CTX310
In Development
CRISPR Therapeutics
Overview
An in vivo gene-editing therapy delivered by lipid nanoparticles that knocks out the ANGPTL3 gene in the liver to lower triglycerides and LDL cholesterol — a potential one-time treatment for severe dyslipidemia and cardiovascular risk, and the lead of CRISPR's in vivo pipeline.
Phase 1. Positive data reported in November 2025 showed triglycerides down up to ~84% and LDL down up to ~87%, with durable ANGPTL3 editing; published in NEJM. A US IND has been cleared and a further update is expected in H2 2026.