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A Phase 1 trial presented at the American Heart Association's 2025 Scientific Sessions showed a one-time CRISPR-Cas9 infusion could switch off a liver gene involved in fat metabolism, cutting LDL cholesterol by nearly half.

Which gene did this 2025 CRISPR gene-editing trial target to lower cholesterol and triglycerides?

ANGPTL3
The therapy, CTX310, used CRISPR-Cas9 to permanently switch off ANGPTL3 in the liver, producing an average LDL cholesterol reduction of about 49% and a triglyceride reduction of about 55% in the 15-patient trial, with no serious adverse events reported. Results were published simultaneously in the New England Journal of Medicine.

The trial targeted ANGPTL3, a gene that normally makes a protein restraining enzymes that clear fat particles from blood. Using CRISPR to permanently disable it in liver cells let the body clear LDL cholesterol and triglycerides more efficiently, producing a dramatic drop from a single infusion rather than a daily pill.

PCSK9 is a different, already well-known cholesterol target used by existing injectable drugs, and APOB encodes the main structural protein of LDL particles itself, targeted by other experimental therapies, but neither was edited in this trial.

A single treatment producing a lasting effect, unlike statins taken indefinitely, is the appeal here, though long-term safety and durability still need years of follow-up to confirm.

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