A single infusion of gene editing therapy YOLT-101 can reduce bad cholesterol by 52.3% and the effect lasts for half a year

September 5, 2026, 11:44

The illustration depicts gene-editing therapy acting on liver cells, potentially reducing "bad cholesterol" in the blood. (Illustration by this newspaper)

[Compiled by Chen Cheng-liang / Comprehensive Report] Could hyperlipidemia patients one day avoid daily medication? A recent early-stage human trial found that a new gene-editing therapy, administered via a single intravenous infusion, reduced "bad cholesterol" in the blood by an average of 52.3% after 24 weeks in patients receiving the highest dose, with this reduction sustained throughout the six-month follow-up period.

The study results were published in the medical journal Nature Medicine. Conducted in collaboration between Renji Hospital, affiliated with Shanghai Jiao Tong University School of Medicine, and YolTech Therapeutics, the study tested YOLT-101 in patients with familial hypercholesterolemia who had poorly controlled blood lipid levels. These patients suffer from chronically elevated low-density lipoprotein cholesterol (LDL-C), predisposing them to premature atherosclerosis and cardiovascular disease.

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The study tested an in vivo gene-editing therapy called "YOLT-101," which uses lipid nanoparticles to deliver gene-editing tools into liver cells to target the "PCSK9" gene. PCSK9 hinders the liver's ability to clear bad cholesterol from the blood. The research team aims to enhance the liver's cholesterol-clearing capacity by silencing this gene, thereby enabling a single treatment to provide long-lasting effects.

The trial enrolled six patients, each receiving a single intravenous infusion at one of three different dosage levels. Results showed that in the highest-dose group, the three patients experienced an average 48.9% reduction in bad cholesterol by week 4, increasing to a 52.3% reduction by week 24. The study also found that PCSK9 concentrations decreased with increasing dosage, indicating that the gene editing produced the intended biological effect.

The focus of this study is not merely on temporarily lowering cholesterol, but on testing whether a single gene-editing intervention can sustain lipid-lowering effects for a longer duration. Based on the 24-week results, the reduction in the highest-dose group did not significantly wane, though it remains unclear how many years the effect will last.

Phase I Trial with Only Six Participants Cannot Yet Replace Existing Lipid-Lowering Drugs

Regarding safety, no Grade 3 or higher adverse events occurred during the study. More common reactions included transient infusion-related symptoms and elevated liver enzymes. The research team believes the current results support further development, but this therapy remains in the early stages of human clinical trials.