For Regulators Only - Free to Attend
The Asia-Pacific region is witnessing rapid advancements in the field of gene therapies, offering promising avenues for the treatment of various diseases. However, ensuring the safety, efficacy, and quality of these innovative therapies relies heavily on the quality of critical raw and starting materials used in their manufacturing processes.
The U.S. Pharmacopeia (USP) and Northeastern University (NEU), as an Asia-Pacific Economic Cooperation (APEC) Center of Excellence for Biotherapeutics and Advanced Therapies and Priority Work Area Champions, are hosting a training program focused on the qualification of raw and starting materials for gene therapies. This training aims to foster dialogue, collaboration, and capacity-building among regulators and stakeholders to address the evolving regulatory challenges and opportunities in this rapidly advancing field.
Day 1 - September 29, 2025
8:30 AM Opening Remarks Youngju Choi (NIFDS), Fouad Atouf (USP), Jared Auclair (NEU)
8:45 - 9:30 AM Introduction to Gene Therapies Srinivasan Kellathur, Roche
• Current trends and innovations in gene therapies
• Importance of starting materials, raw and ancillary materials in manufacturing
9:30 - 10:30 AM Session 1: Terminology and Definitions Anthony Blaszczyk, USP
• Define starting, raw, and ancillary materials per ICH guidelines
• Compare materials used in small-molecule drugs vs. advanced therapies
• Excipients, compendial grade materials, and applicable standards
• Quality considerations for starting materials including viral vectors
10:30 AM - 12:00 PM Session 2: Regulatory Expectations Jinwook Kang (NIFDS), Atsushi Nishikawa (PMDA), Ilona Reischl (EMA)
Overview of regulatory frameworks across Asia-Pacific, with perspectives from Korea, Japan, and Europe on raw material expectations for advanced therapies.
1:30 - 3:00 PM Session 3: Selection and Sourcing Srinivasan Kellathur, Roche
• Risk-based approaches to material suitability assessment
• Supplier and regulator perspectives on vendor qualifications
• Qualification requirements for human- and animal-derived materials
• Risk management tools and quality considerations
Day 2 - September 30, 2025
9:10 - 11:00 AM Session 4: Lifecycle Management Jared Auclair, Northeastern University
• Case studies on common lifecycle issues and material substitution
• Regulatory expectations for post-approval changes
• Comparability studies and change management
• Additional expectations for formulated products
12:30 - 2:30 PM Session 5: Industry Case Studies Gayatri Sharma (CellVec), Sun Jae Kwon (ENCell), Natalie Ward (Bristol Myers Squibb)
• Practical applications covering requirements for starting, raw, and ancillary materials including plasmid DNA, transfection reagents, and cell lines for virus production.
3:00 - 3:15 PM Closing Discussion
• Summary of learnings and future directions