ELRIG-Forum 2024: Abstracts

Lisa Mutz, Progen, Heidelberg

The Enzyme-Linked Immunosorbent Assay (ELISA) has a long history in the field of immunoassays. Yet, in the ever-evolving landscape of AAV gene therapy, its continued relevance is worth checking. This presentation aims to highlight the enduring significance of ELISA in the context of AAV gene therapy development.

Over the last decades, the AAV ELISA has proven to be an indispensable tool for AAV analytics since the measurement of total capsid titers is still obligatory to ensure a safe and efficient gene transfer. However, there are additional safety measures to be considered during the development process of AAV gene therapies, which can also easily be analyzed by ELISA e.g. serological analysis of pre-existing immunity. PROGEN´s AAV Titration ELISA has had a strong impact on the measurement of AAV capsid titers and demonstrated time and time again the proven strength of the method. In light of the progressing needs of the steadily evolving field of AAV gene therapy, alternative immunoassays show high potential considering its integration with and contribution to new set-ups and technologies such as Automation, Simple Plex, and the intriguing possibility of Luminescence-based methods - which will all be summarized in this presentation.

Given the progress of AAV gene therapy, this presentation shows what the future of ELISA might look like. Join us as this talk uncovers the enduring value of this "oldie" in the context of AAV gene therapy and provides insights into why it will continue to be the "goldie" in this promising field.

Stephan Kirchmaier, Promega, Walldorf

Bioluminescent tools are renowned for their versatility, sensitivity, and widespread application in research and development. Promega has accumulated decades of experience in developing effective bioluminescent technologies. These technologies seamlessly integrate biochemical, cell-based, and in vivo applications using a consistent reporter. Attendees will discover our latest advancements in T Cell therapy and AAV-based gene therapy.

Stephan Maughan, Refeyn Ltd., Oxford OX4 6FF, UK

In this talk, you will learn about two new additions to the analytical toolbox for biologics: Mass photometry and macro mass photometry. These technologies enable rapid analysis of samples in solution, using small quantities of starting materials. Both techniques analyze samples at the level of single proteins or particles, and through measurement of mass or a proxy for mass, they can identify distinct populations of analytes and provide useful insights at key stages of workflows.
Mass photometry characterizes samples of adeno-associated virus (AAV), antibodies and other biomolecules. Compatible with GMP environments for AAV analytics, it can resolve and quantify populations of empty, full, partially filled and overfilled capsids – providing empty/full measures and estimates of titer for each population. For laboratories developing therapeutic antibodies, mass photometry can give insights into antibody-antigen interactions and resolve complex stoichiometries. It can also promptly detect degradation and aggregation, as demonstrated by recent forced degradation studies. 
Macro mass photometry is a new technology that characterizes large viral vectors, such as adenovirus (AdV) and lentivirus (LVV). Ideal for informing process development in preclinical settings and early drug development, its vector analysis capabilities include the assessment of sample purity and stability.