Exosomes in Medicine: A Critical Appraisal of Therapeutic Potential, Current Applications, and Translational Hurdles Publisher



Moghadam R ; Moghadam M ; Shahmir C ; Seyhoun I ; Zolbin M M
Source: Regenerative Engineering and Translational Medicine Published:2026

Abstract

Background: Exosomes have transitioned from being considered cellular waste to becoming central mediators in intercellular communication, sparking intense investigation across medicine fields. However, as the exosome research expands, the need for critical appraisal of the evidence base has become more important. Main Body: We first learn about the fundamental biological properties of exosomes, heterogeneity, complex cargo, and context-dependent functionality. We then survey therapeutic applications across major disease areas, distinguishing the evidence level of these findings. Subsequently, we analyze the translational landscape, clinical trial trends, manufacturing hurdles (scalability, isolation purity, and batch consistency), regulatory uncertainty, and finally, safety considerations requiring resolution. Conclusion: The translational gap between preclinical promise and clinical reality remains substantial. Meaningful progress requires moving beyond descriptive cataloging toward rigorous, reproducible, and regulation-aligned development. Emerging engineering strategies, artificial intelligence applications, and alternative exosome sources offer potential paths forward, but must be evaluated against realistic benchmarks. This review works towards a foundation for a realistic assessment of where exosome-based medicine stands and what strategic directions offer the most viable paths toward approved therapeutics. Lay Summary: Exosomes are nano-sized particles secreted by almost all cell types. No longer regarded as the cellular waste that they were once thought to be, exosomes are now known to be important messengers transporting proteins, genetic material, and other molecules between cells in the development of normal function and disease. Because their contents reflect the status of the cell types producing them, exosomes also act as liquid biopsies, giving a non-invasive window into disease states. They also hold promise as therapies: capable of crossing barriers such as the blood-brain barrier and delivering treatments directly to target cells. This makes them exciting alternatives to stem cell therapies and drug delivery systems in applications that could range from cancer and heart disease to neurological disorders and skin products. There are indeed many challenges that remain to be resolved, such as large-scale production, quality control, storage, regulation, and safety concerns, as some exosomes can promote diseases. In general, exosomes represent a field that is quickly unfolding and promises to dramatically alter the dimensions of diagnosis and treatment as research unfolds. © The Author(s), under exclusive licence to The Regenerative Engineering Society 2026.