Sidra Medicine introduces Qatar’s first advanced gene-editing therapy for sickle cell disease and thalassemia

First Patient Onboarded for Treatment

21 April 2026—Doha, Qatar: Sidra Medicine, a member of Qatar Foundation, has been qualified by Vertex Pharmaceuticals as one of a of a limited number of hospitals in the world, to administer Casgevy, a groundbreaking, one-time CRISPR/Cas9-based gene therapy treatment. 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).

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. 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 U.S. 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.

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. 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. 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. For this group, where conventional treatment is no longer enough, Casgevy offers something genuinely new: a one-time intervention aimed at the root cause.

Dr. Chiara Cugno, Acting Chief of Pediatric Hematology, Oncology and BMT 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. Our team at Sidra Medicine has worked closely with Vertex to ensure that all clinical, ethical, and technical processes are in place to deliver this treatment safely and effectively.”

Sidra Medicine’s approval as a Casgevy treatment center means that eligible patients in Qatar will now have access to this one-time, potentially curative therapy closer to home. It has just onboarded its first patient who is currently undergoing assessment for the therapy.

Dr. Ahmed Al Hammadi, Chair of Pediatric 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-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.”

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 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.

The treatment journey

The treatment journey at Sidra Medicine will begin with the safe collection of a patient’s stem cells through apheresis. The cells, in collaboration with Vertex, are then sent to expert laboratories, where they undergo gene editing and validation using the CRISPR/Cas9 technology. Once edited, the cells are returned to Sidra Medicine and preserved until infused.

Before infusion, the patient receives chemotherapy to prepare their body to accept the new cells. The gene-edited cells are then infused back into the patient, where they begin to grow in the bone marrow and start producing healthy red blood cells.

This complex and highly specialized process is followed by a comprehensive post-treatment recovery and long-term monitoring plan led by Sidra Medicine’s multidisciplinary team in pediatric hematology, oncology, nephrology, and the Good Manufacturing Practice (GMP) facility. For more information about Sidra Medicine’s Hematology, Oncology and Bone Marrow Transplant Services, please visit here. For international patients, please email International.Services@sidra.org.