Transkaryotic Therapies Inc. on Monday said it has received anon-exclusive license for the gene for a blood clotting protein,Factor IX, from British Technology Group plc.
The license covers use of the gene in non-viral DNA genetransfer, according to K. Michael Forrest, Transkaryoticpresident and chief executive officer. The privately heldCambridge, Mass., company will make undisclosed up-front androyalty payments.
Forrest said Transkaryotic will incorporate the gene into itsproprietary gene therapy system to develop a therapy forhemophilia B. In the company's approach, a sample of thepatient's skin is sent to Transkaryotic's laboratory, where cellsare genetically engineered to produce Factor IX. The modifiedcells are then injected under the patient's skin, and thepatient's own cells produce sufficient quantities of Factor IX toprevent bleeding crises.
Hemophilia B, inherited from the mother, appears in about onein 30,000 people. It is caused by a defect in the gene encodingFactor IX. About 3,000 U.S. patients and some 12,000 in otherdeveloped countries suffer bleeding crises, Forrest said. Thesepatients are treated by increasing their Factor IX levelsthrough the intravenous administration of products derivedfrom blood plasmas. These products cost $10,000 to $100,000per year and subject patients to the risk of exposure to blood-borne diseases, he said.
Phase I/II trials of Transkaryotic's therapy will start in mid-1993, Forrest said.
Genetic Therapy Inc. (NASDAQ:GTII) has also licensed Factor IXfrom British Technology. The agreement, which was announcedin January, grants GTII exclusive rights to use the gene forFactor IX in retroviral vectors for gene therapy to treathemophilia B. -- Steve Usdin
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