The Cas9 protein together with RNA and DNA (orange; artist's illustration). A new therapy uses the CRISPR-Cas9 gene editing system to treat a rare genetic disease called hereditary angioedema.Credit: Alfred Pasieka/Science Photo Library The therapy, named lonvoguran ziclumeran (lonvo-z), was designed to treat one form of a genetic disorder that causes episodes of painful and potentially dangerous swelling in the throat and other parts of the body. Lonvo-z deploys the CRISPR-Cas9 gene-editing system to disable the gene that produces prekallikrein, a protein made in the liver that promotes blood-vessel leakage. Intellia, headquartered in Cambridge, Massachusetts, has applied for approval of lonvo-z to the US Food and Drug Administration (FDA), which is expected to issue a decision by 10 March. If approved, the therapy will become the world's first CRISPR medicine to be delivered directly inside the body, or in vivo, John Leonard, Intellia's chief executive, told investors on 12...
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