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SC-CAR.CLEC2Av1 is an investigational chimeric antigen receptor (CAR) T-cell therapy targeting C-type lectin domain family 2 member A (CLEC2A). Developed by Seattle Children's Hospital in collaboration with Fred Hutchinson Cancer Center, the therapy is designed to treat pediatric and adult patients with relapsed or refractory acute myeloid leukemia (AML), particularly those with KMT2A rearrangements or monosomy 7. CLEC2A is a cancer-specific antigen highly expressed in these high-risk AML subtypes but absent in normal, healthy tissues, minimizing the risk of on-target, off-tumor hematopoietic toxicity. SC-CAR.CLEC2Av1 utilizes a fully human antibody binder (developed at the University of Pittsburgh) and is delivered via a lentiviral vector. It is currently being evaluated in the Phase 1 ADVANCE-01 clinical trial.
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