Opus Genetics, Inc. (Nasdaq: IRD) issued a press release on October 15, 2026, detailing clinical data presented at the Retina in Rio 2026 conference held in Rio de Janeiro, Brazil. The company provided updates on two ongoing gene therapy programs: OPGx-BEST1 and OPGx-LCA5.
Regarding the OPGx-BEST1 program, which is in a Phase 1/2 trial for Best vitelliform macular dystrophy and autosomal recessive bestrophinopathy, the company presented data from the initial low-dose Cohort 1. The data included results from five participants with follow-up ranging from three to six months. Among four participants who completed retinal sensitivity testing, three demonstrated improvements in the treated transitional zone, an area surrounding damaged retinal tissue. The company noted that improvements in retinal sensitivity were associated with reductions in intraretinal fluid characteristic of BEST1-associated disease.
For the OPGx-LCA5 program, an investigational gene therapy for Leber congenital amaurosis type 5, the company presented data from the ongoing Phase 1/2/3 trial. The presentation included results from six participants treated at a low dose of 1E10 vector genomes per eye, including three adults and three adolescents. Adults demonstrated sustained improvements in visual acuity through 24 months, while pediatric participants showed restoration of cone-mediated vision with sensitivity improvements reaching normal ranges through six months. Both groups also demonstrated improved light sensitivity as measured by full-field stimulus testing.
Based on these findings, Opus Genetics advanced OPGx-LCA5 into Phase 3 development using the low dose without further dose escalation. The company stated that the Phase 3 clinical trial’s natural history and treatment cohorts are fully enrolled, with dosing initiation expected in the fourth quarter of 2026. Additionally, the company reported that the OPGx-BEST1 Phase 1/2 trial has completed enrollment in the high-dose Cohort 2 with 11 participants.
