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 TKT Completes Licensing Program For Blood Coagulation
 CAMBRIDGE, Mass., Sept. 14 /PRNewswire/ -- Transkaryotic Therapies

Inc. (TKT) today announced that it has entered into a collaboration and licensing agreement with Genetics Institute Inc. that provides TKT with certain rights to the Factor VIII gene for the development of gene therapy products to treat hemophilia A, a blood-clotting disease that affects one-in-10,000 males worldwide. This agreement completes TKT's blood coagulation gene licensing program for the three genes -- Factor VIII, Factor IX and gamma carboxylase -- necessary for the commercialization of gene therapy products for hemophilia A and B.
 Under terms of the agreement, TKT and Genetics Institute will collaborate in the development and commercialization of non-viral gene therapy products for the treatment of hemophilia A. TKT will have the exclusive right to market such Factor VIII gene therapy products in all countries of the world, except Europe. Genetics Institute will have manufacturing rights of the products in Europe. TKT retains worldwide exclusive manufacturing rights for the products and will sell them to Genetics Institute for European distribution. The companies will jointly develop and fund a clinical program designed to meet U.S., Canadian and European regulatory requirements.
 "With the completion of this collaboration agreement, TKT has enhanced its position as a strong player in the development and commercialization of gene therapy products for blood coagulation disorders. Our goal is to develop non-viral-based gene therapy products that we believe will provide a major improvement over existing treatments for hemophilia," said K. Michael Forrest, president and chief executive officer of TKT. "TKT plans to initiate its hemophilia clinical program in 1994 with an IND filing for a gene therapy product for the treatment of hemophilia B. We expect the data generated in the hemophilia B trials will have a significant impact on our hemophilia A program," he said.
 Genetics Institute also has a presence in the field of blood coagulation therapy. It produces the recombinant Factor VIII concentrate used in the manufacture of Baxter Healthcare Corp.'s Recombinate(TM) antihemophilic factor (recombinant) product, sold by Baxter in the United States and several European countries. Genetics Institute continues to have an active research and development program in the area of blood coagulation therapy. In early 1993, Genetics Institute announced that it has embarked on a program to bring recombinant Factor IX to market.
 Factor VIII is a naturally occurring blood protein that is absent or deficient in people suffering from hemophilia A, the most prevalent form of hemophilia. Approximately 18,000 patients in the United States (about 70,000 in the developed world) who have this disease experience acute, often life-threatening bleeding episodes. These bleeding crises can occur many times each year, usually triggered by trauma that would be well-tolerated by an individual not afflicted with the disease. Sometimes the bleeding events are spontaneous. Patients in crisis are currently treated with recombinant Factor VIII products such as Recombinate(TM) or through intravenous administration of purified products derived from donated blood plasma.
 TKT is developing a novel approach to the treatment of hemophilia in which a physician would remove a small sample of the patient's skin cells and send it to TKT, where the cells would be genetically modified to produce Factor VIII or Factor IX. Four to six weeks later, the modified cells would then be returned to the physician for injection under the patient's skin. If successful, the implanted cells would produce a constant supply of clotting factor in quantities sufficient to reduce or eliminate bleeding crises, resulting in a greatly improved quality of life for the patient. In pre-clinical studies, TKT has succeeded in engineering normal cells that, following a single implantation, produce therapeutic levels of human growth hormone for the lifetime of laboratory animals. Similar studies with the hemophilia factors are underway at TKT.
 Genetics Institute (NASDAQ: GENIZ) is a leading biopharmaceutical firm engaged in the discovery and development of human pharmaceuticals through recombinant DNA and other technologies. The company has a diversified portfolio of licensed and proprietary products at various stages of development, including treatments of anemia, hemophilia, cancer, bone damage, heart disease, inflammatory conditions and immune system disorders.
 Transkaryotic Therapies Inc. is dedicated to the development and commercialization of non-viral gene therapy products for the long-term treatment of a broad range of human diseases. TKT also is developing powerful new technologies which will enable the surgically precise modification of human chromosomes and the rapid, cost-effective isolation of genes responsible for common diseases.
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 /CONTACT: Lorraine Ruff of Stoorza, Ziegaus & Metzger, (work) 619-236-1332, or (home) 619-755-5610/

CO: Transkaryotic Therapies Inc.; Genetics Institute Inc. ST: Massachusetts IN: MTC SU: PDT

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Publication:PR Newswire
Date:Sep 14, 1993

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