STAT+: 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.

In 2012, when Charpentier and Jennifer Doudna published their landmark paper on editing DNA in a test tube with CRISPR, almost no one believed that a treatment based on the technology would be reaching patients in little more than a decade.

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