Genetic Therapy for Rare Disease

Chair

Kimberly Goodspeed, Assistant Professor in the Departments of Pediatrics, Neurology, and Psychiatry, UT Southwestern Medical Center

9am Presentation: Developing Innovative Therapies for Patients

Raj Prabhakar, Chief Business Officer, Senior Vice President, Rocket Pharmaceuticals

9.15am Fireside Chat: Reducing Manufacturing Costs through Innovation

• Reducing Raw Material Cost of Goods
• Efficiencies with Platform Scale Up & Facility Simplification

David Radspinner, Chief Executive Officer, VintaBio

9.30am Presentation: Accelerating the Advancement of Novel Gene Therapies in Rare Disease

• Advancing safe and durable gene therapies with non-viral platform technologies
• Potential advantages of liver-directed gene therapy in rare disease
• Opportunity to address pediatric and other hard-to-treat patient populations

Brent Warner, President, Gene Therapies, Poseida Therapeutics

9.45am Q&A