8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
8:55 am
CHARACTERIZATION CHALLENGES IN GENE THERAPY COMPARABILITY
9:00 am Assessing Product Comparability Using Advanced Characterization Techniques
Synopsis
- Highlighting the challenge of assessing gene therapy product Critical Quality Attributes (CQA) that may appear similar but differ in potency or genome integrity
- Leveraging advanced characterization techniques like genome integrity or residual contamination analyses using multiplex ddPCR or NGS for effective product comparability studies
- Comparing empty/full/partial capsid content with genome heterogeneity for product comparability
9:30 am Enhancing Gene Therapy Characterization: Multi-Step Gradient Separation
Synopsis
- Utilizing multiple gradient steps to achieve enhanced separation in gene therapy products
- Exploring separation of partials and aggregation for comprehensive product characterization
- Investigating the impact of environmental factors on gene therapy products, including heat, cold, and packaging-induced stresses, to assess aggregation and profile changes
10:00 am Morning Break & Networking
11:00 am Combination of orthogonal methods to characterize empty to full capsid ratio for analytical comparability
Synopsis
- Addressing the challenges for the analytical comparability study of AAV-related gene therapy products
- Exploring the risk-based assessment of AAV Critical Quality Attributes (CQAs)
- Comparing orthogonal methods to characterize empty to full capsid ratio for product comparability
11:29 am
DELVING INTO ANALYTICAL CHALLENGES TO OVERCOME COMPARABILITY ISSUES
11:30 am Navigating Challenges in CMC Package Submissions
Synopsis
- Addressing the challenge of limited batch data, including the proposal to manufacture additional batches for compatibility testing
- Emphasizing the need for forward-thinking strategies in transitioning from experimental processes to commercially viable ones
- Exploring the regulatory landscape and how regulatory agencies, such as the FDA, respond to innovative approaches
- Highlighting the importance of risk assessment in justifying the analytical approach and how this can influence regulatory decision-making
12:00 pm Lunch & Networking
12:59 pm
UNDERSTANDING THE SIGNIFICANCE OF CRITICAL QUALITY ATTRIBUTES (CQAS) IN GENE THERAPY COMPARABILITY
1:00 pm Navigating the Future of Gene Therapy: Ensuring Dynamic Manufacturing and Analytical Capabilities for Robust Comparability Packages
Synopsis
- Embracing the dynamic nature of the gene therapy industry
- Recognizing that gene therapy programs are unique and must be treated on a case-by-case basis
- Strategizing how to proactively plan for years ahead to keep pace with the rapidly evolving industry
1:30 pm Exploring How CQAs Affect the Function and Potency of AAV Gene Therapy Drug Products
Synopsis
- Delving into the intricacies of specific CQAs relevant to AAV gene therapy products
- Exploring case studies demonstrating the correlation between CQAs and therapeutic outcomes
- Assessing how specific CQAs, such as viral titer, capsid integrity, and transgene expression, directly influence the functional and potency aspects of AAV gene therapy
1:55 pm
ADVANCING ASSAY DEVELOPMENT FOR GENE THERAPY COMPARABILITY
2:00 pm Mastermind Session: Strategic Assay Development and Implementation for Effective Comparability
Synopsis
- Addressing the challenges of having appropriate assays to demonstrate comparability between manufacturing and clinical batches
- The impact of process changes on the assessment and need for reliable assays during late-stage development
- Leveraging the experiences of companies and agency feedback in the development and acceptance of novel assays for gene therapy comparability