UK Approves World’s First CRISPR Gene Therapy for Blood Disorders

According to a recent Reuters article, the United Kingdom has authorized the use of a gene therapy called Casgevy to treat sickle-cell disease and another inherited blood disorder in patients over the age of 12. The move marks the first instance of approval for a medicine using CRISPR, which was awarded the Nobel Prize in 2020. Sickle cell disease and β-thalassemia result from genetic errors affecting the production of hemoglobin, crucial for transporting oxygen in red blood cells. 

Casgevy, administered by modifying a patient’s bone marrow stem cells in a laboratory and then infusing them back after conditioning treatment, has shown promise in restoring healthy hemoglobin production during clinical trials. The UK’s Medicines and Healthcare products Regulatory Agency stated that the therapy showed positive results and had no significant safety concerns during trials.