The American Society of Gene and Cell Therapy’s 2025 Annual Meeting
New Orleans, LA, USA
May 13–17, 2025
Join Genedata experts at the 28th Annual Meeting of ASGCT.
Don't miss the opportunity to see how Genedata Selector® enables biopharmaceutical organizations to make data-driven go/no-go decisions regarding cell and gene therapy products' safety, quality, and integrity with significant time savings. By providing out-of-the-box pipelines for automated NGS data analysis, Genedata Selector empowers scientists to implement biosafety testing in-house without the need for extensive bioinformatics training.
For more information about Genedata solutions or to set up a meeting, please contact us.
Recommended Posters
Leveraging Next-Generation Sequencing for CMC of Gene and Cell Therapy Products
Presenter: Thipa Asvarak, Genedata Inc., Boston, MA, USA
Poster number: 834
In the rapidly evolving field of gene and cell therapies, Chemistry, Manufacturing, and Controls (CMC) is instrumental. The development and production of such therapeutics present significant challenges, including shorter shelf life, Critical Quality Attributes (CQAs) assessment, manufacturing complexity, logistics and cost, and stringent regulatory compliance. By harnessing the power of NGS technology, biopharmaceutical companies can produce high-quality cell and gene therapies (CGT) that meet good manufacturing practice (GMP) requirements and clinical standards, ensuring that CMC concerns are addressed. Biopharma and biotech organizations can significantly reduce development timelines and improve product quality by enabling the NGS multi-attribute method (MAM) to monitor several CQAs of CGTs simultaneously for e.g. adventitious agent detection, vector integration and genome integrity assessment.
For this to succeed, such overarching NGS platforms need to be implemented in a validated in-house environment. However, to successfully validate such platforms, teams need to consider several matters, including variability in methods, instruments, and protocols, robust data handling, and adherence to regulatory compliance. Strategic partnerships are crucial to succeed in this mission.
In this poster, we present best practices based on three use cases of leading biopharmaceutical companies that are on a successful path to validate NGS platforms in-house for CGT development.
Leveraging Short- and Long-Reads NGS Technologies In-House for Optimal Quality Control in Manufacturing of Gene and Cell Therapies
Presenter: Ziwei (Coco) Li, Genedata AG, Basel, Switzerland
Poster Number: 974
Biopharmaceutical companies are increasingly adopting next-generation sequencing (NGS)-based assays in the manufacturing process of cell and gene therapies to assess Critical Quality Attributes (CQAs), such as the integrity and safety of the product. Regulatory agencies, such as the FDA or EMA, can request thorough information from biopharmaceutical companies about the CQAs of the product during the IND application.
Besides the broad adaptation of short read technology, long-read sequencing has further advanced the field of NGS by enabling the sequencing of long DNA fragments. Long-read NGS-based assays can be used for CGTs to, for example, detect fusion or truncation events, ensuring product safety and efficacy, fully sequence plasmids and vectors to verify the orientation of gene inserts, and ensure that correct and error-free adeno-associated viruses (AAVs) are packaged into capsids.
Here we report on how to automate, bring to a validated environment in-house, and optimally use both short- and long-read sequencing in manufacturing QC assays of CGTs. We show how both techniques should complement each other to achieve optimal outcomes. We demonstrate how to simplify and streamline all NGS workflows in a GMP environment in-house, ensuring reliable results, faster turnaround times, and improved ROI. This contributes to safer, more efficient, and ethical biotherapeutic development.
Poster Session:
Date: May 13, 2025
Time: 6:00 PM - 7:30 PM
Room: Poster Hall Hall I2
