Presentation Description: The developability of novel biological modalities require a careful consideration of risks during preclinical development. The identification of risks, mitigation strategies and candidate rank-ordering and optimization needs to occur in parallel with other developability efforts. The clinical data can be reverse translated to drive a better developability for future next generation molecules. There is a gap in communication between early discovery/developability preclinical teams and clinical teams, that leads to a high attrition of molecules at later stage. The root cause behind the attrition needs analysis and back and forth communication to improve the future generation of molecules and ensuring Quality by Design In this talk the Failure More and Effects analysis (FMEA) process provides the multi-dimensional approach of defining risk, and determining causes, effects and mitigation strategies. The product examples that will be discussed as case studies include CAR-T cell based therapy and Adeno-associated virus (AAV) expressing hemophilia factor VIII.
Describe the key touchpoints during preclinical developability where the risk assessment and mitigation strategies can be implemented
Define the process of FMEA and how to apply that during preclinical development of novel biological modality to mitigate the risk
Learn how to harness and mine the clinical data and reverse translate it to influence the early discovery and development activities
A systematic process of risk assessment across the drug development life cycle
The process of anticipating and mitigating failures