Abstract

Steps to Translate EV-Based Therapeutics into Clinics

Eva Rohde, Head of Department for Transfusion Medicine, Director of GMP Laboratory, Paracelsus Medical University Salzburg

Extracellular vesicles from mesenchymal stromal cells (MSC-EVs) are being investigated as novel promising biologic drug candidates. In her talk, Eva Rohde will highlight the pharmaceutical and clinical development of naïve umbilical cord-derived MSC-EVs as candidate therapeutics since about 10 years. Therapeutic concepts for selected target diseases are based on observed anti-inflammatory, anti-fibrotic, and neuroprotective biological activities of MSC-EVs in various in vitro and in vivo models. Non-clinical safety and efficacy data required for clinical evaluation include pharmacodynamic, pharmacokinetic and toxicological results for the selected routes of administration and specific indications. Pilot clinical experiments to confirm the EV-associated prevention of secondary tissue damage following acute traumatic injury or chronic (neuro)degeneration and the developmental road for first-in-human clinical trials are presented.