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

•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. [Display omitted] Donor-derived platelets are u...

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Veröffentlicht in:Blood advances 2022-12, Vol.6 (23), p.6056-6069
Hauptverfasser: Sugimoto, Naoshi, Nakamura, Sou, Shimizu, Shin, Shigemasa, Akiko, Kanda, Junya, Matsuyama, Nobuki, Tanaka, Mitsunobu, Hayashi, Tomoya, Fuchizaki, Akihiro, Nogawa, Masayuki, Watanabe, Naohide, Okamoto, Shinichiro, Handa, Makoto, Sawaguchi, Akira, Momose, Dai, Koh, Ki-Ryang, Tani, Yoshihiko, Takaori-Kondo, Akifumi, Eto, Koji
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container_end_page 6069
container_issue 23
container_start_page 6056
container_title Blood advances
container_volume 6
creator Sugimoto, Naoshi
Nakamura, Sou
Shimizu, Shin
Shigemasa, Akiko
Kanda, Junya
Matsuyama, Nobuki
Tanaka, Mitsunobu
Hayashi, Tomoya
Fuchizaki, Akihiro
Nogawa, Masayuki
Watanabe, Naohide
Okamoto, Shinichiro
Handa, Makoto
Sawaguchi, Akira
Momose, Dai
Koh, Ki-Ryang
Tani, Yoshihiko
Takaori-Kondo, Akifumi
Eto, Koji
description •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. [Display omitted] 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 to rare HPA-1 mismatch in Japan, we developed an ex vivo good manufacturing process (GMP)–based production system for an induced pluripotent stem cell–derived platelet product (iPSC-PLTs). Immortalized megakaryocyte progenitor cell lines (imMKCLs) were established from patient iPSCs, and a competent imMKCL clone was selected for the master cell bank (MCB) and confirmed for safety, including negativity of pathogens. From this MCB, iPSC-PLTs were produced using turbulent flow bioreactors and new drugs. In extensive nonclinical studies, iPSC-PLTs were confirmed for quality, safety, and efficacy, including hemostasis in a rabbit model. This report presents a complete system for the GMP-based production of iPSC-PLTs and the required nonclinical studies and thus supports the iPLAT1 study, the first-in-human clinical trial of iPSC-PLTs in a patient with allo-PTR and no compatible donor using the autologous product. It also serves as a comprehensive reference for the development of widely applicable allogeneic iPSC-PLTs and other cell products that use iPSC-derived progenitor cells as MCB.
doi_str_mv 10.1182/bloodadvances.2022008512
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[Display omitted] 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 to rare HPA-1 mismatch in Japan, we developed an ex vivo good manufacturing process (GMP)–based production system for an induced pluripotent stem cell–derived platelet product (iPSC-PLTs). Immortalized megakaryocyte progenitor cell lines (imMKCLs) were established from patient iPSCs, and a competent imMKCL clone was selected for the master cell bank (MCB) and confirmed for safety, including negativity of pathogens. From this MCB, iPSC-PLTs were produced using turbulent flow bioreactors and new drugs. In extensive nonclinical studies, iPSC-PLTs were confirmed for quality, safety, and efficacy, including hemostasis in a rabbit model. This report presents a complete system for the GMP-based production of iPSC-PLTs and the required nonclinical studies and thus supports the iPLAT1 study, the first-in-human clinical trial of iPSC-PLTs in a patient with allo-PTR and no compatible donor using the autologous product. 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[Display omitted] 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 to rare HPA-1 mismatch in Japan, we developed an ex vivo good manufacturing process (GMP)–based production system for an induced pluripotent stem cell–derived platelet product (iPSC-PLTs). Immortalized megakaryocyte progenitor cell lines (imMKCLs) were established from patient iPSCs, and a competent imMKCL clone was selected for the master cell bank (MCB) and confirmed for safety, including negativity of pathogens. 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subjects Animals
Antigens, Human Platelet
Blood Platelets - metabolism
Hematopoietic Stem Cell Transplantation - adverse effects
Humans
Induced Pluripotent Stem Cells - metabolism
Platelet Transfusion - adverse effects
Rabbits
Regular
Thrombocytopenia - etiology
title Production and nonclinical evaluation of an autologous iPSC–derived platelet product for the iPLAT1 clinical trial
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