DoH Abu Dhabi launches 1st gene therapy trial for rare eye disease

DoH Abu Dhabi 1st gene therapy trial for rare eye disease-GCC Business News
Image credits: WAM | Cropped by GBN
By Desk Reporter, GCC Business News

The Department of Health – Abu Dhabi (DoH Abu Dhabi) has launched the funding of a UAE-first clinical trial for a novel gene therapy for MerTK-related retinitis pigmentosa (RP) developed by Opus Genetics.

A groundbreaking initiative by DoH Abu Dhabi aims to address a rare inherited eye disease that causes progressive vision loss and eventual blindness, with no current approved treatments. This effort underscores Abu Dhabi’s commitment to advancing solutions for rare conditions and solidifies its position as a global hub for life sciences and biomedical innovation.

The pioneering clinical trial is a collaboration between the DoH Abu Dhabi, the Authority of Social Contribution – Ma’an, and key partners including Opus Genetics (a US-based biopharmaceutical company), M42’s Innovative Research Oversight and Support (IROS) division, and Cleveland Clinic Abu Dhabi. It highlights a shared commitment to healthcare research and innovation.

Cleveland Clinic Abu Dhabi will serve as the clinical site, with IROS overseeing trial design, patient recruitment, and clinical operations. Clinical development is set to begin in 2026, marking a significant step forward for rare disease treatment in the region.

The study will assess the safety, efficacy, and long-term potential of an innovative AAV-based gene therapy developed by Opus Genetics. With this groundbreaking therapy, DoH Abu Dhabi targets the MerTK gene, which, when mutated, disrupts protein recycling in the retina, leading to progressive vision loss and eventual blindness.

Adeno-associated viruses (AAVs), recognized for their proven safety, precision, and effectiveness in gene delivery, hold significant promise for treating inherited retinal diseases that currently have no approved treatments. This therapy represents a potential breakthrough in providing hope for patients suffering from these rare conditions.

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Dr. Noura Al Ghaithi
Undersecretary – DoH Abu Dhabi

“In collaboration with our international and local partners, we are proud to support the development of a novel therapy that could transform the lives of patients living with inherited blindness. This milestone brings renewed hope to patients and reinforces our position as a trusted destination for advanced healthcare and life sciences research.”

Abdullah Al Ameri, Director-General of the Authority of Social Contribution – Ma’an, said that, “Supporting the health sector by deploying community funds to projects that impact lives is one of the Authority of Social Contribution – Ma’an’s key strategic objectives to foster community engagement and improve quality of life. Clinical trials are imperative to advance healthcare, and we’re proud to be part of this groundbreaking project, which has the potential to shift the trajectory of many people’s lives.”

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