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Analyzing the purity of adeno-associated virus (AAV)-based gene therapy products requires effective methods for detecting host proteins, DNA, and incomplete viral particles in samples. It is a critical step in the manufacturing of AAV therapies and ensures that the final products meet the relevant standards for safety, efficacy, and consistency.
In this on-demand webinar, experts from Regeneron Pharmaceuticals discuss two novel methods for characterizing AAV-based gene therapy products. Dr. Song Nie discusses a new workflow for robust analysis of AAV samples that combines wide-window data-dependent acquisition (WWA) with single-pot, solid-phase-enhanced sample-preparation (SP3). This method improves host cell protein (HCP) identification with low-input sample preparation, as Dr Nie will present in a recent case study detecting hundreds of HCPs in a commercial AAV1 sample.
Additionally, we focus on a broadly applicable, denatured size exclusion chromatography (dSEC-MS) method that effectively separates viral proteins across different AAV serotypes. Dr. Tim Tiambeng will show how the method identifies both AAV DNA and viral particle components from empty and full AAV samples. This tool helps scientists streamline both development and analytical quality control processes for AAV gene therapies.
About the presenters
Song Nie, PhD, Sr. Principal Scientist, Analytical Chemistry, Regeneron Pharmaceuticals, Inc.
Dr. Nie earned his PhD from the Shanghai Institutes for Biological Sciences (SIBS), followed by postdoctoral studies at the University of Michigan and the Pacific Northwest National Laboratory (PNNL). Currently, Dr. Nie is a Senior Principal Scientist at Regeneron, bringing more than 20 years of expertise in utilizing Liquid Chromatography-Mass Spectrometry (LC-MS) for post-translational modifications analysis (PTMs) in a variety of samples, biomarker discovery, and biological therapeutic drug characterization. Dr. Nie has developed multiple methods to address the challenges in biological therapy characterization and impurities identification to improve drug development and process.
Tim Tiambeng, PhD, Sr. Scientist, Analytical Chemistry, Regeneron Pharmaceuticals, Inc.
Timothy-Neil Tiambeng, PhD, is a senior scientist at Regeneron Pharmaceuticals. He received a B.S. in chemistry from the University of California–Irvine. He completed his doctoral degree in chemistry with Professor Ying Ge at University of Wisconsin–Madison, where he developed analytical techniques and tools for the analysis of low-abundance proteoforms from clinical samples. At Regeneron, he focuses on developing LC-MS based analytical methods to support development and characterization of biotherapeutic drug candidates.