Key questions (and answers) about the historic approval of a CRISPR-based medicine
History just happened.
For the first time, a regulator has cleared a treatment using CRISPR, the gene editing technology, for patients. The regulator is the United Kingdom’s Medicines and Healthcare products Regulatory Agency. The product is Casgevy, a treatment for sickle cell disease and beta thalassemia, two blood disorders. It was developed by CRISPR Therapeutics, the Swiss company co-founded by Nobel laureate Emmanuelle Charpentier, and Vertex Pharmaceuticals, a large Boston-based biotech firm.
