Viral vector integrity analysis – Advancements in gene therapy development скачать в хорошем качестве

Viral vector integrity analysis – Advancements in gene therapy development

Round table discussion - Gene therapy holds promise for treating various genetic diseases. In recent years, more than 30 gene therapies have been approved by the FDA, and more than 2000 are currently in development at various pipeline stages around the globe. Recombinant Adeno-Associated Viral (AAV) vectors are widely used as a gene delivery mechanism to introduce therapeutic genes in patients. Accurate and reproducible characterization of such vectors is critical to ensure correct clinical dosing, safety, and treatment efficacy. Genome integrity can influence vector quantitation results depending on the regions targeted for analysis. In addition, truncated vectors may not result in a functional product. In this roundtable, our experts will discuss the available methods to assess vector integrity and the advantages dPCR-based solutions can bring. They will share perspectives on multiplex assay design to accomplish this task and related challenges, as well as discuss the importance of reference standard materials and assay qualification.