Unlock Next-Level Gene Therapy Insights with ddPCR
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Biodistribution is a critical step in developing safe and effective gene therapies, but traditional qPCR methods can fall short in sensitivity and precision. Droplet digital PCR (ddPCR) is changing the game, and a white paper from Smithers Pharmaceutical Development Services shows exactly how.
The paper details the development and GLP-compliant validation of a ddPCR assay for AVGN7, a Smad7 gene therapy designed to combat muscle wasting. From primer/probe optimization to rigorous validation across accuracy, precision, specificity, recovery, and stability, the study provides a clear, step-by-step roadmap. The assay delivers outstanding sensitivity, detecting as few as 2.5 copies per reaction, while maintaining linearity up to 80,000 copies and reliable performance across multiple tissue types and storage conditions.
Beyond AVGN7, this white paper serves as a model for other gene therapy programs, offering practical guidance on navigating regulatory challenges and implementing high-precision ddPCR assays. Aligning with Global CRO Council (GCC) recommendations and exceeding standard validation metrics, it’s a must-read for researchers pushing the boundaries of gene therapy development.
Download the full white paper now to see how ddPCR can elevate your biodistribution studies and accelerate your program.