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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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