Overview
- In a 15-person Phase 1 study of CTX310, the four participants on the highest dose saw LDL fall about 49–50% and triglycerides drop roughly 55% within weeks.
- The investigational therapy targets the liver to disable the ANGPTL3 gene using a lipid nanoparticle delivery of CRISPR molecular scissors.
- Short‑term safety was generally acceptable, with transient nausea or back pain, one reversible liver‑enzyme rise, and one death reported months later that investigators said was unrelated.
- Results were published in the New England Journal of Medicine and presented at the American Heart Association meeting, with trial sites in the U.K., Australia and New Zealand.
- CRISPR Therapeutics and study investigators plan Phase 2 trials as early as 2026 and will follow participants long term, consistent with FDA recommendations for extended surveillance of gene‑editing therapies.