Doha, April 21 (QNA) -  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).

Chief Medical Officer at Sidra Medicine Prof. Ibrahim Janahi 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. The therapy is available to children in 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 the first-in-class CRISPR/Cas 9 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.

Executive Country Manager at Vertex GCC Hisham Hagar 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. We are confident that this treatment will offer the potential for a durable and impactful treatment option for eligible patients with transfusion-dependent beta thalassemia and severe sickle cell disease in Qatar."

Sidra Medicine currently follows 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 said: "Our goal is to ensure that every child treated at Sidra Medicine receives not only the most advanced therapies but also compassionate, family-centered 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. By integrating innovation with compassion, we are redefining what it means to deliver excellence in pediatric care."

Chief Research Officer at Sidra Medicine Prof. Khalid Fakhro added that: "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 personalized care for children living with rare and inherited diseases. This is a testament to our national commitment to advancing genomic medicine and positioning Qatar as a leader in precision health across the region."

Casgevy has already been successfully administered to patients globally outside of clinical trials, achieving transfusion independence and significant improvements in quality of life. With this milestone, Sidra Medicine reaffirms its vision to become a regional center of excellence in pediatric cell and gene therapy, expanding access to advanced, personalized, and life-changing treatments. (QNA)