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HA-1 TCR-T is a first-in-human, investigational engineered T cell therapy in which patient or donor T cells are genetically modified to express a T cell receptor (TCR) specifically recognizing the minor histocompatibility antigen HA-1 in the context of HLA-A*0201. The therapy targets leukemic cells (primarily in acute myeloid leukemia and other hematologic malignancies) that express HA-1, while sparing normal, non-hematopoietic tissues because HA-1 expression is restricted to hematopoietic cells. The engineered TCR is delivered via lentiviral vector, and both CD4+ and CD8+ T cells can be targeted by including a CD8 co-receptor to enhance class I TCR activity in CD4+ cells. The therapy also incorporates a safety switch and a tracking marker. The main developer groups are Seattle Children’s Research Institute and academic collaborators. HA-1 TCR-T is in Phase 1 clinical investigation post allogeneic hematopoietic stem cell transplantation for relapsed acute leukemia, with early data suggesting safety and feasibility and some patients achieving or maintaining remission.
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