Eduard Ayuso: MANUFACTURING AND QUALITY CONTROL OF ADENO-ASSOCIATED VIRAL VECTORS
Location:Seminar Room 1 and 2, Children’s Medical Research Institute (CMRI), 214 Hawkesbury Rd, Westmead
Eduard Ayuso, Team Leader, Innovative Vectorology; Scientific Director Translational Vector Core (CPV), University of Nantes (Nates, France)
Seminar Title: MANUFACTURING AND QUALITY CONTROL OF ADENO-ASSOCIATED VIRAL VECTORS
Host: Leszek Lisowski
Abstract: The market approval of adeno-associated virus (AAV)-based gene therapy products in Europe and USA constitutes an evidence that the field is progressing from proof-of-concept studies toward clinical development. Concurrent with this increasing success and application, regulatory agencies have enhanced the stringency of their requirements concerning the quality and purity of these novel drugs.
Manufacturing of AAV vectors at high quantities and fulfilling current good manufacturing practices (GMP) is still a challenge, and several technological platforms are competing for this niche. Here, I will describe the most commonly used upstream (production) and downstream (purification) methods to produce AAV vectors and available options to scale up these processes for industrial applications.
The safety and efficacy profiles of viral-based drugs not only depend on the purification steps that ensure an efficient removal of product- and process-derived impurities, but also on the development of robust and precise assays for the detection and quantification of these impurities. In fact, even purified vector stocks contain impurities such as DNA, detergents, antibiotics or proteins. In this presentation, I will also discuss the most common analytical tests used for the characterization of the safety, efficacy, purity and identity of drug products as well as the development of next generation methodologies for advanced quality controls.