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**GPC2 CAR T** is an autologous chimeric antigen receptor (CAR) T-cell therapy targeting glypican-2 (GPC2), a cell surface oncoprotein overexpressed on neuroblastoma and other embryonal tumors. It uses a second-generation CAR construct derived from the D3-GPC2 antibody (specific for a tumor-unique conformational epitope), incorporating a CD28 hinge/transmembrane domain and 4-1BB/CD3ζ costimulatory domains for enhanced persistence and cytotoxicity. Developed by researchers at the Children's Hospital of Philadelphia (CHOP) and University of Pennsylvania, with support from Gilead Sciences and the National Cancer Institute, it demonstrates potent selective killing of GPC2-high tumor cells in preclinical models, minimal off-tumor toxicity, and is currently in phase 1 trials for relapsed/refractory high-risk neuroblastoma (NCT05650749), medulloblastoma (NCT07087002), and retinoblastoma.
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