アプリケーションノート：Genedata Selectorによる遺伝子治療製品の開発と製造

Compared to traditional therapeutics, gene therapies are novel biotherapeutics that aim to treat, prevent, and potentially cure diseases by correcting underlying genetic disorders. Gene therapy focuses on manipulating, introducing, or deleting genes, whether in vivo or ex vivo. Ensuring the efficacy and safety of gene therapies is critical to their ability to achieve their intended purpose. The advent of Next-Generation Sequencing (NGS) has transformed the biopharmaceutical industry as a powerful tool for assessing critical quality attributes (CQAs) to confirm the biosafety, identity, and potency of biotherapeutics. NGS-based assays provide an unbiased, sensitive, and more rapid approach than traditional methods, and require significantly less biological input material.

Genedata Selector^® is an end-to-end software solution that transforms data into actionable results for all NGS-based assays. As a sequencing technology-agnostic platform, it simplifies sample and data management while enabling automated analysis, intuitive visualization, and the integration of NGS data with other data types. Genedata Selector is a single source of truth that can be easily integrated into cGMP environments to provide reports for internal documentation and regulatory submissions.