Leveraging rAAV bioprocess understanding and next generation bioanalytics development

Recombinant adeno-associated (rAAV) vector-based gene therapy has been the focus of intense research driven by the safety profile and several recent clinical breakthroughs. As of April 2021, there are two rAAV-based gene therapies approved and more than two-hundred active clinical trials (approximately thirty in Phase III). However, the expected increase in demand for rAAV vectors still poses several challenges. Discussed herein are key aspects related to R&D needs and Chemistry, Manufacturing and Control (CMC) efforts required to attend this growing demand. Authors provide their perspective on strategic topics for rAAV-based therapies success: scalability and productivity; improved safety; increased process understanding combined with development of orthogonal bioanalytics that are able to identify, monitor and control Critical Quality Attributes (CQAs) during bioprocessing.

Automated summary

This article discusses the R&D needs and Chemistry, Manufacturing and Control (CMC) efforts required for the growing demand of recombinant adeno-associated (rAAV) vector-based gene therapy. The key aspects mentioned include scalability and productivity improvement, enhanced safety, and increased process understanding along with the development of orthogonal bioanalytics for identifying, monitoring, and controlling Critical Quality Attributes (CQAs) during bioprocessing.