Platform Solutions to Enhance the Safety, Efficacy, and Scalability of Gene Edited Cell Therapies

Since the discovery and description of CRISPR based gene-editing, there has been an explosion in pre-clinical and clinical stage programs focused on the development of gene-edited cell therapies.2023 will likely witness the first ever FDA approval and commercial launch of a CRISPR gene-edited cell therapy. However, there remain many outstanding questions about the design space, manufacturing, and analytical requirements for developing safe and effective CRISPR gene-edited cell therapies. This brief talk will review several novel platform technologies, including high-fidelity Cas9 enzyme, nanoplasmid based HDR donor template, HDR enhancers, and rhAmpSeq for analytical characterization of on-target and off-target edits; this talk will also discuss real-world case studies highlighting the role of these platform technologies in enabling the development of safer and more effective CRISPR gene-edited cell therapies.