Sidra Medicine Brings Gene Therapy Casgevy to Qatar Patients

Sidra Medicine, a member of Qatar Foundation, has been selected by Vertex Pharmaceuticals as one of a limited number of hospitals worldwide to deliver Casgevy, a one-time gene therapy based on CRISPR/Cas9 technology.

The hospital confirmed that Casgevy is now available in Qatar for patients aged 12 years and above who are living with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).

Chief Medical Officer Prof. Ibrahim Janahi said this step marks a major milestone, as Sidra Medicine becomes the first hospital in Qatar to provide a gene-editing therapy with the potential to offer a functional cure. He added that this achievement strengthens Qatar’s position as a regional leader in advanced and precision medicine.

Casgevy has received approvals from major global health authorities, including the Ministry of Public Health, U.S. Food and Drug Administration, and European Medicines Agency. It is the first approved treatment using CRISPR/Cas9 gene-editing technology to target the root genetic cause of these inherited blood disorders.

Clinical results have shown strong outcomes, including independence from regular blood transfusions for thalassemia patients and significant improvements in the quality of life for people with sickle cell disease.

Hisham Hagar, Executive Country Manager at Vertex GCC, said the launch of Casgevy in Qatar represents a major advancement in treating serious diseases. He highlighted the strong partnership with Sidra Medicine and expressed confidence that the therapy will provide long-lasting benefits to eligible patients.

Sidra Medicine currently treats around 150 to 200 children in Qatar diagnosed with thalassemia and sickle cell disease.

Dr. Ahmed Al Hammadi, Chair of Pediatric Medicine, said the hospital aims to provide not only advanced treatments but also compassionate, family-centered care. He noted that Casgevy represents both a scientific breakthrough and a commitment to improving the lives of young patients.

Chief Research Officer Prof. Khalid Fakhro added that this achievement reflects Qatar’s focus on precision health, where treatment is based on each patient’s genetic profile. He said bringing therapies like Casgevy to Qatar supports the country’s goal of becoming a leader in genomic medicine in the region.

Casgevy has already been successfully used in patients globally outside clinical trials, helping many achieve transfusion independence and better quality of life. With this development, Sidra Medicine aims to expand access to advanced, personalized treatments and strengthen its role as a regional center for gene and cell therapy.