Characterizing the Stability of Gene Therapy Products at Ultra Low Volume

Gene therapies are no longer an “emerging market,” nor is it just a buzzword for biological therapy developers as new novel therapies are coming online every day. Getting to market first and safely beating the competition is more critical than ever. Characterizing gene therapy candidates at early-stage development is the ideal scenario but has been cost-prohibitive due to the high-volume requirements of antiquated biophysical characterization techniques—one mL of sample costs thousands of dollars to produce. Early assessment for critical quality attributes (CQA) such as drug stability, subvisible product aggregation, and inherent impurities ensures drugs are developed with the foresight to prevent potential immunogenic responses.

During this GEN webinar, our distinguished guest Dr. Bernardo Cordovez will tell us more about an instrument—Aura^® GT—that his company created to characterize gene therapies for stability and CQAs using a minuscule amount of sample. With this novel platform, drug products and particles can be quantitated for size and count, imaged by microscopy for morphological features, and identified via labeled fluorescence by fluorescence membrane microscopy, using a fraction of the sample requirement compared to other methods. Dr. Cordovez will also discuss the technique of fluorescently labeling DNA payloads to understand its impact on subvisible aggregate formation for AAV product stability and shelf life. As a result, gene therapy developers finally have the cutting-edge tools required to develop advanced science and medicine.

A live Q&A followed the presentation, offering a chance to pose questions to our expert panelist.