Qatar has taken a landmark step in precision medicine after Sidra Medicine became the first hospital in the country — and one of a select few worldwide — to be qualified by Vertex Pharmaceuticals to administer Casgevy, a revolutionary one-time gene therapy that uses Nobel Prize-winning CRISPR/Cas9 technology to treat inherited blood disorders.
Casgevy is now available in Qatar for patients aged 12 and above living with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), with Sidra Medicine having already onboarded its first patient for assessment.
Prof Ibrahim Janahi, chief medical officer at Sidra Medicine, said: "We are proud to be chosen as the first hospital in Qatar to deliver a gene editing therapy with the potential for a functional cure. This milestone reinforces Qatar's position as a regional leader in advanced medicine and precision health. Through our collaboration with Vertex, we are bringing the world's first approved medicine using the Nobel Prize winning CRISPR/Cas9 technology to treat transfusion-dependent beta thalassemia and severe sickle cell disease."
Approved by the Ministry of Public Health in Qatar, as well as the US Food and Drug Administration, the European Medicines Agency, and other agencies, Casgevy is the first-in-class CRISPR/Cas9 gene-edited therapy that targets the genetic root cause of these inherited blood disorders. The therapy has shown transformative outcomes for patients with sickle cell disease and independence from regular blood transfusions for patients with transfusion-dependent beta thalassemia.
Hisham Hagar, executive country manager at Vertex GCC, said: "The launch of the CRISPR/Cas9 therapy in Qatar represents a significant leap forward in our efforts against serious diseases. This achievement is a direct result of our invaluable partnership with Sidra Medicine and reinforces Vertex's mission to transform lives through scientific innovation."
Sidra Medicine currently follows approximately 150 to 200 children in Qatar diagnosed with thalassemia and sickle cell disease. While not all of them need gene therapy, Casgevy is reserved for patients with the most severe form of SCD or TDT — those still experiencing frequent pain crises, stroke risk, or the cumulative burden of lifelong transfusions despite the best medical care.
Dr Chiara Cugno, acting chief of paediatric Haematology, Oncology and Bone Marrow Transplant and director of the Advanced Cell Therapy Core at Sidra Medicine, said: "The introduction of Casgevy marks a historic advancement for people living with transfusion-dependent beta thalassemia and severe sickle cell disease. This therapy offers not only hope but the potential for a functional cure, by addressing these diseases at their genetic origin."
Sidra Medicine's approval as a Casgevy treatment centre means that eligible patients in Qatar will now have access to this one-time, potentially curative therapy closer to home.
Dr Ahmed al-Hammadi, chair of paediatric medicine at Sidra Medicine, said: "Our goal is to ensure that every child treated at Sidra Medicine receives not only the most advanced therapies but also compassionate, family-centred care throughout their journey. Casgevy represents more than a scientific breakthrough. It embodies our commitment to improving the lives of young patients living with these challenging conditions."
Prof Khalid Fakhro, chief research officer at Sidra Medicine, added: "This milestone exemplifies our precision health vision, where treatment is guided by each patient's unique genetic makeup. By bringing transformative gene therapies like Casgevy to Qatar, Sidra Medicine is leveraging cutting-edge research and clinical practice to deliver personalised care for children living with rare and inherited diseases."
Casgevy has already been successfully administered to patients globally outside of clinical trials, achieving transfusion independence and significant improvements in quality of life.