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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Phase 1b Study of VERVE-201 in Patients with Refractory Hyperlipidemia
NCTID
NCT06451770
(View at clinicaltrials.gov)
Description
VT-20101 is an Open-label, Phase 1b, Single-ascending dose study that will evaluate the safety of VERVE-201 administered to patients with Refractory Hypercholesterolemia. VERVE-201 uses base-editing technology designed to inactivate the expression of the ANGPTL3 gene in the liver and lower circulating low-density lipoprotein cholesterol (LDL-C). This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population.
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Development Status
Active
Indication
Hypercholesterolemia
Disease Ontology Term
DOID:13810
Compound Name
VERVE-201
Sponsor
Verve Therapeutics, Inc.
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
36
Results Posted
Not Available
Therapy Information
Target Gene/Variant
ANGPTL3
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 + GalNAc
Editor Type
ABE
Dose 1
Undisclosed dose 1
Dose 2
Undisclosed dose 2
Dose 3
Undisclosed dose 3
Dose 4
Undisclosed dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2024-06-04
Completion Date
2027-03
Last Update
2025-02-13
Participation Criteria
Eligible Age
18 Years - 70 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
Canada,United Kingdom
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
First patient dosed November 2024, expected update 2H 2025
Resources/Links
News and Press Releases
Verve Therapeutics Announces Pipeline Progress and Anticipated 2025 Milestones
Preclinical Publications
(Presentation) An investigational in vivo base editing medicine targeting ANGPTL3, VERVE-201, achieves precise, and durable liver editing in nonclinical studies - EAS 2024
(Presentation) An investigational in vivo base editing medicine targeting ANGPTL3, VERVE-201, achieves potent and LDLR-independent liver editing in mouse models - ESC 2023
(Presentation) An in vivo CRISPR base editing therapy to inactivate ANGPTL3: nomination of a development candidate for VERVE-201 - ESC 2022
(Presentation) Preclinical Data Supporting Potential Efficacy of VERVE-201, an Investigational Base Editing Medicine Targeting ANGPTL3 - ACC 2023