First-in-human prime editing rewrites DNA inside liver to treat Wilson disease

Prime Medicine, based in Cambridge, Massachusetts, has performed the first in-vivo prime-editing procedure on a person suffering from Wilson disease, a genetic disorder that leads to dangerous copper accumulation. The treatment precisely rewrites the faulty ATP7B gene in liver cells, eliminating the mutation that blocks copper excretion. The trial's initial patient was treated earlier this month, and the company plans to publish early data next year.

Rhonda Rowland, president of the Wilson Disease Association, hailed the breakthrough, while biomedical engineer Krishanu Saha warned that the method entails distinct risk trade-offs. In-vivo editing could make therapies cheaper and faster than the conventional ex-vivo cell-editing model, yet no gene-editing drug has yet received FDA approval for such use. The FDA currently approves only one CRISPR-Cas9 therapy, Casgevy, for blood disorders, and an in-vivo CRISPR-Cas9 candidate for hereditary angioedema is under review.