USP launches solutions package to support adeno-associated virus (AAV) manufacturing

Lack of standardization slows the development and commercialization of AAV-based gene therapies.

April 28, 2025

Rockville, MD - The U.S. Pharmacopeia (USP) has launched a package of reference standards, materials, and other resources to provide clarity for developers and manufacturers of AAV-based gene therapies.

These resources can help AAV manufacturers standardize quality assessment and control throughout the production lifecycle, from raw and starting materials to release and stability. Standardized analytical methods and appropriately qualified materials can support efficient product development and characterization.

"Innovative AAV-based therapies are approved and in development for many therapeutic categories, including previously untreatable genetic and rare diseases," said Fouad Atouf, Ph.D., Senior Vice President of Global Biologics for USP. "But manufacturers tell us that they face unique technical and regulatory challenges that complicate their path to market and to delivering value for patients with these products."

Along with physical reference materials and standards, USP will publish proposed General Chapter <1067> AAV Best Practices for the Manufacture and Quality Control of Recombinant Adeno-Associated Virus (rAAV) Gene Therapy Products. The general chapter describes best practices for the structure, manufacture, and quality control of rAAV products for in vivo gene delivery applications.

USP also recently launched a Gene Therapy Analytical Guide, which features all USP quality and safety resources for AAV gene therapy developers and manufacturers. These resources include a list of commonly used methods, as well as USP reference standards and materials that are associated with each analytical method, where applicable.

Members of the USP Biologics team will host a booth at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting to discuss these new releases and other solutions for manufacturers. ASGCT 2025 takes place May 13 - 17 in New Orleans, LA at the New Orleans Ernest N. Morial Convention Center.

USP will continue to engage with industry and regulatory stakeholders to identify challenges and develop new standards to address these areas of need.

Media contact: mediarelations@usp.org 

About US Pharmacopeia
USP is an independent scientific organization that collaborates with the world's top experts in health and science to develop quality resources and standards for medicines, dietary supplements, and food ingredients. Through our resources, standards, advocacy and education, USP helps increase the availability of quality medicines, supplements and foods for billions of people worldwide.