Sascha Karassek, Charles River Laboratories

Potency testing for advanced therapy medicinal products (ATMPs) is both a guideline requirement and a challenge. Often the mechanism of action (MoA) is very complex and may not be fully understood and the reflection with in vivo fate is difficult to demonstrate. With a matrix approach, various aspects of product characteristics can be assessed. On the one hand, transcriptional, translational, and functional levels can be covered, and, on the other hand, various potential MoAs can be addressed. Brief case studies on bioactivity determination for AAV, lentivirus and a plasmid product using the matrix approach are presented.