Short Education Webinar Series

The Society of Toxicologic Pathology (STP) Internet Committee-Web-based Education Subcommittee (WBEC) is pleased to offer these quarterly Short Education Webinar Series as an educational tool to foster collaboration among the toxicologic pathology community. The eighth of the series will focus on Scientific and Regulatory Considerations in Safety Evaluation of Gene Therapy Products in Preclinical Studies which features four presentations and a panel discussion from a half-day Continuing Education Course at the STP 37^th Annual Symposium held in Indianapolis, IN (June 2018).

Progress in understanding the molecular bases of human health and disease in recent decades has flourished the field of Gene Therapy (GT) to offer new possibilities for treating, and even curing, a plethora of medical conditions such as monogenic disorders and metabolic diseases. GT is a therapeutic intervention to genetically alter or modify living cells by means of gene delivery achieved using either viral vectors or non-viral vectors, with the former constituting market share majority. Although gene therapy is conceptually attractive, adverse and even fatal iatrogenic complications have marred the initial enthusiasm of clinical successes. The properties of investigational viral vector-based gene therapy product (VGTP), such as their integration potentials, pose safety concerns unique from those of small molecule drugs and other macromolecule biologics. These products carry risks associated with ectopic or unregulated expression of the transgene, genotoxicity and cell transformation associated with integrative vectors, immunogenicity and inflammatory host tissue responses, long term persistence, and off-target distribution. These risks are generally evaluated in preclinical studies as part of a comprehensive preclinical safety program prior to administration in humans. However, safety assessment for these products can be challenging due to the lack of standardized approaches. A primary goal of this session is to introduce this product class to the toxicologic pathology community and provide a forum for discussion of the scientific and the regulatory considerations in the evaluation of host responses to GT products.