Deciphering Empty-Full Characterization: A Comparative Analysis of Available Analytical Methods - Seguridad Mania.com - España y América Latina

Empty full ratio of AAV particles in a gene therapy drug is an important critical quality attribute. Due to the complexity of biological production process specifically, viral vector production mechanisms, AAV particles are quite heterogeneous. Purification process can’t entirely separate empty, partial and full particles from the process and hence a significant amount of empty and partial particles gets retained in the final product, potentially compromising quality of the product and raising safety concerns. The complexity of the challenge is understandable as the as gene therapy drugs contain multimeric protein assembly enclosing a genetic material. Where the actual process of DNA packaging remains loosely understood. There are various analytical technologies available for the characterization of these particles but almost every technology has some limitations, either in terms of the sample requirement, quality of information, precision, affordability, complexity of the assay or for their suitability in the GMP environment. AUC maintains its central role in this process and remains as a gold standard but at the same time has limited applicability across various phases of drug development due to its limited throughput and high sample requirements. There are various alternative technologies coming into play, including DLS, CDMS and Mass spectrometry, mass photometry and others. In this webinar we will discuss opportunities and challenges in various analytical technologies in characterizing this critical quality attribute of AAV vector for gene therapy.