8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
9:00 am Panel Discussion: Setting the Stage: Understanding the Evolving Landscape of Viral Vector Process Development
Synopsis
- Delving into an overview of industry growth and the latest advancements shaping process development
- Addressing the hurdles in balancing quality, cost-efficiency, and regulatory demands as companies move from lab to commercial-scale production
- Exploring emerging technologies, including automation, PAT, and AI-driven analytics, that are redefining the future of viral vector production
Driving Down Upstream Process Development Costs Through Improved Viral Vector Quality & Yield Optimization
9:45 am Optimizing Viral Vector Production: Balancing High Titers and Maintaining Potency
Synopsis
- Maximizing Viral Yields Without Sacrificing Quality: Strategies for increasing vector production, such as cell line engineering and process optimization, while minimizing the formation of defective particles
- Controlling Capsid Ratios for Consistency: Ensuring the proper empty-to-full capsid ratio to maintain viral vector potency and reduce risks related to immunogenicity and safety
- Addressing Impurities and Vector Purity: Implementing advanced purification techniques to remove unwanted impurities that arise from high-titer production, ensuring high-quality vectors suitable for therapeutic applications
10:15 am The Rise of Modular Reagent Manufacturing — Scalable Solutions to Accelerate Novel Gene Therapy Development
Synopsis
Novel gene therapy development is focused on increasingly individualized and diverse solutions, requiring smaller, more customized batches of reagents and buffers to be used across the clinical pipeline as companies scale from research to process development and into commercialization. Existing bioprocessing infrastructure was not designed to manufacture these new therapies, forcing the industry to look for research and clinical-grade reagent formulations that are easily customizable, scalable, and efficiently produced. Find out more about what criteria novel therapy developers should be considering when evaluating reagent solutions.
10:45 am Morning Break & Speed Networking
Synopsis
As this community re-unites for the third time, this session will provide valuable networking time with your peers, enabling you to forge new and lasting connections.
11:45 am Technological Advances in Gene Therapy: Reducing Uncertainty in Dosing with dPCR
Synopsis
- Transition from qPCR to dPCR: Discuss how digital PCR (dPCR) has improved accuracy in measuring viral genome titers
- Dose Precision Improvements: Highlight the reduction in uncertainty from 50% to less than 15% using dPCR, allowing for more reliable dosing
- Impact on Gene Therapy Products: Explore how this innovation enhances understanding and quality control in gene therapy development
12:15 pm Industrialization of AAV Manufacturing by Xcite® Transient and Stable Production Platforms
Synopsis
- Overview of Lonza’s Xcite® AAV transient transfection and stable producer cell line (PCL) platforms
- Case study of AAV production by the transient transfection platform to meet your targets
- Case study of the development of AAV stable PCL with superior productivity
12:45 pm Lunch Break & Networking
Innovations in Downstream Processing to Improve Purification & Process Efficiency
1:45 pm Round Table Discussion: Enhancing Efficacy through Reduced Immunogenicity with Innovative Capsid Designs
Synopsis
- Capsid Engineering Techniques: Cutting-edge methodologies, such as rational design and directed evolution, enable strategic modifications to AAV capsids for improved immune evasion and targeting
- Reduced Immunogenicity: Tailored capsid modifications can significantly diminish immunogenic epitope presentation, reducing T and B cell activation and enhancing the efficacy of AAV vectors in gene therapy
- Improved Delivery Efficiency: Engineered variants like PHP.B/eB AAV not only lower immunogenicity but also enhance delivery across the blood-brain barrier, crucial for effective treatments of neurological conditions
2:15 pm Overcoming Impurity Challenges Downstream in Viral Vector Production: Advances in Purification & Analytical Techniques
Synopsis
- Discuss the challenges associated with removing empty capsids and other impurities in viral vector production, emphasizing their impact on therapeutic efficacy
- Explore advancements in purification methods, moving away from traditional ultracentrifugation to more efficient techniques that support high-purity vector production at scale
- Examine the development of high-throughput, sensitive assays for deep characterization of viral vectors, focusing on critical quality attributes (CQAs) such as genome integrity, potency, and the identification of various impurities, particularly during scale-up processes
2:45 pm Afternoon Break & Poster Session
Ensuring Process & Product Consistency in Viral Vector Manufacturing for Robust Development & Reliable Outcomes
3:45 pm Ensuring Cell Line Stability for Consistent Lentiviral Vector Production Genetic Engineering for Stability
Synopsis
- Discuss techniques for stabilizing producer cell lines, including stable transfection and selection methods, to ensure consistent vector yield and quality over extended production cycles
- Minimizing Genetic Drift: Explore strategies to reduce genetic variation and loss of transgene expression over time, which can lead to inconsistent LVV production and reduced quality
- Long-Term Culture Conditions: Highlight the importance of optimizing culture conditions (e.g., media, cell density) and process monitoring to maintain cell line performance and stability during large-scale, long-term production
4:15 pm The Use of a Suspension Packaging Cell Line for Lentiviral Vector Production and Generation of Stable Producer Cell Lines
Synopsis
- Strategies employed to select clones with the best productivity and scalability profile
- Process intensification: A simplified perfusion process for a shorter seed train
- Example of a robust end-to-end biomanufacturing process for a lentiviral vector expressing a CAR