VERVE-101: CRISPR-Based Gene Editing Therapy Shows Promise in Reducing LDL-C and PCSK9 Levels in Patients With HeFH
A single infusion of a CRISPR-based gene editing therapy significantly reduced LDL-C and PCSK9 levels in patients with heterozygous familial hypercholesterolemia (HeFH), based on findings from the VERVE-101 trial presented Nov. 12 at AHA 2023. In presenting the findings, Andrew Bellinger, MD, PhD, said they provide the first proof-of-concept for in vivo DNA base editing in humans.