OPGx-BEST1

June 16, 2026: Opus Genetics, Inc. (NASDAQ: IRD) hosted an R&D Science Forum outlining five inherited retinal disease (IRD) gene therapy programs and upcoming clinical catalysts.

OPGx-RDH12 (RDH12-associated Leber congenital amaurosis): Preclinical studies showed in vivo delivery of functional RDH12 enzyme with restored structure/function in a small animal model. Clinical testing in the U.S. is expected to start in Q4 2026 (funded by the RDH12 Alliance).

OPGx-LCA5 (LCA5-associated LCA): In an ongoing Phase 1/2 trial, visual acuity improved and was maintained in adults over 24 months and improved in pediatrics over 6 months; FST showed durable vision improvement and microperimetry suggested increased sensitivity and fixation movement toward the fovea. Run-in recruitment for a pivotal Phase 3 is ongoing; Phase 3 dosing is expected to begin in Q4 2026, alongside manufacturing of clinical/commercial drug product.

OPGx-BEST1 (BEST1 retinal degeneration; BVMD and ARB): Phase 1/2 Cohort 1 (3 BVMD, 2 ARB) dosing completed in May 2026; 3-month topline data are expected in September 2026.

OPGx-MERTK (MERTK retinitis pigmentosa): Preclinical evidence of preserved retinal structure/function and outer nuclear layer thickness in a small animal model; clinical testing at Cleveland Clinic Abu Dhabi expected in Q1 2027 (funded by a consortium led by Abu Dhabi’s Healthcare Research and Innovation Fund).

OPGx-RHO (autosomal-dominant RHO retinitis pigmentosa): “Silence and replace” approach; clinical testing expected globally in 2H 2027.