The UK has become the first country in the world to approve a therapy based on Crispr gene editing, with the regulator authorising a treatment for sickle cell disease and beta thalassaemia.
The Medicines and Healthcare products Regulatory Agency has approved the therapy, called Casgevy, which was developed by Vertex Pharmaceuticals and Crispr Therapeutics. The drug could be used to replace 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, leading to a Nobel Prize in 2020.
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