Abstract

From Bench to GMP Production: Limitations and Pitfalls during Process Transfer of Viral Vectors for Gene Therapies

Ryan Schweller, Senior Scientist, Upstream Process Development, FUJIFILM Diosynth Biotechnologies

Cell and gene therapies continue to offer new and innovative approaches to address a myriad of conditions ranging from cancer to ultra-rare genetic disorders. In recent years, important advancements in the development of gene therapy products have been driven by the identification and understanding of new gene delivery vectors. As with most novel therapeutic platforms, the process development, manufacturing and characterization of gene therapy products present unique challenges. At FUJIFILM Diosynth Biotechnologies, we work with many clients, ranging from small biotech start-ups and academic institutions to large pharma, to develop scalable processes to support pre-clinical, clinical, and eventual commercial manufacture of viral vector-based gene therapies and vaccines. However, consistent pitfalls exist across this technology transfer process that can significantly affect project timelines. This presentation will describe the critical steps to transfer processes from the laboratory to GMP manufacturing, highlighting the key factors and often overlooked requirements that could impact successful execution of a project. We hope to initiate new discussions that could help accelerate future therapies to market.