10:30am Chair Introduction:

Chairperson: Chris Williams, Principal Engineer, DeSci Applied Research     

10:35am Presentation: Manufacturing considerations for novel vectors designed for enhanced expression & genetic payload capacity

• Novel vector design can be implemented for enhanced expression and payload capacity of CGT products
• However, these CGT products can be complex often requiring dual vector strategies and small molecule additives
• There is thus a need for a strategic & comprehensive CMC development plan to navigate complexities, de-risk and drive PD, formulation and analytics.
• Understanding both the Science and the Regulatory Landscape to successfully bring your CGT products to the market can be critical.

Takis Athanasopoulos,Chief Executive Officer, Gene & Cell Ltd., Vice President, Gene Therapy Research & Vector Technology, Vigeneron

10:50am Presentation: How a Configurable Platform and Data-based Process Development Tool Kit Overcomes AAV Production Challenges

• The benefits of a platform approach, and the importance of standardization across the industry 
• However, there are limitations to standardization and a “one-size-fits-all” approach is not always possible 
• Introducing the concept of “optimization by design”: intentionally building optimization into Platform processes to quickly fine tune the manufacturing platform for specific constructs, based on program/client needs and requirements 

Vera Araujo, Senior Director, Business Development, Andelyn Biosciences

11:05am Presentation: Using Stable Producer Cell Lines for the Manufacturing of Lentiviral Vectors

• Fully developed adherent cell culture bioreactor perfusion process at scale for Phase 1 studies 
• High yield purification process including sterile filtration of highly concentrated solutions 
• Optimized suspension bioreactor perfusion process for further process intensification  

Tobias Brandt, Senior Scientist, Viral Vector Process Development, CSL Behring

11:20am Presentation: How to Tackle Gene Therapy Production Bottleneck through AAV Flexible Platforms

• In this presentation, you will discover real-world examples and case studies where AAV flexible platforms have been successfully implemented to overcome production bottlenecks. 
• The Design of Experiments (DoE) approach tool was used to optimize AAV flexible platforms, enabling systematic evaluation of process parameters and formulation variables to enhance production efficiency, yield, and cost efficiency. 
• The transient transfection process for rAAV production was seamlessly scaled up from a shake flask to a 1000L scale bioreactor, using a right-first-time approach. 

Hanna P. Lesch, Chief Technology Officer, Exothera

11:35pm Closing Panel with Q&A

With all session participants joined by: Dale Stibbs, Post-Doctoral Fellow, UCL