These promising results have determined the start of a phase 1 single-ascending dose to evaluate the safety of VERVE-101, a CRISPR base-editing drug assembled by a messenger RNA for an adenine base editor and a guide RNA and designed to block PCSK9 in patients with HeFH, atherosclerotic cardiovascular disease, and uncontrolled hypercholesterolemia [131]. This evidence concerns the gene PCSK9 and familial hypercholesterolemia.