We are very pleased to share the following developments on REGENEXBIO’s first-in-human gene therapy program for the treatment of CLN2 Batten disease.
REGENEXBIO announced that the first patient was dosed in the Phase 1/11 ocular trial of RGX-381 at Great Ormond Street Hospital (GOSH) in the UK. RGXRGX-381 is an investigational one-time AAV gene therapy for the treatment of ocular manifestations of CLN2 disease, using the NAV® AAV9 to deliver the TPP1 gene directly to the retina.
Initial data from this trial is expected to be shared in 2024.
REGENEXBIO also shared an update on the RGX-181 targeted gene therapy program for brain/central nervous system (CNS). Physicians in Brazil continue to follow the first child with CLN2 disease dosed with RGX-181 under a single-patient investigator-initiated study.
REGENEXBIO expect investigators to report initial interim data from this study, including 6-month results, at the upcoming SSIEM (Society for the Study of Inborn Errors of Metabolism) Annual Symposium in late August.
RGX-181 is an investigational one-time AAV therapeutic for the treatment of CLN2, using the NAV AAV9 vector to deliver the TPP1 gene directly to the CNS.
For further information please see REGENEXBIO’s press release here: