Life threatening diseases can be treated using novel cell and gene therapy based medicines. These drugs have a different mechanism of action in the body but also very different drug development processes to conventional tablets and capsules. Consequently, there is a unique requirement for more insightful analytical techniques to help progress research and development in this area. Malvern Panalytical Ltd. have developed, the newly launched Zetasizer Ultra, which can specially facilitate the development of Adeno-Associated Viral Vector (AAV) based gene therapies. To ensure that AAV based medicines are effective in patients, it is important to measure and monitor the total particle concentration throughout the research, development and manufacturing processes. The new Zetasizer Ultra can help analyze the total particle concentration of AAVs in a more efficient and robust manner compared to traditional ELISA based assays.

Ultragenyx Pharmaceutical Inc. are currently investing in the development of AAV based gene therapies. They have recognized the benefits of utilizing the Zetasizer Ultra in their processes and will be presenting their findings at the Bioprocessing Summit in Boston 12-19th August. Alongside this, Malvern Panalytical Ltd. will be exhibiting at the conference to showcase these capabilities. If you are working in this emerging field of AAVs please come and speak with us or alternatively, connect with peers like Ultragenyx Pharmaceutical Inc. working with the Zetasizer Ultra.

Date/time: Thursday 15th August- 10:45

Presentation title: Determining the Total Particle Titer of AAV by Multi Angle Dynamic Light Scattering

Presenter: Wesley Concepcion, Research Associate II, Analytical Development, Ultragenyx

Abstract: AAV capsid titers define the patient exposure to the therapies and are typically determined by commercial ELISA assays. ELISA-based capsid titer assays tend to have poor accuracy due to the lack of well-characterized reference materials. Quantifying total particles by Multi Angle Dynamic Light Scattering is an orthogonal way to directly determine AAV capsid titers. This presentation will cover the method development and provide comparison between different capsid titer methods.

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