Advancing AAV: Novel Synthesis and Sequencing Solutions for Gene Therapy Research

Interest in cell and gene therapy-based disease prevention and treatment has increased rapidly over the last few decades, however there are still many hurdles to overcome and further progress to be made in the field. In this presentation, Dr. Andrea O’Hara will discuss GENEWIZ’s innovative gene therapy solutions across the clinical development pipeline, highlighting adeno-associated virus (AAV) plasmid DNA sequencing (RUO/GLP), preparation, and synthesis solutions for recombinant AAV (rAAV) production, which includes confirmation, maintenance, and correction of inverted terminal repeat (ITR) regions. She will also talk about GENEWIZ’s proprietary Sanger & next generation viral genome sequencing confirmation solutions post-AAV packaging. This includes sequencing validation of packaged materials to confirm rAAV vector integrity and identify DNA contaminants in AAV library preparation utilizing short- and long-read whole genome sequencing solutions. Finally, she will discuss novel approaches to stratify and characterize host-immune response and measure rAAV expression during and after treatment.

Learning Objectives:

1. The structure of the AAV genome and how it's used in gene therapy research

2. The challenges associated with AAV usage, and proprietary solutions that can be used to overcome these obstacles

3. The importance of Illumina® short-read and PacBio® long-read sequencing technology for rAAV quality control