Strengthen Total AAV Potency Assurance with NGS Sequence Analysis

Demonstration of therapeutic potency is critical for successful development and commercialization of viral-vector based products. In fact, the potency assay is one of the most critical cornerstone assays for successful clinical programs.

However, depending on the intended indication, in vivo-based potency assays may be difficult to design and implement successfully. In these instances, a total potency assurance strategy may be used to infer potency indirectly. This webinar explores how next-generation sequencing can support potency assurance using adeno-associated virus as a model.

Total potency assurance assays focus on the Critical Quality Attributes (CQA’s) for the specific type of molecule being assessed and combine multiple assays which serve different functions. For example, a total potency assurance package may include assays for particle assessment, nucleic acid assessment, functional titer and many other applications.

The featured speaker will introduce how a Next Generation Sequencing (NGS) based approach can be integrated into a total potency assurance package using AAV as a model. The speaker will demonstrate NGS’s ability to serve as a sequence purity assay and how quantitation of sequence variants, or vector sub-populations, can be achieved with suitable precision and accuracy. Finally, the webinar will present data to support the validation of such an assay as a quantitative assessment suitable for phased trials and commercial release in accordance with parameters consistent with ICH Q2(R2), and an approach to product-specific validation which has been accepted within the context of regulatory filings.

Register for this webinar to learn how potency assurance can incorporate NGS-based sequence purity testing to support AAV therapy development, validation and commercial release.

Speaker

Rebecca Bova, PhD, Senior Scientist, Head of CNAT (Custom NGS Assays Team), MilliporeSigma

Dr. Rebecca Bova is a Senior Scientist with the Next Generation Sequencing group at MilliporeSigma. In her current role, Rebecca leads a custom assay team which develops NGS-based assays for testing multiple attributes for biologically derived products. She also provides technical support to clients around our broad spectrum of NGS offerings, including identity testing, AAT and gene editing testing. Rebecca joined the organization in 2021 from the Uniformed Services University of the Health Sciences, where she performed her postdoctoral research. Rebecca earned her MS and PhD degrees in Molecular and Cell Biology from the University of Connecticut.

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