Keynote: Analytical Challenges with Gene Editing in In vivo and Ex vivo Gene Therapy

Gene editing therapies have been in the research and development arena of many universities, research institutes and biopharmaceutical companies for over 20 years with the goal of dramatically improving the quality of life and in some cases curing a variety of common and rare genetic diseases. As more knowledge is gained, the reality of these drugs becoming viable therapeutics is creating unique challenges for many CMC functions ranging from process and analytical development, to quality and regulatory for which prior knowledge is not available and the science and technology may not be able to adequately address all of the concerns related to product quality and patient safety. In this presentation, a general review of the challenges of in-vivo and ex-vivo gene editing will be discussed followed by examination of some case studies focusing ‘real-world’ challenges encountered during the drug design, development, and manufacturing of gene editing therapies. Finally, recommendations on areas for future improvements on getting safe and effective medicines to patient in a timely manner will be discussed. These include, developing better manufacturing and measurement systems through collaborations between drug developers and equipment manufactures. And second, by creating better understanding and alignment of expectations between drug developers and regulators to ensure timely delivery of these life changing drugs to the patient who need them.