Gene-editing trials test a possible one-time fix for high cholesterol

Early-stage CRISPR studies that target liver genes such as ANGPTL3 and PCSK9 have lowered LDL and triglycerides in small groups, and larger, longer trials are being planned.

Scientists are testing gene-editing as a potential one-time treatment for high cholesterol. The research is at an early stage and has been tried in only a few dozen people so far. Two companies are developing CRISPR-based infusions to switch off liver genes linked to high cholesterol, including ANGPTL3 and PCSK9. Small studies have shown rapid drops in LDL cholesterol and triglycerides, but longer follow-up and larger trials are needed. Key facts: - In one study, 15 adults received a single infusion carrying CRISPR to the liver; participants given the highest dose saw LDL and triglycerides fall by about half within two weeks. - Verve Therapeutics, a unit of Eli Lilly, reported a similar LDL reduction in a small PCSK9-targeted study, and some Verve participants have remained lowered after two years of follow-up. - Gene edits in liver cells are considered permanent in principle, and researchers note edited cells and their progeny can retain the changes. - Researchers have flagged safety questions: CRISPR-based therapies have limited long-term human data, delivery particles can irritate or inflame the liver, and unintended off-target edits have not been ruled out. - Companies are expanding their work: U.S. study sites are opening for one program and a next-step study of the ANGPTL3 approach is expected later this year, with sites to be announced. Summary: If findings hold up in larger and longer studies, gene-editing could produce lasting reductions in harmful cholesterol for people at high risk. Major questions about long-term safety and durability remain, and researchers plan expanded trials and extended follow-up to assess those outcomes.