Exploring the charge heterogeneity in recombinant adeno-associated virus with imaged isoelectric focusing

Recombinant Adeno-associated Virus is one of the leading platforms for monogenic disease therapy. One of the attractive features of rAAV is its wide tropism, owing in part to its surface property where charged amino acids are not conserved.  In addition, charged regions are also involved during its transduction of host cells. As a result, the characterization of its charged surface is necessary for understanding of rAAV function. Here we present the exploration of the charge profile of rAAV and give a fingerprint of the charge profile, and demonstrate the potential application of charge fingerprinting for formulation development under elevated temperature and pH stress.

• Analytical challenges for formulation development of AAV 
• 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 
• 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- formulation works by iCIEF

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.

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Julie Wei

Associate Director, Analytical Development at Ultragenyx Gene Therapy

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