8:00 am Registration & Morning Refreshments

8:50 am Chair’s Opening Remarks

8:55 am
Outlining Up & Coming Challenges to Futureproof Your Gene Therapy Process Development

9:00 am Optimization of Upstream Approaches for Improved AAV Yield in Mammalian Cell Culture Platform

  • Pranav Joshi Associate Director - Upstream Process Development, Gene Therapy Program at The University of Pennsylvania


  • Exploring new methods to increase rAAV yields and reduce costs
  • Utilizing a DOE methodology to optimise rAAV production
  • Distinguishing the optimal transgene, packaging, and helper plasmid ratios

9:30 am Advancing a Rapid, High Throughput Screening Platform & Platform Filtration for Optimization of Transient Transfection

  • David McNally Head of Vector Technology, University of Massachusetts Chan Medical School


  • Optimizing the use of high throughout methods to reduce waste and cost
  • Dig-deep into using screening platforms to reduce time to clinic
  • Examine the latest advances in filtration platforms to reduce pressure on downstream purification

10:00 am Looking into the Future: Moving Towards Non-Viral Gene Therapies

  • Steve Greenway Director Upstream Process Development, Vesigen Therapeutics


  • Overview of Vesigen Therapeutics’ ARMMS technology as a novel modality for non-viral delivery of therapeutics
  • Managing scalable upstream production of ARMMs using transient transfection or stable producer cell lines
  • Exploring the advantages of ARMMs as a delivery platform for gene therapies

10:30 am Morning Refreshments

10:45 am
Leveraging High Quality Product During Downstream Purification

11:00 am Panel Discussion: Optimizing the Path: Advances in Downstream Process Development for Gene Therapy


  • Examining empty: full characterization techniques
  • Discussing the effect of filtration techniques on the quality attributes of the quality attributes
  • Utilizing analytics to help optimize downstream process development

11:30 am Creation of a High-Yield Gene Therapy Production Platform Using DOE, & Optimization of Downstream Processing


  • Exploring the optimization of rAAV production in suspension cells, utilizing DOE methodology
  • Increasing rAAV production using cell culture additives
  • Developing optimal methods for rAAV affinity purification

12:00 pm Improving the Efficiency of Recovery During Downstream Process Development


  • Examining methods to achieving a high percentage of full capsids
  • Navigating prime methods to remove remaining aggregates from the titer
  • Exploring the most contents of partially full capsids & the most efficient way of removing them from the titer

12:30 pm Lunch

1:00 pm
Utilizing Analytical Development to Inform & Improve Processes

1:30 pm Improving Quality Assurance by Design – ELN Template Configuration


  • Documentation practices can be, and should be, intuitive and intrinsically valued.
  • Automated ELN templates bridge the gap between flexibility and control in experimentation.
  • Quality documentation at the earliest stages of development is increasingly important.

2:00 pm Optimizing AAV production and purification for gene therapy to treat cancers

  • Margaret Liu Professor at the William G. Lowrie Department of Chemical and Biomolecular Engineering, James Comprehensive Cancer Centre


  • Optimizing suspensions for AAV production process development
  • Exploring liquid chromatography based AAV purification development
  • Making the most of AAV evaluation to ensure scalability

2:30 pm The Benefits of Creating a Potency Assay Early on in Development

  • Po-Yi Ho Senior Scientist, Analytical Development, Carbon Biosciences


  • Navigating a realistic timeline for analytical development
  • Streamlining your approach for potency assay development
  • Supporting process development with analytical testing capability in early startup environment
  • Ensuring potency assays have been developed prior to IND to prevent scale-up roadblocks

3:00 pm Regulatory Expectations for Comparability in Gene Therapy

  • Scott Burger Principal & Founder, Advanced Cell & Gene Therapy


  • Overview of the July 2023 FDA Draft Guidance on Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products
  • Practical considerations for comparability studies – determining when comparability is needed, appropriate analytical methods and study design, and effective data analysis strategies
  • Recommendations for productive interactions with FDA when planning comparability

3:30 pm Chair’s Closing Remarks

3:40 pm End of Conference Day Two