GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical Trial

ABSTRACT Extracellular vesicle (EV)‐enriched secretomes are emerging as a new and innovative therapeutic option in the field of regenerative medicine. The clinical use of EV‐enriched secretome‐based products requires manufacturing processes and quality control (QC) testing that comply with current g...

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Main Authors: Camille Humbert, Chloé Cordier, Iouri Drut, Michele Hamrick, Jacquelyn Wong, Valérie Bellamy, Justine Flaire, Kiranmayee Bakshy, Florent Dingli, Damarys Loew, Jérôme Larghero, Jean‐Roch Fabreguettes, Philippe Menasché, Nisa K. Renault, Guillaume Churlaud
Format: Article
Language:English
Published: Wiley 2025-08-01
Series:Journal of Extracellular Vesicles
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Online Access:https://doi.org/10.1002/jev2.70145
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author Camille Humbert
Chloé Cordier
Iouri Drut
Michele Hamrick
Jacquelyn Wong
Valérie Bellamy
Justine Flaire
Kiranmayee Bakshy
Florent Dingli
Damarys Loew
Jérôme Larghero
Jean‐Roch Fabreguettes
Philippe Menasché
Nisa K. Renault
Guillaume Churlaud
author_facet Camille Humbert
Chloé Cordier
Iouri Drut
Michele Hamrick
Jacquelyn Wong
Valérie Bellamy
Justine Flaire
Kiranmayee Bakshy
Florent Dingli
Damarys Loew
Jérôme Larghero
Jean‐Roch Fabreguettes
Philippe Menasché
Nisa K. Renault
Guillaume Churlaud
author_sort Camille Humbert
collection DOAJ
description ABSTRACT Extracellular vesicle (EV)‐enriched secretomes are emerging as a new and innovative therapeutic option in the field of regenerative medicine. The clinical use of EV‐enriched secretome‐based products requires manufacturing processes and quality control (QC) testing that comply with current good manufacturing practice (GMP). The goal of this work was to develop a robust and reproducible large‐scale GMP‐compliant process for the production of an EV‐enriched secretome derived from cardiovascular progenitor cells (CPC), including the vesiculation of CPC, purification and concentration of the product; and sterilising filtration. QC strategies for in‐process and release testing of an investigational medicinal product (IMP) were developed to guarantee quantity, safety, purity and identity. The IMP showed biological activity and was non‐immunogenic in vitro, and showed no signs of toxicity or tumour development in vivo. The IMP was approved for use in a single‐centre Phase I clinical trial by the French National Agency for Medicines and Health (ANSM) for the treatment of heart failure. The IMP is stored between –65°C and –85°C and can be easily diluted by the hospital pharmacy for infusion to the patient. This work represents a major advance for the use of CPC derived EV‐enriched secretomes as a biological drug for cardiac clinical applications. Trial Registration: ClinicalTrials.gov identifier: NCT05774509
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spelling doaj-art-a4fd52e36d75423f9850d15248b7fcd62025-08-26T07:24:44ZengWileyJournal of Extracellular Vesicles2001-30782025-08-01148n/an/a10.1002/jev2.70145GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical TrialCamille Humbert0Chloé Cordier1Iouri Drut2Michele Hamrick3Jacquelyn Wong4Valérie Bellamy5Justine Flaire6Kiranmayee Bakshy7Florent Dingli8Damarys Loew9Jérôme Larghero10Jean‐Roch Fabreguettes11Philippe Menasché12Nisa K. Renault13Guillaume Churlaud14Centre MEARY de Thérapie Cellulaire et Génique de l'Assistance Publique – Hôpitaux de Paris (AP‐HP) Direction de la Recherche Clinique et de l'Innovation (DRCI) Paris FranceCentre MEARY de Thérapie Cellulaire et Génique de l'Assistance Publique – Hôpitaux de Paris (AP‐HP) Direction de la Recherche Clinique et de l'Innovation (DRCI) Paris FranceCentre MEARY de Thérapie Cellulaire et Génique de l'Assistance Publique – Hôpitaux de Paris (AP‐HP) Direction de la Recherche Clinique et de l'Innovation (DRCI) Paris FranceFUJIFILM Cellular Dynamics, Inc. (FCDI) Madison Wisconsin USAFUJIFILM Cellular Dynamics, Inc. (FCDI) Madison Wisconsin USAParis Centre de Recherche Cardiovasculaire (PARCC) Institut National de la Santé et de la Recherche Médicale (INSERM) Université Paris Cité Paris FranceCentre MEARY de Thérapie Cellulaire et Génique de l'Assistance Publique – Hôpitaux de Paris (AP‐HP) Direction de la Recherche Clinique et de l'Innovation (DRCI) Paris FranceFUJIFILM Cellular Dynamics, Inc. (FCDI) Madison Wisconsin USAInstitut Curie Université Paris Sciences & Lettres (PSL) Centre de Recherche CurieCoreTech Spectrométrie de Masse Protéomique Paris FranceInstitut Curie Université Paris Sciences & Lettres (PSL) Centre de Recherche CurieCoreTech Spectrométrie de Masse Protéomique Paris FranceCentre MEARY de Thérapie Cellulaire et Génique de l'Assistance Publique – Hôpitaux de Paris (AP‐HP) Direction de la Recherche Clinique et de l'Innovation (DRCI) Paris FranceCentre MEARY de Thérapie Cellulaire et Génique de l'Assistance Publique – Hôpitaux de Paris (AP‐HP) Direction de la Recherche Clinique et de l'Innovation (DRCI) Paris FranceParis Centre de Recherche Cardiovasculaire (PARCC) Institut National de la Santé et de la Recherche Médicale (INSERM) Université Paris Cité Paris FranceFUJIFILM Cellular Dynamics, Inc. (FCDI) Madison Wisconsin USACentre MEARY de Thérapie Cellulaire et Génique de l'Assistance Publique – Hôpitaux de Paris (AP‐HP) Direction de la Recherche Clinique et de l'Innovation (DRCI) Paris FranceABSTRACT Extracellular vesicle (EV)‐enriched secretomes are emerging as a new and innovative therapeutic option in the field of regenerative medicine. The clinical use of EV‐enriched secretome‐based products requires manufacturing processes and quality control (QC) testing that comply with current good manufacturing practice (GMP). The goal of this work was to develop a robust and reproducible large‐scale GMP‐compliant process for the production of an EV‐enriched secretome derived from cardiovascular progenitor cells (CPC), including the vesiculation of CPC, purification and concentration of the product; and sterilising filtration. QC strategies for in‐process and release testing of an investigational medicinal product (IMP) were developed to guarantee quantity, safety, purity and identity. The IMP showed biological activity and was non‐immunogenic in vitro, and showed no signs of toxicity or tumour development in vivo. The IMP was approved for use in a single‐centre Phase I clinical trial by the French National Agency for Medicines and Health (ANSM) for the treatment of heart failure. The IMP is stored between –65°C and –85°C and can be easily diluted by the hospital pharmacy for infusion to the patient. This work represents a major advance for the use of CPC derived EV‐enriched secretomes as a biological drug for cardiac clinical applications. Trial Registration: ClinicalTrials.gov identifier: NCT05774509https://doi.org/10.1002/jev2.70145extracellular vesicles (EV)GMP manufacturinglarge‐scale process developmentquality controlsecretome
spellingShingle Camille Humbert
Chloé Cordier
Iouri Drut
Michele Hamrick
Jacquelyn Wong
Valérie Bellamy
Justine Flaire
Kiranmayee Bakshy
Florent Dingli
Damarys Loew
Jérôme Larghero
Jean‐Roch Fabreguettes
Philippe Menasché
Nisa K. Renault
Guillaume Churlaud
GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical Trial
Journal of Extracellular Vesicles
extracellular vesicles (EV)
GMP manufacturing
large‐scale process development
quality control
secretome
title GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical Trial
title_full GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical Trial
title_fullStr GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical Trial
title_full_unstemmed GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical Trial
title_short GMP‐Compliant Process for the Manufacturing of an Extracellular Vesicles‐Enriched Secretome Product Derived From Cardiovascular Progenitor Cells Suitable for a Phase I Clinical Trial
title_sort gmp compliant process for the manufacturing of an extracellular vesicles enriched secretome product derived from cardiovascular progenitor cells suitable for a phase i clinical trial
topic extracellular vesicles (EV)
GMP manufacturing
large‐scale process development
quality control
secretome
url https://doi.org/10.1002/jev2.70145
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