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NOVEMBER 17, 2023

Novel Gene Therapy Slashes LDL in Patients With Hypercholesterolemia

By Gina Shaw 
VERVE-101, a novel gene therapy using liquid nanoparticles to edit the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene of hepatocytes, reduced low-density lipoprotein (LDL) cholesterol concentrations by 55% at its highest dose, with the results maintained six months after treatment, according to the headline-making phase 1 HEART-1 trial, presented at the 2023 American Heart Association (AHA) Scientific Sessions, held in Philadelphia. 

The trial is the first in