Non-Viral Solutions for Gene Therapy

Chair

Adam Pfieffer, Vice President of Strategy, Project Farma

10am Presentation: Non-viral Platform Innovations in Pursuit of Functional Cures

• Non-viral approach enables delivery of larger genes
• Advantages in tolerability, potency, speed to clinic and costs
• Non-viral gene insertion, delivery and whole gene integration are key to unlock patient potential

Brent Warner, President, Gene Therapy, Poseida Therapeutics

10.15am Presentation: How to retain superior cell health and function following multiple gene-modifications for a cell therapy modalities.

• Solupore is an automated, non-viral transfection process that enables complex, multi-step editing for next-generation cell therapy products.
• Following editing with Solupore, cells demonstrate enhanced in vitro and in vivo functional response and cytotoxicity compared with existing editing technologies.
• Solupore can deliver complex cargos to a variety of cell types, including T cells and iPSCs

Lisa O’Flynn, Sr. Director, Process and Analytical Development, Avectas

10.30am Presentation: Lipid Nanoparticles As A Delivery System For Mrna Therapeutics For The Treatment Of Rare Genetic Disorders

• Lipid Nanoparticles composition and formulation for targeted delivery
• Route of administration of LNPs
• Examples in the clinic for mRNA encapsulated in LNPs

Paolo Martini, Chief Scientific Officer, International Therapeutics Research Centers, Moderna

10.45am Presentation Title: Non-Viral Manufacturing of Gene Modified CAR T Therapies Using TcBuster™

• Learn how gene modified cell therapies can be manufactured at scale using the TcBuster transposon system, specialty media, and liquid cytokines
• Untouched CAR T cell expansion utilizing the G-Rex® 100M bioreactor
• Utilization of Simple Plex™ assays for CAR T cell functional assessment on Ella™, an automated ELISA platform

David Hermanson, Director of Cell and Gene Therapy Applications, Bio-Techne