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
9:30 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
10:00 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.
11:00 am
AAV Transient Transfection & Producer Cell Line Platforms to Accelerate Your Path to IND
Presented by Lonza
Synopsis
- Proprietary cell line, plasmids and process to boost AAV productivity, packaging efficiency and yield
- Meeting AAV quality requirements with improved analytics
- The next-generation AAV PCL manufacturing platform to meet high quality and demand at low cost
11:30 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
12:00 pm Optimizing Viral Amplification Efficiency: Enhancing Transfection and Packaging for Higher Yields
Synopsis
- Improving Transfection Efficiency: Explore strategies to enhance transfection methods, focusing on optimizing DNA quality, selecting the right transfection reagents, and fine-tuning cell types to maximize the efficiency of viral vector production
- Streamlining Viral Packaging: Discuss the critical role of packaging cell lines in viral vector production, and how optimizing packaging conditions can increase both yield and infectivity, addressing variability in packaging efficiency
- Scaling Amplification for Consistency: Address the challenges of scaling viral amplification from lab to production scale, including maintaining high transfection and packaging efficiency while minimizing batch-to-batch variability and optimizing vector potency
12:30 pm Lunch Break & Networking
Innovations in Downstream Processing to Improve Purification & Process Efficiency
1:30 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
2:30 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
3:00 pm Afternoon Break & Networking
Ensuring Process & Product Consistency in Viral Vector Manufacturing for Robust Development & Reliable Outcomes
4:00 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
4:30 pm Enhancing Reproducibility: Standard Operating Procedures & Quality Controls in Viral Vector Development
Synopsis
- Explore the critical role of standardized operating procedures (SOPs) in optimizing viral vector production workflows, reducing variability, and ensuring consistent outcomes across multiple development batches
- Examine best practices for quality control that integrate advanced analytical techniques and real-time monitoring, ensuring the integrity and efficacy of viral vector products throughout the development lifecycle
- Discuss how adherence to SOPs and rigorous quality controls can accelerate regulatory approval processes, ultimately paving the way for successful commercialization of viral vector-based therapies