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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study of VERVE-101 in Patients with Familial Hypercholesterolemia and Cardiovascular Disease
NCTID
NCT05398029
(View at clinicaltrials.gov)
Description
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.
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Development Status
Active
Indication
Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Disease Ontology Term
DOID:13810
Compound Name
VERVE-101
Sponsor
Verve Therapeutics, Inc.
Funder Type
Industry
Recruitment Status
Active not recruiting
Enrollment Count
44
Results Posted
Not Available
Therapy Information
Target Gene/Variant
PCSK9
Therapy Type
Gene editing
Therapy Route
In-vivo
Mechanism of Action
Gene inactivation
Route of Administration
Intravenous
Drug Product Type
MRNA, LNP
Target Tissue/Cell
Hepatocyte
Delivery System
Lipid encapsulation
Vector Type
LDLR
Editor Type
ABE8.8m
Dose 1
0.1 mg/kg
Dose 2
0.3 mg/kg
Dose 3
0.45 mg/kg
Dose 4
0.6 mg/kg
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2022-05-19
Completion Date
2025-06
Last Update
2024-12-30
Participation Criteria
Eligible Age
18 Years - 75 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
New Zealand,United Kingdom
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
This program is deprioritized in favor of VERVE-102
Resources/Links
Clinical Publications
(Corporate Presentation) Transforming the Care of Cardiovascular Disease Through Single-course Gene Editing Medicines Interim Results from heart-1 Trial of VERVE-101
News and Press Releases
Verve Therapeutics Announces Pipeline Progress and Anticipated 2025 Milestones
Preclinical Publications
Potent, Specific, and Durable Liver Editing of PCSK9 in Preclinical Studies of the CRISPR Base Editing Medicine VERVE-101 - Japanese Atherosclerosis Conference 2024
Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models
(Presentation) Proof-of-concept for in vivo Base Editing to Inactivate the PCSK9 Gene and Lower LDL-Cholesterol in Humans - TIDES 2024
(Presentation) Characterization of Guide RNA Site Consistency Across Ancestries and the Potential for Off-Target Editing with the Clinical-Stage Base Editing Medicine, VERVE-101 - ASGCT 2024