Gene therapy has emerged as a groundbreaking approach for addressing a wide array of genetic disorders. With over 30 gene therapies approved by the FDA and more than 2000 in various stages of development globally, the potential for transformative treatments continues to expand. Recombinant Adeno-Associated Viral (AAV) vectors serve as a vital gene delivery mechanism for introducing therapeutic genes into patients. Precise and reproducible characterization of these vectors is crucial to ensure accurate clinical dosing, safety, and therapeutic efficacy. The integrity of the viral genome plays a pivotal role, as variations in genome regions targeted for analysis can influence vector quantitation results, and truncated vectors may fail to produce a functional therapeutic product.
Agathos Biologics recently participated in a panel roundtable discussion hosted by QIAGEN, titled “Viral Vector Integrity Analysis – Advancements in Gene Therapy Development.” The 48-minute session brought together leading experts to explore cutting-edge methods for assessing vector integrity, with a focus on the advantages of digital PCR (dPCR)-based solutions. Key topics included the design of multiplex assays and the challenges involved in their implementation, the significance of reference standard materials and assay qualification, and innovative approaches to genome integrity evaluation. The event concluded with a live Q&A session, providing attendees the opportunity to engage directly with the panel of experts.