Production and nonclinical evaluation of an autologous iPSC–derived platelet product for the iPLAT1 clinical trial

ABSTRACT: Key Points • GMP-based iPSC-platelet production using imMKCL master cells from an aplastic anemia patient with alloimmune transfusion refractoriness.• Extensive nonclinical evaluation confirmed the safety, quality, and efficacy of the patient iPSC-platelet product. Visual Abstract Abstract Donor-derived platelets are used to treat or prevent hemorrhage in patients with thrombocytopenia. However, ∼5% or more of these patients are complicated with alloimmune platelet transfusion refractoriness (allo-PTR) due to alloantibodies against HLA-I or human platelet antigens (HPA). In these cases, platelets from compatible donors are necessary, but it is difficult to find such donors for patients with rare HLA-I or HPA. To produce platelet products for patients with aplastic anemia with allo-PTR due

SUBMITTER: Sugimoto N 

PROVIDER: S-EPMC9706535 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature