Innovative capillary electrophoresis techniques for gene therapy R&D

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 still 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

Chris Heger PhD

Senior Manager, Applications Science, Analytical Solutions Division at Bio-Techne

Chris Heger, Senior Manager of Applications Science for the Analytical Solutions Division of Bio-Techne, leads a team chartered with creating scientific collateral, fostering collaborations, training, and coming up with new applications. Chris has expertise in immunoassays, capillary electrophoresis, antibody design and production, and chromatography. In recent years, his team has focused on developing analytical solutions for cell and gene therapy researchers.
Chris received his doctoral degree in Pharmacology from Cornell University and completed his post-doctoral training at the National Cancer Institute. Working in the Antibody and Protein Purification Unit, Chris oversaw antibody development projects, from immunogen design to final product. A unique aspect of his postdoctoral training was his role in the evaluation of new technologies, serving as the gateway for cutting edge instrumentation to get into use at the NCI/NIH. Through this role, Chris became well-acquainted with ProteinSimple and after completing his postdoctoral training, began working for ProteinSimple (now part of Bio-Techne).

Julie Wei

Associate Director, Analytical Development at Ultragenyx Gene Therapy

Dr. Wei is the Associate Director of Analytical Development at Ultragenyx Gene Therapy. Dr. Wei has her PhD from Brandeis University, where she studied protein structure and functional relationship with NMR, and is the discoverer of the mechanism of O2-CO2 conversion in P450 proteins. She has held patent in processing in the area of cell culture SEC method, cell-culture CE-MS method, in-silico formulation screening, and a patent in preventative therapy in Alzheimer’s disease. Prior to her role at Ultragenyx, Dr. Wei was the Director of Analytical Formulation Sciences at HJB, a Consultant for biotech companies, and she served ten years in the department of Technology Development at Biogen. Her current focus at Ultragenyx, is the development of chemical and biophysical methods suited for characterization of adeno-associated virus for gene delivery therapy. The methods she developed at Ultragenyx include: SEC, CE-SDS, UV, AUC, and LC-MS, which all became quality methods for the release of those products.

Watch now

SPEAKERS

Chris Heger PhD

Senior Manager, Applications Science, Analytical Solutions Division at Bio-Techne

Julie Wei

Associate Director, Analytical Development at Ultragenyx Gene Therapy

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