DoH Abu Dhabi, ADSCC introduce advanced gene-editing therapy for sickle cell disease and thalassemia

Abu Dhabi just turned a page in medical history. For the first time in the UAE, a powerful new gene-editing treatment — CASGEVY — is stepping into the spotlight, thanks to the Department of Health - Abu Dhabi and the Abu Dhabi Stem Cells Center (ADSCC). This cutting-edge therapy takes aim at two inherited blood disorders that have long haunted families across the region: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

The first patient is set to begin this revolutionary treatment at Yas Clinic Hospital this April. And while it might sound like science fiction, what's unfolding is real — and it's reshaping the region's healthcare map.

How does it work?

CASGEVY is a one-and-done gene therapy built on CRISPR/Cas9, a Nobel Prize-winning gene-editing technology. Here's the simple version of how it works:

• Doctors extract the patient's own blood stem cells.
• Scientists then edit the genes in the lab, correcting the defect at the root.
• The edited cells are put back into the patient's body.
• Those cells then begin producing healthy red blood cells — offering lasting relief.

It's like rewiring the body's blood production system to fix the problem at its core.

"This revolutionary treatment... we aim to offer as one of the first advanced gene therapies in the country," said Dr. Maysoon Al Karam, Chief Medical Officer at ADSCC. "Patients with genetic disorders such as thalassemia and sickle cell disease will greatly benefit."

Why does it matter?

Sickle cell and beta-thalassemia can mean constant pain, repeated hospital visits, and lives lived in the shadow of chronic illness. Now, with CASGEVY, there's the promise of:

• Fewer hospitalizations
• Less dependency on blood transfusions
• A real chance at normalcy

"This partnership represents a significant step toward bringing transformative medicines to patients in the UAE," said Hisham Hagar, Senior Country Manager at Vertex GCC.

What's more, the treatment is being covered by insurance — a major move by the Department of Health to make sure life-changing care doesn't stay out of reach.

The context

Abu Dhabi has been quietly building its reputation as a global health and biotech hub. This milestone marks yet another stride in that direction.

The DoH has rolled out regulations and safety guidelines aligned with top international standards, ensuring that gene therapies like this one are delivered safely and ethically. As Her Excellency Dr. Noura Khamis Al Ghaithi puts it, "Our goal is to establish the world's most efficient healthcare system through the adoption of the latest innovations."

This moment didn't come out of nowhere. It's the result of years of strategic planning, investment, and partnership — including with Vertex Pharmaceuticals, one of the giants in the biotech world. The UAE's leadership sees this not just as medicine but as vision in action — using science to tackle some of the region's most stubborn health challenges.

The Emirates Drug Establishment (EDE) has already approved CASGEVY for patients 12 and up who suffer from severe forms of SCD and TDT.