CLEC2A CAR T cells

**CLEC2A CAR T cells** are an investigational autologous T cell therapy engineered with a chimeric antigen receptor (CAR) targeting CLEC2A, a leukemia-restricted antigen highly enriched in KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML), particularly high-risk subtypes in infants, children, and treatment-related AML in adults. The CAR utilizes a fully human VH single domain antibody binder cloned into a construct with an IgG4 hinge, intermediate spacer, and 4-1BB/CD3ζ costimulatory domain, enabling potent target-specific cytotoxicity and cytokine production (IL-2, IFNγ, TNFα) against CLEC2A-positive AML cells, including low antigen density models, without activity against CLEC2A-negative cells. Preclinical data from in vitro co-cultures and in vivo xenograft models (e.g., OCI-AML2 and MOLM13 in NSG mice) demonstrate robust anti-leukemia efficacy, durable persistence, and survival benefit without evidence of hematotoxicity due to CLEC2A's absence in normal hematopoietic cells and tissues. Developed through proteo-genomic discovery analyzing over 3,000 patient samples, it represents a novel immunotherapy avoiding myeloablation risks associated with shared AML antigens, with GMP manufacturing complete and first-in-human trials planned for early 2026 in Seattle (lentiviral vector) and Rome (retroviral vector).[1][2][3]

Other names

CLEC2A-directed CAR T cellsCLEC-2A-directed CAR T cellsCLEC 2A-directed CAR T cellsCLEC2A-targeting CAR T cellsCLEC-2A-targeting CAR T cellsCLEC 2A-targeting CAR T cellsCLEC2A-CAR TCLEC-2A-CAR TCLEC 2A-CAR T