USP standards to support AAV quality testing and analytical characterization of AAV content utilizing mass photometry and orthogonal techniques

Gene therapy represents one of the fastest growing biotherapeutics worldwide, with AAV at the forefront. This rapid growth is constantly challenging CMC approaches, from manufacturing through release testing. The challenges are amplified by the need for best practices and test methods that are harmonized across the field. In AAV testing, the capsid content attribute (empty-full) is one of the biggest challenges, due to the great variety of test methods and underlying physicochemical and metrological principles.

 

Reference standards are a critical component for gene therapy analytical method development, as they help ensure consistent method performance. Well characterized AAV reference standards have been difficult to reliably source. The United States Pharmacopeia (USP) has now developed gene therapy reference materials that support AAV stakeholders from raw material qualification through product release. In this webinar, Dr. Anthony Blaszczyk will discuss the first USP AAV reference materials available, which will support AAV stakeholders from raw material qualification through product release, including AAV standards for empty/full assessment, capsid titer, genomic titer, residual plasmid quantification, endonuclease activity, and plasmid topology.

 

In addition, Drs. Paul Getty and Lauren Tomlinson will discuss Pharmaron's strategy utilising mass photometry for rapid process development as well as in-process controls during large scale AAV manufacture and compare mass photometry to other orthogonal techniques used during the AAV manufacturing process.

 

Learning Objectives