By Paul Cashen, Senior Bioprocess Specialist and Britta Manser, Manager Intensified Processing
After several successful approvals of gene therapy products in the past years, 2020 saw some clinical trials face regulatory setbacks. The reason for these rejections primarily seems to be the lack of sufficient data in the chemistry, manufacturing and controls (CMC) documentation. The interpretation and implementation of the various frameworks released for advanced therapy medicinal products (ATMPs) seems to be unclear to the industry.
This framework shows how QbD can be applied to adeno-associated virus (AAV) gene therapy products and collects initial thoughts on where data gaps or interpretation uncertainties of the regulatory framework impede the chances of regulatory approval.