03:30pm Chair Introduction

Michael Lawlor, Professor, Medical College of Wisconsin

03:35pm Presentation: USP Standards for Plasmid DNA as a Starting Material for Cell and Gene Therapy

• Overview of USP general chapters development
• New chapter will address gaps in current guidance for plasmids used as a starting material
• Best practices for sourcing, qualification, and testing of plasmid DNA

Lili Belcastro, Senior Principal Scientist, Material Sciences & Engineering, Cell Therapy Development, Bristol Myers Squibb

03:50pm Presentation: Plasmid DNA Supply for AAV Gene Therapy

• Preparing for gene therapy industry success
• Critical considerations and advanced technologies for AAV plasmid production
• Evaluation and selection of a plasmid CMDO partner

Emma Bjorgum, Vice President, Client Services, DNA, Aldevron

04:05pm Presentation: Use of Pathology and Protein Expression Endpoints in Tissue to Evaluate the Efficacy of Gene Therapy

• 1. Discuss how understanding disease pathology in experimental animals and humans assists in efficiently identifying useful structural study endpoints
• 2. Explain how tissue analysis strategies can be adapted in situations where key tools (such as specific antibodies) are not available
• 3. In the context of manufacturing, how can histological or protein expression assays be used to understand the impact of process changes

Michael Lawlor, Professor, Medical College of Wisconsin

04:20pm Presentation: Designing Quality and Scalability Into Upstream Processes for Successful AAV Manufacturing

• Improving AAV yield and quality by focusing on key step of upstream process: optimizing scalability of transient transfection step
• Engineering optimized AAV plasmids to improve both infectivity and quality of viral particles
• Developing  a robust and GMP compliant, yet cost-effective AAV manufacturing process to reduce cost per dose

Roel Gordijn, Chief Commercial Officer, Polyplus

04:35pm Closing Panel with Q&A

With all session participants joined by: 

Peter Francis, Chief Medical Officer and Chief Scientific Officer, Ray Therapeutics