At the 2026 meeting of the Association for Research in Vision and Ophthalmology (ARVO), Opus Genetics presented clinical and preclinical data from multiple inherited retinal disease programs, including updated findings from its investigational gene therapy OPGx-LCA5.

In an oral presentation, Tomas S. Aleman, MD, reported 6-month data from an ongoing phase 1/2/3 study evaluating OPGx-LCA5 in pediatric patients with Leber congenital amaurosis type 5. According to investigators, treated patients demonstrated restoration of cone-mediated visual function after a single subretinal injection despite severe baseline disease.

The company reported more than 30-fold improvements in cone sensitivity across treated patients, along with gains in visual acuity relative to untreated eyes. Additional findings included improvements in pupillary light responses, orientation and mobility testing, and patient-reported functional vision. Investigators said the therapy was well tolerated, with no dose-limiting toxicities and only mild adverse events.

Opus also presented early clinical data from OPGx-BEST1, including a reported 12-letter visual acuity improvement in 1 treated adult participant, as well as preclinical updates from mutation-independent RHO programs for autosomal-dominant retinitis pigmentosa.

The company said it expects topline cohort 1 data from its phase 1/2 OPGx-BEST1 study in September 2026.