A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease
Status:
Not yet recruiting
Trial end date:
2024-12-01
Target enrollment:
Participant gender:
Summary
VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety
of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH),
atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia.
VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene
in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to
HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101
in this patient population.