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PHOX2B (A*24) CAR-T is an investigational chimeric antigen receptor (CAR) T-cell therapy being developed by Myrio Therapeutics for the treatment of neuroblastoma and other pediatric cancers. The therapy is engineered to recognize a specific peptide derived from the Paired-like homeobox 2b (PHOX2B) protein, a transcription factor that is highly and specifically expressed in neuroblastoma cells. This peptide is presented on the cell surface by the Human Leukocyte Antigen (HLA) allele A*24 (HLA-A*24). By targeting this peptide-HLA complex, the CAR-T cells can effectively identify and eliminate neuroblastoma cells that express PHOX2B, even though the protein itself is located intracellularly. This approach leverages T-cell receptor-like recognition to expand the range of targetable antigens beyond traditional surface proteins.
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