CRISPR therapy CTX310 cuts LDL cholesterol 52.5% for a year in 15-patient trial

October 3, 2026 at 07:56 AM1 min

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A single dose of the experimental gene therapy CTX310 reduced LDL cholesterol by 52.5% and triglycerides by 47.8% after 12 months in a 15-patient trial. Results published in the New England Journal of Medicine showed no serious adverse events linked to the therapy. The effect persisted for at least a year, according to study author Luke Laffin of Cleveland Clinic.

Key Facts

CTX310 uses CRISPR-Cas9 to switch off the ANGPTL3 gene in liver cells after a single intravenous administration.
The trial enrolled 15 patients with lipid metabolism disorders who had responded insufficiently to drug treatment.
At the highest dose, LDL cholesterol remained 52.5% lower and triglycerides 47.8% lower after 12 months.
No serious adverse events associated with CTX310 were recorded during the year of observation.
Trial participants are planned to be monitored for another 15 years.

Trial Results

The study involved 15 patients with lipid metabolism disorders who had responded insufficiently to drug treatment. After 12 months, patients who received the highest dose of CTX310 had, on average, LDL cholesterol levels that remained 52.5% lower and triglyceride levels that were 47.8% lower. During the year of observation, the researchers recorded no serious adverse events associated with CTX310.

Mechanism and Duration

The therapy uses CRISPR-Cas9 gene-editing technology and, following a single intravenous administration, reaches liver cells and switches off the ANGPTL3 gene, which is involved in regulating blood fat levels. Cleveland Clinic cardiologist and study author Luke Laffin said the duration of the effect proved impressive. The trial participants are planned to be monitored for another 15 years.

Experimental Status

CTX310 remains an experimental treatment, and this is an early-phase clinical trial involving only 15 people. Researchers still need to confirm its long-term safety and effectiveness in a much larger number of patients.