An Overview of rAAV Vector Product Development for Gene Therapy

The drug product development pathway involves proof-of-concept efficacy studies, toxicology studies, and human clinical trials conducted in preparation for commercialization. For time and financial efficiency, a perspective of clinical use, product licensure, and commercialization is beneficial at the outset. For gene transfer products, an understanding of the unmet need for the disease target, the market and competing products, the vector configuration, patient population profile, organ target, delivery method, and time and resources involved is essential. Addressing these variables leads to interconnected strategies and plans for business and finance, regulatory affairs, product supply, R&D, clinical investigations, and commercial launch. Establishing an organization with experienced personnel who have defined responsibilities, departments that are working together but are independently responsible, a network of consultants and contract organizations, and a high level of communication is essential to ensure the quality of the product and protection of patients and give the highest chance for success.