Genzyme Corp. has made a 10 percent equity investment in aprivate company based in the Netherlands to develop and market agene therapy for treatment of Gaucher's disease.Financial terms of the agreement between Cambridge, Mass.-basedGenzyme and IntroGene B.V., of Rijswijk, were not disclosed. Inreturn for licensing the technology, Genzyme will provideIntroGene with research, milestone and royalty payments.Kathleen Rinehart, Genzyme's spokeswoman, said the collaborationcombines IntroGene's stem cell gene therapy with her company'sexperience with Gaucher's disease, which is caused by the lack ofthe enzyme, glucocerebrosidase (GCR). Symptoms of thepotentially fatal disease include enlarged liver, anemia, fatigue andorthopedic problems.Genzyme has two products, Ceredase and Cerezyme, on the marketto treat Type I Gaucher's disease. Both drugs are designed toreplace the missing enzyme.The gene therapy, Rinehart said, is aimed at correcting the geneticdefect responsible for not producing the enzyme. IntroGene'stechnology involves employing a retrovirus to introduce the GCRenzyme into stem cells in the bone marrow. In theory, as new cellsare produced, they will contain GCR.Whereas enzyme replacement treatments, such as Ceredase andCerezyme, must be conducted frequently, Rinehart said genetherapy may be a longer-lasting approach.She said Genzyme and IntroGene don't anticipate launching clinicaltrials with the gene therapy for "a couple of years." IntroGenecurrently is conducting preclinical research.Other companies developing gene therapy for Gaucher's diseaseinclude Genetic Therapy, of Gaithersburg, Md., and TargetedGenetics, of Seattle. _ Charles Craig
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