Sidra medicine: Sidra Medicine, a member of Qatar Foundation, announced today that it has been qualified by Vertex Pharmaceuticals as one of a limited number of hospitals in the world, to administer Casgevy, a groundbreaking, one-time CRISPR/Cas9-based gene therapy treatment. The hospital said that Casgevy is now available in Qatar for patients aged 12 years and older, living with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).
According to Qatar News Agency, Chief Medical Officer at Sidra Medicine Prof. Ibrahim Janahi expressed pride in being the first hospital in Qatar to offer a gene editing therapy with potential for a functional cure. He emphasized that this milestone strengthens Qatar's role as a regional leader in advanced medicine and precision health. The collaboration with Vertex brings the world's first approved CRISPR/Cas9 medicine to treat these inherited blood disorders to Qatar and the broader MENA region.
Approved by the Ministry of Public Health in Qatar (MOPH), as well as the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other agencies, Casgevy is a first-in-class CRISPR/Cas9 gene-edited therapy targeting the genetic cause of these conditions. The therapy has shown significant results for sickle cell disease patients and provides independence from regular blood transfusions for those with transfusion-dependent beta thalassemia.
Executive Country Manager at Vertex GCC Hisham Hagar noted that the launch of the CRISPR/Cas9 therapy in Qatar marks a significant advancement in the fight against serious diseases. This achievement highlights the successful partnership with Sidra Medicine and aligns with Vertex's mission to transform lives through scientific innovation. The treatment promises to offer a substantial and lasting treatment option for eligible patients in Qatar.
Sidra Medicine currently manages the care of approximately 150 to 200 children in Qatar diagnosed with thalassemia and sickle cell disease. Chair of Pediatric Medicine at Sidra Medicine Dr. Ahmed Al Hammadi shared that their goal is to ensure that every child treated receives the most advanced therapies along with compassionate, family-centered care. Casgevy stands as more than a scientific breakthrough, embodying the commitment to improve the lives of young patients with these challenging conditions.
Chief Research Officer at Sidra Medicine Prof. Khalid Fakhro remarked that this milestone exemplifies Sidra Medicine's precision health vision, where treatment is tailored to each patient's unique genetic profile. By introducing transformative gene therapies like Casgevy to Qatar, Sidra Medicine leverages cutting-edge research and clinical practice to deliver personalized care, reinforcing Qatar's commitment to advancing genomic medicine and positioning the country as a leader in precision health in the region.
Casgevy has already been successfully administered to patients globally outside of clinical trials, achieving transfusion independence and marked improvements in quality of life. With this achievement, Sidra Medicine reaffirms its vision to become a regional center of excellence in pediatric cell and gene therapy, enhancing access to advanced, personalized, and life-changing treatments.