Last week, in a global milestone for medicine, U.K. regulators approved the first-ever CRISPR gene-editing drug. The treatment, from Vertex and CRISPR Therapeutics, will be licensed under the brand name Casgevy in the U.K. for patients with sickle cell disease (SCD) and beta thalassemia.
Through CRISRP gene editing, Casgevy, or exa-cel, provides a fix at the DNA level by editing the offending genes in a lab and then reinfusing the hemoglobin-producing cells into patients. The clinical evidence shows its benefits might last a lifetime, as the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) noted in a statement announcing the approval on Thursday.
In the U.S., the Food and Drug Administration was already expected to approve the CRISPR drug by the end of the year to treat sickle cell disease, and by spring 2024 to treat beta thalassemia, barring any unforeseen consequences. The MHRA's clearance might speed up that process and fuel questions and concerns about the role of gene editing in medicine as groundbreaking science hits the market.
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