Opus Genetics reports positive Phase 1/2 low-dose Cohort 1 data for OPGx-BEST1 gene therapy in BEST1 retinal disease
IRD•FDA discussions and development path
FDA discussions supported a potential pivotal endpoint framework, clearing a path toward Phase 3 planning with dosing targeted to start in 2027.
Higher-dose cohort and market opportunity
Higher-dose Cohort 2 was over-enrolled; dosing is expected to finish in Q4 2026, with topline 3-month results due in Q2 2027.
New epidemiology work pointed to a larger symptomatic BEST1 patient pool, supporting a bigger commercial opportunity than prior estimates.
Positive early data from low-dose Cohort 1
Opus Genetics reported positive early results from the low-dose Cohort 1 of its Phase 1/2 OPGx-BEST1 eye-gene therapy trial.
The treated patients showed clinically meaningful visual-function gains, with retinal-structure improvements in most participants; safety was favorable with no serious events.
