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Scientific journal publication

Extracellular Vesicles as Next-Generation Diagnostics and Advanced Therapy Medicinal Products

Stawarska, Agnieszka; Bamburowicz-Klimkows, Magdalena; Rundén-Pran, Elise; Dusinska, Maria; Cimpan, Mihaela Roxana; Rios-Mondragon, Ivan; Grudzinski, Ireneusz P.

Publication details

Journal: International Journal of Molecular Sciences, vol. 25, 6533, 2024

Arkiv: hdl.handle.net/11250/3136118
Doi: doi.org/10.3390/ijms25126533

Extracellular vesicles (EVs) hold great promise for clinical application as new diagnostic and therapeutic modalities. This paper describes major GMP-based upstream and downstream manufacturing processes for EV large-scale production, also focusing on post-processing technologies such as surface bioengineering and uploading studies to yield novel EV-based diagnostics and advanced therapy medicinal products. This paper also focuses on the quality, safety, and efficacy issues of the bioengineered EV drug candidates before first-in-human studies. Because clinical trials involving extracellular vesicles are on the global rise, this paper encompasses different clinical studies registered on clinical-trial register platforms, with varying levels of advancement, highlighting the growing interest in EV-related clinical programs. Navigating the regulatory affairs of EVs poses real challenges, and obtaining marketing authorization for EV-based medicines remains complex due to the lack of specific regulatory guidelines for such novel products. This paper discusses the state-of-the-art regulatory knowledge to date on EV-based diagnostics and medicinal products, highlighting further research and global regulatory needs for the safe and reliable implementation of bioengineered EVs as diagnostic and therapeutic tools in clinical settings. Post-marketing pharmacovigilance for EV-based medicinal products is also presented, mainly addressing such topics as risk assessment and risk management.