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GTAD Boston 2023—Gene Therapy Analytical Development Summit

Boston, MA, USA
November 27–30, 2023

Meet Genedata experts at GTAD.

Don't miss out on the opportunity to see how Genedata solutions efficiently support the whole biopharmaceutical R&D workflow. This includes accelerating NGS-based cell line development and biosafety, and streamlining biopharma mass spectrometry workflows across instruments and organizations, delivering high-quality results with significant time and cost savings.

For more information about Genedata solutions or to set up a meeting, please contact us.

Recommended Posters

Leveraging NGS for Critical Quality Attribute Measurement of Gene Therapy Products

Thipa Asvarak1, David Killen1, Thomas Hartsch2
1Genedata Inc., US | 2Genedata AG, Switzerland
Tuesday, November 28 | 3:30 pm

Available gene therapies have had remarkable outcomes, yet there have been multiple cases where some therapeutics have led to unforeseen outcomes and clinical holds. To avoid that and guarantee that the intended therapeutics are developed, in-process testing is required to ensure these therapeutics maintain critical quality attributes (CQAs). For gene therapies, CQAs include identity, integrity, and safety. Establishing and monitoring these attributes throughout development and manufacturing can help to ensure the product developed is safe and fit for its purpose. Regulatory authorities such as the FDA and the EMA also request detailed documentation regarding CQAs when companies apply for an IND. For this, a streamlined assay portfolio and the right technology that allows the simultaneous determination of multiple attributes of therapeutics would be highly beneficial. Here we present Next-Generation Sequencing (NGS) as the multi-attribute method for gene therapy products, as it eliminates the need for implementing several different assays to determine CQAs by streamlining multiple assays. The vast amount of useful information obtained from NGS makes it easier, faster, and more cost-efficient to examine the critical quality attributes of gene therapy products.

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Automated Peptide Mapping and Intact Mass Analysis Data Processing Workflows for Streamlined MS-Based Characterization of AAV Capsids

Catherine Evans1, Stephen Kok2, Amy Claydon3
1Genedata AG, Switzerland | 2Genedata Inc., US | 3Genedata Ltd., UK
Tuesday, November 28 | 3:30 pm

Adeno-associated virus (AAV) vectors are often the preferred platform for therapeutic gene delivery, due to their high infectivity, low immune response, and high overall safety compared to other viral vectors. However, to ensure vector stability and potency of gene therapy products, characterization of AAV capsids — for example, by monitoring potential stability hot spots through accurate assessment of deamidation and phosphorylation across multiple samples — is crucial during candidate selection and process development. Mass spectrometry-based analytical methods enable in-depth capsid characterization at both the protein and peptide level and consequently are increasingly being used for serotype confirmation and heterogeneity characterization. Here, we present automated peptide mapping and intact mass analysis workflows for MS data processing, analysis, and reporting that streamline MS-based characterization of AAV capsids and facilitate downstream deployment of novel cell and gene therapies.

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