Bahrain became the second country in the world to approve Casgevy, a CRISPR-based therapy for sickle cell disease and transfusion-dependent beta-thalassaemia. The decision came on 2 December 2023, and no other country in the Middle East had acted before it.
Casgevy, known generically as exagamglogene autotemcel, was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. It is the first licensed therapy built on CRISPR/Cas9 gene editing, a technique whose inventors won the 2020 Nobel Prize in Chemistry. Both conditions are inherited blood disorders that weigh heavily on health and life expectancy, and the treatment is described as a potential functional cure.
Bahrain says the approval followed a rigorous review of the therapy’s safety, quality and efficacy.
How the treatment works
The process runs in stages. First, injections push the patient’s bone marrow to make large numbers of stem cells. Those cells are edited so they go on to produce red blood cells carrying functional haemoglobin. After quality and safety testing, the edited cells are transplanted back into the patient’s bloodstream. Before that, the patient goes through a full assessment and a blood exchange transfusion to prepare the marrow, and the harvested cells travel to a laboratory for the genetic work. Afterward, doctors watch closely while the new cells settle into the marrow and begin producing healthy cells.
From approval to a first patient
The Bahrain Oncology Centre (BOC) was the hospital that took on the task. In 2024 it received Vertex accreditation for its bone marrow transplant and cellular therapy services, which allowed it to offer Casgevy as an accredited centre of excellence. The accreditation came after a review of documents the centre submitted and an on-site audit.
On 16 February 2025, Bahrain’s National Communication Centre announced that the BOC had treated a sickle cell patient with the therapy. It described this as the first successful treatment of its kind outside the United States.
Health Minister Dr. Jaleela bint AlSayed Jawad Hasan said: “This milestone exemplifies Bahrain’s commitment to integrating global medical innovations into our national healthcare strategy.” Brigadier Dr. Shaikh Fahad bin Khalifa bin Salman Al Khalifa, Commander of the Royal Medical Services, said the treatment “provides renewed hope for patients with complex blood disorders.”
Dr Edward Rowland, chief executive of the BOC, spoke for the clinical team. “We are honoured to bring CRISPR-based therapy to patients in Bahrain and beyond,” he said. The programme itself was a joint effort of the Ministry of Health, the Royal Medical Services, Government Hospitals and the National Health Regulatory Authority.
The centre says it can now offer Casgevy to patients with sickle cell disorder and beta thalassaemia from all over the globe. Anyone living with either condition should talk to their doctor about whether this option fits their situation.
Sources
- Bahrain Makes History with First Successful CRISPR-Based Sickle Cell Treatment Outside the US
- Bahrain Oncology Centre Pioneers Sickle Cell and Thalassemia Gene Therapy
