New gene therapy cuts harmful cholesterol after a single dose

A single dose of an experimental CRISPR-based therapy produced a sustained reduction in harmful cholesterol, according to results from a clinical trial reported by agencies. The treatment, known as CTX310, remained effective for at least a year after administration.

CTX310 targets a gene in liver cells responsible for producing ANGPTL3, a protein involved in regulating blood-fat levels. By reducing production of the protein, the therapy seeks to mimic a naturally occurring genetic variation found in some people, which is associated with lower fat levels and a reduced risk of cardiovascular disease.

The trial involved 15 people with severe cholesterol or triglyceride disorders. Each participant received one intravenous dose, and researchers monitored their health for 12 months. One participant died 179 days after receiving the treatment, but researchers concluded that the death was not related to CTX310.

The other 14 participants did not develop new serious side effects during the follow-up period. The most notable results came from four participants who received the highest dose, although the supplied report ends before giving the full details of their results.