Platelet-derived purified exosome product (PEP): A clinical-grade lyophilized extracellular vesicle product with broad therapeutic potential, including kidney repair

Abstract Extracellular vesicle (EV)-based therapies are rapidly emerging as novel, regenerative cell-free strategies for treating diseases. A current good manufacturing practice (cGMP)-compliant, platelet-derived EV, known as purified exosome product (PEP), has been evaluated in several ongoing preclinical and clinical applications. PEP offers multiple advantages, including abundance and ambient temperature stability. Across 22 published studies, PEP demonstrated pro-angiogenic, anti-inflammatory, and pro-proliferative properties, yielding promising findings in wound, musculoskeletal, cardiovascular, and pulmonary conditions. PEP has been evaluated in phase 2 clinical trials for diabetic foot ulcers and phase 1 clinical trials for various wounds, osteoarthritis, and acute myocardial infarction. However, the therapeutic potential of PEP in kidney disease remains unexplored. Here, we summarize prior and ongoing PEP investigations and introduce PEP reparative effects in a murine model of diabetic kidney disease (DKD), the predominant cause of kidney failure globally. Preliminary findings reveal that intravenous PEP administration reduces kidney fibrosis and hyperglycemia in murine DKD and attenuates fibrogenesis in human proximal tubular cells in vitro. Collectively, PEP demonstrates robust capacity as a regenerative platform with promise to delay disease progression and restore health across multiple organ systems. Further large-scale investigations are warranted to support clinical translation of this promising EV-based technology.