Abu Dhabi launches first UAE clinical trial for gene therapy treating inherited blindness

Abu Dhabi is funding the UAE's first clinical trial for a gene therapy that could treat a rare inherited eye disease. The treatment targets MerTK-related retinitis pigmentosa (RP), a condition that causes progressive vision loss and blindness with no approved treatments available.

The Department of Health – Abu Dhabi announced the trial in partnership with the Authority of Social Contribution - Ma'an. The therapy was developed by US company Opus Genetics and will be tested at Cleveland Clinic Abu Dhabi starting in 2026.

How does it work?

The experimental therapy uses adeno-associated viruses (AAVs) to deliver healthy genes into cells. These viruses are considered safe and effective carriers for gene therapy.

The treatment targets the MerTK gene, which normally helps recycle proteins in the retina. When this gene is mutated, it can't do its job properly, leading to vision loss over time. The therapy aims to replace the faulty gene with a working version.

The trial will test both safety and effectiveness in patients with this specific form of retinitis pigmentosa.

Why does it matter?

This condition affects about 5% of the population in the Middle East region, making it more common there than in other parts of the world. Currently, patients have no treatment options and face inevitable blindness.

The trial could provide the first real chance to stop or slow vision loss for these patients. If successful, it would represent a major breakthrough for treating inherited blindness.

"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," said George Magrath, CEO of Opus Genetics.

The context

The trial is part of Abu Dhabi's push to become a global center for life sciences research. Several organizations are working together on the project:

• Opus Genetics provides the experimental therapy
• Cleveland Clinic Abu Dhabi will run the clinical trial
• M42's IROS division will oversee trial design and patient recruitment
• The Department of Health and Ma'an authority are providing funding

Gene therapy for inherited eye diseases has shown promise in recent years. AAV-based treatments are becoming more common because they can deliver therapeutic genes directly to the cells that need them.

The trial is expected to begin recruiting patients in 2026. If successful, it could lead to the first approved treatment for this form of inherited blindness.