"Abu Dhabi Health" Launches the first clinical trial for a gene therapy for a rare eye disease

ABU DHABI, March 23 / WAM / The Department of Health – Abu Dhabi, the regulatory body for the healthcare sector in the emirate, launched the first clinical trial of its kind in the UAE to treat retinitis pigmentosa disease associated with the MerTK gene, using an innovative gene therapy developed by Opus Genetics, in a qualitative step that reflects the accelerating trend towards adopting the latest medical solutions for rare diseases.

This hereditary disease is a rare retinal disease that leads to gradual loss of vision and may lead to complete blindness, in the absence of approved treatments so far, which gives this experience exceptional importance as a glimmer of hope for patients and a translation of Abu Dhabi’s commitment to promoting medical innovation and consolidating its position as a leading global center in life sciences and biotechnology.

This experience embodies a strategic cooperation between the Department of Health – Abu Dhabi and the Community Contribution Authority – together, and it represents an ambitious initiative that brings together the American company “Opus Genetics”, which specializes in developing biopharmaceuticals, the Department of Supervision and Innovative Research Support “AIROS” affiliated with the M42 Group, and the Cleveland Clinic Abu Dhabi Hospital, with the aim of accelerating the development of advanced genetic treatments for hereditary retinal diseases that affect about 5% of the region’s population.

The trial will be implemented at Cleveland Clinic Abu Dhabi, while the Innovative Research Supervision and Support Department (AIROS) will manage the study design, patient recruitment, and oversee clinical operations.

The clinical trial focuses on evaluating the safety and effectiveness of gene therapy based on the use of adeno-associated viral vectors, which is a widely approved technology due to its high levels of safety and efficiency in delivering healthy genes into cells.

The treatment targets the MerTK gene, mutations in which disrupt the mechanism of recycling proteins within the retina, causing gradual deterioration in visual ability.

The trial activities are expected to start during the year 2026, in a pivotal step in terms of treating rare diseases in Abu Dhabi and the region.

Her Excellency Dr. Noura Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, confirmed that the emirate continues to consolidate its leadership in precision medicine and innovative treatments for rare diseases by launching the first clinical trial to treat this disease, noting the role of these initiatives in transforming scientific capabilities into tangible results that benefit patients, and that cooperation with local and international partners enhances the development of treatment, opens new horizons, and restores hope to those suffering from hereditary blindness, as well as supporting Abu Dhabi’s position in health care and life sciences.

His Excellency Abdullah Al Ameri, Director General of the Community Contributions Authority – Ma’an, explained that supporting the health sector is among the Authority’s priorities to enhance community participation and quality of life, noting that directing contributions to projects with a tangible impact makes a real difference in the lives of individuals, expressing his pride in the Authority’s role in enabling this project and embodying Abu Dhabi’s vision of investing in medical innovation.

Dr. George McGrath, CEO of Opus Genetics, said that the launch of the first clinical trial for targeted gene therapy for the MerTK gene in the country represents a turning point for patients and the field of hereditary retinal diseases, stressing that this cooperation combines scientific innovation and clinical excellence to address urgent medical needs, pointing out that the study provides a real opportunity to change the course of a disease associated with inevitable loss of vision.

Dr. Fahd Al Marzouqi, CEO of the Integrated Health Solutions Platform of the M42 Group, stressed that the experiment represents a pivotal step in strengthening Abu Dhabi’s position as a global center for innovation in biotechnology, pointing to the role of partnerships and clinical excellence in transforming scientific achievements into therapeutic solutions that meet urgent needs and contribute to shaping the future of rare disease research.

Dr. Georges Pascal Habre, CEO of Cleveland Clinic Abu Dhabi, explained that research and clinical trials represent a fundamental driver of healthcare transformation, stressing the hospital’s commitment to promoting innovation, improving quality of life, and providing an advanced environment for conducting studies, which supports Abu Dhabi’s position in the life sciences and precision medicine.

• For more: Follow Khaleejion 24 Arabic, Khaleejion 24 English, Khaleejion 24 Live, and for social media follow us on Facebook and Twitter