UAE Launches First Gene Therapy Trial for Rare Blindness

A rare inherited eye disease that slowly steals people's vision may finally have hope, thanks to a groundbreaking clinical trial launching in Abu Dhabi.

The Department of Health Abu Dhabi announced funding for the UAE's first gene therapy trial targeting MerTK-related retinitis pigmentosa, a condition that causes progressive vision loss and eventual blindness. Right now, no approved treatments exist for this disease.

The trial brings together US-based Opus Genetics, Cleveland Clinic Abu Dhabi, and the M42 group's research division. Clinical testing is expected to begin in 2026, marking a major milestone for rare disease treatment in the region.

The experimental therapy uses adeno-associated viruses (AAVs) to deliver healthy genes into retinal cells. These viruses are already recognized as safe and effective tools for treating inherited diseases. The treatment targets the MerTK gene, which fails to properly recycle proteins in the retina when mutated.

Inherited retinal diseases affect approximately five percent of the population in the Middle East region. This makes the trial particularly relevant for local communities who have long needed solutions.

Cleveland Clinic Abu Dhabi will serve as the clinical site where patients receive treatment. The M42 group will handle trial design, patient recruitment, and clinical operations to ensure the study meets rigorous safety standards.

The collaboration also includes the Authority of Social Contribution Ma'an, which is deploying community funds to support healthcare projects that impact lives. This partnership model demonstrates how public and private sectors can unite around urgent medical needs.

The Ripple Effect

This trial positions Abu Dhabi as a serious player in biotech innovation and precision medicine. By hosting cutting-edge research, the emirate is creating infrastructure that will attract more clinical trials and healthcare breakthroughs.

For families affected by inherited blindness, the trial represents something even more valuable: the first real chance to change a disease trajectory that has historically meant inevitable vision loss. Success here could pave the way for treating other genetic eye conditions.

The investment also signals to researchers worldwide that Abu Dhabi welcomes advanced life sciences projects. This could accelerate development of treatments for other rare diseases that disproportionately affect regional populations.

One trial may seem small, but it carries the hopes of countless patients who've been told nothing can be done—and now, finally, something can.