Approaches for Addressing rAAV Immunotoxicity and Overcoming GMP Manufacturing Challenges
Adeno-associated virus (rAAV) is a key viral vector vehicle used extensively in clinical gene therapy studies. Notable clinical successes in these studies have led to regulatory approval in both the US and Europe.
Examples include Zolgensma® for treating spinal muscular atrophy and Roctavian® for hemophilia A. Success in clinical development may not be solely measured by regulatory approval, but also encompasses the knowledge gained from understanding the root cause of serious adverse events (SAEs), like rAAV immunotoxicity.
In this webinar. Dr Irene Ferreira, Head of CGT Platform Innovation & Management at Revvity Gene Delivery (formerly SIRION Biotech), together with Mathias Kahl, Head of Process Development at IDT Biologika, will discuss:
- Underlying causes of rAAV immunotoxicity, like CpG motifs, and vector design strategies to overcome it.
Critical process parameters and quality attributes to consider for designing a scalable GMP manufacturing process for clinical Phase 1 up to commercial.
A live Q&A session will follow the presentations, offering you a chance to pose questions to our experts.