Extracellular Vesicles as Therapeutic Agents: Progress and Challenges in Translational Research
XU Mingzhi1,2#, WANG Yue1,2#, HE Wei1*, ZHANG Jianmin1,2*
EVs (extracellular vesicles), as natural nanoscale bioactive carriers with favorable biocompatibility and certain targeting properties, can effectively deliver bioactive cargo to recipient cells and regulate their functions. These biological features of EVs make them promising candidates for innovative drug development. This review provides a comprehensive overview of the historical development and contemporary advancements in EVbased therapeutics, encompassing fundamental pharmacological mechanisms, clinical application potential, and key challenges in industrial-scale translation. Accumulating evidence indicates that EVs possess unique therapeutic advantages in neurological disorders, cancer immunotherapy, and tissue regeneration, with several candidate products having already advanced to clinical trial stages. However, the industrial translation of such drugs still faces multiple bottlenecks, including difficulties in standardizing the production process and quality control systems, as well as the lack of comprehensive regulatory guidelines globally. This article systematically reviews the development pathway and current translation status of EV-based drugs, proposes a development strategy based on the QbD (quality by design) concept, and discusses key considerations in non-clinical research to align with regulatory requirements. Finally, it calls for collaborative efforts among scientists, entrepreneurs, and regulatory authorities to jointly advance the development of innovative EV-based therapeutics, enhance the drug evaluation process, and promote product translation, thereby pioneering a new frontier in drug development.
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