GoKawiilBase-editing therapy durably silences ANGPTL3 gene to cut lipids in primates
Researchers developed a CRISPR adenine base editor delivered via a GalNAc-lipid nanoparticle that makes a single DNA edit in liver cells to inactivate ANGPTL3, a gene linked to high LDL cholesterol and triglycerides. In nonhuman primates, a single infusion cut circulating ANGPTL3 protein by over 90%, an effect sustained for three years, while animal models showed LDL-C reductions of 40-50% and triglyceride reductions of 50-70%, with no detectable off-target edits found in vitro.