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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
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

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