In a groundbreaking development, researchers in Australia have spearheaded the first human trial of an innovative gene-editing therapy designed to significantly reduce bad cholesterol levels. This therapy, known as CTX310, targets individuals suffering from challenging lipid disorders by cutting bad cholesterol and triglycerides in half.

The clinical trial utilized CRISPR-Cas9 technology, employing fat-based particles to deliver gene-editing tools directly into the liver, effectively deactivating the ANGPTL3 gene. This gene silencing leads to a decrease in LDL (bad) cholesterol and triglycerides, both of which are associated with an increased risk of heart disease, as highlighted in a recent announcement from Monash University.

Conducted at the Victorian Heart Hospital, in collaboration with Monash University, this phase 1 trial involved 15 participants aged between 18 and 75, all of whom experienced lipid disorders that are difficult to manage. The trial spanned across Australia, New Zealand, and Britain, according to the report from Xinhua news agency.

Remarkably, the highest dosage of CTX310 resulted in an average decrease of 50% in LDL cholesterol and 55% in triglycerides following a single treatment, with these beneficial effects lasting for over 60 days post-treatment. Overall, participants observed nearly a 60% reduction in both LDL cholesterol and triglycerides across various dosages, with only mild and temporary side effects reported.

Notably, CTX310 is the first treatment to achieve substantial reductions in both LDL cholesterol and triglycerides simultaneously, representing a significant advancement for individuals with mixed lipid disorders characterized by elevated levels of both markers. This finding was published in the New England Journal of Medicine.

According to Stephen Nicholls, Director of the Victorian Heart Hospital and principal investigator of the study, “The potential for a single-course treatment with enduring effects could revolutionize our approach to heart disease prevention.”

He further noted that this innovative treatment could simplify the care process, lower ongoing costs, alleviate pressure on healthcare systems, and enhance patients’ quality of life. Looking ahead, Nicholls expressed intentions to expand future trials of CTX310 to include larger and more diverse patient demographics.