CRISPR Therapeutics presented positive durability data from its Phase 1a trial for CTX310. The company presented this data at the European Society of Cardiology (ESC) Congress 2026. CTX310 is an in-vivo gene-editing therapy. It targets the ANGPTL3 gene to lower triglycerides (TG) and low-density lipoprotein (LDL) cholesterol. The New England Journal of Medicine concurrently published the findings.
The data covered a one-year follow-up period. A single CTX310 treatment resulted in significant, lasting reductions in ANGPTL3, TG, and LDL levels. At the highest dose, patients showed a mean reduction of 79% for ANGPTL3. Patients also showed a 53% reduction for LDL. TG levels saw a 48% reduction. Patients reportedly tolerated the treatment well. No serious adverse events related to the therapy occurred.