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AAV-CAR-T cells
- Development stage
- Preclinical
- Lead developer
- Fred Hutchinson Cancer Center
- Modality
- CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-CAR-T cells represent an experimental in vivo gene therapy platform designed to generate chimeric antigen receptor (CAR) T cells directly within a patient's body. This approach utilizes engineered adeno-associated virus (AAV) vectors, such as the T-cell-tropic variants Ang3 and Ang6, to deliver CAR-encoding genetic material to circulating T cells, bypassing the complex and costly ex vivo manufacturing processes associated with traditional CAR-T therapies. To ensure stable and long-term transgene expression in proliferating T cells, some versions of this technology incorporate the Sleeping Beauty (SB) transposase-transposon system. Preclinical research has explored this modality for the treatment of T-cell leukemia (using CD4-targeting CARs), HIV, solid tumors, and autoimmune diseases. The platform aims to provide a more accessible and scalable alternative to conventional cell therapies by transforming the patient's own immune system into a therapeutic factory through a direct viral injection.
- Other names
- AAV delivering CAR gene therapyACGin vivo CAR-T cell therapy
02
Targets
CD4 (T lymphocyte surface antigen)
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