifestations of CLN2 disease, the most common form of
About this update from Regenxbio Inc.
[{"type":"text","content":"RGX-181 and RGX-381 are potential one-time AAV Therapeutics for the treatment of the CNS and ocular manifestations of CLN2 disease, the most common form of Batten diseasePatient dosed under a single-patient investigator-initiated study of RGX-181Company announces approval of CTA for RGX-381 from the UK Health Authority and plans to initiate a Phase I/II clinical trial in the first half of 2023ROCKVILLE, Md., Dec. 21, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced a comprehensive program update to outline its progress and development plans for RGX-181 and RGX-381, both being developed as potential one-time gene therapies for the treatment of different manifestations in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, the most common form of Batten disease.\nREGENXBIO announced today that physician investigators at the Hospital de Clinicas in Porto Alegre, Brazil have dosed the first child with CLN2 disease with RGX-181 in a single-patient, investigator-initiated study. As of December 20, 2022, RGX-181 is reported to be well-tolerated in this patient with no drug-related SAEs. Hospital de Clinicas is also part of ongoing clinical trials of RGX-111 for the treatment of MPS I and RGX-121 for the treatment of MPS II.\nRGX-181 is an investigational one-time AAV Therapeutic designed to use the NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system (CNS), which could induce sustained levels of TPP1 to treat the neurodegenerative manifestations of CLN2 disease such as progressive loss of language, intellectual abilities, and motor skills.\n\"We are pleased to have the opportunity to participate in the first-in-human study of RGX-181 for patients with CLN2,\" said Roberto Giugliani MD, PhD, Hospital de Clinicas. \"Patients with this devastating disease need new treatment options with the potential for longer lasting effects on the CNS, and we hope that this gene therapy benefits this patient community,\" added Carolina Fischinger, MD, PhD, primary investigator of the single-patient study.\nREGENXBIO also announced today that a clinical trial application (CTA) has been accepted by the UK Health Authority to support a first-in-human, open-label, dose-escalation Phase I/II clinical trial to evaluate the safety and tolerability, as well as the effect on retinal anatomic and func...