The first patient received a dose of the high-precision gene editor in a new clinical trial for Wilson disease. Researchers hope it’s a one-and-done fix.
Prime editing is finally ready for prime time. Introduced in 2019, the CRISPR-based gene editor wowed scientists with its versatility and precision. A search-and-replace engine for the human genome, it can swap individual DNA letters, insert new genetic instructions, or delete chunks, all with minimal damage to the genome. In theory, it could correct up to 89 percent of known genetic variants associated with disease.
Now, scientists at Prime Medicine in Cambridge, Massachusetts, are testing the technology’s ability to make edits inside the body for the first time. In a global clinical trial for Wilson disease, the team is giving participants a prime editor that directly rewrites DNA in liver cells. The first patient received an infusion earlier this month, but results showing whether it’s safe and how well it works aren’t expected until next year.
The therapy has “the potential to offer patients a one-time curative treatment, ideally before irreversible liver and neurological damage occur,” Mohammed Asmal, chief medical officer of Prime Medicine, said in a press release.
Gene editing made history in 2024 when regulators approved Casgevy for the inherited blood disorders sickle cell disease and beta thalassemia. It was the first CRISPR-based treatment to receive the green light. Casgevy uses CRISPR-Cas9 to edit blood stem cells outside the body, switching on a fetal form of a blood protein that compensates for the faulty adult version.
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