Overview
- Results published in The New England Journal of Medicine and presented at the American Heart Association meeting detail a 15-person Phase 1 study in the UK, Australia, and New Zealand.
- At the highest dose, LDL cholesterol fell about 49% and triglycerides about 55% within weeks, with effects maintained for at least two months and reported out to six months in early follow-up.
- The infusion, called CTX310, uses lipid nanoparticles to deliver CRISPR to the liver to knock out ANGPTL3, with dose-dependent reductions in the ANGPTL3 protein confirmed.
- Short-term tolerability appeared acceptable, with transient infusion reactions and a temporary liver enzyme rise; one participant with advanced heart disease died months later and investigators judged the death unrelated.
- CRISPR Therapeutics plans to seek U.S. clearance to begin Phase 2 in 2026, and participants will undergo long-term safety monitoring consistent with FDA recommendations for gene-editing therapies.