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VNX-201

Development stage
Preclinical
Lead developer
Vironexis Biotherapeutics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration
Intravenous
01

Overview

VNX-201 is an investigational AAV-delivered T-cell immuno-gene therapy being developed by Vironexis Biotherapeutics for the treatment of neuroblastoma. Utilizing the proprietary TransJoin™ platform, VNX-201 is designed to deliver a genetic payload via an adeno-associated virus (AAV) vector that programs endogenous T cells *in vivo* to recognize and attack neuroblastoma cells. This approach aims to provide the therapeutic benefits of T-cell-mediated immunotherapy while bypassing the complex and costly *ex vivo* manufacturing processes associated with traditional CAR-T cell therapies. By enabling the patient's own body to produce tumor-targeting T cells following a single administration, VNX-201 represents a potential 'off-the-shelf' gene therapy solution for solid tumors. As of early 2024, the program is in the discovery stage of development.

02

Targets

CD3 (T-cell surface glycoprotein CD3)GD2

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