For the First Time, DNA Was Edited Directly Inside a Patient’s Body

October 31 — The US biotech company Prime Medicine announced in early October that it had used prime editing to edit DNA directly inside a human body for the first time in the world, treating a patient with Wilson’s disease. Initial clinical results are expected in 2027.

Wilson’s disease is an autosomal recessive disorder of copper metabolism; the core problem is that the body cannot excrete excess copper, which then deposits in the liver, brain and cornea. Current drugs are not curative and require lifelong treatment. Liver transplant is the only cure, but it is limited by organ supply and carries a high surgical risk.

In a global clinical trial for Wilson’s disease, the team rewrote DNA directly in the participant’s liver cells. The first patient was treated early this month, but results on safety and efficacy are not expected until next year at the earliest.

“This therapy has the potential to offer patients a one-time curative treatment, ideally before irreversible damage occurs in the liver and nervous system,” chief medical officer Mohammed Asmal said in a press release.

Prime Medicine is a US biotech company dedicated to creating and delivering next-generation gene-editing therapies. It is deploying its proprietary Prime Editing platform, described as a versatile gene-editing technology.

Prime Editor is designed to make the right edit at the right place in a gene while minimising unwanted DNA changes. The company says it has the potential to repair almost all types of genetic mutation and to work across many different tissues, organs and cell types.