Phase 1 open-label trial enrolled 15 adults with refractory dyslipidemia.

CTX310 targets ANGPTL3 using lipid nanoparticle–delivered CRISPR-Cas9 mRNA.

Highest dose (0.8 mg/kg) reduced LDL by 49% and triglycerides by 55% at 60 days.

ANGPTL3 protein levels decreased by 73% at 30 days in the same group.

LDL responses ranged from 19.5% to 86.6% reduction across participants.

No serious adverse events related to CTX310 were observed; infusion reactions occurred in 3 patients.

Trial conducted at 6 sites in Australia, New Zealand, and the United Kingdom.