Abu Dhabi Administers UAE’s First Gene Therapy for Blood Disorders

Abu Dhabi on January 5, 2026 recorded a milestone for healthcare in the UAE with the first administration of a gene-therapy injection for inherited blood disorders. The therapy, CASGEVY, uses CRISPR-Cas9 gene editing to address the genetic cause of conditions such as sickle cell disease and beta thalassemia, marking a shift from long-term symptom management to targeted correction at the DNA level. The procedure was delivered through a collaboration involving Yas Clinic – Khalifa City, the Abu Dhabi Stem Cells Center (ADSCC), and Vertex Pharmaceuticals, under oversight of the Department of Health – Abu Dhabi (DoH).

CASGEVY Marks Shift From Supportive Care to Genetic Correction

For decades, inherited blood disorders have largely been managed through supportive interventions such as regular transfusions, iron chelation, and pain control. CASGEVY represents a different approach by aiming to correct the underlying genetic fault rather than repeatedly treating downstream effects. For patients and families, this raises the prospect of fewer hospital visits, reduced dependence on transfusions, and improved long-term quality of life, subject to eligibility and clinical outcomes.

How the Gene Therapy Process Works in Practice

The approach uses a patient’s own stem cells, which are collected and then edited in controlled laboratory conditions. CRISPR-Cas9 acts like molecular scissors, targeting and modifying specific DNA segments associated with disease. The corrected cells are then returned to the patient, where they can repopulate the bone marrow and begin producing healthier blood cells over time. This autologous method is designed to reduce rejection risks because the cells originate from the same individual, though it still requires specialist facilities and careful monitoring.

Target Diseases and Wider Implications for Regional Health

The first administration focused on inherited blood disorders with high clinical burden, notably beta thalassemia and sickle cell disease. The rollout also signals broader potential for gene-based interventions in conditions prevalent in the region, including spinal muscular atrophy and haemophilia, where genetic therapies are increasingly being explored or adopted globally. Abu Dhabi’s investment in life sciences, supported by initiatives such as the Emirati Genome Program, has strengthened the clinical infrastructure and data foundations needed for advanced precision medicine.

Imporatnt Facts for Exams

• CRISPR-Cas9 is a gene-editing technology used to target specific DNA sequences for modification.
• Sickle cell disease involves abnormal “C-shaped” red blood cells that can block blood flow and cause pain crises.
• Beta thalassemia is an inherited disorder causing reduced haemoglobin production and severe anaemia.
• Gene therapy commonly uses a patient’s own stem cells (autologous therapy) to reduce immune rejection risk.

Phased Rollout and Access Through Specialised Centres

Health authorities in Abu Dhabi are following a phased strategy to expand access while maintaining safety and quality controls. Early delivery is restricted to designated centres of excellence equipped with specialised laboratories and multidisciplinary haematology teams. Over the next 6 to 12 months, the programme is expected to widen through additional certified hospitals and expanded training for medical staff. Cost, long-term follow-up, and equitable access are expected to remain central considerations as the UAE moves from a first-of-its-kind milestone towards broader clinical availability.