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

  • 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

10:30 am
DELVING INTO ANALYTICAL CHALLENGES TO OVERCOME COMPARABILITY ISSUES

11:00 am 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

11:30 am Analytical Methodology Advancements in Gene Therapy Development to Aid Comparability

Synopsis

  • Achieving seamless transition from R&D to manufacturing to maintain product quality and consistency
  • Ensuring methodological rigor for quality control
  • Regulatory engagement: FDA alignment on analytical validation

Speaker to be announced soon.

12:00 pm Lunch & Networking

1:00 pm
UNDERSTANDING THE SIGNIFICANCE OF CRITICAL QUALITY ATTRIBUTES (CQAS) IN GENE THERAPY COMPARABILITY

1:30 pm Exploring How CQAs Affect the Function and Potency of AAV Gene Therapy Drug Products

  • Chen Zhou Principal Research Scientist, 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

1:45 pm
DELVING INTO THE KEY CONSIDERATIONS FOR DESIGNING COMPARABILITY STUDIES

2:00 pm Round Table Discussions: Analyzing the Latest Update from the FDA Review on the Comparability Guidelines

  • Wendy Larson Director, CMC Regulatory Affairs, Astellas Innovation Management LLC

Synopsis

Join us for an enlightening session as we delve into the recent update from the FDA on comparability guidelines and its potential implications for the evolving landscape of gene therapy development. In this session, we will explore diverse viewpoints from experts in the field, shedding light on the key facets of the FDA’s latest guidelines and how they may impact the gene therapy industry.

Explore diverse viewpoints on the FDA’s latest comparability guidelines and their potential impact on gene therapy development

Investigate the practicality and effectiveness of the FDA’s recent guidelines in streamlining the comparability assessment process

Navigate the relationship between the guidelines and the risk assessment strategies employed by industry stakeholders

Strategize how the FDA’s perspectives on comparability will evolve over time

2:30 pm
ADVANCING ASSAY DEVELOPMENT FOR GENE THERAPY COMPARABILITY

2:45 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

3:30 pm Chair’s Closing Remarks