Regenxbio announces program update for RGX-181 and RGX-381

REGENXBIO 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, or CLN2, disease, the most common form of Batten disease. REGENXBIO announced 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, 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. REGENXBIO also announced that a clinical trial application, or 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 functional outcomes, of the subretinal delivery of RGX-381 for the treatment of ocular manifestations of CLN2 disease. The trial will be conducted at Great Ormond Street Hospital in London and the Company expects to initiate the Phase I/II clinical trial in the first half of 2023.