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OCI-001 is an autologous chimeric antigen receptor (CAR) T-cell therapy specifically engineered to target CD1a, a protein highly and specifically expressed on the surface of cortical T-cell acute lymphoblastic leukemia (coT-ALL) cells. Developed by Onechain Immunotherapeutics, a spin-off from the Josep Carreras Leukaemia Research Institute, this therapy involves the genetic modification of a patient's own T cells to express a CAR that recognizes CD1a. Unlike many other T-cell targets, CD1a expression is restricted to a specific stage of thymocyte development and is not found on mature T cells or hematopoietic stem cells, which potentially reduces the risk of severe T-cell aplasia and off-target toxicities. OCI-001 is currently being evaluated in the Phase 1 CARxALL clinical trial for patients with relapsed or refractory CD1a-positive T-ALL and T-cell acute lymphoblastic lymphoma.
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