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PLAT-01 is an autologous, CD19-targeted chimeric antigen receptor (CAR) T-cell therapy developed by Seattle Children's Hospital for the treatment of pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL). The therapy involves the genetic modification of a patient's own T cells using a lentiviral vector to express a CAR construct specific to the CD19 protein, which is highly expressed on B-cell malignancies. This specific construct is engineered with a dual-costimulatory signaling architecture, incorporating both CD28 and 4-1BB domains alongside the CD3ζ signaling chain to optimize T-cell activation, expansion, and long-term persistence. A distinguishing feature of this product is the inclusion of a truncated epidermal growth factor receptor (EGFRt) sequence. EGFRt serves as a non-immunogenic selection marker during manufacturing and acts as a safety 'termination switch,' enabling the selective depletion of the CAR T cells via the administration of cetuximab in the event of severe treatment-related toxicities such as cytokine release syndrome.
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