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PHOX2B PC-CAR T is a novel peptide-centric chimeric antigen receptor (PC-CAR) T cell therapy designed to target neuroblastoma. This therapy specifically recognizes a tumor-specific peptide derived from the transcriptional regulator and neuroblastoma dependency gene PHOX2B, presented on the surface of cancer cells by MHC molecules. Unlike traditional CAR-T therapies that target extracellular antigens, this approach enables targeting of intracellular oncoproteins previously considered "undruggable." Preclinical studies demonstrated complete and targeted elimination of neuroblastoma tumors in mouse models. The therapy is being developed by Myrio Therapeutics in collaboration with researchers at Children's Hospital of Philadelphia (CHOP), with first-in-human clinical trials now underway for relapsed or refractory neuroblastoma[1][3][4][6].
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