Bahrain Reports First Administration of CASGEVY in the Middle East

The Bahrain Oncology Centre announced a major milestone on Sunday with the successful administration of CASGEVY - the world's only approved therapy using CRISPR gene-editing technology - to treat a sickle cell disease patient, marking the first CASGEVY administration in the Middle East region.

By: Karen O'Hanlon Cohrt - Feb. 19, 2025

Lay summary - first CRISPR therapy administered in Middle East

Doctors in Bahrain have successfully used a gene-editing therapy called CASGEVY to treat a patient with sickle cell disease, marking the first time this treatment has been used in the Middle East.

CASGEVY is the world's first approved therapy that uses CRISPR, a revolutionary gene-editing technology. It works by modifying blood stem cells taken from the patient to increase production of a special type of haemoglobin that the body normally only makes during foetal development. This helps compensate for the defective haemoglobin causing sickle cell disease.

CASGEVY treatment involves collecting the patient's blood stem cells, editing them in a laboratory, and then returning them to the patient. In clinical trials, CASGEVY eliminated painful crisis episodes in 97% of patients for at least a year. This milestone highlights Bahrain's growing role in advanced medical treatments, with their oncology center now accredited to offer this therapy to patients from across the region.

Meet one of the first CASGEVY patients at the 2nd CRISPR Medicine Conference, CRISPRMED25, in Copenhagen, Denmark, April 8-11, 2025.

The Bahrain Oncology Centre (BOC) announced a major milestone on Sunday with the first successful administration of the only approved gene-editing therapy, CASGEVY, outside of the United States. The patient was treated at the Bone Marrow and Stem Cell Transplant Unit at the Royal Medical Services in BOC.

CASGEVY, jointly developed by Vertex Pharmaceuticals and CRISPR Therapeutics, was first approved for the treatment of sickle cell disease (SCD) in the United Kingdom in November 2023. It works by reactivating foetal haemoglobin (HbF) production to compensate for the lack of functional adult haemoglobin seen in SCD and the related haemoglobinopathy beta thalassemia.

CASGEVY: Mechanism, efficacy, and global regulatory approvals

The development of CASGEVY built on decades of research which revealed that HbF is highly expressed and critical during foetal development, but then rapidly suppressed early in life. This research also led to the discovery that the B-cell lymphoma/leukaemia 11A gene, BCL11A, acts as a negative regulator of HbF expression. CASGEVY is a one-time, potentially curative treatment that works by disrupting BCL11A to increase the production of HbF.

In the Phase 3 clinical trial of CASGEVY in severe SCD (CLIMB SCD-121), treatment with CASGEVY eliminated vaso-occlusive crises in 97% of patients with SCD for a period of 12 months or more (1).

Bahrain's first administration of CASGEVY follows a number of regulatory approvals across the world. The UK’s Medicines and Healthcare products Regulatory Agency was the first to approve the therapy in November 2023 for both SCD and beta thalassaemia, with Bahrain following suit on 2nd December 2023. The United States FDA approved CASGEVY for SCD in December 2023 and expanded its approval to include beta thalassaemia in January 2024. Saudi Arabia’s Food and Drug Authority (SFDA) and the European Medicines Agency granted approvals for both conditions in early 2024, followed by Canada’s Drug Agency in September 2024.

Sickle cell disease (SCD) results from a single-point mutation in the haemoglobin subunit beta (HBB) gene and is associated with a range of symptoms and life-limiting complications. Vaso-occlusive crises (VOEs or VOCs) are the most commonly encountered complication in severe SCD, and these occur when small blood vessels get blocked because of sickled red blood cells which tend to be stiffer than healthy cells, and because the defective form of haemoglobin seen in SCD (haemoglobin S) renders the red blood cell membranes sticky.

Daily treatment for SCD patients is limited to symptom control, dietary intervention and pain management. While a bone marrow transplant from a healthy matched donor has traditionally been the only (albeit slight) hope of a cure for the haemoglobinopathies, gene-editing technology unleashes the possibility to cure a patient using their own cells. Central to many of the current approaches is reactivation of foetal haemoglobin (HbF) expression.

Administering CASGEVY in Bahrain

In 2024, BOC received Vertex accreditation for its bone marrow transplant and cellular therapy services, enabling it to provide CASGEVY, as an accredited centre of excellence, to SCD and beta thalassaemia patients from all over the globe.

According to the BOC’s press release, administering CASGEVY to patients in Bahrain involves a multi-stage process that begins with stimulating the patient's bone marrow to produce stem cells, using CRISPR-Cas technology to disrupt BCL11A in these cells ex vivo, and then returning the edited cells to the patient after comprehensive safety testing.

We will continue to share news about gene-editing therapies as new developments emerge. In the meantime, and for a complete overview of current gene editing clinical trials, check out CRISPR Medicine News' Clinical Trials Database.

References:

Frangoul H, Locatelli F, Sharma A, et al; CLIMB SCD-121 Study Group. Exagamglogene Autotemcel for Severe Sickle Cell Disease. N Engl J Med. 2024 May 9;390(18):1649-1662.

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