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Development of extracellular vesicle-based medicinal products: A position paper of the group “Extracellular Vesicle translatiOn to clinicaL perspectiVEs – EVOLVE France”

2021; Elsevier BV; Volume: 179; Linguagem: Inglês

10.1016/j.addr.2021.114001

1872-8294

Amanda Silva, Marie Morille, Max Piffoux, Surendar Arumugam, Phlippe Mauduit, Jérôme Larghero, Arnaud Bianchi, Kelly Aubertin, Olivier Blanc‐Brude, Danièle Noël, Émilie Velot, Célia Ravel, Céline Élie-Caille, Anna Sebbagh, Chantal M. Boulanger, Claire Wilhelm, Gabriel Rahmi, Isabelle Raymond‐Letron, Kondareddy Cherukula, Tristan Montier, Christophe Martinaud, Jean‐Marie Bach, Olivier Favre‐Bulle, Jolanda Spadavecchia, Christian Jørgensen, Philippe Menasché, Clotilde Aussel, Joël Chopineau, Mathilde Mosser, Matti Ullah, Nicolas Sailliet, Nathalie Luciani, N. Mathieu, Pierre‐Emmanuel Rautou, Sophie Brouard, Wilfrid Boireau, Sébastien Jauliac, Marianne Dedier, Jean‐Hugues Trouvin, Florence Gazeau, Marina Trouillas, Juliette Peltzer, Antoine Monsel, Sébastien Banzet,

Inhalation and Respiratory Drug Delivery

Extracellular vesicles (EV) are emergent therapeutic effectors that have reached clinical trial investigation. To translate EV-based therapeutic to clinic, the challenge is to demonstrate quality, safety, and efficacy, as required for any medicinal product. EV research translation into medicinal products is an exciting and challenging perspective. Recent papers, provide important guidance on regulatory aspects of pharmaceutical development, defining EVs for therapeutic applications and critical considerations for the development of potency tests. In addition, the ISEV Task Force on Regulatory Affairs and Clinical Use of EV-based Therapeutics as well as the Exosomes Committee from the ISCT are expected to contribute in an active way to the development of EV-based medicinal products by providing update on the scientific progress in EVs field, information to patients and expert resource network for regulatory bodies. The contribution of our work group "Extracellular Vesicle translatiOn to clinicaL perspectiVEs - EVOLVE France", created in 2020, can be positioned in complement to all these important initiatives. Based on complementary scientific, technical, and medical expertise, we provide EV-specific recommendations for manufacturing, quality control, analytics, non-clinical development, and clinical trials, according to current European legislation. We especially focus on early phase clinical trials concerning immediate needs in the field. The main contents of the investigational medicinal product dossier, marketing authorization applications, and critical guideline information are outlined for the transition from research to clinical development and ultimate market authorization.