Exosome-based drug delivery : a next-generation platform for cancer, infection, neurological and immunological diseases, gene therapy and regenerative medicine

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Serrano, Dolores R. and Juste, Francisco and Anaya, Bryan J and Ramirez, Bianca I and Sánchez-Guirales, Sergio A. and Quispillo, John M. and Hernandez, Ester M. and Simon, Jesus A. and Trallero, Jose M. and Serrano, Celia and Rawat, Satyavati and Lalatsa, Katerina (2025) Exosome-based drug delivery : a next-generation platform for cancer, infection, neurological and immunological diseases, gene therapy and regenerative medicine. Pharmaceutics, 17 (10). 1336. ISSN 1999-4923 (https://doi.org/10.3390/pharmaceutics17101336)

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Abstract

Exosomes naturally derived extracellular vesicles have emerged as powerful bio-nanocarriers in precision medicine. Their endogenous origin, biocompatibility, and ability to encapsulate and deliver diverse therapeutic payloads position them as transformative tools in drug delivery, gene therapy, and regenerative medicine. This review presents a comprehensive analysis of exosome-based therapeutics across multiple biomedical domains, including cancer, neurological and infectious diseases, immune modulation, and tissue repair. Exosomes derived from stem cells, immune cells, or engineered lines can be loaded with small molecules, RNA, or CRISPR-Cas systems, offering highly specific and low-immunogenic alternatives to viral vectors or synthetic nanoparticles. We explore endogenous and exogenous loading strategies, surface functionalization techniques for targeted delivery, and innovations that allow exosomes to traverse physiological barriers such as the blood–brain barrier. Furthermore, exosomes demonstrate immunomodulatory and regenerative properties in autoimmune and degenerative conditions, with promising roles in skin rejuvenation and cosmeceuticals. Despite their potential, challenges remain in large-scale production, cargo loading efficiency, and regulatory translation. Recent clinical trials and industry efforts underscore the accelerating momentum in this field. As bioengineering and manufacturing platforms mature, exosome-based therapeutics are poised to become a central pillar of next-generation personalized medicine. This review offers critical insights into current technologies, therapeutic mechanisms, and future directions to unlock the full translational potential of exosomes in clinical practice.

ORCID iDs

Serrano, Dolores R., Juste, Francisco, Anaya, Bryan J, Ramirez, Bianca I, Sánchez-Guirales, Sergio A., Quispillo, John M., Hernandez, Ester M., Simon, Jesus A., Trallero, Jose M., Serrano, Celia, Rawat, Satyavati and Lalatsa, Katerina ORCID logoORCID: https://orcid.org/0000-0003-4791-7468;
CORE (COnnecting REpositories)