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VERVE-102
- Development stage
- Phase 2
- Lead developer
- Verve Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
VERVE-102 is a novel, investigational in vivo base editing medicine developed as a single-course gene editing therapy to permanently turn off the PCSK9 gene in the liver. By using an adenine base editor and a guide RNA (gRNA) targeting PCSK9, both encapsulated in a lipid nanoparticle (LNP) and administered via intravenous infusion, it aims to durably reduce disease-driving LDL cholesterol (LDL-C). The therapy uses Verve Therapeutics' proprietary GalNAc-LNP delivery technology for targeted delivery to liver cells. Its primary indication is for patients with heterozygous familial hypercholesterolemia (HeFH), premature coronary artery disease (CAD), or hyperlipidemia who require additional LDL-C lowering. The mechanism of action involves permanent disruption of the PCSK9 gene, leading to reduced production of PCSK9 protein and increased LDL receptor activity, thereby lowering blood LDL-C levels[1][2][4][5][6].
- Other names
- base editing therapy targeting cardiovascular diseasein vivo base-editing cardiovascular medicine
02
Targets
ASGPR (Asialoglycoprotein Receptor 1)PCSK9 (Proprotein convertase subtilisin/kexin type 9)
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