Abu Dhabi Launches UAE’s First Gene Therapy Trial

Department of Health – Abu Dhabi has announced the launch and funding of the UAE’s first clinical trial for a new gene therapy targeting a rare inherited eye disease known as Retinitis Pigmentosa.

The therapy, developed by Opus Genetics, focuses on patients suffering from MerTK-related retinitis pigmentosa, a condition that leads to gradual vision loss and can eventually cause blindness. Currently, there are no approved treatments for this disease.

This first-of-its-kind clinical trial highlights Abu Dhabi’s growing role in advanced healthcare and life sciences. The initiative is a joint effort between the Department of Health – Abu Dhabi and Authority of Social Contribution - Ma'an, aiming to support innovation in treating rare diseases.

The project also involves key partners, including M42 through its research division IROS, and Cleveland Clinic Abu Dhabi, which will serve as the main clinical site for the trial.

The study will test the safety and effectiveness of a gene therapy that uses adeno-associated viruses (AAVs) to deliver a healthy version of the MerTK gene into retinal cells. When this gene is mutated, it fails to recycle proteins properly in the eye, leading to progressive vision damage.

Clinical activities for the trial are expected to begin in 2026. Researchers aim to develop a treatment that could slow down or even stop the progression of the disease.

Experts say this trial could be a major breakthrough for patients with inherited retinal diseases, which affect around 5% of people in the region. It also strengthens Abu Dhabi’s position as a global hub for medical research, precision medicine, and innovative therapies.

Officials from all partner organisations said the collaboration reflects a shared goal of turning scientific research into real treatments that can improve patients’ lives and offer hope to those with currently untreatable conditions.