Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3
Authors' conclusion
Editing of ANGPTL3 was associated with few adverse events and resulted in reductions from baseline in ANGPTL3 levels.
Study context
- Population
- Adults with uncontrolled hypercholesterolemia, hypertriglyceridemia or mixed dyslipidemia on maximally tolerated lipid-lowering therapy≥60 days follow-up
- Participants
- 15 adults
- Comparison
- CTX310 at 0.1, 0.3, 0.6, 0.7 or 0.8 mg/kg
- Outcome
- Adverse events, including dose-limiting toxic effects
Methodological detailsascending-dose
- Ascending-dose
Main finding
As reported in the abstract
Adverse events, incl. dose-limiting toxic effects
Serious adverse events (1 spinal disk herniation; 1 sudden death 179 days after 0.1 mg per kilogram dose)
2
13%
Infusion-related reactions
3
20%
Transient aminotransferase elevation (3–5× baseline; elevated at baseline), peak day 4, back to baseline by day 14
1
7%
Further findings
| Outcome | Values |
|---|---|
| Percent change in ANGPTL3 level, by dose | 0.1 mg per kilogram mean percent change 9.6% (range −21.8–71.2) · 0.3 mg per kilogram mean percent change 9.4% (range −25.0–63.9) · 0.6 mg per kilogram mean percent change −32.7% (range −51.4–-19.4) · 0.7 mg per kilogram mean percent change −79.7% (range −86.8–-72.5) · 0.8 mg per kilogram mean percent change −73.2% (range −89.0–-66.9) |
Safety
- Serious adverse events
- 2 (13%)
- Infusion-related reactions
- 3 (20%)
- Transient aminotransferase elevation
- 1 (7%)
Original publication
Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3. N Engl J Med. Published 8 November 2025. doi:10.
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