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GD2-CAR T cells with G3BP1 knockdown

Development stage
Preclinical
Lead developer
Weill Cornell Medicine
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration
Intravenous
01

Overview

GD2-CAR T cells with G3BP1 knockdown are an experimental cellular immunotherapy designed to treat GD2-positive solid tumors, including glioblastoma and neuroblastoma. These cells are genetically modified to express a chimeric antigen receptor (CAR) that recognizes the disialoganglioside GD2, a surface antigen frequently overexpressed in neuroectodermal tumors. Additionally, they incorporate an inducible knockdown system (typically using an NFAT promoter-driven shRNA) targeting Ras GTPase-activating protein-binding protein 1 (G3BP1). G3BP1 is a scaffold protein essential for the assembly of stress granules, which are cytoplasmic RNA-protein complexes that form in response to environmental stressors like hypoxia or chronic antigen stimulation. The knockdown of G3BP1 is intended to modulate these stress-buffering pathways to potentially enhance T cell persistence and function, although preclinical data suggests that G3BP1 may be necessary for optimal CAR T cell efficacy in certain solid tumor models.

Other names
GD2-specific CAR T cells with G3BP1 knockdownGD-2-specific CAR T cells with G3BP1 knockdownGD 2-specific CAR T cells with G3BP1 knockdownG3BP1-knockdown GD2-CAR T cellsG-3BP1-knockdown GD2-CAR T cellsG 3BP1-knockdown GD2-CAR T cells
02

Targets

GD2

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