Opus Genetics reports early positive data from gene therapy eye trial

Opus Genetics, Inc. (NASDAQ: IRD) announced positive three- and six-month results from the low-dose Cohort 1 of its Phase 1/2 clinical trial, BIRD-1, evaluating gene therapy 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. All five showed improvement in at least one visual function measure, including best-corrected visual acuity, low-luminance visual acuity, contrast sensitivity, or microperimetry. Structural improvements were observed in four of the five participants. The company noted that greater functional gains were seen in participants with less advanced disease.

OPGx-BEST1 showed no serious adverse events, no dose-limiting toxicities, no intraocular inflammation, and no significant safety-laboratory findings. All treatment-related adverse events were mild or moderate.

In August 2026, Opus Genetics met with the U.S. Food and Drug Administration to discuss potential pivotal trial endpoints. The company said it aligned with the FDA on a potential primary endpoint based on a microperimetry improvement of at least 3 decibels in at least five prespecified locations, paired with a patient-reported outcome measure in a randomized, controlled trial. The company expects to begin Phase 3 participant dosing in 2027.

Cohort 2, evaluating OPGx-BEST1 at a higher dose of 4.5 x 10⁹ vg/eye, has enrolled eight participants, exceeding its original target of five. Dosing is expected to be completed in the fourth quarter of 2026, with three-month topline data anticipated in the second quarter of 2027.

New epidemiology research commissioned by the company estimated approximately 23,600 symptomatic BEST1 patients in the U.S. and approximately 45,400 globally, figures described as substantially higher than prior estimates.

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