March 23, 2026 —
The Department of Health – Abu Dhabi has announced the launch and funding of a first-of-its-kind clinical trial in the United Arab Emirates evaluating an AAV-based gene therapy for MerTK-related retinitis pigmentosa (RP) developed by Opus Genetics. The initiative marks a major milestone for rare disease research in the region and reflects Abu Dhabi’s growing role as a global hub for biomedical innovation and life sciences.
Retinitis pigmentosa is a group of inherited retinal disorders characterized by progressive degeneration of photoreceptor cells in the retina, leading to gradual vision loss and, in many cases, eventual blindness. Mutations in the MerTK gene disrupt the ability of retinal pigment epithelial cells to properly recycle photoreceptor outer segments, ultimately causing retinal degeneration. Currently, there are no approved treatments for MerTK-associated RP, leaving patients with limited therapeutic options.
The investigational therapy uses an adeno-associated virus (AAV) vector to deliver a functional copy of the MerTK gene to retinal cells. AAV vectors are widely used in gene therapy due to their favorable safety profile and their ability to efficiently deliver therapeutic genes into target tissues, making them particularly suitable for treating inherited retinal diseases.
This pioneering trial is the result of a collaborative effort between several regional and international partners. The Department of Health – Abu Dhabi and the Authority of Social Contribution – Ma’an are supporting the program, while the clinical trial will be conducted at Cleveland Clinic Abu Dhabi. The Innovative Research Oversight and Support (IROS) division of the M42 group will oversee the trial design, patient recruitment, and operational management.
Clinical development activities are expected to begin in 2026, marking an important step toward advancing treatments for inherited retinal diseases in the Middle East. These conditions are estimated to affect approximately 5% of the population in the region, highlighting a significant unmet medical need.
The trial will evaluate the safety and efficacy of Opus Genetics’ AAV-mediated gene therapy in patients with MerTK-related retinitis pigmentosa. By delivering a healthy copy of the MerTK gene directly to retinal cells, the therapy aims to restore the biological processes necessary for maintaining photoreceptor function and potentially slow or halt disease progression.
Leaders involved in the initiative emphasized the broader significance of the program. The Department of Health – Abu Dhabi noted that the project demonstrates the Emirate’s commitment to supporting advanced therapies and improving outcomes for patients with rare diseases. Partners from the Authority of Social Contribution – Ma’an and M42 highlighted the importance of collaborative innovation and community-supported funding to accelerate breakthroughs in healthcare.
Executives from Opus Genetics also underscored the importance of the trial for patients living with MerTK-related RP. For many individuals affected by this rare genetic disease, the study represents the first potential opportunity to alter the course of progressive vision loss.
By bringing together global biotechnology expertise, clinical infrastructure, and regional investment in innovation, the trial represents a significant step forward in advancing AAV gene therapy for inherited retinal diseases and expanding access to cutting-edge treatments in new regions of the world.
