piggyBac CAR19 T cells

piggyBac CAR19 T cells are an investigational cell therapy product where patient or donor T cells are genetically engineered ex vivo using the piggyBac transposon system to express a chimeric antigen receptor (CAR) targeting CD19, a surface antigen expressed on B cells. The piggyBac transposon system is a non-viral gene transfer method that allows for efficient and cost-effective genetic modification of T cells by integrating the CAR gene via electroporation with plasmids encoding both the CAR transgene and the piggyBac transposase. The CAR construct commonly employs a second-generation design, typically incorporating a 4-1BB or CD28 co-stimulatory domain and a CD3ζ signaling tail. These engineered T cells are designed to recognize and kill CD19-expressing B cell malignancies such as acute lymphoblastic leukemia (ALL) and diffuse large B cell lymphoma (DLBCL). Clinical trials and case studies have demonstrated feasibility, significant anti-tumor activity, and the potential for durable remissions. However, two documented cases of T cell lymphoma arising from piggyBac CAR19 T cells have led to increased scrutiny and voluntary trial suspension for some protocols, related to concerns about insertional mutagenesis and transformation risk unique to the non-viral system[1][2][3][5][6][7].

Other names

piggyBac-generated CAR19-T cellspiggyBac CD19 CAR-T cellsPB CAR19 T cells