8:30 am
Morning Coffee

Optimizing Upstream Processes to Enhance Scalable Viral Vector Manufacturing

9:20 am Chair’s Opening Remarks

  • Jessie Sun Director, Upstream Process Development, Ultragenyx Pharmaceutical

9:30 am Developing Baculovirus Expression Vectors to Decrease Time to IND Filing

  • Shamik Sharma Director, Cell Culture Development, Voyager Therapeutics

Synopsis

  • Introducing challenges associated with baculovirus expression vector development
  • Identifying strategies to address lengthened development times
  • Uncovering proof of concept data supporting how the identified strategies can improve development time and stability

10:00 am Process Development & Scale Up Of rAVVs Processes: Opportunities for Acceleration & Cost Reduction

Synopsis

  • 400x scale up of the rAAV production process: lessons learned and best practices
  • Optimization of affinity chromatography for capsid recovery and impurity removal
  • Accelerated process development/characterization through digitization and high-throughput enablement

10:15 am Panel Discussion: Comparing the Triple Transfection Process to Viral Based Production System to Ensure Quality, Scalability & Productivity

Synopsis

  • Examining the advantages and disadvantages of triple transfection and viral based production systems
  • Navigating the production systems in early stage versus late stage development
  • How do you approach the differences in production systems in relation to systemic or localized administration?

11:00 am
Speed Networking & Morning Coffee

12:00 pm Process Development & Scale Up Of rAVVs Processes: Opportunities for Acceleration & Cost Reduction

  • Ran Li Associate Director, Packgene

Synopsis

  1.  π-Alpha 293 AAV High-yield Production Platform: Improved scalability & Biosafety
  2. π-Omega and π-Lambda Plasmid Production Platforms: Higher Yield and Quality with Lower Cost
  3. Case study will be discussed with a successful scale up of a developed process from bench-scale to clinical-scale

12:15 pm Shall We Relieve the Painful rAAV Process Development & Regulatory Concerns In Advance?

  • Xiaozhi Ren Director, Cell Line & Process Development, Vedere Bio II

Synopsis

  • Selecting an idea HEK293 suspension cell line for triple transfection while considering regulatory guidance, upstream cell culture, AND downstream purification
  • Introducing regulatory guidance preferred HEK293 suspension clone candidates that demonstrate zero aggregation under high-density cell culture and significantly low nutrient consumption
  • Demonstrating a full spectrum cell line evaluation example to meet both upstream and downstream’s demands Meeting Regulatory Expectations, On & Below

12:45 pm
Networking Lunch Break

Meeting Regulatory Expectations, On & Below the Lines

1:45 pm Establishing Product Quality Standards for AAV Vectors to Enable Smooth Gene Therapy Regulatory Filing

  • Susan D’Costa Executive VP & Global Head, Technology, Alcyone Therapeutics

Synopsis

  • Highlighting the significance of holding and understanding CQAs early on to allow comparability throughout process changes and batch
  • Outlining the landscape of regulatory expectations for viral vectors, such as contaminant levels of host protein, adventitious virus, and empty/full specifications
  • How can you best defend your product meeting critical attributes with regulatory bodies?

2:15 pm Enabling Large-scale LV vector production to Achieve Improved Process Speed and Cost

Synopsis

  • Streamlining your choice of bioreactor and upstream production platform foryour product
  • How can you ensure good scalability between the full-scale and the small-scale processes?
  • Highlighting critical process parameters and key steps in production that you should optimize to ascertain a successful LV vector production

At the end of the session, we will ask each group to share feedback and open up for further Q&As.

3:00 pm Chair’s Closing Remarks

  • Jessie Sun Director, Upstream Process Development, Ultragenyx Pharmaceutical

3:15 pm End of Summit