Downstream processing 2024

Displacement chromatography for enrichment of rAAV genome-containing capsids using weak organic acid

Cell & Gene Therapy Insights 2024; 10(9), 1317–1335

DOI: 10.18609/cgti.2024.150

Published: 24 October
Research Article
Tamara Zekovic, Paul Greback-Clarke, Eric Vorst, Eva Graham, Jordan Hobbs, Robert Tikkanen, Hunter Reese, Amith Naik, Rashmi Bhangale, Carlos Cruz-Teran, Christian Denis, Mayur Jain, Thomas Guarinoni, César Trigueros Fernandez, Jacob Smith, David Knop, Joshua Grieger

Separation of empty and genome-containing particles is a challenging requirement for recombinant adeno-associated virus gene therapy vectors. Strong anion exchange chromatography operated with a linear salt concentration gradient to achieve a high degree of resolution has emerged as the replacement for traditional density-gradient ultracentrifugation to improve efficiency, consistency, and scalability. Herein, an innovative mechanism of anion exchange chromatography using weak organic acid present in the load is shown to selectively displace empty capsids into the flowthrough, enabling an isocratic elution and eliminating operational challenges associated with linear gradients. This separation technique was applied to AAV2 and AAV8 serotypes using three chromatographic media (monoliths, resin, and membranes) and showed comparable genome-containing capsid enrichment levels to that of density gradient ultracentrifugation. Processes for both serotypes were successfully transferred from lab to production scale.