Abstract

Cost-Effective GMP Production of Extracellular Vesicles

Marcin Jurga, Chief Scientific Officer, Exo Biologics

The therapeutic effect of Extracellular Vesicles (EVs) has been demonstrated in numerous non-clinical studies. The efficacy and safety of EVs have been proven in different animal models including treatment of Bronchopulmonary Dysplasia (BPD), among other indications. Currently, the most advanced projects are being translated into the clinic to confirm the safety and efficacy of EVs in humans. This requires pharmaceutical grade EVs, manufactured to be compliant with Good Manufacturing Practice (GMP). In the emerging EV field, there are various methods and instruments qualified for the production of GMP-grade EVs and most of the manufacturers use their own proprietary techniques. It is important for a biotech company to develop a robust and cost-effective process, capable of producing EVs on different scales, starting from low-volume, which can be used in the non-clinical phase, and moving on to a large-scale production for clinical trials and ultimately commercial drug manufacture. Considering that EVs as biological drug products are defined by their production process, any major changes in the manufacturing method can have an impact on the drug product identity and activity. EXO Biologics has developed a GMP-approved custom platform for EV manufacturing, based on a closed and scalable bioreactor system. The production process is flexible and allows for a manual production with a low-volume bioreactor during the early phases of drug development. Subsequently, the process can be scaled up to a fully automated large-volume system for commercial manufacturing, without changing the characteristics of the product. The platform can be used for production of multiple EV types, including the 1st generation of non-modified EVs, which are currently being manufactured for clinical tests in BPD, and also engineered EVs designed for enhanced activity and drug delivery.