The gene therapy industry urgently needs standardized, reproducible methods for measuring AAV full-to-total (FTT) capsid ratios, as current interlaboratory variability of up to 25% undermines confidence in critical quality attributes, delays regulatory approvals, and increases development costs.
Initiate a project in collaboration with NIIMBL and their member organizations to conduct an interlaboratory study to investigate methods used for characterizing the distribution of intact capsids in preparations of AAV vectors used in gene therapy products.
Assess and harmonize, where possible, the use of analytical methods and platforms across industry, as a variety of technologies are currently being used to perform these analyses
By implementing standardized, harmonized analytical methods for measuring AAV full-to-total (FTT) capsid ratios across laboratories, as demonstrated in this project, an organization will reduce interlaboratory variability by up to 60% (from ≈25% SD to <10% SD), enabling consistent characterization of this critical quality attribute to improve regulatory confidence and accelerate gene therapy product approvals
Enhance the ability of developers and regulators to assess data across laboratories and organizations, ultimately reducing data variability and increasing confidence in reported values.
Lehman, S., Vreeland, W., Blaszczyk, A., Adams-Hall, S, Ahuja, S., Arnaout, A, Balduf, H., Budyak, I., Carbonell, R., Charlebois, T., Cleveland, T., Deng, J., Doyle, B., Duewer, D., Elger, C., Fagan, J., Fuo, T., Haller, J., Hilgenfeld, L., Hoang, V., Huldin, A., Hyatt, M., Jacques, J., Kar, S., Kedia, Sandeep., Kokona, B., Liu, A., Ma, L., McCarthy, D., Noble, E., Oettl, V., Pla, Andrew., Powers, T., Richardson, J., Ripple, D., Runnels, H., Ruppert, R., Semmelmann, F., Sims, C., Singh, S., Vogt, A., Wenzel, S., Whitaker, N., Yang, Z>< and Zhuang, B. (2024). Interlaboratory Measurement of Adeno-Associated Virus: Comparative Quantification of Full and Empty Capsids. Human Gene Therapy. https://doi.org/10.1089/hum.2024.124
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United States Pharmacopeial Convention
Eli Lilly and Company
Federal Stakeholder: National Institute of Standards and Technology
Genentech, Inc.
Merck Sharp & Dohme LLC
NIIMBL
Pfizer, Inc.
Sanofi
Spark Therapeutics, Inc.
