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Autologous piggyBac-mediated anti-CD19 CAR-T cells are an investigational cell therapy designed to treat CD19-positive B-cell malignancies, primarily acute lymphoblastic leukemia (ALL). This therapy utilizes the piggyBac transposon system, a non-viral gene delivery platform, to stably integrate a chimeric antigen receptor (CAR) gene into a patient's own T cells. The CAR is engineered to recognize the CD19 antigen, a protein widely expressed on the surface of B-cell leukemia and lymphoma cells. By using a non-viral approach, developers aim to simplify the manufacturing process, reduce costs associated with viral vector production, and potentially generate T cells with a more robust memory phenotype. This specific program is being advanced through clinical trials in Japan, sponsored by the Japan Agency for Medical Research and Development (AMED) in collaboration with Nagoya University and Shinshu University, focusing on pediatric and adult patients with relapsed or refractory ALL.
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