Department of Health – Abu Dhabi Launches Uae’s First Gene Therapy Clinical Trial for Inherited Eye Disease

ADM NEWS | Department of Health — Abu Dhabi launches UAE’s first gene therapy clinical trial for inherited eye disease Department of Health — Abu Dhabi, the regulator of the healthcare sector in the emirate of 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 5 per cent of the population in this region.

The launch of the trial coincides with global efforts to raise awareness of rare diseases and accelerate research for conditions that collectively affect millions worldwide. 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.

Her Excellency Dr. Noura Al Ghaithi, Undersecretary of the Department of Health — Abu Dhabi, said: “By hosting the first MerTK gene therapy clinical trial in the UAE, Abu Dhabi is not only demonstrating its leadership in precision medicine and rare disease innovation but also turning scientific possibility into a tangible reality for patients.

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.” George Magrath, M.D., Chief Executive Officer of Opus Genetics, said: “Launching the UAE’s first clinical trial for our MerTK gene therapy is a defining moment for patients and for the field of inherited retinal disease.

This collaboration with the Department of Health — Abu Dhabi and our partners brings together scientific innovation, clinical excellence, and a shared commitment to addressing serious unmet needs. 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.” Cleveland Clinic Abu Dhabi will serve as the clinical site, equipped with advanced diagnostic imaging, surgical expertise and specialised retinal disease clinics, 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. Dr. Fahed Almarzooqi, Chief Executive Officer of M42’s Integrated Health Solutions, said: “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. This collaboration reflects our mission to accelerate access to transformative therapies and to shape the future of rare disease research from the heart of the region.” Dr. Georges-Pascal Haber, Chief Executive Officer, Cleveland Clinic Abu Dhabi, said: “Clinical research is the engine of transformation in healthcare.

Our participation in this trial reflects Cleveland Clinic Abu Dhabi’s long-standing commitment to advancing medical innovation and improving lives through science. This partnership reinforces our 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.”