Gene therapy using adeno-associated viruses (AAVs) is transforming medicine by treating previously incurable genetic disorders. However, verifying the quality of these therapies remains challenging. Current methods for checking AAV DNA are inadequate – they're either too imprecise to detect important genetic changes or too slow and expensive for routine use.
This project develops quality control techniques using long-read DNA sequencing technology from Oxford Nanopore Technologies and Pacific Biosciences. Unlike current methods, these platforms can read entire AAV genomes in a single pass, revealing crucial details about their structure and composition.
Enabling earlier detection of vector genome issues during production
Accelerating manufacturing troubleshooting with detailed sequence information
Reducing development timelines by 30-50% through faster analytical turnaround
Enhancing product safety and consistency across the biopharmaceutical ecosystem
Supporting regulatory submissions with comprehensive genomic characterization data
This initiative is projected to cut AAV development timelines by up to 50%, significantly reduce quality control costs, and strengthen product safety and consistency. By enabling rapid, high-accuracy genomic analysis, it positions manufacturers to accelerate market entry and realize substantial long-term savings across the gene therapy pipeline.
Validated protocols for AAV genome sequencing achieving >99.99% consensus accuracy, with detection of variants at
Optimized sample preparation workflows for both AAV plasmids and packaged vectors, including methods for ITR preservation
Control materials with engineered variants for method benchmarking and validation
End-to-end computational pipeline for processing long-read AAV data, incorporating UMI consensus algorithms
Comprehensive documentation for laboratory workflows and analytical methods
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Johns Hopkins University
