Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use

Extracellular vesicles (EVs) derived from stem and progenitor cells may have therapeutic effects comparable to their parental cells and are considered promising agents for the treatment of a variety of diseases. To this end, strategies must be designed to successfully translate EV research and to de...

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Bibliographic Details
Published in:International journal of molecular sciences 2017-06, Vol.18 (6), p.1190
Main Authors: Gimona, Mario, Pachler, Karin, Laner-Plamberger, Sandra, Schallmoser, Katharina, Rohde, Eva
Format: Article
Language:English
Subjects:
Animals
Biological activity
Biological products
Biological Products - isolation & purification
Biological Products - pharmacology
Biological Products - therapeutic use
Cancer therapies
Cell Fractionation - methods
Cell Fractionation - standards
Cell-Derived Microparticles
Clinical trials
Disease
Drug development
Drug dosages
Epidermolysis bullosa
Exosomes
Extracellular Vesicles
Good Manufacturing Practice
Humans
Manufacturing
Mesenchyme
Opinion
Progenitor cells
Skin
Stem cells
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Summary:Extracellular vesicles (EVs) derived from stem and progenitor cells may have therapeutic effects comparable to their parental cells and are considered promising agents for the treatment of a variety of diseases. To this end, strategies must be designed to successfully translate EV research and to develop safe and efficacious therapies, whilst taking into account the applicable regulations. Here, we discuss the requirements for manufacturing, safety, and efficacy testing of EVs along their path from the laboratory to the patient. Development of EV-therapeutics is influenced by the source cell types and the target diseases. In this article, we express our view based on our experience in manufacturing biological therapeutics for routine use or clinical testing, and focus on strategies for advancing mesenchymal stromal cell (MSC)-derived EV-based therapies. We also discuss the rationale for testing MSC-EVs in selected diseases with an unmet clinical need such as critical size bone defects, epidermolysis bullosa and spinal cord injury. While the scientific community, pharmaceutical companies and clinicians are at the point of entering into clinical trials for testing the therapeutic potential of various EV-based products, the identification of the mode of action underlying the suggested potency in each therapeutic approach remains a major challenge to the translational path.
ISSN:1422-0067
1661-6596
1422-0067
DOI:10.3390/ijms18061190