Session Details

Q&O Session 1: Qualification, Regulation of Gene Editing Tools in Cell & Gene Therapy
Thursday, May 28, 2020 11:00 AM - 12:15 PM
Quality & Operations
Recent advances in genome editing technologies have substantially improved our ability to make changes in the genomes of eukaryotic cells. Viral vectors and programmable nucleases are already revolutionizing our ability to interrogate the function of the genome. This session provides an overview of current progress in qualifying and regulating targeted genome editing technologies as they are being used clinically to correct or introduce genetic mutations to treat diseases that are refractory to traditional therapies. Preclinical assessment and GMP manufacturing of these technologies as well as the regulatory requirements for FIH clinical trials utilizing these technologies will be discussed.