Opus Genetics launches phase 1/2 MERTK gene therapy trial

Opus Genetics has launched its clinical trial evaluating OPGx-MERTK gene therapy for MERTK-related retinitis pigmentosa (RP). The trial is funded through Abu Dhabi’s Healthcare Research and Innovation Fund.

OPGx-MERTK is an investigational adeno-associated virus (AAV)-based gene therapy designed to deliver a functional copy of the MERTK gene to retinal cells. The trial is being launched in Abu Dhabi, in collaboration with the Department of Health (DoH), Cleveland Clinic Abu Dhabi, the Innovative Research Oversight and Support (IROS) division of the M42 group, and the Authority of Social Contribution, Ma’an. Cleveland Clinic Abu Dhabi will be the clinical site for the trial.¹

The company noted that the Opus MERTK program is expected to commence clinical development activities in 2026.¹

George Magrath, MD, CEO of Opus Genetics, commented on the launch, saying, “Launching our clinical trial for a MERTK gene therapy is a defining moment for patients and for the field of inherited retinal disease. […] For patients living with MERTK-related retinitis pigmentosa, this trial represents the first real opportunity to potentially change the course of a disease that has historically led to inevitable vision loss.”

Dr Fahed Al Marzooqi, CEO of M42's Integrated Health Solutions, added, “This trial represents a pivotal step in strengthening Abu Dhabi’s position as a global hub for biotech innovation. Through strategic partnerships and clinical excellence, we are translating scientific breakthroughs into real-world treatments for patients with urgent, unmet needs.”

At the end of 2025, Opus Genetics completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the US Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene.²

The trial is a phase 1/2 open-label, ascending-dose study of the safety and efficacy of OPGx-LCA5. Recently, the company released positive 3-month pediatric data, showing significant measurable gains in vision in teenagers and maintenance of visual gains to 18 months in adults who were treated earlier.

Additionally, at the end of 2025, the company dosed the first patient in its OPGx-BEST1 phase 1/2 clinical trial, BIRD-1, for Best disease (BEST1).

BIRD-1 is a multi-center, adaptive, open-label, dose-exploring study evaluating the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in patients with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). The trial will consist of 2 dosing cohorts and will explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.²

Initial data from the trial is expected in the first quarter of 2026.

References:

1. Opus Genetics Launches Gene Therapy Clinical Trial for MERTK-related Retinitis Pigmentosa. Published January 27, 2026. Accessed January 27, 2026. https://ir.opusgtx.com/press-releases/detail/516/opus-genetics-launches-gene-therapy-clinical-trial-for-mertk-related-retinitis-pigmentosa

2. Harp MD. Opus Genetics doses first patient in phase 1/2 clinical trial, BIRD-1, evaluating OPGx-BEST1. Published November 17, 2025. Accessed January 27, 2026. https://www.ophthalmologytimes.com/view/opus-genetics-doses-first-patient-in-phase-1-2-clinical-trial-bird-1-evaluating-opgx-best1