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One-Time CRISPR Therapy Cuts LDL and Triglycerides in First Human Trial

The single infusion edits the liver gene ANGPTL3 to trigger rapid lipid drops, with long-term follow-up planned.

Overview

  • Preliminary Phase 1 results presented at the American Heart Association meeting showed CTX310 lowered stubborn LDL cholesterol and triglycerides after a single intravenous dose.
  • The 15-participant trial in Australia, New Zealand, and the United Kingdom tested ascending doses from 0.1 to 0.8 mg/kg with at least 60 days of follow-up for all participants.
  • Lipid levels began falling within two weeks and remained reduced through the 60-day analysis window.
  • At the highest dose, average reductions reached roughly 50–60%, while a separate report cited peak drops near 80% at higher doses across the full cohort.
  • Reported side effects were mild, with no serious safety signals to date, and investigators plan Phase 2 studies plus one-year trial monitoring and 15-year safety surveillance in line with FDA guidance.