This poster will present the fit-for-purpose approach to validate a GLP-compliant ddPCR duplex assay for quantifying AVGN7, a Smad7 gene therapeutic for muscle wasting, in mouse tissues. The mouse TATA-box binding protein (mTBP) was used as reference gene for total DNA normalization. This poster was created alongside AAVogen, Inc., which develops the proprietary gene therapeutic for muscle wasting (AVGN7) that is discussed herein.
The biodistribution of adeno associated virus (AAV) gene therapy vectors are traditionally monitored using real-time quantitative PCR. More recently, droplet digital (dd)PCR was introduced as an additional platform to quantify nucleic acids. This latter technology has distinct advantages including higher sensitivity and absolute quantification but is underutilized due to lacking regulatory guidance and meaningful examples in the literature.
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