Development and validation of a model gene therapy biodistribution assay for AVGN7 using digital droplet polymerase chain reaction

Development and validation of a model gene therapy biodistribution assay for AVGN7 using digital droplet polymerase chain reaction

Smithers Pharmaceutical Development Services experts Agostinho G. Rocha, Ph.D. and Joyce Chen recently published a scholarly article in Molecular Therapy Methods of Clinical Development discussing the development and validation of a model gene therapy biodistribution assay for AVGN7 using digital droplet polymerase chain reaction.

Biodistribution assessments are required components of investigational and final drug or biological approval applications with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Japan Pharmaceutical and Medical Devices Agency (PMDA), and other regulatory authorities. 

Described herein is the development, optimization, and validation of a ddPCR assay for quantifying AVGN7 vector genomes in tissue extracts and whole blood. Our report follows the white paper published by the Global CRO Council in Bioanalysis (GCC) and outlines the critical processes within a typical commercial timeline. It describes assay performance, acceptance criteria, and metrics expected from several regulatory authorities. It also addresses challenges to the technology and serves as a model for guiding development of other gene therapy programs.

Download the article below: 
Development-and-validation-of-a-model-gene-therapy-biodistribution-assay-Smithers-2023

Rodgers, B. D., Herring, S. K., Carias, D. R., Chen, J., Rocha, A. G. (2023, June 8). Development and validation of a model gene therapy biodistribution assay for AVGN7 using digital droplet polymerase chain reaction. Molecular Therapy - Methods & Clinical Development.

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