The First Crispr Medicine Just Got Approved

The gene-editing therapy, called Casgevy, uses Crispr to prevent debilitating pain in patients with sickle cell disease. It also eliminates the need for regular blood transfusions in people with beta thalassemia.

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 other genetic conditions, as well as certain types of cancers and even HIV. It works by making targeted cuts in DNA.

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.

The authorization from the UK Medicines and Healthcare products Regulatory Agency is for sickle cell patients with recurrent pain crises and for those with the most serious form of beta thalassemia who are 12 years and older. Vertex and Crispr Therapeutics estimate that about 2,000 people in the UK are eligible for the therapy.

The UK agency greenlit the groundbreaking treatment after “a rigorous assessment of its safety, quality and effectiveness,” it said in a statement. However, the approval is conditional for one year. Vertex and Crispr Therapeutics will need to provide more data on the therapy’s safety and efficacy for it to remain available.

Both sickle cell disease and beta thalassemia are genetic conditions caused by errors in the genes for hemoglobin, the vital protein found in red blood cells that carries oxygen throughout the body. Sickle cell disease disproportionately affects people of African and Caribbean descent, while beta thalassemia mainly affects people of Mediterranean, South Asian, Southeast Asian, and Middle Eastern origin.

Source: www.wired.com