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GPC2-targeted CAR T cells are an investigational, autologous chimeric antigen receptor (CAR) T-cell therapy designed to target glypican-2 (GPC2), an oncofetal cell surface heparan sulfate proteoglycan. GPC2 is highly expressed in neuroblastoma and other pediatric solid tumors, such as medulloblastoma and retinoblastoma, while showing minimal expression in normal tissues. The therapy involves genetically engineering a patient's own T cells to express a second-generation CAR construct, typically combining a GPC2-binding single-chain variable fragment (such as the D3 or CT3 clones) with a CD3ζ signaling domain and a costimulatory domain (such as 4-1BB or CD28). GPC2-targeted CAR T cells are being evaluated in Phase 1 clinical trials for relapsed or refractory neuroblastoma, medulloblastoma, and other GPC2-positive pediatric malignancies.
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