OPGx-MERTK / Opus Genetics - LARVOL DELTA

OPGx-MERTK (The Manila Times) - "OPGx-MERTK is designed to restore critical retinal pigment epithelium metabolic functions....In preclinical studies, OPGx-MERTK provided evidence of retinal preservation of structure/function and preserved outer nuclear layer thickness in a small animal model of retinal degeneration....This program is expected to initiate clinical testing at the Cleveland Clinic Abu Dhabi in the first quarter of 2027."

New trial • Preclinical • Retinitis Pigmentosa

Opus Genetics Launches Gene Therapy Clinical Trial for MERTK-related Retinitis Pigmentosa (GlobeNewswire) - "The clinical trial will evaluate the safety and efficacy of OPGx-MERTK, an investigational adeno-associated virus (AAV)-based gene therapy developed by Opus Genetics. The therapy is designed to deliver a functional copy of the MERTK gene to retinal cells....The trial is funded through Abu Dhabi’s Healthcare Research and Innovation Fund. Clinical development activities will commence at Cleveland Clinic Abu Dhabi in 2026."

New trial • Retinitis Pigmentosa

OPGx-MERTK - Advancing with Non-Dilutive Support (The Manila Times) - "Non-dilutive funding up to $2 million received from the Retinal Degeneration Fund to advance OPGx-MERTK, targeting retinitis pigmentosa caused by MERTK mutations."

Financing • Retinitis Pigmentosa

Evaluation of MERTK gene therapy in RCS rats following a single bilateral subretinal injection. (ASGCT 2025) - "These data demonstrate that in the RCS rat model of retinal degeneration, 1E10 vg/eye OPGx-MERTK effectively preserved the photoreceptors and retinal function. Disease Focus of Abstract:Eye Disorders"

Gene therapy • Preclinical • Gene Therapies • Hematological Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • MERTK

Opus Genetics Announces Presentations on Inherited Retinal Disease Programs at Medical Conferences in May (GlobeNewswire) - "In this rat model of retinal degeneration, subretinal administration of OPGx-MERTK (1E10 vg/eye) demonstrated effective preservation of both photoreceptors and retinal function....This preclinical study evaluated the efficacy of OPGx-MERTK in a Royal College of Surgeons (RCS) rat model, which exhibit a RPE phagocytosis defect due to a natural mutation in MERTK, following a single bilateral subretinal injection....Results from a preclinical study evaluating the safety and efficacy of OPGx-BEST1 in a canine model of BEST1 related IRD will be presented....Opus plans to commence a Phase 1/2 trial of OPGx-BEST1 in 2025 and aims to obtain preliminary data by Q1 2026."

New P1/2 trial • Preclinical • Macular Degeneration • Retinitis Pigmentosa