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 Hyperlipidemia. 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) and triglycerides. This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population. (Show More)
Development Status	Active
Indication	Hypercholesterolemia
Disease Ontology Term	DOID:13810
Drug Product Name	VERVE-201
Sponsor	Verve Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	36 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	ANGPTL3
Therapeutic Modality	Gene editing
Therapy Route	in vivo
Mechanism of Action	Gene inactivation
Route of Administration	Intravenous
Drug Product Type 🔄	Encapsulated mRNA
Target Tissue/Cell	Hepatocyte
Gene Delivery System Type 🔄	Endocytosis
Non-Viral Vector Type	LNP (LDLR + GalNAc)
Editor Type 🔄	ABE mRNA
Dose 1 🔄	Undisclosed dose escalation, 4 levels

Eligible Age	18 Years - 70 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Refractory hypercholesterolemia * Refractory hypertriglyceridemia Exclusion Criteria: * Active or history of chronic liver disease * Current treatment with monoclonal antibody targeting ANGPTL3 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

No.of Trial Sites 🔄	16
Locations 🔄	Canada,Netherlands,United States,South Africa,United Kingdom,Australia

Has US IND	True
FDA Designations
Recent Updates	Verve was acquired by Lilly in July 2025