8:00 am Registration & Morning Refreshments
8:50 am Chair’s Opening Remarks
8:50 am
Successfully Scaling-up the Transfection Process for Viral Gene Therapies
9:00 am Gene Therapy Manufacturing Challenges & Scale-Up
Synopsis
- Reviewing the Pinnacle PCL Platform 2000L batch process
- Deep-dive into the challenges in manufacturing & possible solutions
- Overcoming the impacts & effects of scale-up
9:30 am Session Reserved for Mirus Bio More details to follow
10:00 am A Case Study in Commercial Site Build with Process Scale-Up Execution
Synopsis
- Discussing benefits associated with owning/building a site for future production
- Exploring the challenges associated with scale-up & process transfer
- Examining case study results & associated optimization techniques
10:30 am Morning Refreshments & Speed Networking
Synopsis
A session dedicated to taking advantage of face-to-face networking time. This an exciting opportunity to get an understanding of who else is overcoming similar challenges within the gene therapy process development space.
11:00 am
Ensuring High Quality Transgene Packing Through Selecting the Optimal Plasmid Ratio
11:30 am Achieving Optimal Complex Size Regardless of Operation Scale for Transient Transfection
Synopsis
- Exploring the impact of transfection complex size on productivity & product quality in gene therapy manufacturing
- Implementing scale-independent transfection through strategic control of complex assembly
- Enhancing process robustness through automation and process analytical tools
12:00 pm Session Reserved for Culture Biosciences
Culture Biosciences - Innovation Partner
Culture Biosciences has developed proprietary 250mL and 5L bioreactors for microbial, protein therapeutic, and viral vector cell culture applications. Our services have been used by over 75 customers and 20,000 bioreactor runs and counting. Here we describe experiments that our team performed using an AAV transfection-based process. We standardized transfection complex addition via peristaltic pump and manual syringe additions with no significant differences observed between methods. We also evaluated process robustness by increasing agitation rate which resulted in no significant difference in viral titer between both conditions. The next set of experiments will be to further define process robustness and optimization by altering additional process parameters in bioreactors.
12:15 pm Revolutionizing AAV Manufacturing – Enhancing Quality While Reducing Costs with TruStableTM Cell Line
Synopsis
- Cutting-edge Developments in TruStable Cell line Technology
- Optimizing Process: Intensification and Scaling Up for Higher Productivity
- Process automation: Manufacturing in a Box
12:45 pm Session Reserved for Form Bio – more details to follow
1:00 pm Lunch
2:00 pm Workshop: Beginning with the End in Mind: Establishing a Clear Quality Target Product Profile Prior to R&D can Prevent Roadblocks in Process Development
Synopsis
This session will act as a platform to allow attendees to re-evaluate their approach to upstream design by looking at strategies to look forward to commercialization & manufacturing prior to R&D & Upstream Development. This will allow you to gain key insights to help with your future development strategies.
Join this session to learn:
- Addressing the need for cooperation between development stages in the pre-competitive space
- Achieving clarity in the characteristics of the quality target product profile prior to development
- Doubling down on development strategies that are adaptable to the challenges provided by gene therapy
- Discussing the benefits of early readouts from the bioreactor in ensuring high quality titer