(294b) Leveraging Bioprocessing Fundamentals for the Development of Autologous Cell-Based Gene Therapies

In many ways, the current state of process development for cell-based gene therapies mirrors the early years of protein therapeutic bioprocessing. Similar to challenges faced when the first biologics products were emerging, gene therapy companies are tasked with understanding complex products and developing robust, scalable manufacturing processes. Many of the critical advances that have been made over the past 30 years to understand more traditional cell culture processes, as well as the structured approach of Quality by Design, can be applied to gene therapy products in development. The challenges of autologous cell-based gene therapy additionally include patient-to-patient (raw material) variability, complex supply chains, and a requirement to develop novel analytical methods. The demonstration of biochemical, biophysical and cellular comparability across multiple facilities can be challenging. This presentation will summarize the advances made in the process and product development of autologous cell-based gene therapies.