The VT-1001 trial (NCT05398029) is a phase-1b clinical trial assessing the safety of a CRISPR-based therapy using base editing to disrupt the PCSK9 gene in the liver, aiming to reduce PCSK9 and LDLc in patients with heterozygous familial hypercholesterolemia and uncontrolled coronary heart disease [43]. This evidence concerns the gene PCSK9 and familial hypercholesterolemia.