Early human studies of CRISPR-based therapies targeting lipid pathways produced large reductions in LDL and triglycerides, and several Phase 1 programs targeting ANGPTL3 are now underway. A small cohort study presented at the American Heart Association and published in the New England Journal of Medicine reported dramatic lipid drops in 15 patients treated with a CRISPR approach. Separately, a Phase 1 trial of CRISPR-Cas9 editing targeting ANGPTL3 is active and registered, marking parallel clinical efforts aimed at durable lipid lowering. Sponsors include CRISPR Therapeutics and other gene-editing developers advancing single-dose or one-time approaches. Investigators flagged liver-toxicity signals and cautioned the data remain early; safety monitoring, potential off-target effects, and patient acceptance of permanent edits are central near-term questions. ANGPTL3 inhibition reduces hepatic lipid secretion and is a recognized mechanistic route to lower LDL and triglycerides.
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