CTX310 (gene therapy)

2025: Technology Development

On November 10, 2025, the results of the first clinical trial of gene therapy for the treatment of elevated cholesterol were presented. Scientific work was carried out at the Monash University in Australia.

According to the press service of the university, CTX310 therapy is aimed at blocking the ANGPTL3 gene. This gene is responsible for regulating blood lipid levels. Some people with congenital ANGPTL3 changes have naturally low cholesterol without harm to their health.

As part of the therapy, a method of genetic modification of CRISPR is used, which provides delivery to hepatocytes of specialized molecules that block the target gene.Clinical trials involved persons aged 18 to 75 years. Each patient received a single treatment at different dosages.

𝓲

Фото: Freepik

Within two weeks of the therapy being introduced, levels of low-density lipoprotein (LDL), so-called "bad" cholesterol, and triglycerides dropped significantly, according to the official Monash University portal. Rates remained stable throughout the follow-up period. The average reduction in LDL and triglyceride levels was almost 60%, and the maximum recorded result reached 80%. Representatives of the University assure that the procedure showed a high safety profile: side effects were mild and resembled flu symptoms.

The authors of the study will continue to follow the first volunteers for the first year, and then for 15 years. In parallel, subsequent phases of clinical trials will be conducted to confirm the long-term effect and safety of treatment. Earlier, according to Science Daily, in preclinical trials in the United States, scientists managed to reduce cholesterol by 50% in three days, which also demonstrates the potential of gene therapy in this area.^[1]

Notes