Analytical Solutions for AAV Vector Characterization

Fast and reproducible AAV titer measurement is essential for ensuring safety and efficacy of your gene therapy product.

In partnership with PROGEN, we have automated the AAV titration ELISA, delivering rapid and efficient viral quantitation with superior specificity. These assays eliminate the need for time-consuming manual steps, such as standard curve preparation, so you can easily monitor AAV physical titer throughout development.

With potency being one of the most important CQAs for gene therapy analysis, it is crucial to have a robust assay that provides highly reproducible results.

Traditional methods, such as ELISAs, Western blots and flow cytometry, have limitations, including limited quantitation, large sample volume requirements, and low throughput.

By switching to an automated platform, you can get fast and reproducible protein quantitation that’s simple to transfer to other groups and sites.

The presence of empty or partially filled AAV particles can compromise the purity and safety of the final product in gene therapy.

Traditional methods, such as analytical ultracentrifugation assays (AUC) and ELISAs, are often unreliable, insensitive, and not easily scalable for manufacturing needs.

By transitioning to an automated solution, you can get consistent and sensitive quantification of empty/full capsid ratios, with scalability for adapting to AAV manufacturing workflows.

Validating AAV identity is critical for selecting the right vector for targeted gene delivery, as AAV serotypes exhibit different tropisms and immunogenic profiles, as well as individuals having pre-existing immunity to specific serotypes.

As you scale up your workflows, the low-throughput and lengthy processes of SDS-PAGE, Western blotting, and mass spectrometry may cause bottlenecks.

By moving to an automated method, you not only expedite your process, but also receive high resolution and reproducible data using smaller sample volumes.

Detecting and quantifying residual proteins, such as host cell proteins (HCPs) from HEK293 cells or endonucleases from AAV vector preparation, is crucial for ensuring the purity of AAV products.

High sensitivity platforms are necessary to identify low levels of impurities that can impact product efficacy, alter quality, and potentially trigger immune reactions in gene therapy applications.

Regulatory agencies strongly recommend biodistribution studies to characterize engineered therapy products.

The RNAscope™ in situ hybridization (ISH) technology is an ideal solution for detecting AAV vector DNA and therapeutic transgene mRNA expression with morphological context, addressing critical questions on tissue biodistribution, cellular tropism, and vector performance in vivo. Easily evaluate dosing, safety and efficacy, and functional markers using RNAscope ISH.