Combining best practices and analytical reference materials to tackle challenges in AAV manufacturing

The gene therapy industry is booming, as evidenced by the hundreds of AAV-based gene therapies in clinical trials and even more in development. This rapid increase creates a high demand for plasmids and other starting materials. However, manufacturing challenges and compressed development timelines, compared to traditional biologics, make scaling production difficult from early development stages. Despite these challenges, gene therapy products must still comply with existing regulations. Yet, the absence of standards and validated reference materials in this field complicates the development and implementation of methods to achieve compliance in a quality control environment. Standards-setting organizations, like the National Institute of Standards and Technology (NIST) and the United States Pharmacopeia (USP), are well-equipped to address these complex issues by developing tools to ensure the quality of AAV-based gene therapies. This article discusses the ongoing efforts by these organizations to create documentary standards and reference materials, facilitating consistent and predictable manufacturing processes for AAVs.

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