Formulation, manufacturing and regulatory strategies for extracellular vesicles-based drug products for targeted therapy of central nervous system diseases

ABSTRACT Introduction Extracellular vesicles (EVs) are a family of natural nanosize vesicles that transport biological cargos, including DNA, RNA, protein, and lipids. In recent years, EVs have attracted a lot of attention for their capability to function as a drug delivery system (DDS). While clinical trials have been conducted, the techniques to formulate, process, and quality control EVs-based drug products still have the potential to be improved, especially in large scale production. Areas covered We will introduce and discuss EVs biology, their potential role in CNS pathologies under different conditions, and recent methods of isolation. We will then provide a detailed discussion on current benchtop formulation methods, quality control methods, and regulatory affairs for EVs-based drug products. Expert opinion Despite being a hot topic, EVs-based drug products have not received any approval from regulatory agencies. In this review, we will provide general guidance to help EVs-based drug products to ‘move from bench to bedside’.