Saturday, February 22, 2025

Bahrain Makes History with First Successful CRISPR-Based Sickle Cell Treatment Outside the US

The Bahrain Oncology Centre has made medical history by successfully treating a sickle cell disease patient using CRISPR-based gene-editing therapy, Casgevy. This marks the first successful treatment of its kind outside the United States, positioning Bahrain as a global leader in precision medicine and innovative healthcare solutions.

Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first licensed therapy to utilise CRISPR/Cas9 gene-editing technology, whose inventors were awarded the 2020 Nobel Prize in Chemistry. The therapy offers a potential functional cure for SCD and transfusion-dependent beta-thalassaemia —inherited blood disorders that significantly impact health and life expectancy.

This achievement follows Bahrain’s landmark decision on 2 December 2023 to become the second country globally and the first in the Middle East to approve Casgevy for the treatment of SCD and TDT. The approval was granted after a rigorous evaluation of the therapy’s safety, quality, and efficacy.

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The multi-stage transplant process involves stimulating the patient’s bone marrow to produce large quantities of stem cells using specialised injections, then editing the stem cells to produce red blood cells carrying functional haemoglobin. The third and final stage is transplanting the edited cells back into the patient’s bloodstream after rigorous quality and safety testing. This process can be used to treat Thalassaemia (Mediterranean anaemia).

HE. Dr. Jaleela bint AlSayed Jawad Hasan, Minister of Health, said: “This milestone exemplifies Bahrain’s commitment to integrating global medical innovations into our national healthcare strategy. By fostering partnerships across institutions, we are delivering on our mandate to provide access to life-changing therapies for all beneficiaries and positioning Bahrain as a hub for innovative medical care, in line with the visionary directives of His Majesty King Hamad bin Isa Al Khalifa and His Royal Highness Prince Salman bin Hamad Al Khalifa, the Crown Prince and Prime Minister.”

“Bahrain is proud to be at the forefront of cutting-edge healthcare advancements in the region. The successful administration of Gene edited autologous stem cell transplant demonstrates our dedication to providing access the most advanced treatments and reinforces our position as a regional hub for precision medicine,” said Brigadier Dr. Shaikh Fahad bin Khalifa bin Salman Al Khalifa, Commander of the RMS. “This provides renewed hope for patients with complex blood disorders and reinforces the Kingdom’s growing focus on healthcare innovation.”

“As the clinical team responsible for delivering this groundbreaking treatment, we are honoured to bring CRISPR-based therapy to patients in Bahrain and beyond. Our success reflects the Bahrain Oncology Centre’s dedication to combining advanced technology with world-class expertise. This achievement, validated by our international accreditation in bone marrow transplantation, underscores our mission to transform patient outcomes through precision medicine and collaboration with global pioneers,” said Dr Edward Rowland, CEO of Bahrain Oncology Centre.

SOURCE: GlobeNewswire

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