Regulatory and CMC Alignment for Gene Therapy Manufacturing

Chair

Khandan Baradaran, Vice President, Regulatory CMC, Ultragenyx

4pm Presentation Title: Navigating the Manufacturing and Associated Regulatory Challenges for Advancing Gene Therapies

• Emphasizing the need for forward-thinking strategies in transitioning from experimental processes to commercially viable ones.
• Exploring the regulatory landscape, addressing the challenge of limited historical data, and comparability of manufacturing batches.
• Highlighting the importance of risk assessment in justifying the analytical approach and how this can influence decision-making.

Samarendra Mohanty, Chief Scientific Officer, NanoScope Therapeutics

4.15pm Presentation Title: How to Expedite your Gene Therapy Program: Platform Manufacturing Approaches.

• Ensure alignment with phase-appropriate plasmid DNA, AAV, and LVV manufacturing best practices
• Adopt an established platform approach for speed and predictability
• Jump-start production with off-the-shelf products

Ramin Baghirzade, Senior Director, Gene Therapy, Charles River Laboratories

4.30pm Presentation: How to Improve the Accessibility of Genetic Medicines through Better Manufacturing

• How to overcome critical manufacturing challenges when scaling up and out
• Process improvements for higher yield and better quality
• Molecular improvements for enhanced productivity and efficacy
• Enhanced analytics for better understanding of CQAs and CPPs

John Maslowski, Chief Commercial Officer, Forge Biologics

4.45pm Q&A