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

  • Sarang Brahma Analytical Development (Mol Bio) Group Lead, AskBio

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

  • Megan Chartier Associate Engineer - Gene Therapy Downstream Process Development, Sarepta Therapeutics

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

  • Ya Fu APL, Spark Therapeutics

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

  • Chen Zhou Principal Scientist - Research, AbbVie

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

2:45 pm Chair’s Closing Remarks