UAE’s First Gene Therapy Trial Targets Inherited Eye Disease
A gene therapy clinical trial targeting retinitis pigmentosa caused by MerTK mutations will be conducted in Abu Dhabi.
A clinical trial for a gene therapy targeting retinitis pigmentosa caused by mutations in the MerTK gene has been launched in Abu Dhabi, marking the first study of its kind in the UAE.
The trial focuses on a rare inherited eye condition that leads to progressive vision loss and blindness, for which no approved treatments currently exist for this specific subtype.
The programme is led by the Department of Health – Abu Dhabi in collaboration with the Authority of Social Contribution – Ma’an, biotechnology company Opus Genetics, M42 and Cleveland Clinic Abu Dhabi.
Cleveland Clinic Abu Dhabi will serve as the clinical site for the study, while M42’s Innovative Research Oversight and Support division will oversee trial design, patient recruitment and clinical operations. Clinical development activities are expected to begin in 2026.
The study will assess the safety and efficacy of the gene therapy, which uses an adeno-associated virus vector to deliver a functional copy of the MerTK gene directly to retinal cells.
According to the Department of Health – Abu Dhabi, inherited retinal diseases affect approximately 5% of the population in the region.
