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In February, the US Food and Drug Administration (FDA) proposed a radical rethink of how scientists, physicians and manufacturers develop personalized genetic therapies. The regulator’s suggested introduction of a ‘plausible mechanism pathway’ should increase incentives for drug companies to develop personalized treatments, including gene-editing therapies for rare disorders (see go.nature.com/4mnmgzq).
Some 350 million people worldwide live with one of more than 5,000 genetic diseases1. Many of these conditions could, in theory, be treated with personalized therapies, which correct a single genetic mutation in a person’s DNA using the CRISPR gene-editing tool. In February 2025, KJ Muldoon, a baby born in the United States with a life-threatening metabolic disorder, became the first person to receive this type of personalized treatment2.
Yet, so far, the bespoke nature of such therapies has posed a huge obstacle to their spread. Much as a letter needs to be labelled with the right address, so CRISPR needs to be programmed to find and repair a mutation using a tiny string of nucleic acids called a guide RNA, which can be...