Sept. 19, 2000--Targeted Genetics Corporation (Nasdaq:TGEN - news) today announced that the Company has received notification from the U.S. Patent and Trademark Office regarding the issuance of a patent relating to its synthetic gene delivery technology. The patent, U.S. No. 6,120,794, titled ``Emulsion and micellar formulations for the delivery of biologically active substances to cells'' was issued to the University of Pittsburgh and is exclusively licensed to Targeted Genetics.
The patent covers the composition and formulation of a lipid-based gene delivery system that is resistant to association with and degradation by blood components. The technology described in the patent should therefore have broad utility in the development of gene delivery systems designed to be delivered intravenously, a crucial route of delivery for the development of advanced gene delivery systems. Additionally, the formulation allows for the incorporation of other proteins, peptides or targeting elements that can be used to route the delivery system to specific cell or tissue types.
``This patent is the first to cover a new class of synthetic gene delivery systems and we believe that vectors based on this technology will have applications in a variety of diseases for which current gene therapy approaches are not practical,'' said Dr. Ralph Paul, Ph.D., Senior Scientist, Research at Targeted Genetics. ``A vector that can be delivered systemically may be useful in treating metastatic cancer, a disease in which local gene delivery is not practical. We already are exploring this approach using another one of our synthetic gene delivery systems, LPD. Additionally, the formulation described in this patent is particularly suitable for the addition of targeting molecules and thus may provide the foundation on which to build the first specifically tissue-targeted gene delivery systems. These are the types of next-generation technologies we are evaluating in Emerald Gene Systems, our joint venture with Elan Corporation, plc.''
To date, lipid-based vectors have been subject to rapid degradation in the bloodstream. This characteristic of these vectors has limited their utility to local or regional gene delivery. Loco-regional delivery may be effective for treating diseases such as head and neck cancer or ovarian cancer. A lipid-based delivery system capable of circulating through the bloodstream would expand the potential indications in which gene delivery may provide therapeutic benefit.
``The addition of another gene delivery technology to our portfolio of vectors will likely expand our product opportunities,'' said H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics. ``The best way to achieve therapeutic benefit is to match the properties of the vector with the requirements of the treatment. We have multiple vector systems, each with its own properties, and this should allow us to pick the most appropriate gene delivery system for a given disease. Furthermore, as we move our existing product candidates through preclinical and clinical development, we continue to expand our underlying gene delivery technologies. We believe that this broad platform of vectors and our ability to move programs rapidly from the bench to the clinic will enable us to capitalize on the enormous potential of gene therapy and gene delivery technologies... |
