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The UK has become the first country in the world to approve a therapy based on editing the Crispr gene, with the regulator approving a treatment for sickle cell anemia and beta thalassemia.
The Medicines and Healthcare products Regulatory Agency has approved the therapy called Casgevy, developed by Vertex Pharmaceuticals and Crispr Therapeutics. The drug could be used as a replacement for bone marrow transplants.
Crispr is a flexible and efficient gene editing tool based on the bacterial immune system. Scientists Jennifer Doudna and Emmanuelle Charpentier discovered Crispr in 2012 and received the Nobel Prize for it in 2020.