Overview
- Fifteen adults in the UK, Australia, and New Zealand received a single CTX310 infusion that edits the liver gene ANGPTL3.
- At the highest dose, LDL cholesterol and triglycerides fell by about 50% within two weeks and remained lowered through the 60-day observation window.
- The findings were presented at the American Heart Association meeting and published in The New England Journal of Medicine.
- Short-term safety findings included minor infusion reactions and one transient liver-enzyme rise; one participant died months later from preexisting heart disease, which investigators judged unrelated.
- Crispr Therapeutics is leading development, with 15-year safety follow-up required and a Phase II trial planned for 2026 to test broader populations and durability.