LONDON — Regulators in the U.K. on Thursday approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia, making it the world’s first therapy built on the revolutionary gene-editing technology and ushering in a new era of genetic medicine.
The authorization of the therapy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is itself not a surprise. Clinical trials have shown the one-time treatment enabled many sickle-cell patients to live free of debilitating pain crises and relieved thalassemia patients of needing regular blood transfusions, raising hopes that they have been effectively cured. But it also stands as a major achievement, coming just over a decade after the first and seminal lab experiments that showed CRISPR’s potential as a gene-editing tool were published.
In granting conditional approval to the therapy, which will have the brand name Casgevy and is also known as exa-cel, the U.K.’s Medicines and Healthcare products Regulatory Agency jumped ahead of its counterparts in the U.S. and Europe, which are also reviewing the medicine, in greenlighting the globe’s first CRISPR treatment. The MHRA’s authorization is for patients who are at least 12 years old.