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GD2-CART01 is an autologous, third-generation chimeric antigen receptor (CAR) T-cell therapy targeting disialoganglioside GD2, which is highly expressed on neuroblastoma cells. It incorporates two costimulatory domains (CD28 and 4-1BB) and includes an inducible caspase 9 suicide gene as a safety switch that can be activated to rapidly eliminate the CAR T cells if dangerous side effects occur. The therapy has shown promising results in treating relapsed or refractory high-risk neuroblastoma, with clinical trials demonstrating feasibility, safety, and potential sustained antitumor effects.
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