Gene therapy platform for blinding retinal diseases — with LCA5 Phase 3 enrolled and BEST1 Phase 1 data positive — contingent on single-arm Phase 3 meeting FDA's evidentiary bar

Opus Genetics is building a gene therapy pipeline for rare inherited retinal diseases, with OPGx-LCA5 for Leber congenital amaurosis — a childhood-onset blinding condition — as the lead: its Phase 1/2 pediatric cohort showed approximately 1.5 log-unit cone sensitivity gains reaching normal ranges after a single dose, and FDA RDEP acceptance allows a single-study approval path. The thesis resolves on whether that single-arm eight-participant Phase 3 satisfies FDA's evidentiary standard for effectiveness. The primary approval-path risk is the reliance on natural history as external control in a small adaptive trial, where unexplained variability in the control arm could undermine the regulatory case.