First-in-human trial of CRISPR gene-editing therapy safely lowered cholesterol, triglycerides
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Updates every hour. Last Updated: 22-Dec-2025 11:11 ET (22-Dec-2025 16:11 GMT/UTC)
In a Phase 1, first-in-human trial, a one-time infusion of an investigational CRISPR-Cas9 therapy targeting angiopoietin-like protein 3 (ANGPTL3) was safe and reduced LDL cholesterol by nearly 50% and reduced triglycerides by about 55%.
In a small, short-term, Phase 2 clinical trial in adults, a first-of-its-kind medication reduced triglyceride levels in most patients by more than 60%. Called DR10624, the medication activates three different receptors linked to triglycerides: FGF21, glucagon and GLP-1 receptors.