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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study of VERVE-102 in Patients with Familial Hypercholesterolemia or Premature Coronary Artery Disease
NCTID
NCT06164730
(View at clinicaltrials.gov)
Description
VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.
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Development Status
Active
Indication
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Disease Ontology Term
DOID:13810
Compound Name
VERVE-102
Sponsor
Verve Therapeutics, Inc.
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
36
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
Delivery System
Lipid encapsulation
Vector Type
LDLR + GalNAc
Editor Type
base editor
Dose 1
0.3mg/kg
Dose 2
0.45mg/kg
Dose 3
0.6mg/kg
Dose 4
Undisclosed dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2023-12-01
Completion Date
2026-08
Last Update
2024-11-12
Participation Criteria
Eligible Age
18 Years - 70 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
8
Locations
New Zealand,Canada,United Kingdom,Australia
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Initial data for the Heart-2 Phase 1b clinical trial of VERVE-102 targeting PCSK9 expected in second quarter of 2025; Dosing in the Heart-2 trial has moved to the 0.6 mg/kg cohort; IND cleared by FDA in March 2025
Resources/Links
News and Press Releases
Verve Therapeutics Announces Pipeline Progress and Anticipated 2025 Milestones
Verve Therapeutics Announces Dosing of First Patient in Heart-2 Phase 1b Clinical Trial Evaluating VERVE-102
Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-102, an Investigational Gene Editing Medicine Designed to Durably Lower Cholesterol After a Single Dose
Preclinical Publications
(Presentation) Proof-of-concept for in vivo Base Editing to Inactivate the PCSK9 Gene and Lower LDL-Cholesterol in Humans - TIDES 2024
Protocol
(Presentation) Design of Heart-2 A Phase 1b clinical trial of VERVE-102, an in vivo base editing medicine delivered by a GalNAc-LNP and targeting PCSK9 to durably lower LDL cholesterol - AHA Scientific Sessions 2024