Gene therapy practical considerations in drug development and evaluation

Gene therapy products and treatment regimens are fast developing to treat a wide variety of indications such as anticancer and genetic disorders. Unlike the small molecule where the traditional toxicology program has been well established, these new modalities require a science driven approach to develop a program that will both satisfy the regulatory needs and also to evaluate the full toxicity profile of the material. Regulatory guidelines are available and are indeed a good starting point when considering the needs for a gene therapy product. The seminar will focus on the development of gene therapy products using adeno-associated virus based (AAV) . In recent years, AAV products have achieved both clinical and regulatory approval. In this seminar, we will focus on the practical considerations for the conduct of these studies, pulling on experience gained in the conduct of these studies. We will look at the study designs, in-life conduct, and clinical and anatomic pathology endpoints.