To: Mike McFarland who wrote (483 ) 10/18/2006 8:32:05 AM From: Mike McFarland Read Replies (1) | Respond to of 557 Engelhardt lab again (there was a paper in Nature Biotech from that lab just a few months ago, also see a patent in 2002 (#6436392). Newly Issued Targeted Genetics Patent Enables More Effective Delivery of Recombinant AAV Vectors Wednesday October 18, 7:30 am ET SEATTLE, WA--(MARKET WIRE)--Oct 18, 2006 -- Targeted Genetics Corporation (NASDAQ:TGEN - News) today announced the issuance of a new patent that describes a novel approach for improving the efficiency with which adeno-associated viral (AAV) vectors deliver genes to target cells. This approach covers small molecule compounds and methods that enhance the trafficking of AAV vectors to the nucleus of a host cell. Transport to the nucleus is necessary for expression of delivered genes. The patent, U.S. #7,122,335, is issued to the University of Iowa Research Foundation and is exclusively licensed to Targeted Genetics. "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," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "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." 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, commented: "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. 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." Targeted Genetics was the first to bring AAV vectors into clinical trials. AAV is a naturally occurring virus that is not associated with any disease in humans. AAV vectors can efficiently deliver genetic information to numerous cell types and can be engineered to carry a variety of DNA sequences. These vectors are highly stable, persist in cells for extended periods of time, and carry an extremely low frequency of integration into host-cell DNA. ---------- that other patent: Targeted Genetics gets additional patent for AAV vectors Saturday, August 24, 2002 08:00 IST Seattle, WA Targeted Genetics Corporation announced the issuance of another patent related to the Company's leading adeno-associated virus (AAV) technology platform. U.S. patent #6,436,392, titled "Adeno-Associated Virus Vectors," was issued to the University of Iowa Research Foundation and is exclusively licensed to Targeted Genetics. The patent covers the use of two AAV vectors to deliver DNA sequences that, once inside a cell, are used to make a single protein. This approach allows AAV vectors to be used to deliver genes that ordinarily would be too large to fit inside a single vector, expanding the potential applications of AAV-based gene delivery.