Abu Dhabi has achieved a historic medical milestone by delivering the UAE’s first gene therapy treatment for inherited blood disorders, administering CASGEVY to eligible patients. This revolutionary therapy addresses severe sickle cell disease and beta thalassaemia conditions prevalent in the Middle East, Africa, and South Asia by correcting the underlying genetic defect responsible for abnormal haemoglobin.

Unlike traditional approaches relying on regular blood transfusions, medications, and symptom control, CASGEVY offers a potentially curative option. The process involves harvesting a patient’s hematopoietic stem cells, genetically editing them in a lab to restore normal haemoglobin function, and reinfusing the modified cells. Over time, these cells produce healthier red blood cells, alleviating chronic pain, fatigue, organ damage, and transfusion dependence.

The landmark procedure took place at Yas Clinic–Khalifa City, in close collaboration with the Abu Dhabi Stem Cells Center and under oversight from the Department of Health  Abu Dhabi. CASGEVY, developed by Vertex Pharmaceuticals, received UAE regulatory approval for patients 12 years and older with severe forms of the disorders.

Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, stated: “These innovations give patients new hope and reduce the need to travel abroad for complex treatments,” emphasizing Abu Dhabi’s goal to expand advanced therapy availability locally.

Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, added: “This is a major step forward for patients who have lived with these conditions since childhood.”

Hisham Hagar, Vertex Pharmaceuticals’ regional head, noted the therapy “addresses the root cause of the disease rather than managing symptoms alone.”

Patients undergo rigorous screening, stem cell mobilization/collection, myeloablative conditioning, reinfusion, and long-term monitoring. Access is facilitated through insurance and reimbursement programmes, making it feasible within the UAE ecosystem.

This UAE-first enhances Abu Dhabi’s reputation in regenerative medicine and stem cell research, building on facilities like the Abu Dhabi Stem Cells Center. As more patients are evaluated, it promises improved quality of life, reduced healthcare burdens, and potential medical tourism inflows.