Gene therapy and capillary UHPLC-MS
Date: Friday, September 24, 2021
Time: 03:00 PM Central European Summer Time
Duration: 1 hour
The field of gene therapy is rapidly growing and introduces new challenges in the analytical characterization. Monoclonal antibodies are recognized as large molecules, but they are indeed dwarfed in comparison to the size of an intact viral particle. The full viral particle will contain an oligonucleotide payload which will also need to be characterized in addition to the protein capsid, bringing in additional analytical requirements.
A further challenge to the analytical chemist is the reduced concentration of the sample in comparison to the mg/ml levels of a monoclonal antibody in a therapeutic drug formulation. These problems and more will be addressed, showing the advances that have already been made and the future possibilities in a range of analytical characterization techniques used in gene therapy product analysis. Full and Empty ratio analysis, titer analysis, component characterization, peptide mapping for PTM analysis with sequence confirmation and oligonucleotide analysis will be addressed, including UHPLC and LCMS possibilities. The implementation of an easy to use capillary flow platform to increase sensitivity is introduced.
Key learning points;
· UHPLC based characterization techniques for adeno associated viruses
· HRMS based characterization techniques for adeno associated viruses
· Techniques for dealing with low concentration samples