The lucky break behind the first CRISPR treatment The world's first commercial gene-editing treatment is set to start changing the lives of people with sickle-cell disease. It's called Casgevy, and it was approved last month in the UK. US approval is pending this week. The treatment, which will be sold in the US by Vertex Pharmaceuticals, employs CRISPR, which can be easily programmed by scientists to cut DNA at precise locations they choose. But where do you aim CRISPR, and how did the researchers know what DNA to change? That's the lesser-known story of the sickle-cell breakthrough, which doesn't rely on fixing the genes responsible for the mutation that leaves patients' hemoglobin molecules misshapen. Instead, it's a kind of molecular bank shot—thankfully, one with a happy ending. Read the full story. —Antonio Regalado Read more about the sickle-cell breakthrough: + I received the new gene-editing drug for sickle cell disease. It changed my life. As a patient enrolled in a clinical trial for Vertex's new exa-cel treatment, Jimi Olaghere was among the first to experience CRISPR's transformative effects. Read the full story. + The first CRISPR cure might kick-start the next big patent battle. Vertex Pharmaceuticals plans to sell a gene-editing treatment for sickle-cell disease. A patent on CRISPR could stand in the way. Read the full story. |
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