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PTA001 is an autologous chimeric antigen receptor (CAR) T-cell therapy targeting the CD19 antigen, developed by Shanghai Tong Ren Hospital. The therapy involves the genetic modification of a patient's own T cells using a lentiviral vector to express a second-generation CAR construct. This construct consists of an anti-CD19 single-chain variable fragment (scFv) for antigen recognition, coupled with a CD28 costimulatory domain and a CD3-zeta signaling domain to facilitate T-cell activation and persistence. PTA001 is primarily investigated for the treatment of relapsed or refractory CD19-positive B-cell malignancies, including B-cell acute lymphoblastic leukemia (B-ALL) and various B-cell lymphomas. Upon intravenous administration, the engineered T cells recognize and bind to CD19 on the surface of malignant B cells, triggering targeted cytotoxicity and tumor cell lysis.
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