Presented by Pallavi Karnik, Analytical lead, Cell and Gene Therapy, Intas Pharmaceuticals Ltd.
With a total of six approved rAAV-based therapies and more than 200 ongoing clinical trials, analytical characterization of rAAV-vectors is subject of intense, progressive research. Plasmids are critical raw materials for rAAV generation. Viral vectors and any starting material (including plasmids) are required to be well characterized and QC tested before release, to understand the relationship between product quality attributes, manufacturing processes, patient safety and efficacy. They must comply with quality attributes, backed up by suitably validated, precise and repeatable analytical methods. Additionally, these require extensive characterization to demonstrate product comparability and consistency. Phase appropriate analytical method development and validation is intended to provide insights on product and process characteristics required to develop a Quality Target Product profile (QTPP) and identify the Critical Quality Attributes (CQA’s). Analytical methods establish the functional relationship between CQAs and process parameters, to aid in process optimization, and ultimately assure product quality by design. Given the variety of AAV serotypes and the complexity of rAAV-based products with diverse mechanisms of action, appropriate methods are necessary to address the challenges of both biomanufacturing, e.g., transfection efficiency, removal of empty capsids and, clinical use e.g., transduction efficiency. This webinar is focused on the analytical method development strategy for viral vectors, with an emphasis on need for early method development and use of orthogonal techniques to identify CQA’s