VERVE-101, a novel gene therapy using liquid nanoparticles to edit the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene, reduced low-density lipoprotein (LDL) cholesterol concentrations by 55% at its highest dose, with the results maintained six months after treatment, according to the phase 1 HEART-1 trial, presented at the AHA Scientific Sessions 2023, in Philadelphia.
The HEART-1 trial (ClinicalTrials.gov Identifier: NCT05398029) is the first in humans to employ a very