Opus Genetics reports positive Phase 1/2 low-dose Cohort 1 data for OPGx-BEST1 gene therapy in BEST1 retinal disease

PUBT · 2d ago
Opus Genetics reports positive Phase 1/2 low-dose Cohort 1 data for OPGx-BEST1 gene therapy in BEST1 retinal disease
  • 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.
  • 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 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.


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Opus Genetics Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: 202609090700PRIMZONEFULLFEED9823429) on September 09, 2026, and is solely responsible for the information contained therein.