GD2-CART01 (Ospedale Pediatrico Bambino Gesù)

GD2-CART01 (GD2 CAR T-cells (CD28.4-1BB)) is an autologous, third-generation chimeric antigen receptor (CAR) T-cell therapy targeting the disialoganglioside GD2, which is highly expressed in neuroblastoma, pediatric brain tumors, and various sarcomas. The CAR construct incorporates CD28 and 4-1BB costimulatory domains alongside the CD3ζ signaling domain to enhance T-cell activation, persistence, and anti-tumor efficacy. Additionally, the therapy is engineered with an inducible caspase-9 (iC9) suicide gene safety switch (licensed from Bellicum Pharmaceuticals), which can be activated by the dimerizing agent rimiducid (AP1903) to rapidly induce apoptosis of the CAR T-cells in the event of severe toxicity. GD2-CART01 was developed by the Bambino Gesù Children's Hospital and is currently being evaluated in clinical trials for pediatric patients with relapsed or refractory high-risk neuroblastoma, pediatric brain tumors (such as medulloblastoma and diffuse midline glioma), and other GD2-positive solid tumors.

Other names

CAR.GD2.CD28.4-1BB.CD3zGD2 CAR T-cells (CD28.4-1BB)iC9-GD2-CAR T-cellsiC-9-GD2-CAR T-cellsiC 9-GD2-CAR T-cellsthird-generation GD2-CAR T cells