Prime Medicine reports first in-body prime-editing treatment
The experimental Wilson disease treatment aims to rewrite DNA in liver cells inside the body. Prime Medicine expects the first trial results in 2027.
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The experimental Wilson disease treatment aims to rewrite DNA in liver cells inside the body. Prime Medicine expects the first trial results in 2027.
Prime Medicine announced that it treated the first participant in its Wilson disease trial earlier in October 2026, according to Nature’s 22 October report. Nature describes the treatment as the first known use of prime editing—a method for rewriting DNA—directly inside a person’s body.
The biotechnology company had previously used prime editing in a separate trial involving two people with a rare immune disorder. In that trial, it edited blood stem cells outside the body and then returned them to the participants. The Wilson disease trial instead aims to correct a mutation in liver cells while those cells remain inside the body.
Wilson disease is caused by mutations in ATP7B, a gene that provides instructions for making a protein that transports excess copper out of the liver. Without treatment, copper can build up and cause liver and nerve damage, as well as neurological disorders.
Current treatment involves copper-clearing molecules and other medicines. Rhonda Rowland, identified in the October report as president of the Wilson Disease Association, says patients want a one-time treatment instead of that complicated regimen. Correcting the faulty gene could, in theory, offer a lasting solution.
A technique called base editing can change individual DNA letters, but it cannot make the particular corrections needed for the most common ATP7B mutations. Prime editing can insert, delete and rewrite segments of DNA, giving it the flexibility needed to attempt those repairs.
Editing cells inside the body is called in vivo editing. Nature explains that this approach could make treatment cheaper and faster to manufacture and administer than extracting each person’s cells, editing them in a laboratory and returning them. Those possible advantages are not yet demonstrated benefits of this trial.
Giving the treatment does not by itself establish that it corrected DNA, is safe or improves patients’ health. Prime Medicine expects to announce the study’s first results in 2027.
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