Drug intelligence / Profile preview

VERVE-301

Development stage
Preclinical
Lead developer
Verve Therapeutics
Modality
Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

VERVE-301 is a novel, investigational in vivo gene editing therapy designed as a single-course treatment to permanently inactivate the LPA gene in the liver. Its primary goal is to reduce blood levels of lipoprotein(a) [Lp(a)], which is a genetically validated, independent risk factor for atherosclerotic cardiovascular disease (ASCVD), ischemic stroke, thrombosis, and aortic stenosis. The therapy uses Verve’s proprietary GalNAc-LNP delivery technology and incorporates a novel gene editor. By targeting the LPA gene directly at the DNA level, VERVE-301 aims to provide durable reduction of Lp(a) with one-time administration—a significant advance over current lipid-lowering therapies that have minimal or no effect on Lp(a). Development of VERVE-301 is being advanced through an exclusive research collaboration between Verve Therapeutics and Lilly[1][2][4][5][7].

02

Targets

LPA (Lysophosphatidic acid)

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