OPGx-BEST1 demonstrated a favorable safety and tolerability profile with no serious adverse events or dose-limiting toxicities observed 

All five participants demonstrated clinically meaningful improvement in visual function, with structural improvements observed in four participants

FDA aligned on ≥3 decibels microperimetry improvement in conjunction with patient reported outcomes as a potential pivotal endpoint 

Clinically meaningful best-corrected visual acuity improvements in 3 of 5 participants and retinal sensitivity improvements on microperimetry observed in 3 of 4 evaluable participants

Cohort 2 over-enrolled, with dosing expected to be completed in Q4 2026 and topline 3-month data expected in Q2 2027

Cash runway into 2029 expected to support multiple clinical inflection points and opportunities for priority review vouchers

Webcast and conference call today at 8:00 a.m. ET with management and Key Opinion Leader and retinal specialist, Mark Pennesi, M.D., PhD.

RESEARCH TRIANGLE PARK, N.C., Sept. 09, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (NASDAQ:IRD) (the "Company" or "Opus Genetics"), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced positive 3- and 6-month results from the low-dose Cohort 1 of BIRD-1, its ongoing Phase 1/2 clinical trial evaluating OPGx-BEST1 in patients with BEST1-related retinal diseases, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).

Cohort 1 enrolled five participants treated at 1.5 x 10⁹ vg/eye: three participants with BVMD who have reached three months of follow-up and two with ARB who have reached six months of follow-up. All five participants demonstrated clinically meaningful improvement in visual function, measured as one or more of the following: best-corrected visual acuity (BCVA), low-luminance visual acuity (LLVA), contrast sensitivity (CS) or microperimetry, an advanced eye test that maps how well the central part of the retina sees light. Structural improvements were also observed across four participants. The greatest functional gains were observed in participants with less advanced disease, supporting the potential benefit of treating patients while viable retinal tissue remains. The Company expects to announce 6-month data for the three participants with BVMD in Q2 2027.