Post by : Badri Ariffin

Sidra Medicine, part of the Qatar Foundation, has been chosen by Vertex Pharmaceuticals as one of the select hospitals worldwide to provide Casgevy, a revolutionary one-time gene therapy utilizing CRISPR/Cas9 technology.

The hospital announced the availability of Casgevy in Qatar for patients aged 12 years and older suffering from transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).

Prof. Ibrahim Janahi, Chief Medical Officer, highlighted this development as a groundbreaking achievement, making Sidra the first hospital in Qatar to offer a gene-editing therapy that could potentially serve as a functional cure. He emphasized that this progress underscores Qatar’s emerging leadership in advanced and precision medicine in the region.

Casgevy has received endorsements from leading global health organizations, including the Ministry of Public Health, U.S. Food and Drug Administration, and European Medicines Agency. It stands as the first sanctioned treatment employing CRISPR/Cas9 gene-editing technology to address the genetic origins of these inherited blood disorders.

Clinical evidence indicates remarkable outcomes, including the independence of thalassemia patients from regular blood transfusions and notable enhancements in the quality of life for those with sickle cell disease.

Hisham Hagar, Executive Country Manager at Vertex GCC, stated that the introduction of Casgevy in Qatar marks a significant milestone in treating critical diseases. He praised the solid collaboration with Sidra Medicine, expressing optimism that the therapy will yield enduring benefits for eligible patients.

Currently, Sidra Medicine is caring for approximately 150 to 200 children in Qatar who are diagnosed with thalassemia and sickle cell disease.

Dr. Ahmed Al Hammadi, Chair of Pediatric Medicine, remarked that the hospital is dedicated to delivering not just progressive treatments but also compassionate, family-oriented care. He asserted that Casgevy signifies both a scientific innovation and a steadfast commitment to enhancing the lives of young patients.

Prof. Khalid Fakhro, Chief Research Officer, further stated that this milestone mirrors Qatar’s focus on personalized healthcare, where treatments are tailored according to individual genetic profiles. He mentioned that introducing therapies like Casgevy in Qatar aids the nation's aspiration to become a pioneer in genomic medicine in the region.

Casgevy has already demonstrated success in patients globally beyond clinical trials, allowing many to achieve transfusion independence and an improved quality of life. With this initiative, Sidra Medicine aims to broaden access to personalized advanced treatments and strengthen its position as a regional hub for gene and cell therapy.

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