Recombinant adeno-associated virus (rAAV) vectors have been widely used for in vivo gene therapy, with Luxturna™ and Zolgensma™ already approved by FDA as commercial products. In order to quickly characterize and test AAV gene therapy products, a platform approach for AAV vector genome titer quantification, residual human host cell DNA quantification and sizing evaluation, and replication competent AAV (rcAAV) detection has been established.
Adopting real-time PCR, as well as ddPCR, technologies allows for targeting to sequences other than gene of interest (GOI) region, along with using reference materials available from ATCC or biological materials. Part two of this four-part series examines data from in-house development and generic validation studies and discuss individualizing platform analytical methods based on a specific AAV gene therapy product.
Yan Zhi, PhD
Director, Scientific Services
View the other webinars in this series:
- Analytical Ultracentrifugation for Characterization of AAV Gene Delivery Vectors (On Demand)
- Advanced Mass Spectrometry-based Characterization of Adeno-Associated Viruses and Next Generation Biologics (On Demand)
- Viral Clearance Challenges for Gene Therapy Products (On Demand)
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