Drug intelligence / Profile preview

INXN-4001

Development stage
Phase 1
Lead developer
Triple-Gene
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Intracoronary
01

Overview

INXN-4001 is an investigational **non-viral multigenic plasmid gene therapy** developed by Triple-Gene and Precigen for heart failure. The single triple-effector plasmid is designed to constitutively express human stromal cell-derived factor 1 alpha, vascular endothelial growth factor 165, and S100 calcium-binding protein A1 in cardiac tissue. These transgenes are intended to promote progenitor-cell recruitment, angiogenesis, and cardiomyocyte calcium handling, respectively, thereby addressing pathological myocardial remodeling. It was administered to the cardiac ventricle by retrograde coronary sinus infusion in a completed first-in-human Phase 1 study in patients with end-stage heart failure supported by a left ventricular assist device. ([prnewswire.com](https://www.prnewswire.com/news-releases/precigen-triple-gene-provides-six-month-follow-up-data-from-phase-i-study-of-inxn-4001-a-multigenic-investigational-therapeutic-candidate-for-heart-failure-301107258.html))

02

Targets

VEGFR-1 (Vascular endothelial growth factor receptor 1)CXCR4 (C-X-C motif chemokine receptor 4)VEGFR2 (Vascular endothelial growth factor receptor 2)

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