Science · 2026-10-23 · Morning briefing

Prime editing alters DNA inside a human for the first time

For the first time, a gene-editing technique has been used to alter DNA directly inside a person's body. Prime Medicine, a biotechnology firm in Massachusetts, announced it had treated the first patient in a clinical trial using 'prime editing,' a method more precise than standard CRISPR-Cas9. The therapy aims to correct a mutation in liver cells that causes Wilson disease, a rare genetic disorder leading to a toxic build-up of copper.

The treatment marks a significant step for in vivo gene therapies, which edit genes within the body rather than modifying cells in a laboratory. To date, the US Food and Drug Administration has approved only one gene-editing therapy, Casgevy. It treats two inherited blood disorders by editing a patient's cells externally before they are reinfused.

Prime Medicine expects to release initial results from the study next year. The FDA is also scheduled to rule early next year on a separate in vivo CRISPR therapy for a different condition, hereditary angioedema. For patients, the advance is profound; the president of the Wilson Disease Association said she "never dreamed" she would see such a treatment in her lifetime.

Summary written by Cup of News with AI from the sources below. Check the originals for the full story.

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• A first for CRISPR: powerful prime editing alters DNA directly in the human body
• Prime Editing Clears IP Fight, Sets Q3 Filing for First Liver-Targeted AATD Drug
• Prime Editing: The Next Frontier in Genetic Medicine
• Scientists improve nearly every aspect of prime editing, moving it closer to treating more genetic diseases
• Gene-Editing Biotech Prime Medicine Strikes Up Cell Therapy R&D Alliance With Bristol Myers Squibb

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