Enabling Gene Therapy In Europe: Building the Infrastructure and Ecosystem for Breakthrough Medicines

15:30 Chair Introduction

Ry Leahy, Vice President of Research & Head of Partnerships, Phacilitate

15:35 Presentation: Company Creation in Italy

• Sofinnova Partners and Telethon strategy
• The gap and the opportunity in Italy -
• Why Italy and why now
• From a Small Reality to Global Reach: The Importance of attracting talents and international investors,
• AAVantgarde, a study case of success

Tizianna Rossetti, Partner, Telethon Fund Strategy, Sofinnova Partners

15:50 Presentation: What Does Big Pharma Look for in Advanced Therapies?

• Trends, Economics (New Modalities & Big Pharma Thinking)
• Ex-US markets - if you can crack Europe
• What would it take for gene therapies to succeed?

Joshi Venugopal, General Manager & Head of Europe, Novartis Gene Therapies

16:05 Presentation: Flexibility in Gene Therapy Manufacturing: Connecting Demand Volatility to an Evolving Technology Landscape

• Recombinant adeno-associated viral vector therapies (rAAV) offer potentially durable and efficacious treatment across a wide range of devastating diseases which can have wildly different requirements in clinical and commercial application

• rAAV products also have challenges related to quality and process yield that remain a key area of focus for the industry today resulting in rapid evolution of manufacturing technologies

• Here we examine the intersection of manufacturing technologies with changes in indication, route of administration, and CMC regulatory requirements for a rAAV based manufacturing plant

Kyle Zingaro, Head of Gene Therapies Process, UCB

16:20 Panel Discussion with all session participants

Michael Baker, Senior Director, Viral Gene Therapy, FUJIFILM Diosynth Biotechnologies