anti-CD33 CAR-NK (KLRC1 KO)

Anti-CD33 CAR-NK (KLRC1 KO) is an experimental cell therapy consisting of primary natural killer (NK) cells that have been dual-modified: (1) lentivirally transduced with a chimeric antigen receptor (CAR) targeting CD33 (a myeloid cell surface antigen), and (2) subjected to CRISPR/Cas9-mediated knockout of the KLRC1 gene, which encodes the inhibitory immune checkpoint receptor NKG2A. By eliminating NKG2A expression, the therapy aims to prevent suppression of NK cell killing activity via the HLA-E–NKG2A immune checkpoint axis, which is commonly exploited by AML cells to evade immune destruction. The drug is a biologic/cell therapy targeting CD33 on leukemic blasts, intended for the treatment of acute myeloid leukemia (AML). It has demonstrated potent antileukemic activity against AML cell lines and primary patient-derived blasts both in vitro and in vivo in preclinical studies. The research has been published in Nature Communications (2024) and presented at ASH (2025), with academic groups including the University of Pennsylvania and Dana-Farber Cancer Institute driving development.

Other names

anti-CD33 CAR-NK cells with KLRC1 knockoutanti-CD-33 CAR-NK cells with KLRC1 knockoutanti-CD 33 CAR-NK cells with KLRC1 knockoutCD33-targeted CAR-NK cells (KLRC1 KO)CAR-KO-NK