Adeno-associated viruses (AAV) are increasingly popular in gene therapy due to their mild immune response, ability to infect a broad range of cells, and overall safety (Naso et al., 2017). Due to the complexity of the delivery vector, a series of robust analytical approaches are required to ensure correct assembly and packaging prior to product release.

First Segment:
Visualize, detect, and quantify AAV and LV vector biodistribution and transgene expression in preclinical animal models with RNAscope™ and BaseScope™ in situ hybridization technologies.  By combined RNAscope probes to AAV vector and transgene mRNA, vector copy number and expression can be visually assessed and quantified simultaneously in each cell of an intact fixed tissue.  BaseScope enables specific detection of even single nucleotide differences and can easily differentiate AAV human transgene from non-human primate and other animal ortholog sequences of high homology.  These assays are enabling more informative and efficient preclinical gene therapy development and contributing critical data in IND-enabling studies.

Second segment:
Explore various capillary electrophoresis techniques that can be easily implemented along the development path to support gene therapy development.  The Cell and Gene Therapy Catapult in London developed an advanced analytical platform for AAV vector characterization, addressing industry challenges in 1) assay precision through implementation of next generation technologies, 2) standardization and throughput via automation, and 3) enabling in-line product characterization in closed processes through the development of process analytical technologies.  We will highlight data showcasing the impact of the post translational modification on AAV capsids that correlates with a loss in vector activity and can affect capsid assembly and transduction efficiency.  Methods to detect serotype identity and empty versus full capsid ratios will also be discussed.

Post symposium Round Table discussion with featured Application Scientists and Account Managers will be available.