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CRISPR-CAR33-1
- Development stage
- Preclinical
- Lead developer
- CRISPR 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
CRISPR-CAR33-1 is an experimental allogeneic, CRISPR/Cas9 gene-edited chimeric antigen receptor T-cell (CAR-T) therapy targeting the CD33 (Siglec-3) antigen. Developed by CRISPR Therapeutics, it is designed for the treatment of acute myelogenous leukemia (AML). The therapy utilizes healthy donor-derived T cells that undergo three specific genetic modifications: disruption of the TRAC locus to prevent graft-versus-host disease (GvHD), site-specific insertion of the anti-CD33 CAR construct into the TRAC locus for regulated expression, and disruption of the beta-2-microglobulin (B2M) locus to eliminate MHC class I expression and prevent host-mediated rejection. Preclinical studies demonstrate that CRISPR-CAR33-1 exhibits potent tumor cell lysis and high levels of interferon-gamma and interleukin-2 secretion upon engagement with CD33-positive AML cells.
- Other names
- allogeneic anti-CD33 CAR-T cells
02
Targets
CD33 (Myeloid cell surface antigen CD33)
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