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PRAME (A*02) CAR-T is a discovery-stage chimeric antigen receptor (CAR) T-cell therapy being developed by Myrio Therapeutics for the treatment of neuroblastoma and other pediatric cancers. The therapy utilizes T cells engineered to express a CAR that specifically recognizes the PRAME (Preferentially Expressed Antigen in Melanoma) peptide when presented by the HLA-A*02 (Human Leukocyte Antigen A*02) complex. PRAME is a cancer-testis antigen that is highly expressed in various solid and hematologic malignancies but has limited expression in normal adult tissues, making it an attractive target for immunotherapy. By targeting the peptide-HLA complex, this approach allows the CAR-T cells to recognize intracellular tumor antigens that are typically inaccessible to conventional surface-targeting CARs, potentially expanding the range of treatable pediatric solid tumors.
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