Clinical Trial

Disease: Familial Hypercholesterolemia, FH, (NCT05398029)

Disease info:

Familial hypercholesterolemia (FH) is a condition associated with significantly elevated low-density lipoprotein (LDL) cholesterol (LDL-C) or "bad cholesterol" and an increased risk of early onset of coronary artery disease if not sufficiently treated. Most commonly, individuals have heterozygous familial hypercholesterolemia (HeFH), caused by a single DNA variant (alteration) for FH that they have inherited from one (affected) parent. In rare cases, an individual can have homozygous familial hypercholesterolemia (HoFH), which is caused by having two causal FH DNA variants, where one variant is inherited from each (affected) parent. Individuals with HoFH typically have a more severe form of disease.

Having FH greatly increases the risk of hardening of the arteries (atherosclerosis), which can lead to heart attacks, strokes and other vascular conditions. Untreated individuals with FH have a 20-fold increased risk for coronary artery disease (CAD).

FH is treatable and the associated cardiovascular disease is largely preventable with early and intensive treatment, using statins, additional drugs, and other means. Other non-statin medications include PCSK9 inhibitors, ezetimibe, and bempedoic acid. These are effective treatments for individuals with FH who have a persistently elevated LDL-C despite treatment with maximally tolerated statin therapy.

Early identification and treatment of individuals with FH is key to preventing cardiovascular disease. Underdiagnosis of FH is a problem in most countries as high cholesterol can be an invisible and undetected problem until it leads to coronary artery disease.


The incidence of familial hypercholesterolemia (FH) is estimated at about 1/300 persons worldwide but is 7/100 in persons with premature ischemic heart disease (IHD) and founder effect such as French Canadians, Finns, and Afrikaners.
Official title:
Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia


Number: 781-970-6833,   



New Zealand
Auckland, New Zealand
Christchurch, New Zealand

United Kingdom
London, United Kingdom


Study start:
Jul. 5, 2022
44 participants
Gene editing method:
Base editors
Type of edit:
Gene knock-out
Proprotein Convertase Subtilisin-Kexin Type 9 (PCSK9)
Delivery method:
Lipid-based nanoparticle (LNP) - In-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting


VERVE-101 is designed to be a one-time treatment, delivered via intravenous infusion, that uses adenine base editing to safely and precisely turn off PCSK9 in the liver and permanently lower LDL-C levels to treat coronary heart disease. By making a single, precise, A-to-G substitution in the PCSK9 gene in the liver, VERVE-101 is designed to completely inactivate the target gene.

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. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.

Last updated: May. 28, 2024
Search CRISPR Medicine