Abu Dhabi Makes History With First CRISPR Gene Therapy for Blood Disorders

Abu Dhabi Administers First CRISPR Gene Therapy for Inherited Blood Disorders

On January 5, 2026, Abu Dhabi marked a major milestone in healthcare with the first administration of a CRISPR-based gene therapy for inherited blood disorders in the United Arab Emirates. The therapy, CASGEVY, was delivered to a patient suffering from a genetic blood condition, signalling a transition from lifelong symptomatic treatment to direct correction of disease at the genetic level.

The procedure was carried out through a coordinated effort involving Yas Clinic – Khalifa City , the Abu Dhabi Stem Cells Center , and Vertex Pharmaceuticals , under regulatory supervision of the Department of Health – Abu Dhabi .

From Symptom Management to Genetic Repair

Inherited blood disorders such as sickle cell disease and beta thalassemia have traditionally been managed through repeated blood transfusions, iron chelation therapy, and pain management. CASGEVY represents a fundamentally different therapeutic model by targeting the root genetic cause rather than managing symptoms indefinitely.

By correcting defective DNA sequences, the therapy offers the potential for reduced hospital dependence, fewer transfusions, and improved long-term outcomes, although eligibility and sustained clinical monitoring remain essential.

How the Gene Therapy Is Delivered

The treatment follows an autologous approach, meaning the patient’s own stem cells are used. These cells are first extracted and then genetically edited in specialised laboratories using CRISPR-Cas9 technology, which functions as a precise molecular tool to modify disease-causing DNA segments.

Once corrected, the stem cells are infused back into the patient, where they can repopulate the bone marrow and begin generating healthier blood cells. Using the patient’s own cells helps minimise immune rejection risks, though the procedure requires advanced infrastructure and expert oversight.

Target Diseases and Broader Health Impact

The initial application of CASGEVY focuses on high-burden inherited disorders such as beta thalassemia and sickle cell disease, both of which have significant prevalence in parts of the Middle East. The development also highlights wider potential for gene therapies in conditions like haemophilia and spinal muscular atrophy, where genetic interventions are increasingly advancing globally.

Abu Dhabi’s growing investment in genomics and precision medicine, supported by initiatives such as population-level genetic research programmes, has strengthened the scientific and clinical base needed to support such advanced treatments.

Phased Expansion Through Specialised Centres

Health authorities have adopted a phased rollout strategy to ensure patient safety and treatment quality. For now, gene therapy delivery is limited to certified centres of excellence equipped with specialised laboratories and multidisciplinary haematology teams.

Over the next 6 to 12 months, access is expected to expand gradually as more facilities receive certification and healthcare professionals undergo specialised training. Long-term follow-up, affordability, and equitable access are expected to remain key policy considerations as gene therapy moves from a landmark achievement to wider clinical use.

Important Facts for Exams

• CRISPR-Cas9 is a gene-editing technology that allows precise modification of DNA.

• Sickle cell disease is characterised by abnormally shaped red blood cells that obstruct blood flow.

• Beta thalassemia causes reduced haemoglobin production, leading to chronic anaemia.