CRISPR-CAR33-3

CRISPR-CAR33-3 (often associated with the CTX330 program) is an investigational allogeneic, CRISPR/Cas9 gene-edited chimeric antigen receptor T-cell (CAR-T) therapy targeting the CD33 (Siglec-3) antigen. Developed by CRISPR Therapeutics, the therapy is designed to treat myeloid malignancies such as acute myeloid leukemia (AML). The manufacturing process involves three specific genetic modifications using CRISPR/Cas9: disruption of the T-cell receptor alpha constant (TRAC) locus to reduce the risk of graft-versus-host disease (GvHD), site-specific insertion of the anti-CD33 CAR construct into the TRAC locus to enhance potency and safety, and disruption of the beta-2-microglobulin (B2M) locus to eliminate MHC class I expression, thereby preventing host-versus-graft rejection and improving CAR-T cell persistence. Preclinical data indicates that these cells exhibit potent tumor cell lysis and effector cytokine secretion (IFN-gamma and IL-2) upon engagement with CD33-positive AML cells.

Other names

Allogeneic anti-CD33 CAR-T cellsAnti-CD33 CRISPR-edited CAR-TAnti-CD-33 CRISPR-edited CAR-TAnti-CD 33 CRISPR-edited CAR-T