Recombinant AAV Vectors for Hepatic Gene Therapy
Introduction The liver is the target of a number of different viral infections and other disorders. Despite the impressive progress in biomedical sciences during the last decades, the therapy of many liver diseases remains unsatisfactory. There is a need for efficient alternative therapeutic approaches and in recent years gene therapy has emerged as a promising method to treat human diseases. A challenging issue for successful application of gene therapy to these conditions is the selection of a safe and effective vector for transducing normal liver cells or malignant liver cells in vivo or ex vivo. Technology description Dr. Mark Kay and his collaborators have developed novel methods and vectors for transducing liver cells. Adeno associated virus vectors are non-pathogenic human parvoviruses that have been engineered to be effective gene therapy vectors. The advantages of the use of AAV-based vectors are that they transduce both dividing and nondividing cells and achieve long-term expression of therapeutic genes with no apparent adverse effects. This invention also permits expression of diffusible polypeptides in the liver which provides access to the circulation and permits systemic delivery of therapeutic proteins and polypeptides of interest that are encoded by an exogenous or endogenous polynucleotide of a rAAV vector. Business Opportunity This technology carries the potential to treat several diseases affecting the liver including, but not limited to hepatitis C, which has a worldwide therapeutic market of $4 billion, hepatitis B, which is expected to reach $1.3 billion across the seven major markets by 2016 and hemophilia, which has a global treatment market that is projected to be $8.7 billion by 2012. Stage of Development Studies were carried out in mice using this technology, showing transduced gene expression almost a year post-injection with no evidence of pathology or toxicity.Patents:
US 6,936,243
Type of Offer: Licensing
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