In a groundbreaking Phase 1 trial, a one-time infusion of the CRISPR-Cas9 gene-editing therapy CTX310 safely reduced LDL cholesterol and triglycerides in patients with difficult-to-treat lipid disorders. The treatment uses fat-based particles to deliver the CRISPR mechanism to the liver, where it turns off the ANGPTL3 gene. As a result, LDL cholesterol dropped by nearly 50% and triglycerides by about 55% on average at the highest dose — with effects lasting for at least 60 days. This is significant because it’s the first therapy to simultaneously target both markers effectively, offering hope for patients with mixed lipid disorders. Minor infusion-related reactions occurred in some participants, but no serious safety concerns were raised.
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