Abu Dhabi Administers First Gene-Therapy Injection For Patients With Inherited Blood Disorders

Abu Dhabi has delivered its first gene-editing treatment for inherited blood disorders, a milestone that could change how conditions such as sickle cell disease and thalassaemia are treated in the UAE.

Yas Clinic–Khalifa City, in partnership with the Abu Dhabi Stem Cells Center (ADSCC), has successfully administered the country's first gene-therapy injection for haemoglobin disorders, the clinic said in a press release issued on Monday.

The treatment, called CASGEVY, works by correcting the faulty gene that causes these disorders. It has been approved by the UAE Medicines Agency for patients aged 12 and above who suffer from severe sickle cell disease or require regular blood transfusions due to beta thalassaemia.

Stay up to date with the latest news. Follow KT on WhatsApp Channels.

Health officials said the procedure was carried out under the supervision of the Department of Health – Abu Dhabi.

Eligible patients will be able to access the treatment through insurance and reimbursement programmes in the emirate.

Instead of managing symptoms for life, the therapy targets the problem at its source. Doctors collect a patient's stem cells and correct the genetic error in a specialised laboratory. The repaired cells are then returned to the patient, allowing the body to produce healthier blood cells over time.

Dr Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, said the successful treatment reflects the emirate's focus on bringing advanced medical care closer to patients.

“These innovations give patients new hope and reduce the need to travel abroad for complex treatments,” she said, adding that Abu Dhabi aims to expand access to advanced therapies within its healthcare system.

Inherited blood disorders such as sickle cell disease and thalassaemia are common in parts of the Middle East, Africa and South Asia. Patients often depend on frequent hospital visits, blood transfusions and medication to manage pain, fatigue and other complications.

Dr Maysoon Al Karam, Chief Medical Officer at Yas Clinic, said the introduction of gene-editing therapy could offer a life-changing option for patients who have had few alternatives.

“This is a major step forward for patients who have lived with these conditions since childhood,” she said. The therapy was developed by Vertex Pharmaceuticals, a global biotechnology company. Its regional head, Hisham Hagar, said the treatment could offer long-term relief by addressing the root cause of the disease rather than its symptoms.

Patients receiving the therapy undergo careful screening, followed by the collection and treatment of their cells, reinfusion, and long-term medical monitoring to ensure safety.

ADSCC said offering such treatments in the UAE would help keep care local, improve outcomes and strengthen Abu Dhabi's position in advanced medical treatment and research.

Health authorities said more patients will be assessed for eligibility as the programme expands under existing medical guidelines.

MENAFN05012026000049011007ID1110555480