Gene editing therapy for sickle cell approved in Bahrain.

Bahrain has become the second country to approve the gene editing therapy Casgevy (exagamglogene autotemcel) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), following its approval in the UK last month. The therapy is intended for adults and children aged 12 and older, who are eligible for a stem cell transplant but lack a donor, and those with SCD who have recurrent vaso-occlusive crises (VOCs). The approval of Casgevy in Bahrain demonstrates the country’s dedication to innovation and improving the lives of its citizens. The therapy is currently under priority review for approval in the US, with decisions pending for the European Union and Saudi Arabia.

Casgevy works by increasing the production of fetal hemoglobin, which is more effective at transporting oxygen than adult hemoglobin and can reduce hemoglobin clumping. The therapy involves collecting and engineering patients’ hematopoietic stem cells, which are then modified using the CRISPR gene editing tool to increase fetal hemoglobin production. The modified cells are then returned to the patient through a stem cell transplant. Regulatory approvals have been based on data from ongoing clinical trials, including one evaluating the safety and efficacy of Casgevy in individuals with severe SCD who had experienced at least two VOCs in each of the two years prior to enrollment. The main goal of the trial, which is set to conclude in October 2024, was met, with 29 out of 30 patients being free of VOCs in the first year after treatment.

The approval of Casgevy in Bahrain marks a significant advancement in the available treatment options for patients with SCD and TDT in the country. It reflects Bahrain’s commitment to providing high-quality healthcare and staying at the forefront of scientific breakthroughs. As regulatory decisions from other countries are expected in the near future, further expansions in treatment accessibility may be on the horizon.

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