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CTX310 one-year data show ANGPTL3, LDL and triglyceride cuts last
CRISPR TherapeuticsCTX310 (ANGPTL3)
CRISPR Therapeutics reported one-year follow-up for CTX310, its one-time gene-editing therapy against ANGPTL3, at ESC 2026 and in the New England Journal of Medicine. The reductions from a single infusion held for a year in the highest-dose group, with no new serious treatment-related side effects.
- The phase 1a trial enrolled 15 adults at five dose levels in Australia and New Zealand; the one-year data cover the highest dose (0.8 mg/kg) in 4 participants.
- At one year in that group, ANGPTL3 fell a mean of 78.6%, LDL cholesterol 52.5% and triglycerides 47.8%.
- Three participants had grade 2 infusion reactions that resolved; one had a transient liver enzyme rise soon after dosing with no ongoing liver problems.
- A phase 1b trial is testing a fixed 0.8 mg/kg dose in patients with refractory dyslipidemias, with a severe hypertriglyceridemia cohort update expected in the second half of 2026.