With the finalization of licensing for adeno-associated virus (AAV)vectors from three different institutions, Targeted Genetics has gainedexclusive rights to key technologies related to gene therapy, thecompany has announced. The company is evaluating AAV vectors aspotential in vivo gene delivery systems for diseases such as cysticfibrosis (CF) and restenosis.With this step, Targeted Genetics has completed arrangements initiatedin 1993. In August last year, the company announced that it had signeda collaborative research agreement with the Johns Hopkins UniversitySchool of Medicine to develop AAV vectors for in vivo delivery. Theagreement gave Targeted Genetics the first option on exclusiveworldwide licensing rights to inventions resulting from thecollaboration.In a preclinical study done collaboratively at Johns Hopkins, an AAVvector containing the CF transmembrane regulatory (CFTR) gene wasdelivered directly into the lungs of rabbits. The expression wasmaintained for at least six months, with no side effects. TargetedGenetics now plans to file an investigational new drug applicationtoward the end of the year for the treatment of CF using AAV vectors.Jon Case, the company's director of corporate development, toldBioWorld the collaboration with Johns Hopkins is continuing and othertechnologies might be involved.In July last year, Targeted Genetics also sought an exclusive licensefrom the National Institutes of Health's Office of Technology Transferto a modified AAV capable of expression from a novel promoter in thetreatment of cystic fibrosis. This agreement has now been finalized.Targeted Genetics also concluded a licensing agreement with theMedical College of Ohio giving the company exclusive rights totechnology related to the production of AAV vectors.In all three cases, Targeted Genetics will pay an initial fee when theagreements are executed, as well as annual maintenance fees, milestonefees, and royalties. Case would not disclose further details of theagreements.Targeted Genetics has also entered into a separate non-exclusiveagreement with the University of Florida Research Foundation Inc. todevelop and market products utilizing AAV-related patents. A similaragreement has been signed with the University of Michigan and theToronto Hospital for Sick Children regarding the CFTR gene.Some other companies focusing on gene therapy include Viagene Inc.,Applied Immune Sciences, and Avigen Inc.040894TARGET

-- Philippa Maister

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