Enhancing Gene Therapy Assets with Streamlined NGS Assays in GMP Environment

October 3, 2025

Next-generation sequencing (NGS)-based assays are rapidly becoming the gold standard for assessing the safety and efficacy of gene therapy products and their intermediates. By leveraging a Multiple-Attribute Method (MAM) approach, these assays enable the simultaneous measurement of multiple critical quality attributes (CQAs), offering exceptional analytical depth. However, the complexity involved in analyzing, recording, and sharing NGS results creates a significant bottleneck during the research and development (R&D) and Chemistry, Manufacturing, and Controls (CMC) stages. Regulatory bodies such as the FDA require rigorous justification and detailed documentation of these assays to ensure patient safety. 

Genedata Selector^® addresses these challenges by streamlining bioinformatics workflows across all stages of gene therapy development. Here we demonstrate how essential functionalities such as adventitious agent detection and integration site validation can be simplified early in the process by removing bioinformatics bottlenecks. By implementing a GMP-compliant platform that supports workflows from early discovery through to Investigational New Drug (IND) submission, organizations can avoid platform transitions and maintain consistent, efficient development. This approach accelerates the path to GMP-readiness and delivers a substantial return on investment, helping organizations bring safe and effective gene therapies to market more quickly.