GlobeNewswire·Nov 5, 2026, 7:00 AM ET

Opus Genetics Completes Dosing in Cohort 2 of Phase 1/2 Clinical Trial of OPGx-BEST1 Gene Therapy for Inherited Retinal Diseases

Opus Genetics, Inc. completed dosing in Cohort 2 of the BIRD-1 Phase 1/2 trial for OPGx-BEST1 at a dose of 4.5 × 10⁹ vg/eye, which is three times the Cohort 1 dose.
The higher-dose Cohort 2 enrolled 11 participants, exceeding its original target of five.
Topline three-month data for Cohort 2 is expected in the first quarter of 2027, with a potential pivotal Phase 3 trial anticipated to begin in the second half of 2027.
Cohort 1 results demonstrated favorable safety with no serious adverse events, dose-limiting toxicities, or intraocular inflammation.
All five Cohort 1 participants showed clinically meaningful improvements in visual function, including best-corrected visual acuity, low-luminance visual acuity, or contrast sensitivity.
Structural improvements were observed in four of five Cohort 1 participants, including reductions in vitelliform material for BVMD patients and intraretinal fluid for ARB patients.
The FDA aligned with Opus Genetics on a potential pivotal endpoint requiring at least three decibels of improvement in microperimetry across five or more retinal loci plus a patient-reported outcome.
Phase 3 commercial manufacturing requirements are expected to be completed in early 2027.
OPGx-BEST1 is an investigational gene therapy using an AAV vector to deliver a functional BEST1 gene to treat BEST1-related inherited retinal diseases, for which no approved therapies currently exist.