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Trials suggest a one-shot nanodrug could replace daily cholesterol pills
The approach, tested on 15 volunteers using gene editing carried into liver cells, targets the ANGPTL3 gene that helps regulate blood fat levels.
Two clinical trials in the United States and China are pointing to a potential shift in cholesterol treatment, with researchers exploring a gene-editing nanodrug designed as a single injection rather than a daily pill, according to SCMP Economy.
The therapy, called CTX310 and developed with research led by the Cleveland Clinic in the US and conducted in Australia, New Zealand, and Britain, uses tiny particles to deliver a CRISPR gene-editing tool into liver cells.
SCMP Economy reports that CTX310 targets the ANGPTL3 gene, which influences how much fat circulates in the blood, aiming to better control low-density lipoprotein cholesterol, also known as bad cholesterol, a major driver of heart disease.
In the reported study, CTX310 was tested on 15 volunteers, and the trial design reflects evidence from people born with a natural mutation that inhibits ANGPTL3, who tend to have extremely low cholesterol and triglycerides and rarely develop heart disease.