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CRISPR Therapeutics presents CTX310 durability data at ESC Congress 2026
CRISPR TherapeuticsCTX310 (ANGPTL3)
CRISPR Therapeutics showed one-year data for CTX310, a one-time infusion that uses CRISPR to switch off the ANGPTL3 gene in the liver, a gene whose protein slows the clearing of fats from the blood. All 15 Phase 1a participants had at least a year of follow-up, and at the top dose blood fats stayed well down, supporting the idea that a single treatment could replace lifelong pills or injections.
- Five dose levels from 0.1 to 0.8 mg/kg. At 0.8 mg/kg, mean reductions were 79% for ANGPTL3, 48% for triglycerides and 53% for LDL cholesterol (best individual results: 89%, 78%, 84%).
- No treatment-related serious adverse events and no Grade 3 or worse liver-enzyme rises; three people had Grade 2 infusion reactions and one an allergic reaction that resolved.
- Phase 1b will use a fixed flat dose equivalent to 0.8 mg/kg and focus on severe hypertriglyceridemia; another update is due in the second half of 2026.
- Caveat: 15 people is a small sample, and an edit to DNA is permanent, so long-term safety follow-up matters more than for a drug that wears off.