8:20 am Registration & Morning Coffee
9:20 am Chairs Opening Remarks
IDENTIFYING STRATEGIC PLANNING PERSPECTIVES TO STREAMLINE GENE THERAPY COMPARABILITY STUDIES
9:30 am Exploring the Use of a Platform Approach for Gene Therapy Comparability
Synopsis
• Hearing from Ultragenyx on how the use of a platform approach to comparability could be of benefit
• Discussing the opportunities for application of a platform approach
• Highlighting the major current challenges and benefits of this approach and applications for the future
10:00 am Applying a Quality by Design Methodology Across Product Development to Establish Robust Comparability Plans and Packages
Synopsis
• Common manufacturing changes during product development that would require comparability analyses
• Structuring comparability plans to capture likely risks across all stages of product development
• Executing comparability assessments, including Analytical Comparability, to generate your Comparability Package
10:30 am Morning Refreshments & Speed Networking
Synopsis
This session is an opportunity to connect with peers working gene therapy and hold in-depth conversations with some of the brightest minds to establish meaningful and lasting relationships.
CLARIFYING REGULATORY EXPECTATIONS FOR GENE THERAPY COMPARABILITY
11:30 am Panel Discussion: Clarifying Expectations from Regulators on What Comparable Means – Virtual Session
Synopsis
• Outlining the definition of comparable from a regulators perspective and what this means for your comparability studies
• Applying advice from regulators to your own assessments
• Using this advice to gain foresight for your next comparability studies
12:15 pm Illuminating the Subtle Relationship Between Product & Process Lifecycle Validation & Comparability – Virtual Session
Synopsis
• Setting the scene via product and process validation according to the FDA
• Taking a step back to consider other fundamental elements
• Giving a concrete case with a tech transfer and a new facility in another country
12:45 pm Lunch
1:45 pm Platform Characterization Strategies to Support AAV-Based Gene Therapy Comparability Assessments
Synopsis
• Characterizing AAV protein impurities and determine accurate capsid titer
• Determine genomic integrity, purity and titer with an accurate genome size estimation
• Calculating empty and full capsid ratios with an accurate determination of partial capsids
2:15 pm Global Case Studies of Comparability from Approved Gene Therapy Products
Synopsis
• Hearing from individuals involved in gene therapy regulation from different key regions from across the world
• Discussing differences in regulatory expectations from different regulatory boards and the implications this may have on your comparability
• Understanding regulatory needs for securing approvals in multiple regions and what data you need
2:45 pm Regulatory recommendations when changing manufacturing facilities during early phase development
Synopsis
• Tips when moving your gene therapy program from an academic institution to a commercial facility
• Understanding the unique challenges of comparability study design during early phase development
2:55 pm Afternoon Refreshments & Scientific Poster Session
Synopsis
While the formal presentations of Day One come to an end, the learning doesn't stop as we enter the Scientific Poster Session. This will serve as the perfect opportunity to showcase your recent work to your peers and allow you to learn and share insights in a relaxed atmosphere.
NAVIGATING DIFFICULTIES ASSOCIATED WITH LATE PHASE COMPARABILITY STUDIES
4:00 pm Demonstrating Analytical Comparability in Analytical Development – Virtual Session
Synopsis
• Analytical comparability – Introduction and challenges with Gene therapy applications
• Evolution of assays – Phase appropriateness of methods and use in comparability
• Orthogonal characterization and statistical analysis used in the comparability