Abu Dhabi Begins First Gene Therapy Treatment for Blood Disorders

The Abu Dhabi Stem Cells Center said offering gene-therapy treatments locally will help keep care within the UAE, improve patient outcomes, and strengthen Abu Dhabi’s position in advanced medical treatment and research.

Abu Dhabi has administered its first gene-therapy injection for patients with inherited blood disorders, marking a significant step in advancing treatment options for conditions such as sickle cell disease and beta thalassaemia in the UAE.

The procedure has been carried out at Yas Clinic–Khalifa City in partnership with the Abu Dhabi Stem Cells Center. Health officials said the treatment was administered under the supervision of the Department of Health – Abu Dhabi.

The therapy, known as CASGEVY, works by correcting the faulty gene responsible for haemoglobin 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.

Eligible patients will be able to access the treatment through insurance and reimbursement programmes available in the emirate, health authorities confirmed.

Unlike conventional care that focuses on lifelong symptom management, the gene therapy targets the condition at its source. Doctors collect a patient’s stem cells, correct the genetic defect in a specialized laboratory, and then reinfuse the modified cells, enabling 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 administration reflects the emirate’s commitment to expanding access to advanced medical treatments locally.

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

Inherited blood disorders such as sickle cell disease and thalassaemia are prevalent across parts of the Middle East, Africa, and South Asia. Patients often depend on frequent hospital visits, regular blood transfusions, and medication to manage chronic 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 transformative option for patients with limited alternatives.

“This is a major step forward for patients who have lived with these conditions since childhood,” she said.

The therapy has been developed by Vertex Pharmaceuticals. Hisham Hagar, the company’s regional head, said the treatment addresses the root cause of the disease rather than managing symptoms alone.

Patients undergoing the therapy are subject to careful screening, followed by stem cell collection, treatment, reinfusion, and long-term medical monitoring to ensure safety and effectiveness.

The Abu Dhabi Stem Cells Center said offering gene-therapy treatments locally will help keep care within the UAE, improve patient outcomes, and strengthen Abu Dhabi’s position in advanced medical treatment and research.

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

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