Abu Dhabi opens UAE's first gene therapy trial for MerTK-linked retinal blindness

The Department of Health Abu Dhabi (DOH) has launched the UAE's first gene therapy clinical trial, targeting a rare inherited retinal disorder caused by mutations in the MerTK gene.

What the trial involves

Retinitis pigmentosa linked to MerTK mutations causes progressive degeneration of photoreceptor cells, typically resulting in severe vision loss before age 40. The MerTK protein maintains the retinal pigment epithelium's ability to clear cellular debris. When the gene is mutated, debris accumulates and triggers retinal damage that has, until recently, had no viable treatment.

The therapy delivers a functional copy of the MerTK gene directly to retinal cells, aiming to halt or reverse degeneration. Globally, only a handful of MerTK-specific gene therapy programmes have reached clinical stage. Spark Therapeutics' Luxturna, approved by the FDA in 2017 for a related retinal dystrophy, set the regulatory precedent that DOH and other regional authorities have studied closely.

Why this matters beyond ophthalmology

For medical directors and clinical leads at Abu Dhabi's major hospital groups, the trial confirms that DOH has built a working regulatory pathway for gene therapy in the emirate. That infrastructure is now tested and operational:

• Ethics committee approval for somatic gene therapy protocols
• GMP manufacturing oversight for advanced therapy medicinal products
• Informed consent standards specific to gene therapy trials
• Post-trial monitoring frameworks for rare disease indications

Facilities that want to participate in future oncology or rare disease gene therapy trials have a reference framework to work from.

The strategic context is Abu Dhabi's Healthcare Sector Transformation Programme, which targets reduced medical tourism outflows and positions the emirate as a destination for complex care. Patients who would previously have travelled to London or Houston for experimental treatments now have a local option for this indication.

For health tech founders and life sciences companies, the regulatory risk premium for establishing gene therapy programmes in Abu Dhabi is lower than it was two years ago. The UAE's Federal Law No. 5 of 2012 on Biomedical Research provides the national framework; this trial shows how that law applies to somatic gene therapy in practice.

The competitive picture for Dubai and the Northern Emirates

The Dubai Health Authority (DHA) has not publicly approved a gene therapy clinical trial. No equivalent announcement has come from DHA, creating a visible gap in advanced therapy capability between the two emirates. Abu Dhabi's move creates pressure on DHA to clarify its own approval pathway, particularly as the emirate's plan to build a life sciences cluster at Dubai Science Park depends in part on trial capability.

The Ministry of Health and Prevention (MOHAP), which governs the Northern Emirates, has not signalled a parallel programme. For hospital operators outside Abu Dhabi, the near-term implication is direct: patients presenting with inherited retinal conditions are more likely to be referred into the Abu Dhabi system for advanced care assessments.

DOH has not yet published the trial's patient recruitment criteria, sponsor details, or expected timeline to primary endpoints. Those disclosures will determine whether this trial functions as proof of concept for a broader Abu Dhabi gene therapy pipeline or remains a single-indication milestone.