Registration & Morning Refreshments
BIOASSAYS
9.00 – 12.00
Using a Matrix of In Vitro & In Vivo Potency Assays to Determine Potency as a Function of Capsid Content
Synopsis
• Explore the different types of capsids produced during AAV vector manufacturing, including full, intermediate, and empty capsids
• Review methodologies for determining the potency of AAV products in relation to capsid content, including in vitro and in vivo assays for infectivity, gene expression, and biological activity
• Determine the relative contribution of genome fragment populations to specific potency outcomes to better relate product quality to clinical efficacy
• Discuss the challenges and considerations involved in designing and executing potency assays for capsid populations
• Examine the findings of primary studies assessing the contribution of intermediate capsids to AAV product potency
PHYSICOCHEMICAL PROPERTIES
9.00 – 12.00
Advancements in Residual DNA Detection & Quantification Methods in Gene Therapy
Synopsis
• Discuss the relative advantages and disadvantages of specialized host cell specific DNA kits for residual DNA detection
• Examine the growing adoption of digital PCR for residual DNA testing and its benefits over traditional qPCR methods in overcoming the need for standard curves
• Analyze the implications of FDA regulations regarding a 200-base pair limit on fragment length in residual DNA analysis
• Discuss the advantages and disadvantages of long and short-read sequencing methods to provide qualitative insight alongside quantitative assessments of host cell DNA presence
• Explore strategies for ensuring compliance with evolving regulatory standards while optimizing detection sensitivity and accuracy
PROCESS DEVELOPMENT & CMC
9.00 – 12.00
Using In-Process Analytics to Monitor Emerging CQAs & Guide Upstream Process Development
Synopsis
• Determine what kind of in-process analysis is suitable and sufficient for real-time monitoring during manufacturing and effective process development
• Discuss relevant CQAs to incorporate into your in-process analytical strategy
• Understand to what extent it is beneficial to measure novel CQAs such as empty-full ratios compared to traditional parameters such as genome titer and host-cell DNA
• Develop efficient in-process analytics for efficient chromatographic purification
• Brainstorm the most important point of measurement to determine whether a process is the best by balancing the cost, quality, and timeline
• Discuss how in-process analytics can be used to assess vector stabilization strategies
12.00 Lunch & Networking
1.00 – 4.00
Developing Appropriate Reference Standards for Effective Determination of Relative Potency
Synopsis
• Introduce the importance of reference standards in developing an overall potency strategy
• Develop understanding of lot selection processes for reference material used in potency assays
• Brainstorm strategies for maintaining consistency in potency assays when changing reference standards or manufacturing processes
• Utilize reference standards for comparative analysis to determine relative potency and assay performance
1.00 – 4.00
Utilizing Orthologous Methods for Partial Particle Characterization & Determination of Vector Genome Integrity
Synopsis
In the wake of evolving regulatory demands, the characterization of capsid content demands increased scrutiny. This workshop to address cutting-edge analytical strategies for characterizing partial capsid content and will consider existing methodologies, confront their limitations, and introduce novel techniques like mass photometry.
This workshop will aim to:
• Discuss the intricacies of AAV manufacturing and the significance of monitoring partial particles as impurities
• Explore existing methodologies like ELISA, HPLC, analytical ultracentrifugation (AUC), multiplex ddPCR, Capillary Electrophoresis (CE), and charge-detection mass spectrometry (CDMS), LC-MS and NGS for quantifying partial particles
• Highlight the limitations of current techniques in terms of turnaround time, sample throughout, and data analysis complexity
• Explore the applicability of novel technologies such as mass photometry across multiple AAV serotypes without the need for extensive method parameter adjustments
1.00 – 4.00
Deciphering Empty-Full Characterization: A Comparative Analysis of Available Analytical Methods
Synopsis
Accurately characterizing empty and full viral particles is essential for ensuring product quality and safety. Analytical Ultracentrifugation (AUC) has long been the gold standard for this purpose, but recent advancements have introduced alternative techniques. This workshop will delve into the comparative analysis of various analytical methods, evaluating their overall efficacy in empty-full beyond traditional AUC.
This workshop will aim to:
• Provide an in-depth examination of analytical methods commonly used for empty-full characterization such as DLS, CDMS, Mass Photometry and others
• Weigh up the analytical methods available for empty-full characterization – how do they compare against AUC?
• Explore case studies and real-world examples highlighting the application of each technique
Pre-Conference Day, Workshop F
Deciphering Empty-Full Characterization: A Comparative Analysis of Available Analytical Methods
Speaker
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steven berkowitz
consultant - Independent