December 1, 2020
Hosted by Cell & Gene Therapy Insights

R&D requirements for gene therapy candidates are not that different from other biotherapeutics. To fully characterize these complex molecules, you need analytical instrumentation that you can rely on to make informed, accurate decisions. You need speed, accuracy, and flexibility to support both characterization and release requirements. In the first segment of the technical seminar, we will discuss various capillary electrophoresis techniques that can be easily implemented along the development path to support gene therapy development.

The second segment of the seminar will focus on Adeno-associated virus (AAV) Empty and Full separation using the imaged cIEF method. The separation of AAV Empty and Full by charge-based techniques has been developed on several serotypes using ion-exchange chromatography (IEX). However, sometimes IEX cannot separate serotypes effectively and thus you need to use other techniques. Here we will showcase how imaged cIEF separates an AAV drug substance that could not be separated using other techniques and illustrates the imaged cIEF method development.

Attendees will learn about:
  • Analytical challenges in characterizing gene therapy drug candidates
  • Important analytical considerations in selecting the right tools for the characterization of viral vectors
  • Applications of capillary electrophoresis techniques in the analysis of viral vectors
  • Showcasing the imaged cIEF method’s separation of Empty and Full Adeno-associated virus (AAV)
  • An exploration into what works now, why, and what directions they would like to see the field take in the future, from our two experts in analytical development


Julie Wei

Julie Wei, Ph.D.
Associate Director, Analytical Development
Ultragenyx Pharmaceutical Inc.

Chris Heger

Chris Heger, Ph.D.
Senior Manager, Applications Science
Analytical Solutions Division,  Bio-Techne