The University of Iowa Research Foundation was issued a license to a patent that describes an approach for improving the efficiency with which adeno-associated viral (AAV) vectors deliver genes to target cells. Targeted Genetics has exclusively licensed this patent. This approach covers small molecule compounds and methods that enhance the trafficking of AAV vectors to the nucleus of a host cell.
"As a company focused on developing AAV-based products for important therapeutic indications, we strive to enhance the natural properties of AAV that give these vectors great potential in treating a diverse array of diseases," remarks H. Stewart Parker, president and CEO. "Use of the small molecule compounds described in this patent may increase the transduction efficiency that we can achieve with AAV vectors. In turn, increased transduction efficiency may help to increase the therapeutic effect of AAV-based products and also may allow for smaller doses, which could result in increased tolerability and reduced per-dose cost.
“AAV vectors have demonstrated excellent safety profiles in our clinical trials and have a number of other attributes that make them quite promising as an approach for introducing therapeutic genes into target cells."
"Studies in our laboratory have shown that a number of small molecules have the ability to increase the efficiency of AAV transduction in cell culture and animal models,” adds John Engelhardt, Ph.D., director of the Center for Gene Therapy, professor and head of the department of anatomy and cell biology at the University of Iowa, and an inventor on the patent. “The use of small molecule compounds, several of which already are approved by the FDA for use in other indications, may add to the efficiency of the AAV transduction process, making AAV vectors even more attractive as a platform for developing novel therapies."