Drug intelligence / Profile preview

RACR

Development stage
Preclinical
Lead developer
Virtuoso Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Intravenous, Intratumoral
01

Overview

RACR (Receptor-Associated CRISPR) is an experimental gene therapy platform and therapeutic candidate developed by Virtuoso Therapeutics. The technology utilizes a specialized delivery system where CRISPR/Cas9 components are linked to ligands that target specific cell-surface receptors, such as CD47 or EpCAM. This approach is designed to enable cell-type-specific genome editing, potentially reducing off-target effects associated with systemic CRISPR delivery. By targeting receptors overexpressed on tumor cells, RACR aims to selectively disrupt oncogenic drivers or modulate the tumor microenvironment. The primary focus of the development has been in oncology, specifically for the treatment of solid tumors where precise targeting of malignant cells is critical.

Other names
Receptor-Associated CRISPR
02

Targets

CD47 (Cluster of Differentiation 47)EPCAM (Epithelial cell adhesion molecule)

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