Researchers in an early clinical trial announced that a new gene therapy called VERVE-102 was able to reduce levels of LDL (“bad”) cholesterol and the PCSK9 protein in patients with inherited high cholesterol and premature heart disease.

Using gene-editing technology, the treatment permanently disables the PCSK9 gene in the liver so the body produces less cholesterol. Results showed that a single infusion reduced LDL cholesterol by up to 62% in some patients, with effects lasting at least one year.

Researchers also reported that this newer version of the therapy appeared safer than its predecessor and caused only limited serious side effects. However, they emphasized that further studies are needed to evaluate its long-term safety and effectiveness.

The U.S. Food and Drug Administration (FDA) has granted the therapy Fast Track designation, and phase II clinical trials are expected to begin later this year.

https://www.medpagetoday.com/cardiology/generalcardiology/121439