Two recent clinical trials, one conducted in the United States and another in China, indicate that a single injection could potentially control low-density lipoprotein cholesterol (LDL-C), commonly known as “bad” cholesterol, for extended periods—ranging from a year to possibly a lifetime.

LDL-C is a significant factor in the development of heart disease, the leading cause of death worldwide. Alongside triglycerides, elevated levels of LDL-C increase cardiovascular risk. Currently, millions rely on daily statin pills to manage cholesterol levels. The new investigational therapies, however, aim to provide a long-lasting solution through gene editing.

In a U.S.-led study spearheaded by the Cleveland Clinic and involving research sites across Australia, New Zealand, and the United Kingdom, investigators tested a gene-editing nanodrug called CTX310 on 15 volunteers. This therapy utilizes nanoparticles to deliver a CRISPR gene-editing system directly into liver cells, targeting the ANGPTL3 gene, which regulates blood fat levels.

Some individuals naturally have mutations that inhibit ANGPTL3, resulting in remarkably low cholesterol and triglyceride levels and a reduced incidence of heart disease. CTX310 is designed to replicate this genetic effect. According to results published in The New England Journal of Medicine on August 28, participants receiving the highest dose experienced a sustained average reduction in ANGPTL3 levels of 78.6% one year after a single infusion. Corresponding decreases in LDL-C and triglycerides were 52.5% and 47.8%, respectively. The study also noted that edited liver cells continued to propagate the cholesterol-lowering gene modification, implying a potentially permanent effect.

Separately, a Chinese team from Shanghai Jiao Tong University’s Renji Hospital published findings in Nature Medicine in March focusing on individuals with familial hypercholesterolaemia (FH), a genetic disorder that causes dangerously elevated cholesterol and substantially raises heart attack risk. The researchers used an adenine base editor targeting the PCSK9 gene, which when overexpressed disrupts the liver’s capacity to remove LDL-C from the bloodstream.

In their trial, the high-dose group saw LDL-C levels decrease by an average of 52.3% after 24 weeks, nearly twice the reduction typically achieved with statins. PCSK9 protein levels dropped by 74.4%, without rebounds. Mild and transient side effects such as fever, muscle aches, and slight changes in liver enzyme levels were reported.

Both teams reported that the treatments were generally safe. In the U.S. trial, one participant died unexpectedly 179 days after receiving the lowest dose; investigators determined the death was unrelated to the therapy.

Gene editing has previously demonstrated promise in cancer treatment and is now extending its reach to chronic diseases. Many genes that once conferred survival advantages against conditions like famine or malaria may contribute to metabolic and other health challenges in modern environments. These trials represent initial efforts to “rewrite” genetic instructions for long-term disease mitigation.

Both studies remain under long-term observation to assess durability, safety, and broader applicability of these gene-editing approaches for lipid control.