A single CRISPR infusion held lipids down for a year

CRISPR Therapeutics presented one-year data for CTX310 at the European Society of Cardiology congress on 28 August 2026, with the results published the same day in the New England Journal of Medicine under the title “Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310”.

Fifteen adults received a single intravenous infusion across sequential cohorts, at doses from 0.1 to 0.8 mg/kg of lean body weight. In the highest-dose cohort at one year, ANGPTL3 fell by a mean of 79 percent, with a maximum of 89 percent; triglycerides fell by a mean of 48 percent, maximum 78; and LDL cholesterol by a mean of 53 percent, maximum 84. The company reported no treatment-related serious adverse events.

CTX310 edits the ANGPTL3 gene in the liver, so what is being measured at twelve months is not a drug still circulating but a change made once and left in place. That is the claim the durability read-out exists to test, and the reason a lipid result at one year carries different weight than the same numbers from a daily therapy.

Fifteen people is a Phase 1a. A Phase 1b is running in the United States and elsewhere with a fixed flat dose, and the company says an update covering patients with severe hypertriglyceridemia is due in the second half of 2026.