Transkaryotic Therapies Inc. (TKT) announced Tuesday that ithas entered into a collaboration and licensing agreement withGenetics Institute Inc. (GI) that provides TKT of Cambridge,Mass., with certain rights to GI's Factor VIII gene for thedevelopment of gene therapy products to treat hemophilia A, ablood-clotting disease that affects one in 10,000 malesworldwide.
Under the agreement, TKT and Genetics Institute(NASDAQ:GENIZ) will collaborate in the development andcommercialization of non-viral gene therapy products for thetreatment of hemophilia A. TKT will have the exclusive right tomarket such Factor VIII gene therapy products in all countriesexcept Europe. GI will have manufacturing rights for theproducts in Europe. TKT retains worldwide exclusivemanufacturing rights for the products and will sell them to GIfor European distribution. The companies will jointly developand fund a clinical program designed to meet U.S., Canadianand European regulatory requirements.
The agreement with GI completes TKT's blood coagulation genelicensing program for three genes -- Factor VIII, Factor IX andgamma carboxylase -- that are necessary for thecommercialization of gene therapy products for hemophilia Aand B. TKT has the exclusive worldwide rights to developBritish Technology Group plc's Factor IX gene for non-viralgene therapies. And it licensed the exclusive worldwide rightsto the gamma carboxylase gene from the University of NorthCarolina at Chapel Hill.
Privately-held TKT "plans to initiate its hemophilia clinicalprogram in 1994 with an investigational new drug (IND) filingfor a gene therapy product for the treatment of hemophilia B.We expect the data generated in the hemophilia B trials willhave a significant impact on our hemophilia A program," said K.Michael Forrest, TKT's president and chief executive officer.
-- Jennifer Van Brunt Senior Editor
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