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Gene therapy offers significant potential for addressing various genetic disorders. Over 30 gene therapies have received FDA approval, with more than 2,000 currently in development across different stages of the pipeline worldwide.

Recombinant Adeno-Associated Viral (AAV) vectors are a commonly employed gene delivery system for introducing therapeutic genes into patients. Precise and reliable characterization of these vectors is essential to ensure proper clinical dosing, patient safety, and the efficacy of treatments.

Vector quantitation outcomes can be affected by genome integrity, as results vary depending on the regions analyzed. Additionally, truncated vectors may fail to produce a functional therapeutic product.

This roundtable brings together experts to explore methods for evaluating vector integrity, highlighting the benefits of dPCR-based solutions. Discussions will also cover the design of multiplex assays, the challenges involved, and the critical role of reference standard materials and assay qualification.  

Key topics that are discussed by our experts:

  • Innovative Methods for Gene Therapy: Gain insights into how digital PCR provides precise and efficient vector genome quantification, enhancing safety and efficacy.
  • Practical Insights on Genome Integrity: Discover expert strategies for designing robust multiplex assays and optimizing sequence packaging efficiency to enhance vector performance.
  • Tools to Solve Key Challenges: Explore advanced methods like AUC and mass photometry for assessing full vs. empty capsids, and hear expert answers to pressing technical questions during the Q&A session.