Quality Control Testing, Characterization and Critical Quality Attributes of Adeno‐Associated Virus Vectors Used for Human Gene Therapy

Abstract For adeno‐associated virus (AAV)‐based human gene therapy, challenges for the translation of promising research results to successful clinical development include optimization of vector design and manufacturing processes to ensure that vectors prepared for administration to human subjects have attributes consistent with safe and durable expression. This article briefly reviews quality control methods for routine testing and supplemental characterization of AAV vectors for investigational product development. The relationship of vector and manufacturing process design with product critical quality attributes is discussed.