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

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. This article describes quality control testing and characterization of adeno‐associated virus (AAV) gene therapy vectors for clinical product development. It explores the relationship between AAV vector design and manufacturing process design with product critical quality attributes required for safety and durable efficacious expression, with emphasis on vector immunogenicity.