The first medical treatment that uses Crispr gene editing was authorized [November 16] by the United Kingdom.
The one-time therapy, which will be sold under the brand name Casgevy, is for patients with sickle cell disease and a related blood disorder called beta thalassemia, both of which are inherited. The UK approval marks a historic moment for Crispr, the molecular equivalent of scissors that won its inventors a Nobel Prize in 2020.
Developed by Vertex Pharmaceuticals of Boston and Crispr Therapeutics of Switzerland, Casgevy is meant to prevent episodes of excruciating pain that are typical of sickle cell disease and free people with beta thalassemia of regular blood transfusions. The treatment involves editing a patient’s own cells outside the body and infusing them back in. For some, the therapy may even be a cure.
Europe and the United States are poised to approve the Crispr therapy soon as well. The US Food and Drug Administration has until December 8 to make a decision. On October 31, an advisory committee to the FDA concluded that the treatment was safe for patients.
