Drug intelligence / Profile preview

RB-340-1

Development stage
Preclinical
Lead developer
Refuge Biotechnologies
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

RB-340-1 is an experimental **autologous HER2-directed CAR-T cell therapy** that incorporates an inducible **CRISPR interference** circuit to suppress **programmed cell death protein 1** expression after antigen engagement. The product is engineered to express an anti-HER2 single-chain variable fragment CAR with **CD28** and **CD3 zeta** signaling domains, together with a TEV protease and an sgRNA targeting the **PDCD1** transcription start site; a second construct uses **LAT** fused to **dCas9-KRAB** through a TEV-cleavable sequence so that T-cell activation triggers localized transcriptional repression of PD-1. It was developed by **Ruby Biotech** as a single-product strategy intended for **HER2-positive cancers**, aiming to combine tumor targeting and checkpoint pathway suppression while avoiding the added cost and toxicity of administering separate checkpoint inhibitors.

Other names
human epidermal growth factor receptor 2 chimeric antigen receptor t-cell therapy + programmed cell death protein 1 knockdown systemHER2 chimeric antigen receptor T-cell therapy + PD-1 knockdown systemanti-HER2 CAR-T cell therapy with PD-1 knockdownanti-HER-2 CAR-T cell therapy with PD-1 knockdownanti-HER 2 CAR-T cell therapy with PD-1 knockdown

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