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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.
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.
Miguel Medina, Ph.D.
|Christopher Bunker, Ph.D., MBA
Dr. Christopher Bunker is the Sr. Director of Business Development at Advanced Cell Diagnostics, a Bio-Techne Brand.
|Chris Heger, Ph.D.
Senior Manager, Applications Science
ProteinSimple a Bio-Techne brand