Genome Editing: New, Emerging, and Interesting Developments for Clinical Applications

Abstract

Principles of Genome Editing can be applied in the various areas of medical diagnosis and treatments ---from early process design/development through maintenance of the validated state during commercial manufacturing and post-marketing surveillance. Gene editing and clinical applications comprises of systematically assessing, monitoring, and reviewing manufacturing processes and subsequently monitoring measures to control output risks. Quality risk management (QRM) principles have been described in various FDA’s regulatory guidances for several aspects of good manufacturing practices (GMPs) such as several stages of process validation and verification in the drug product lifecycle including critical quality attributes (CQA’s) and monitoring critical process parameters (CPPs). A CPP is defined as a process parameter whose variability has an impact on a CQA and, therefore, should be monitored or controlled to ensure that manufacturing process produces end product of the desired quality. FDA’s mission is to facilitate the premarket review and evaluation of new genomic products for clinical use. The FDA guidances emphasize quality management approach to design of studies by providing oversight and objective review based on risk-benefit analysis and guides the use of new medical products by providing patients organized data and appropriate labeling information in support of the new product’s intended clinical use [1-3].