Pre-Conference Workshop Day
Tuesday, March 25
8:30 Registration & Light Breakfast
WORKSHOP A
9:00-12:00
WORKSHOP B
9:00-12:00
Comparative Analysis of Gene Therapy Vectors & Innovations Beyond AAV to Achieve
• An analysis of gene therapy vectors including non-AAV viruses, lipid nanoparticles, and mRNA-based approaches, highlighting advancements in safety and efficacy
• Optimizing vector delivery to non-hepatic tissues to overcome liver toxicity issues
• Examination of cutting-edge technologies such as CRISPR, RNA editing, and epigenetic modulation, focusing on their potential advantages and how they address the limitations of current vectors
Abraham Scaria, Chief Scientific Officer, Beacon Therapeutics
Advancing Gene Therapy Manufacturing by Reducing Costs, Enhancing Scalability & Ensuring Quality Control
• Understand scalability challenges and material output needs to reduce budget constraints and optimize program advancement
• Explore cost-saving strategies that lower program costs while maintaining product efficacy and safety
• Prepare for Quality Control challenges as you navigate assay development, tech transfer, specification setting, and industry development
Brad Mickey, Executive Director, Manufacturing and Operations Site Head, Astellas Innovation
12:00 Lunch
WORKSHOP C
1:00-4:00
WORKSHOP D
1:00-4:00
Designing Effective Clinical Trials for Rare Diseases: Strategies & Regulatory Considerations
• Strategies for incorporating natural history data and surrogate endpoints into clinical trial designs, and navigating regulatory requirements to gain FDA approval
• Insights into recent updates on accelerated approval processes, the role of placebo controls, and alternative approaches to support clinical development in small patient populations
• Real-world examples of successful trial designs and regulatory negotiations, focusing on how companies have addressed unique challenges in rare disease trials
Whedy Wang, SVP, Biometrics Clinical Development, Tenaya Therapeutics
Mastering Potency, Purity, & Comparability: Key Strategies for Consistent, High-Quality Gene Therapy Products
• Gain actionable insights into innovative potency assay development to enhance therapeutic effectiveness
• Learn advanced purity testing methods to maintain high standards and meet stringent regulatory requirements
• Understand best practices for comparability studies to ensure consistent product quality and facilitate smooth regulatory approvals
Jie Li, Director of Analytical Development, Vertex Pharmaceuticals