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cssDNA CAR-T
- Development stage
- Preclinical
- Lead developer
- Kano Therapeutics
- Modality
- Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, 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
cssDNA CAR-T is a non-viral genome engineering platform developed by Kano Therapeutics for the generation of chimeric antigen receptor (CAR) T-cells. The technology utilizes circular single-stranded DNA (cssDNA) templates for CRISPR/Cas9-mediated site-specific integration of CAR circuits. This approach is designed to overcome the limitations of viral vectors and double-stranded DNA (dsDNA) templates, such as immunogenicity and payload constraints, supporting transgenes up to 14kb. The platform includes novel nuclear localization features to improve integration efficiency and uses T-cell targeted lipid nanoparticles (T-LNPs) for the co-delivery of Cas9 mRNA, guide RNA, and the cssDNA template. It is intended for both ex vivo manufacturing and in vivo CAR-T cell generation, with potential applications in solid tumors, hematological malignancies, and autoimmune disorders.
- Other names
- circular single-stranded DNA CAR-T
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