Useful information

A single injection for cholesterol — from dreams to first human results

A Phase 1 trial published in the New England Journal of Medicine (November 2025) brings remarkable results: a single infusion of CTX310, a CRISPR-Cas9 therapy developed by Cleveland Clinic and CRISPR Therapeutics, reduced LDL cholesterol by up to 49% and triglycerides by up to 55% in patients with refractory dyslipidemia — without serious adverse events. The mechanism is unique: instead of blocking a protein or interfering with its mRNA, CTX310 permanently edits the ANGPTL3 gene in hepatic cells, mimicking a natural mutation associated with reduced cardiovascular risk throughout life. The therapy is currently in Phase 1b clinical stage, with updates expected in the second half of 2026. It is not approved and does not replace current treatments. Read the full article for clinical context, comparison with statins and PCSK9 inhibitors, and what these data mean for patients. Medical Disclaimer This article presents results from a Phase 1 clinical trial in an early-stage research phase. The therapy described is not approved and is not available as standard treatment. The information is for educational purposes only. Consult your physician for any medical decision.

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