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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
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
Enrollment Count 85 (ESTIMATED)
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
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

Study Record Dates


Current Stage Phase1
Submit Date 2023-12-01
Completion Date 2027-08
Last Update 🔄 2026-03-25

Participation Criteria


Eligible Age 18 Years - 70 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Diagnosis of HeFH or premature CAD Exclusion Criteria: * Homozygous familial hypercholesterolemia * Active or history of chronic liver disease * Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe * Clinically significant or abnormal laboratory values as defined by the protocol
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 21
Locations New Zealand,Canada,United States,United Kingdom,Israel,Australia

Regulatory Information


Has US IND True
FDA Designations Fast Track
Recent Updates Verve was acquired by Lilly in June 2025

Resources/Links