Harnessing Exosomes for Drug Delivery and Regenerative Medicine

Exosome research has rapidly evolved over the past decade, revealing the potential of these nanometer-scale, membrane-bound particles as natural mediators of intercellular communication and as exceptionally promising drug delivery vehicles. Exosomes, a subclass of extracellular vesicles (EVs), possess unique biomolecular compositions and inherent capabilities to cross biological barriers, which are unmatched by synthetic systems such as liposomes or polymeric nanoparticles. However, in line with ISEV guidelines, the broader term EV is increasingly used unless endosomal origin can be demonstrated, as exosomes represent a subtype of small EVs. Recent studies demonstrate that exosomes not only transport proteins, lipids, and nucleic acids effectively but also confer native biocompatibility and stability within biological environments. Despite these advantages, several barriers remain to their clinical realization, including limited understanding of biodistribution, dose-response dynamics, loading efficiency, and the biological heterogeneity inherent to exosome populations. Furthermore, while advances in exosomes derived from different sources, such as cells and milk, show exciting translational promise, the balance between engineering complexity, reproducibility, and regulatory approval continues to define the frontier of exosome-based therapeutics.

This Research Topic aims to elucidate the multifaceted potential of exosome-based drug delivery and explore innovative solutions to overcome translational hurdles. The objective is to gather evidence and perspectives that clarify mechanisms of exosome biogenesis, secretion, uptake, and therapeutic action, as well as to assess how engineered exosomes can be optimized for precise cellular and tissue targeting. Particular emphasis is placed on understanding how exosome source, production scale-up, and modification strategies influence therapeutic efficacy and clinical safety. By integrating insights from molecular biology, bioengineering, and pharmaceutical science, this topic seeks to bridge the gap between laboratory research and regulated clinical application of exosome-based therapeutics.

To gather further insights into the boundaries of exosome drug delivery research, we welcome articles addressing, but not limited to, the following themes:

- Exosome heterogeneity and its implications for regulatory approval and clinical translation

- Scalable manufacturing, purification, and cost-efficient production of therapeutic exosomes

- Strategies to enhance tissue, organ, and cellular targeting for improved biodistribution and efficacy

- Bioengineering approaches to optimize exosome loading and maintain biological function

- Mechanistic studies on dose-response relationships and pharmacokinetics of exosome therapeutics

- Ethical, social, and communication challenges arising from unregulated or non-clinical uses of exosome-based products

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Community Case Study
• Data Report
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Community Case Study
• Data Report
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Policy and Practice Reviews
• Policy Brief
• Review
• Study Protocol
• Systematic Review
• Technology and Code

Keywords: Extracellular vesicles, Exosomes, Drug delivery systems, Nanomedicine, Targeted therapy, RNA delivery, Biocompatibility, Biodistribution, Therapeutic engineering, Clinical translation

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.