Opus Genetics reports gene therapy trial data for two eye diseases

Opus Genetics, Inc. (Nasdaq: IRD) presented clinical data from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at the Retina in Rio 2026 conference in Rio de Janeiro, Brazil, on Oct. 15, 2026.

In the Phase 1/2 BIRD-1 trial of OPGx-BEST1, three of four evaluable participants in the low-dose Cohort 1 showed improved retinal sensitivity in the treated transitional zone, representing 75% of that group. Follow-up ranged from three to six months across five total participants. The company said additional analyses identified retinal structural and imaging characteristics that may help predict treatment response. Enrollment in the high-dose Cohort 2, comprising 11 participants, has been completed.

For OPGx-LCA5, data from the Phase 1/2 trial showed sustained improvements in visual acuity and light sensitivity through 24 months in three adult participants treated for Leber congenital amaurosis type 5. Three pediatric participants showed improvements through six months, including restoration of cone-mediated vision with sensitivity reaching normal ranges. Both groups showed improved light sensitivity compared with untreated eyes.

Participants in the OPGx-LCA5 trial also reported improvements in everyday activities such as recognizing family members and navigating independently.

Based on Phase 1/2 findings, the company advanced OPGx-LCA5 to Phase 3 development at the low dose. Natural history and treatment cohorts for the Phase 3 trial are fully enrolled, with dosing initiation expected in the fourth quarter of 2026. The primary efficacy endpoint will assess retinal sensitivity across 16 central retinal locations.

"These findings strengthen our understanding of how to measure treatment response in IRDs and support the continued advancement of both programs," said George Magrath, M.D., Chief Executive Officer of Opus Genetics.

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