Single CRISPR infusion (CTX310) cut LDL cholesterol and triglycerides by about half for a full year in an early human trial
- First-in-human Phase 1 trial (Cleveland Clinic), 15 patients with lipid disorders not adequately controlled by medications.
- One-time infusion that switches off the liver gene ANGPTL3.
- At the highest dose, after 12 months: LDL -52.5% and triglycerides -47.8% from baseline.
- No serious treatment-related adverse events over the year.
Caveats:
- Only 15 people, Phase 1, still experimental.
- Funded by CRISPR Therapeutics, which also funds the lead author’s institution.
- Long-term safety follow-up will continue for 15 more years.