8:00 am Check-In & Coffee
8:55 am Chair’s Opening Remarks
Uncovering Scalability & Commercialization of Viral Vector Production While Achieving Higher Yields for Enhanced Market Access
9:00 am Round Table Discussion: Bridging Perspectives on Collaborating for Scalable Solutions in Viral Vector Manufacturing
Synopsis
- Capacity Constraints in Bioreactors: Discuss the limitations in operating bioreactors beyond 500 liters and the impact on production scalability
- Inefficiencies in Current Production Strategies: Examine the challenges and costs associated with aggregating multiple smaller runs, highlighting the need for more effective manufacturing solutions
- Innovative Solutions for Tech Transfer: Explore best practices for technology transfer and collaborative approaches between service providers and manufacturers to enhance efficiency and overcome logistical hurdles in scaling up production
10:00 am Enhancing Reproducibility: Standard Operating Procedures & Quality Controls in Viral Vector Development
Synopsis
- Optimize AAV Production with Quality Control – Explore the implementation of cutting-edge analytical techniques to enhance consistency and reproducibility.
- Enhance Viral Vector Quality with Precision Analytics – Dive into full/empty capsid analysis and DNA integrity assessment to ensure potency and safety.
- Shape the Future of AAV Standards – Discover how collaboration between academia, industry, and regulators is driving the development of global quality control frameworks
10:30 am Morning Break & Networking
11:30 am Process intensification of rAAV purification with column-free BioRMB platform
Synopsis
- Introduction to BioRMB – a real moving bed column-free chromatography platform with fully closed single-use flowpath
- AAV purification data with multiple serotypes featuring significant improvements in productivity (10-20X)
- Discussion on mechanisms of action vs status quo column purification that lead to more robust product quality and higher recoveries
11:40 am The Trend of Plasmid-based AAV Manufacturing in HEK293 Cells
Synopsis
- Current challenges in plasmid-based AAV manufacturing platforms
- Technical innovations that improve rAAV titer and purity with reduced cost
- Potential future directions towards off-the-shelf AAV manufacturing
12:10 pm Using NanoFlow Cytometry to Quantify Virus Particles in Complex Solutions
Synopsis
- Quantify and characterize the virus particles in your formulations with the NanoAnalyzer by NanoFCM
- Thousands of individual virus are counted, sized, and interrogated for their cargo even in complex solutions
- Detecting your virus as single particles can enhance your process development efforts by offering an in depth, quantitative picture of the state of the virus before and after process operations
- Requiring very little sample and providing flexibility to measure many different virus attributes the NanoAnalyzer can make a perfect partner for all of virus process development projects
12:25 pm Lunch Break & Networking
Leveraging Advanced Viral Vector Platform Technologies & New Therapeutic Modalities to Reduce Time-To-Market & Delivering More Effective Treatments
1:25 pm Novel Approach to Treating Rare Disease Using an AAV Vector to Deliver Gene Editing Tools to the Brain
Synopsis
- Provide overview of the development of LETI-101, a preclinical stage AAV5-delivered Life Edit® nuclease and guide RNA for mutant allele-specific editing in Huntington’s Disease
- Share approach for assessing manufacturability of LETI-101 through linking preclinical and CMC efforts
- Discuss the importance of biotech “owning” the future CMC problems at an early stage of pharmaceutical development to de-risk clinical trials and commercialization
1:55 pm Building a Plasmid Toolbox to Improve rAAV Productivity and Product Quality in Transient Transfection Platform
Synopsis
- Suspension transient transfection remains the widely used production platform in the industry. Plasmids and host cell are critical biological raw materials in this process.
- Plasmid design optimization and quality attributes can directly impact the productivity and product quality of rAAV.
- Developing plasmid design strategies that can support a variety of capsids and cargos is critical to accelerate process development timeline.
2:25 pm Afternoon Break & Networking
3:00 pm Unlocking Potential: The Promise of Transient Transfection in Viral Vector Production
Synopsis
- Transient transfection enables rapid generation of viral vectors, eliminating the lengthy stable cell line development process and facilitating quicker access to early-stage research and clinical trials
- This method allows for easy modifications and testing of various vector constructs, empowering researchers to tailor vectors for specific applications and enhance performance through iterative design
- With the potential for significant viral yields and reduced production costs, transient transfection offers an efficient alternative for both small-scale and large-scale production of viral vectors, addressing the growing demand in gene and cell therapies
3:30 pm Overcoming Downstream Impurity Challenges in Lentiviral Manufacturing: A Point-of-Use Approach
Synopsis
- Sharing insights from the point of use on common impurities faced during lentiviral vector production, such as residual host cell proteins, DNA, and product-related contaminants, and their practical impact on downstream processes
- Highlighting effective, on-the-ground strategies for dealing with impurities, from optimizing filtration methods to implementing process control techniques that ensure the purity of lentiviral vectors in real-time
- Discussing the challenges and solutions for ensuring impurity control and product consistency during scale-up, with a focus on practical techniques and decisions made in the lab or manufacturing environment to meet regulatory requirements