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Induced pluripotent stem cell-derived platelets (iPSC-PLTs) are an autologous cell therapy developed for the treatment of thrombocytopenia, specifically in patients with aplastic anemia and alloimmune platelet transfusion refractoriness (allo-PTR). The production process involves reprogramming a patient's peripheral blood mononuclear cells into iPSCs, which are then differentiated into immortalized megakaryocyte progenitor cell lines (imMKCLs) through the overexpression of c-MYC, BMI1, and BCL-XL. These imMKCLs are expanded and then induced to undergo thrombopoiesis in turbulent flow bioreactors to generate functional platelets. This approach aims to provide a stable supply of HLA-matched platelets for patients who have developed antibodies against donor platelets. The therapy was evaluated in the first-in-human iPLAT1 clinical trial conducted at Kyoto University Hospital.
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