Gene-editing injection shows promise as one-time cholesterol treatment alternative

Two clinical trials, one led by the Cleveland Clinic in the US and conducted across Australia, New Zealand and Britain, the other in China, have tested gene-editing injections that could replace daily cholesterol pills with a single shot capable of controlling "bad" cholesterol for years or even permanently.

Single-dose therapy targets liver gene

The US-led research tested CTX310, a nanodrug, on 15 volunteers. The therapy uses tiny particles to deliver a CRISPR gene-editing tool directly into liver cells, targeting ANGPTL3, a gene that regulates fat circulation in the blood. People born with a natural mutation that inhibits ANGPTL3 have extremely low cholesterol and triglycerides and rarely develop heart disease; CTX310 mimics that genetic switch.

Both trials used approaches, CRISPR and base editing, to silence lipid genes, resulting in drastic drops in low-density lipoprotein cholesterol (LDL-C) without the need for daily medication.

Why it matters

LDL-C, commonly called "bad" cholesterol, is a major driver of heart disease, the leading cause of death worldwide. Triglycerides, another type of blood fat, compound the risk. For millions of people, the standard treatment is a daily statin pill. A one-time injection that offers long-term or permanent cholesterol control could eliminate the burden of daily medication adherence and potentially reduce cardiovascular deaths on a global scale.

Source: South China Morning Post