Advances in the Druggability and Clinical Translation of Platelets
YAO Shun1,2#, YANG Yongqi1,2#, LIU Cuicui1,2,3, ZHOU Jiaxi1,2, WANG Fei3*, WANG Hongtao1,2,3*
Platelets, as essential anucleate cells of the body, play a central role not only in hemostasis but also in immune regulation and tissue repair. Clinically, platelet transfusion remains a key therapeutic approach for treating thrombocytopenia and related disorders. However, this therapy is still constrained by multiple challenges, including limited supply, short storage lifespan, immune rejection, and potential infection risks. To overcome these bottlenecks, research has increasingly shifted toward in vitro platelet regeneration strategies based on hiPSCs (human induced pluripotent stem cells). With advances such as cytokine-directed induction and biomimetic bioreactors, large-scale and GMP-compliant platelet production has been achieved and is now progressing into clinical validation. Beyond transfusion substitution, hiPSC-derived platelets (iPSC-PLTs) have also shown promising applications in tissue repair, including hair follicle regeneration and wound healing. Moreover, due to their strong targeting ability and excellent biocompatibility, platelets and their derivatives are being actively explored as novel drug delivery vehicles. This review provides a systematic overview of recent advances in platelet research across transfusion replacement, regenerative repair, and drug delivery, with a particular focus on the breakthroughs and challenges of iPSC-PLTs in large-scale production, functional validation, and clinical application. This review further analyze the translational bottlenecks and corresponding strategies, and highlight future prospects for developing functionally customized platelet products, aiming to accelerate the clinical translation of this innovative cell therapy.
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