22 Nov 2023 • The first in-human study of a gene therapy designed to reduce low-density lipoprotein cholesterol has shown a signal that the treatment works in a small group of patients with heterozygous familial hypercholesterolemia (HeFH). The data from the highly anticipated study was presented at an American Heart Association meeting in Philadelphia.

According to interim results of the heart-1 proof-of-concept study, the 10 participants with heterozygous familial hypercholesterolemia (HeFH) showed dose-dependent reductions in blood PCSK9 protein and LDL cholesterol.

MORE ABOUT THIS GENE EDITING THERAPY

• A second-generation CRISPR technology, VERVE-101 is an investigational DNA base editing medicine that is delivered via one IV infusion into blood through lipid nanoparticles.
• Targeted inside hepatocytes, an editor protein and guide then locate the PCSK9 gene, where they perform a single base pair change to permanently turn off PCSK9 production in that liver cell and presumably dampen downstream cholesterol production.

Source: ACC | Read full story