Opus Genetics Launches Gene Therapy Clinical Trial for MERTK-Related Retinitis Pigmentosa in Abu Dhabi

Opus Genetics has announced the launch and funding of a clinical trial evaluating a gene therapy for MERTK-related retinitis pigmentosa (RP), a rare inherited retinal disease that leads to progressive vision loss and eventual blindness.

The study will be conducted in Abu Dhabi, United Arab Emirates, in collaboration with the Department of Health – Abu Dhabi (DoH), Cleveland Clinic Abu Dhabi, the Innovative Research Oversight and Support (IROS) division of M42, and the Authority of Social Contribution – Ma’an. Cleveland Clinic Abu Dhabi will serve as the clinical site.

Addressing an area of high unmet need

MERTK-related retinitis pigmentosa is caused by mutations in the MERTK gene, which impair the retina’s ability to recycle photoreceptor components. Over time, this leads to photoreceptor degeneration and irreversible vision loss.

Key context:

• No approved treatments currently exist for MERTK-related RP
• Around 60,000 patients worldwide are affected
• Inherited retinal diseases impact an estimated 5% of the population in the region

This trial represents the first clinical effort specifically targeting this genetic subtype of RP.

About the investigational therapy: OPGx-MERTK

The study will evaluate OPGx-MERTK, an 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
• Restore cellular recycling processes essential for photoreceptor survival
• Potentially slow or halt disease progression with a one-time treatment

Clinical development activities for the Opus MERTK program are expected to begin in 2026.

Strategic collaboration in Abu Dhabi

Cleveland Clinic Abu Dhabi will support the trial with:

• Advanced retinal imaging and diagnostics
• Specialized retinal disease clinics
• Surgical expertise in ophthalmic gene therapy delivery

Regional health authorities and research organizations are supporting the program as part of Abu Dhabi’s broader push into precision medicine and rare disease innovation.

What company leaders are saying?

George Magrath, MD, CEO of Opus Genetics, said the trial marks a defining moment for patients living with MERTK-related RP and offers the first real opportunity to potentially alter the disease course.

H.E. Dr. Noura Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, noted that hosting the first MERTK gene therapy trial in the UAE demonstrates Abu Dhabi’s leadership in precision medicine and rare disease research.

Dr. Fahed Al Marzooqi, CEO of M42’s Integrated Health Solutions, highlighted the role of strategic partnerships in translating scientific breakthroughs into real-world treatments.

Why this matters?

The launch of this trial signals:

• Continued momentum in ocular gene therapy development
• Growing global investment in genetic subtypes of retinal disease
• Expansion of advanced clinical research capabilities in the Middle East

For patients with MERTK-related RP, it represents a long-awaited step toward a potential disease-modifying therapy.

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