Gene therapy cuts bad cholesterol by half in single treatment

A single dose of the experimental gene therapy CTX310 reduced LDL cholesterol by nearly half and maintained its effect for at least a year, ERR reported, citing a first phase study.

The treatment was tested on 15 adults in the United States, Australia and New Zealand who had difficult-to-control high cholesterol or another serious disorder of fat metabolism.

The strongest results came among the four patients who received the highest dose. Their LDL cholesterol levels fell by an average of nearly 53 percent over one year. Their triglyceride levels, a type of blood fat linked to an increased risk of cardiovascular disease, fell by 48 percent.

The patients tolerated the treatment well. No signs of toxicity or dangerous side effects were observed during the year-long follow-up.

CTX310 uses CRISPR gene-editing technology to switch off a specific gene in liver cells. The researchers based the approach on a naturally occurring phenomenon: some people have a defective version of the gene that protects them against atherosclerosis. The therapy is intended to mimic this inherited protection by creating a permanent genetic change in the liver that helps reduce blood fat levels.

Researchers writing in the New England Journal of Medicine said the findings support CTX310 as a possible one-time, long-term treatment. Further studies are needed to assess its safety and lasting effects.