TKT Receives U.S. Patent for Gene Targeting Technology
CAMBRIDGE, Mass., Feb 13, 2001 /PRNewswire via COMTEX/ -- Transkaryotic Therapies, Inc. (Nasdaq: TKTX chart, msgs) today announced the U.S. Patent and Trademark Office has issued U.S. Patent No. 6,187,305 relating to a gene therapy method where exogenous DNA is inserted into vertebrate cells in vitro through a process called homologous recombination, also known as gene targeting. This technology is used to introduce DNA into a pre-selected site within the cells' genome, as well as a method to turn on a gene which is normally not expressed in the cell or expressed at significant levels. Homologous recombination offers several advantages, including the potential for improved safety and biological activity of the transfected cells.
"Homologous recombination is a major component of our technological base that is being utilized for a variety of purposes, including protein production and gene therapy," stated Douglas A. Treco, Ph.D., Senior Vice President, Research and Development. "TKT's non-viral gene therapy approach holds promise for the production and delivery of proteins to treat a broad range of diseases, including hemophilia and diabetes, and the issuance of this patent further validates the breadth of our technology."
In Transkaryotic Therapy(TM), the Company's non-viral gene therapy system, a small sample of a patient's cells are removed in an outpatient procedure and sent to TKT's manufacturing facility where the cells are genetically- engineered to produce the desired protein for extended periods of time. After the cells and the protein have been tested to ensure both safety and functionality, an appropriate number of genetically-engineered cells are returned to the physician and injected back into the patient. TKT's lead gene therapy program is focused on the delivery of Factor VIII in twelve patients with hemophilia A. A Phase I safety study is ongoing.
A copy of the patent described above is available on the World Wide Web at uspto.gov.
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