CCTx-001

CCTx-001 is a first-in-class, autologous chimeric antigen receptor T-cell (CAR T) therapy engineered to target human interleukin-1 receptor accessory protein (IL-1RAP), which is specifically expressed on leukemic cells in acute myeloid leukemia (AML). The therapy uses patient-derived CD4+ and CD8+ T lymphocytes that are genetically modified to express a novel IL-1RAP-directed CAR. This construct incorporates a proprietary anti-IL-1RAP single-chain variable fragment (scFv), an IgG1 hinge region, CD28 and 4‑1BB co-stimulatory domains, and the CD3ζ signaling domain. CCTx-001 selectively eliminates IL‑1RAP-positive AML blasts and leukemic stem cells while sparing healthy hematopoietic stem cells and circulating immune cells. Preclinical studies have demonstrated its antigen-specific cytotoxicity against AML models, rapid induction of interferon gamma release, inhibition of tumor growth in vivo, expansion/persistence of CAR T-cells correlating with reduced AML burden, and resistance to inhibition by soluble IL‑1RAP. It is being developed as a targeted treatment for relapsed/refractory acute myeloid leukemia[5][2][3][4].

Other names

autologous anti-IL-1RAP CAR T cells