First gene therapy infusion to be administered in UAE

A quiet breakthrough is taking shape in Abu Dhabi. Yas Clinic in Khalifa City, working with the Abu Dhabi Stem Cells Centre and Vertex Pharmaceuticals, has set the stage for the first gene therapy infusion ever carried out in the UAE. At the heart of this moment is CASGEVY, the region's first treatment based on the CRISPR-Cas9 gene-editing method.

This step is more than a scientific first. It is a signal that the country is ready to join the front line of advanced medicine. As Dr. Maysoon Al Karam put it, beginning the nation's first gene-edited therapy during the UAE National Day reflects a shared drive toward progress and better patient outcomes.

How does it work?

Gene therapy might sound like something from a faraway future, yet the process is surprisingly hands-on. It follows a simple arc.

• Doctors collect a patient's stem cells.
• Those cells are edited outside the body with CRISPR Cas9.
• The corrected cells are then returned to the patient through an infusion.

The science behind it is as clean as it is clever. CRISPR Cas9 works like a guided pair of scissors. A guide RNA molecule directs the Cas9 enzyme to a faulty DNA sequence. Cas9 makes a precise cut and allows doctors to fix the genetic error that fuels disease.

The full treatment involves cell mobilisation, strict quality checks, infusion, and long-term monitoring. Nothing is rushed. Every step is measured.

Why does it matter?

For people living with sickle cell disease or transfusion-dependent beta thalassemia, this is more than a new option. It could change the arc of their lives. Some patients have spent years moving in and out of hospitals with few real choices.

Hisham Hagar from Vertex GCC spoke to that reality. He said the collaboration will give patients who have long faced limited options a path to what he called a potential functional cure.

The UAE Medicines Agency has now authorised CASGEVY for patients aged twelve and above. Eligible patients can access the therapy through insurance agreements and reimbursement plans set by the Abu Dhabi Department of Health.

The first infusion is expected in the first week of December. A fitting moment that carries symbolic weight as the country celebrates National Day. It marks a turning point.

The context

The UAE has been steadily building its place in the global life sciences arena. This new chapter shows how far that vision has come. The Abu Dhabi Stem Cells Centre already leads the nation in stem cell transplants and advanced cellular therapies. Its facilities include GMP laboratories, integrated research units, manufacturing capacity, and FACT-accredited environments.

Together with Yas Clinic in Khalifa City, the centre has pushed to reduce the need for patients to travel abroad for complex treatment. By expanding access to regenerative medicine and precision therapies, they offer long-term hope for conditions once seen as lifelong burdens.

The introduction of CASGEVY is not happening in a vacuum. It is part of a broader strategy that blends scientific ambition, national pride, and a rising commitment to medical innovation. And with this first gene therapy infusion, the UAE places itself firmly on the map of countries shaping the future of genetic medicine.