The UAE is ushering in a new era of hope for thalassemia patients with the introduction of the groundbreaking gene therapy CASGEVY, described as a potential “curative gene therapy.” Recently launched in the country, this treatment uses CRISPR technology to edit patients’ stem cells and stimulate fetal hemoglobin production, aiming for a complete cure. So far, two patients have received the therapy, with final results expected by the end of 2025, and early signs are promising.
Doctors emphasize that this therapy is a significant leap forward, offering a chance for patients to live free of constant medical interventions. In parallel, the UAE has also introduced two advanced drugs, luspatercept and mitapivat, which have shown the ability to reduce the need for blood transfusions by 33% and up to 50%, respectively. These treatments are designed to improve patients’ quality of life by minimizing the burdens of frequent hospital visits.
Beyond treatment, the UAE has been proactive in prevention. Genetic screening before marriage has already led to a noticeable decline in the number of new cases, according to health authorities. The Emirates Thalassemia Society is currently building a comprehensive patient database in collaboration with national health agencies to better tailor services and elevate care standards.
During a recent event marking World Thalassemia Day, the Emirates Thalassemia Society gathered experts, patients, and families to discuss the latest advancements and celebrate patient success stories. Sheikh Sheikha bint Saif Al Nahyan, chair of the Society, expressed deep gratitude to the Ministry of Health for its continuous support and for fostering awareness initiatives that have helped curb the spread of genetic diseases.
Dr. Mohamed Abu Haliga, consultant in hematology and bone marrow transplantation at Abu Dhabi Stem Cells Center, explained that CASGEVY works by collecting the patient’s stem cells, genetically modifying them, and then reintroducing them to the body. Meanwhile, Dr. Khaled Muslim from Burjeel Medical City noted that mitapivat, which the UAE helped develop, has shown remarkable effectiveness and is expected to be widely available by the end of this year.
Abdel Baset Mohamed Merdas, vice-chair of the Emirates Thalassemia Society, highlighted ongoing efforts to compile patient data nationwide, which will support targeted care improvements.
With these promising therapies and the country’s strong commitment to prevention, the UAE is setting a regional example in the fight against thalassemia — transforming what was once considered a lifelong condition into a potentially curable disease.
