1st SCD patient outside US receives gene-editing therapy Casgevy
Bahrain positioned as 'hub for innovative medical care,' health minister says
A sickle cell disease (SCD) patient in Bahrain received the gene-editing therapy Casgevy (exagamglogene autotemcel), becoming the first patient outside the U.S. to be treated with the drug.
Bahrain approved Casgevy in late 2023, becoming the first country in the Middle East to approve the therapy and the second to do so worldwide.
“This milestone exemplifies Bahrain’s commitment to integrating global medical innovations into our national healthcare strategy,” Jaleela bint AlSayed Jawad Hasan, Bahrain’s minister of health, said in a press release from the Bahrain Oncology Centre, where the treatment was given. “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.”
Fahad bin Khalifa bin Salman Al Khalifa, commander of Bahrain’s Royal Medical Services, said the milestone “provides renewed hope for patients with complex blood disorders and reinforces the Kingdom’s growing focus on healthcare innovation.”
“Bahrain is proud to be at the forefront of cutting-edge healthcare advancements in the region,” Al Khalifa said, adding that the successful treatment “demonstrates our dedication to providing access [to] the most advanced treatments and reinforces our position as a regional hub for precision medicine.”
Gene-editing therapy enables hemoglobin production
SCD is caused by mutations in a gene that encodes part of hemoglobin, the protein red blood cells use to transport oxygen through the body. These mutations lead to the production of an abnormal form of hemoglobin that clumps in red blood cells, deforming them into the sickle-like shape that gives the disease its name and ultimately drives SCD symptoms.
The disease affects the adult version of hemoglobin. An alternative form of the protein, fetal hemoglobin, is normally produced during early fetal development, but production stops soon after birth. Casgevy uses the gene-editing technology CRISPR/Cas9 to increase the production of fetal hemoglobin in blood cells, allowing the fetal form of the protein to compensate for the defective adult hemoglobin.
Casgevy works by first collecting stem cells, located in a patient’s bone marrow, that are responsible for giving rise to new blood cells. The stem cells are engineered in a lab, then returned to the patient via a stem cell transplant so that they can give rise to red blood cells capable of producing fetal hemoglobin.
“As the clinical team responsible for delivering this groundbreaking treatment, we are honoured to bring CRISPR-based therapy to patients in Bahrain and beyond,” said Edward Rowland, the oncology center’s CEO. “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.”
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