TKT Awarded U.S. Patent for Erythropoietin Gene Therapy - Company Receives Two Additional Gene Therapy Patents -
CAMBRIDGE, Mass., April 11 /PRNewswire/ -- Transkaryotic Therapies, Inc. (NASDAQ:TKTX) announced today that the U.S. Patent and Trademark Office has issued U.S. Patent No. 6,048,524 entitled, "In Vivo Production and Delivery of Erythropoietin for Gene Therapy," U.S. Patent No. 6,048,724 entitled, "Method of Producing Clonal Cell Strains which Express Exogenous DNA Encoding Glucagon-Like Peptide 1," and U.S. Patent No. 6,048,729 entitled, "In Vivo Protein Production and Delivery System for Gene Therapy." The '524 patent relates to gene therapy for the long-term delivery of erythropoietin for the treatment of anemia, the '724 patent relates to the delivery of glucagon-like peptide 1 (GLP-1) for the treatment of diabetes, and the '729 patent relates to non-viral gene therapy technology for the production and delivery of a broad range of therapeutic proteins.
"These patents, in concert with our other gene therapy intellectual property, demonstrate and validate the breadth of our gene therapy technology," said Richard F Selden, M.D., Ph.D., President and Chief Executive Officer of TKT. "As the first group to demonstrate the feasibility of erythropoietin gene therapy, we continue to innovate scientifically and pioneer new treatment modalities."
Erythropoietin is a hormone that stimulates the production of red blood cells. Used to treat anemia, the protein is presently administered by regular intravenous or subcutaneous injection. After typical treatment regimens, levels of erythropoietin fall over time as the protein is cleared from the blood requiring re-administration of the protein. TKT believes that its gene therapy approach to deliver erythropoietin may provide for a continuous, precisely dosed quantity of the protein into the blood, eliminating the need for frequent injections.
GLP-1 is a hormone that has been shown to be effective in stimulating insulin secretion and decreasing blood glucose levels when administered by intravenous and subcutaneous injection for the treatment of diabetes. Patients affected with diabetes are often treated with high levels of insulin to compensate for their decreased insulin sensitivity. TKT believes its gene therapy approach to deliver genetically-engineered cells producing GLP-1 may provide for a continuous increase in blood glucose to potentiate insulin release and, ultimately, control certain forms of diabetes by lowering blood glucose.
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 the Company'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 believes Transkaryotic Therapy is well suited to allow safe and long-term delivery of therapeutic proteins for the treatment of chronic protein deficiency states, including anemia, hemophilia, hypercholesterolemia, and diabetes. This technology is designed to take advantage of a patients' ability to synthesize therapeutic proteins for extended periods, potentially providing improved therapeutic outcome, a reduction of side effects due to overdosing or underdosing of conventional proteins, improved patient compliance, and significant reductions in cost due to less frequent administration.
In November 1998, TKT began the first ever hemophilia gene therapy clinical trial focused on the delivery of Factor VIII to patients with hemophilia A. The Phase I safety study is ongoing and based on preliminary results, there have been no adverse events associated with the treatment. During 2000, six additional patients with hemophilia A will be enrolled in the study.
Copies of the patents described above are available on the World Wide Web at uspto.gov.
Transkaryotic Therapies, Inc. (TKT) is a biopharmaceutical company dedicated to the development and commercialization of products based on its three proprietary development platforms: Gene-Activated(TM) proteins, Niche Protein(TM) products, and Gene Therapy. The Company's gene activation technology is a proprietary approach to the large-scale production of therapeutic proteins, which does not require the cloning of genes and their subsequent insertion into non-human cell lines. TKT's Niche Protein product platform is based on protein replacement for the treatment of rare genetic diseases, a group of disorders characterized by the absence of certain metabolic enzymes. The Company's Gene Therapy technology, known as Transkaryotic Therapy(TM), is focused on the commercialization of non-viral, ex vivo gene therapy products for the long-term treatment of chronic protein deficiency states.
This press release contains forward-looking statements that involve a number of risks and uncertainties. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward- looking statements. Without limiting the foregoing, the words, "believes," "anticipates," "plans," "expects," "intends," and similar expressions are intended to identify forward-looking statements. Important factors that could cause actual results to differ materially from the expectations described in these forward-looking statements are set forth under the caption "Certain Factors That May Affect Future Results" in the Company's Annual Report on Form 10-K for the year ended December 31, 1999 which is on file with the Securities and Exchange Commission and incorporated herein by reference. These important factors include risks as to whether TKT's products will advance in the clinical trials process, the timing of such clinical trials, whether the clinical trial results will warrant continued product development, and whether the Company's products will receive approval from the U.S. Food and Drug Administration or equivalent regulatory agencies, and, if such products receive approval, whether they will be successfully marketed; the results of any patent litigation in which the Company is involved or may become involved; competition; the Company's dependence on collaborators; and the risk that the claims allowed under any issued patents such as the patents referred to above will not be sufficiently broad to protect the Company's technology, that the claims issued under such patents will not be sustained if challenged in court proceedings or otherwise or that third parties will be able to develop products or processes that do not infringe such patents.
GA-EPO(TM), Gene-Activated(TM), Niche Protein(TM), TKT(TM), and Transkaryotic Therapy(TM) are trademarks of Transkaryotic Therapies, Inc.
Please visit our web site at www.tktx.com for additional information about Transkaryotic Therapies, Inc.
CONTACT:
Justine E. Koenigsberg
Manager, Corporate Communications
(617) 349-0271 SOURCE Transkaryotic Therapies, Inc.
-0- 04/11/2000 |
