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TRAC-CAR T cell therapy
- Development stage
- Preclinical
- Lead developer
- Azalea Therapeutics
- 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
TRAC-CAR T cell therapy is an innovative in vivo CAR-T cell engineering platform developed by Azalea Therapeutics, a spinout from UC Berkeley and UCSF. The platform utilizes a dual-vector system to generate site-specific CAR-T cells directly inside the patient's body, eliminating the need for complex ex vivo manufacturing and lymphodepletion. It employs CD3-targeted enveloped delivery vehicles (EDVs) to deliver CRISPR-Cas9 ribonucleoprotein (RNP) complexes that create a double-stranded break at the T-cell receptor alpha constant (TRAC) locus. Concurrently, a T-cell-tropic adeno-associated virus (AAV-T) delivers a promoterless chimeric antigen receptor (CAR) gene template flanked by TRAC homology arms. This approach enables precise, homology-directed repair-mediated integration of the CAR gene under the control of the endogenous TRAC promoter, ensuring regulated, physiological CAR expression. The platform is being developed for the treatment of B-cell malignancies and autoimmune diseases.
- Other names
- TRAC-CAR TTRAC-CAR T cellsTRAC-CAR T-cell therapyin vivo TRAC-CAR T cell therapy
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