Sidra Medicine Launches Qatar’s First CRISPR Gene Therapy for Sickle Cell Disease and Thalassemia
Doha, Qatar: Sidra Medicine, part of the Qatar Foundation, has been designated by Vertex Pharmaceuticals as one of a select few hospitals globally authorized to administer Casgevy, an innovative CRISPR/Cas9-based gene therapy. This treatment is now available in Qatar for patients aged 12 and older suffering from transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).
Pioneering Gene Therapy in Qatar
Prof. Ibrahim Janahi, Chief Medical Officer at Sidra Medicine, expressed pride in the hospital’s selection as the first in Qatar to provide this groundbreaking gene-editing therapy, which has the potential to serve as a functional cure. He emphasized that this milestone underscores Qatar’s leadership in advanced medicine and precision health. Through collaboration with Vertex, Sidra Medicine is introducing the world’s first approved therapy utilizing Nobel Prize-winning CRISPR/Cas9 technology to address these severe blood disorders.
Casgevy has received approval from the Ministry of Public Health in Qatar, as well as from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It is the first CRISPR/Cas9 gene-edited therapy targeting the genetic causes of these inherited conditions. Clinical results have shown significant improvements for patients with sickle cell disease and have enabled transfusion independence for those with TDT.
Significant Developments in Treatment Options
Hisham Hagar, Executive Country Manager at Vertex GCC, noted that the introduction of this therapy in Qatar represents a substantial advancement in combating serious diseases. He highlighted the importance of the partnership with Sidra Medicine in achieving this goal and expressed confidence that Casgevy will provide a durable treatment option for eligible patients in Qatar.
Currently, Sidra Medicine monitors approximately 150 to 200 children diagnosed with thalassemia and sickle cell disease. While not all require gene therapy, Casgevy is specifically intended for patients with the most severe forms of SCD or TDT—those who continue to experience frequent pain crises, are at risk for strokes, or face the burden of lifelong transfusions despite receiving optimal medical care. For these patients, Casgevy offers a novel, one-time intervention targeting the underlying genetic issues.
A Historic Advancement in Pediatric Care
Dr. Chiara Cugno, Acting Chief of Pediatric Hematology, Oncology, and Bone Marrow Transplant at Sidra Medicine, described the launch of Casgevy as a historic advancement for individuals living with TDT and severe SCD. She noted that this therapy not only brings hope but also the potential for a functional cure by addressing the genetic origins of these diseases. The team at Sidra Medicine has collaborated closely with Vertex to ensure that all clinical, ethical, and technical protocols are established for the safe and effective delivery of this treatment.
With the approval to administer Casgevy, eligible patients in Qatar can now access this potentially curative therapy locally. The hospital has already onboarded its first patient, who is currently undergoing assessment for the treatment.
Commitment to Compassionate Care
Dr. Ahmed Al Hammadi, Chair of Pediatric Medicine at Sidra Medicine, emphasized the institution’s commitment to providing advanced therapies alongside compassionate, family-centered care. He stated that Casgevy represents more than just a scientific breakthrough; it embodies the hospital’s dedication to improving the lives of young patients facing these challenging conditions. By merging innovation with compassion, Sidra Medicine aims to redefine excellence in pediatric healthcare.
Prof. Khalid Fakhro, Chief Research Officer at Sidra Medicine, remarked that this milestone exemplifies the institution’s vision for precision health, where treatments are tailored to each patient’s unique genetic profile. By introducing transformative gene therapies like Casgevy to Qatar, Sidra Medicine is advancing its mission to deliver personalized care for children with rare and inherited diseases, reinforcing Qatar’s position as a leader in genomic medicine.
The Treatment Journey
The treatment process at Sidra Medicine begins with the safe collection of a patient’s stem cells through apheresis. These cells are then sent to specialized laboratories in collaboration with Vertex, where they undergo gene editing and validation using CRISPR/Cas9 technology. Once the cells are edited, they are returned to Sidra Medicine and preserved until they are ready for infusion.
Prior to the infusion, patients receive chemotherapy to prepare their bodies for the new cells. The gene-edited cells are then infused back into the patient, where they begin to proliferate in the bone marrow and produce healthy red blood cells.
This intricate and specialized process is followed by a comprehensive post-treatment recovery and long-term monitoring plan, managed 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, inquiries can be directed to International.Services@sidra.org.
About Sidra Medicine
Sidra Medicine is a private, not-for-profit academic healthcare and research institution dedicated to women, children, and young people. Established by the Qatar Foundation for Education, Science, and Community Development, Sidra Medicine is committed to delivering exceptional patient care, conducting innovative biomedical and clinical research, and providing personalized treatment journeys for rare and genetic diseases.
For specialized healthcare services, including pediatric care and rare disease treatment, visit Sidra Medicine or download the Sidra Medicine app from the App Store or Google Play.
Source: www.zawya.com
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Published on 2026-04-21 17:41:00 • By the Editorial Desk
