Opus Genetics outlines five gene therapy programs for retinal diseases

Opus Genetics, Inc. (NASDAQ: IRD) held a Research and Development Science Forum on June 16, 2026, where management presented updates on five gene therapy programs targeting inherited retinal diseases (IRDs).

The company said three programs — OPGx-RDH12, OPGx-MERTK, and OPGx-RHO — are expected to enter clinical testing within the next 12 to 18 months. OPGx-RDH12, targeting a severe form of Leber congenital amaurosis, is expected to begin U.S. clinical testing in the fourth quarter of 2026, with funding supported by the RDH12 Alliance. OPGx-MERTK, targeting autosomal recessive retinitis pigmentosa, is expected to initiate clinical testing at the Cleveland Clinic Abu Dhabi in the first quarter of 2027, funded by a consortium led by Abu Dhabi's Healthcare Research and Innovation Fund. OPGx-RHO, targeting autosomal-dominant retinitis pigmentosa, is expected to begin global clinical testing in the second half of 2027.

For its two programs already in clinical trials, Opus reported that OPGx-LCA5 showed improved and maintained visual acuity in an adult cohort over 24 months and improvement in a pediatric cohort over six months. Dosing in a pivotal Phase 3 trial is expected to begin in the fourth quarter of 2026. For OPGx-BEST1, dosing in the first cohort of a Phase 1/2 trial was completed in May 2026, with three-month topline data expected in September 2026.

According to a Triangle Insights analysis of five key markets, estimated global patient prevalence across the five programs ranges from approximately 3,240 patients for LCA5 to 30,900 for RDH12.

The company said its cash runway is expected to extend into 2029, which it projects will fund multiple clinical milestones. Four clinical data readouts are anticipated in 2027.

Chief Executive Officer George Magrath stated the company has "first-mover advantage across multiple indications, supported by broad intellectual property protection, rare-disease regulatory pathways."

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