Research Project
2422987
Gene therapy has been proposed in the treatment of diseases ranging from cancer and viral infections to metabolic disorders such as cystic fibrosis. The main hurdle to effective gene therapy remains delivery of genetic drugs at therapeutic doses. Adeno- associated viral vectors can be used as gene delivery vehicles in a number of applications. The major limitation to the use of this technology is achieving reproducible vector preparations with high infectious titer and a low ratio of defective particles. To successfully develop this technology, the entire process must be scaleable and yield consistent product free of contaminating adenovirus and wild-type AAV. Immusol plans to develop packaging cell lines for rAAV vector production using flow cytometry to continuously monitor transgene expression. Helper plasmids for rAAV vector production incorporating FACS sortable markers will be constructed and used to test for high titer vector production in transient assays. Stable packaging cell lines based on these plasmids will be developed and screened for stable expression of the necessary helper functions. Packaging cell lines with desirable titer and particle ratios will be adapted for growth in suspension culture and in low or no serum media. PROPOSED COMMERCIAL APPLICATION Immusol plans to commercially develop recombinant Adeno- associated viral vector packaging cell lines for use in gene delivery, including ribozyme delivery, for assorted diseases including infectious and metabolic disease and cancer.
