Practical strategies for overcoming challenges in the development of AAV vectors for gene therapy

Gene therapy promises to treat and potentially cure a disease by correcting its underlying genetic cause. While gene therapies that utilize AAV vectors have shown encouraging results —including a few approved by the FDA and/or the EMA — they are still facing challenges. Particularly for AAV-based gene therapy, researchers are still figuring out the analytical characterization strategy of the viral vectors and quality control parameters. This webinar provides an overview of analytical challenges and how different techniques are used in the characterization of AAV vectors.

Dr. Santosh Khatwani, Associate Director of Analytical Development at Sangamo Therapeutics, tells us about how he approaches analytical characterization of AAV vectors and uses two case studies as examples of the challenges he faces.

Dr. Xiaoying Jin, a Sr. Principal Scientist who has worked at Sanofi for 20 years and the lead of a group that specializes in the characterization of AAV vectors, presents how she uses LC-MS to characterize AAV capsid proteins, which are critical for viral infectivity and vector potency.

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Quantitation of trace levels of DNA released from disrupted Adeno-associated virus gene therapy vectors
Quantitation of trace levels of DNA released from disrupted Adeno-associated virus gene therapy vectors

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