DOH-Abu Dhabi launches UAE’s first gene therapy clinical trial for retinitis pigmentosa

UAE—The Department of Health – Abu Dhabi has announced the launch and funding of the UAE’s first clinical trial for a novel gene therapy targeting MerTK-related retinitis pigmentosa (RP). 

This rare inherited eye disease causes progressive vision loss and eventual blindness.

Currently, no approved treatments exist for this condition, which affects approximately five percent of the population in the region.

The trial, developed by US-based biopharmaceutical company Opus Genetics, marks a significant step forward in the Emirate’s growing ambition to become a global hub for life sciences and biomedical innovation.

A collaborative effort between public and private partners

The initiative is the result of a partnership between the Department of Health – Abu Dhabi and the Authority of Social Contribution – Ma’an, alongside Opus Genetics, the Innovative Research Oversight and Support (IROS) division of the M42 group, and Cleveland Clinic Abu Dhabi.

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

Clinical development activities are expected to begin in 2026.

How the therapy works

The trial will evaluate the safety and efficacy of an adeno-associated virus (AAV)-based gene therapy that targets the MerTK gene.

When mutated, this gene fails to properly recycle protein in the retina, leading to irreversible vision deterioration.

AAV vectors are widely recognised as safe and effective tools for delivering healthy genes into cells, making them a promising avenue for treating inherited diseases like RP.

Voices behind the initiative

Dr Noura Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, expressed pride in supporting a therapy that could transform the lives of patients living with inherited blindness, calling it a milestone that strengthens Abu Dhabi’s reputation as a destination for advanced healthcare research.

Additionally, Abdullah Al Ameri, Director-General of Ma’an, highlighted that deploying community funds toward health projects that directly improve the quality of life is central to the Authority’s strategic objectives.

For his part, George Magrath, CEO of Opus Genetics, described the trial as the first real opportunity to change the course of a disease that has historically led to inevitable vision loss.

Dr Fahed Almarzooqi, CEO of M42’s Integrated Health Solutions, said the trial advances Abu Dhabi’s standing as a global biotech hub by translating scientific breakthroughs into real-world treatments for patients with unmet needs.

Finally, Dr Georges-Pascal Haber, CEO of Cleveland Clinic Abu Dhabi, added that the partnership cements Abu Dhabi’s position at the forefront of precision medicine and breakthrough therapies.