Innovative Delivery Technology for Gene and Cell Therapy

19 Jan 2023
New & Enabling Technologies Track
ViroCell Biologics

11:00am Chair Introduction

Vincent Ling, Head of Search and Evaluation for Drug Delivery Technologies, Takeda


11:05am Presentation: Enabling the Full Potential of Gene Therapy

  • The importance of integrating the drug delivery strategy and the drug development strategy in the earliest stages of development
  • Opportunities and challenges at the interface between delivery technology development and drug development
  • Is there an opportunity for greater pre-competitive collaboration to strengthen the ecosystem for innovative delivery of gene and cell therapy and accelerate innovation?

Pierre Boulas, Head of Medical Devices and Packaging Development, Biogen


11:20am Presentation: How Do We Produce A Healthy and Highly-Functioning Gene-Edited Immune Cell?

  • Solupore is a non-viral transfection process that enables complex, multi-step editing for next-generation cell therapy products
  • Primary cells edited using Solupore® retain high levels of stem-cell memory cells with minimal apoptosis
  • Following editing with Solupore®, cells demonstrate enhanced functional response and cytotoxicity compared with existing transfection technologies

Justin McCue, Chief Technical Officer, Avectas


11:35am Presentation: Acceleration of Vector Manufacturing for Early-Phase Clinical Testing of CAR-T Cell Therapies

  • A rapid inexpensive vector manufacturing process would enable faster early-phase clinical testing of CAR T cell therapies.
  • High-speed centrifugation efficiently purifies γ-retroviral vectors encoding multicistronic expression cassettes produced by transient transfection at a scale sufficient for phase I CAR T cell studies.
  • This simple downstream process combines high recovery, concentrated viral titres and good impurity clearances; and can be bridged to stable producer cell lines for subsequent larger confirmatory clinical studies

Leila Mekkaoui, Principal Scientist, Autolus


11:50am Presentation: Intelligent Programmable Vectors for New Horizons in Gene Therapy 

  • Role of specificity and targeting in the safety of lenti and AAV
  • Improving vector titre to decrease costs
  • Programmable vectors for enhanced safety and efficacy

Farzin Farzaneh, Chief Scientific Officer, ViroCell Biologics


12:05pm Closing Panel with Q&A

With all session participants


Farzin Farzaneh
Chief Scientific Officer
ViroCell Biologics
Justin McCue
Chief Technical Officer
Leila Mekkaoui
Associate Director Synthetic Genomics
Pierre Boulas
Head of Medical Devices and Packaging Development
Vincent Ling
Senior Director