Characterization of Adeno-Associated Virus (AAV) Gene Therapy Products

Abstract

Enumerable types of gene therapies are collectively one of the fastest-growing areas of biopharmaceutical products today. Gene therapies are generally defined as treatments, perhaps even cures, for diseases through the transfer of genetic material to host cells. The analytical technologies that exist to characterize these novel therapies are rapidly advancing. In this column, we will briefly present some of the basic concepts related to this novel product class, specifically related to formulation and delivery, focusing on adeno-associated virus (AAV) formulations. We will then briefly discuss the analytical technologies and approaches most commonly used to characterize these products, specifically on the characterization of AAV-related products. We will briefly discuss what many consider the gold standard in analytical tools, analytical ultracentrifugation (AUC), and then specifically discuss liquid chromatography-mass spectrometry (LC–MS) tools and methods (such as the multi-attribute method, MAM) that are becoming more commonly used in characterization.