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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doaj-8ddde2acacc14634aafe3397c2c6abba2020-11-25T00:59:55ZengMDPI AGInternational Journal of Molecular Sciences1422-00672017-06-01186119010.3390/ijms18061190ijms18061190Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical UseMario Gimona0Karin Pachler1Sandra Laner-Plamberger2Katharina Schallmoser3Eva Rohde4GMP Unit, Spinal Cord Injury and Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), 5020 Salzburg, AustriaGMP Unit, Spinal Cord Injury and Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), 5020 Salzburg, AustriaGMP Unit, Spinal Cord Injury and Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), 5020 Salzburg, AustriaGMP Unit, Spinal Cord Injury and Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), 5020 Salzburg, AustriaGMP Unit, Spinal Cord Injury and Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), 5020 Salzburg, AustriaExtracellular 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.http://www.mdpi.com/1422-0067/18/6/1190extracellular vesiclesexosomesvesicular secretome fractionmesenchymal stromal cellstherapeuticscritical size bone defectepidermolysis bullosaspinal cord injurygood manufacturing practice |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Mario Gimona Karin Pachler Sandra Laner-Plamberger Katharina Schallmoser Eva Rohde |
spellingShingle |
Mario Gimona Karin Pachler Sandra Laner-Plamberger Katharina Schallmoser Eva Rohde Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use International Journal of Molecular Sciences extracellular vesicles exosomes vesicular secretome fraction mesenchymal stromal cells therapeutics critical size bone defect epidermolysis bullosa spinal cord injury good manufacturing practice |
author_facet |
Mario Gimona Karin Pachler Sandra Laner-Plamberger Katharina Schallmoser Eva Rohde |
author_sort |
Mario Gimona |
title |
Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use |
title_short |
Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use |
title_full |
Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use |
title_fullStr |
Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use |
title_full_unstemmed |
Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use |
title_sort |
manufacturing of human extracellular vesicle-based therapeutics for clinical use |
publisher |
MDPI AG |
series |
International Journal of Molecular Sciences |
issn |
1422-0067 |
publishDate |
2017-06-01 |
description |
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. |
topic |
extracellular vesicles exosomes vesicular secretome fraction mesenchymal stromal cells therapeutics critical size bone defect epidermolysis bullosa spinal cord injury good manufacturing practice |
url |
http://www.mdpi.com/1422-0067/18/6/1190 |
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