For families in Qatar and across the broader MENA region living with severe inherited blood disorders, the nearest access point to the world's most advanced gene therapy just moved significantly closer to home. Sidra Medicine has been qualified by Vertex Pharmaceuticals to administer Casgevy — the world's first approved CRISPR/Cas9 gene therapy — making it the first hospital in Qatar to offer this treatment.
The therapy is available for patients aged 12 years and older with transfusion-dependent beta thalassemia and severe sickle cell disease, offering a potential functional cure through a one-time treatment. The hospital has already onboarded its first patient, who is currently undergoing assessment. As the first regional hub for this technology, Sidra Medicine currently manages a cohort of nearly 200 pediatric patients with sickle cell and thalassemia. For those with the most severe cases, Casgevy is proving to be a paradigm shift. Clinical data remains overwhelmingly positive: 97% of sickle cell patients have remained free of severe pain crises for at least a year, while nearly all thalassemia patients have achieved total transfusion independence—with many maintaining these curative results for over five years.
The qualification carries a regional significance that extends beyond Qatar's borders. For decades, families had to travel abroad to access advanced treatments. Having this therapy available locally represents a shift in how advanced medicine is delivered in the Gulf. With its first patient already in assessment, Sidra Medicine's doors are open. The MENA region's access to curative gene therapy has formally begun.