Abu Dhabi launches UAE’s first gene therapy clinical trial targeting MerTK-related retinitis pigmentosa

ABU DHABI, 23rd March, 2026 (WAM) — The Department of Health – Abu Dhabi has announced the launch and funding of a UAE-first clinical trial for a novel gene therapy for MerTK-related retinitis pigmentosa (RP) developed by Opus Genetics.

This rare inherited eye disease causes progressive vision loss and eventual blindness, with no approved treatments currently available. This groundbreaking initiative reflects Abu Dhabi’s commitment to advancing innovative solutions for rare conditions and reinforces the Emirate’s position as a global hub for life sciences and biomedical innovation.

The first-of-its-kind clinical trial is the result of a collaborative effort between the Department of Health – Abu Dhabi and the Authority of Social Contribution – Ma’an, reflecting their shared commitment to advancing healthcare research and innovation in Abu Dhabi.

The initiative brings together Opus Genetics (a US-based clinical-stage biopharmaceutical company), the Innovative Research Oversight and Support (IROS) division of the M42 group, Cleveland Clinic Abu Dhabi to accelerate the development of novel gene therapies for Inherited Retinal Diseases, which affect approximately five percent of the population in this region.

Cleveland Clinic Abu Dhabi will serve as the clinical site, while IROS will oversee the trial design, patient recruitment and clinical operations.

Clinical development activities are expected to commence in 2026, marking a milestone for rare disease treatment in Abu Dhabi and the region.

The study will evaluate the safety and efficacy of a novel AAV-based gene therapy developed by Opus Genetics for this rare condition. The therapy targets the MerTK gene, which fails to properly recycle protein in the retina when it has mutated, leading to progressive vision loss. Adeno-associated viruses (AAVs) are widely recognised as safe and effective vectors for delivering healthy genes into cells, making them a potentially powerful tool for treating inherited diseases.

Dr. Noura Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, said, “In collaboration with our international and local partners, we are proud to support the development of a novel therapy that could transform the lives of patients living with inherited blindness. This milestone brings renewed hope to patients and reinforces our position as a trusted destination for advanced healthcare and life sciences research.”

Abdullah Al Ameri, Director-General of the Authority of Social Contribution – Ma’an, said, “Supporting the health sector by deploying community funds to projects that impact lives is one of the Authority of Social Contribution – Ma’an’s key strategic objectives to foster community engagement and improve quality of life. Clinical trials are imperative to advance healthcare, and we’re proud to be part of this groundbreaking project, which has the potential to shift the trajectory of many people’s lives.”

George Magrath, M.D., Chief Executive Officer of Opus Genetics, said, “For patients living with MerTK-related retinitis pigmentosa, this trial represents the first real opportunity to change the course of a disease that has historically led to inevitable vision loss.”

Dr. Fahed Almarzooqi, Chief Executive Officer of M42’s Integrated Health Solutions, stated, “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.”

Dr. Georges-Pascal Haber, Chief Executive Officer of Cleveland Clinic Abu Dhabi, said that this partnership reinforces Cleveland’s ability to lead groundbreaking population-relevant studies that aim to redefine patient care. “By providing a platform for cutting-edge trials like this one, we are cementing Abu Dhabi’s position as a global hub for life sciences, precision medicine, and breakthrough therapies.”