anti-mutCALR CAR-T

Anti-mutCALR CAR-T is a first-in-class chimeric antigen receptor (CAR) T-cell therapy designed to target mutant calreticulin (mutCALR), a driver neoantigen found in approximately one-third of patients with myeloproliferative neoplasms (MPNs). In these neoplasms, mutCALR binds to the thrombopoietin receptor (TpoR), leading to its activation and the presentation of the mutant protein on the cell surface of hematopoietic stem/progenitor cells (HSPCs) and megakaryocytes. The therapy aims to selectively eradicate mutCALR-positive clones while sparing healthy hematopoiesis. Preclinical studies have demonstrated robust efficacy in human cell lines, patient-derived HSPCs, and xenograft models of acute myeloid leukemia (AML) and MPN. It is being developed by researchers at University College London (UCL) and the University of Oxford.

Other names

anti-mutant calreticulin CAR-TmutCALR CAR-T