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The first medical treatment that uses Crispr gene editing was authorized Thursday 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.
“This is just the start of Crispr therapies. There are a lot more to come,” says Samarth Kulkarni, chairman and CEO of Crispr Therapeutics. Adapted from a naturally occurring defense system found in bacteria, Crispr is being investigated as a way to treat a range of...