Post by : Bianca Suleiman
Sidra Medicine, part of Qatar Foundation, has been chosen by Vertex Pharmaceuticals as one of the select hospitals globally to administer Casgevy, an innovative one-time gene therapy utilizing CRISPR/Cas9 technology.
The hospital announced that Casgevy is now accessible in Qatar for individuals aged 12 and above diagnosed with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).
Prof. Ibrahim Janahi, Chief Medical Officer, remarked that this announcement is a significant achievement, as Sidra Medicine becomes the first facility in Qatar to offer a gene-editing therapy potentially delivering a functional cure. He emphasized that this success bolsters Qatar’s status as a frontrunner in advanced, precise medical treatments.
Casgevy has secured approvals from leading international health organizations, including the Ministry of Public Health, U.S. Food and Drug Administration, and European Medicines Agency. It stands as the inaugural approved therapy employing CRISPR/Cas9 gene-editing technology aimed at addressing the root genetic factors of these inherited blood disorders.
Clinical trials have demonstrated compelling results, showcasing the ability for thalassemia patients to achieve independence from regular blood transfusions and notable enhancements in quality of life for those with sickle cell disease.
Hisham Hagar, Executive Country Manager at Vertex GCC, conveyed that the introduction of Casgevy in Qatar signifies a major leap forward in combating severe illnesses. He praised the solid collaboration with Sidra Medicine and expressed optimism regarding the therapy’s long-term benefits for eligible patients.
Currently, Sidra Medicine caters to approximately 150 to 200 children in Qatar suffering from thalassemia and sickle cell disease.
Dr. Ahmed Al Hammadi, Chair of Pediatric Medicine, remarked that the institution is dedicated to offering not just advanced therapies but also compassionate, family-centered care. He asserted that Casgevy embodies both a scientific milestone and a commitment to enhancing the lives of young patients.
Prof. Khalid Fakhro, Chief Research Officer, highlighted that this milestone exemplifies Qatar’s ambition in precision health, tailoring treatments according to each patient’s genetic profile. He stated that facilitating access to therapies like Casgevy in Qatar further strengthens the country’s aim of becoming a leader in genomic medicine across the region.
Casgevy has already been effectively utilized in patients internationally, outside of clinical trials, aiding many in attaining transfusion independence and improved quality of life. With this advancement, Sidra Medicine intends to broaden access to cutting-edge, personalized treatments while reinforcing its role as a regional hub for gene and cell therapy.
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