Use of CTLA4Ig for Improved Adenovirus-mediated Gene Therapy

Introduction Gene therapy holds great promise for the treatment of many diseases that now lack specific therapies. The primary goal of gene therapy is to introduce additional genetic elements or to replace missing nonfunctional molecular elements in the DNA of a living cell so as to target and repair alterations that lead to disease in man. Recombinant viral vectors are attractive vehicles for gene therapy because they are efficient at transferring genes into somatic tissues. However, immunologic factors result in the rapid loss of gene expression and inhibit secondary gene transfer. Technology description Researchers at the University of Washington have invented a method to prolong the expression of a heterologous gene of interest. Contacting the cell with the soluble CTLA4 molecule inhibits an immune response and enhances the expression of a gene of interest by permitting its prolonged or persistent gene expression without long-term immunosuppression. The use of soluble CTL4 molecules also allows for successful secondary gene expression following a second administration of a gene of interest. CTLA4 may be administered by orally, transdermally, intravenously, intramuscularly, or subcutaneously. Alternately, CTLA4 may be inserted together with the gene of interest in a viral vector and co-expressed. Business Opportunity More than thirty different acquired, monogenic, multigenic, short-term, and chronic diseases, including cancer, cardiovascular disease, hemophilia A & B, cystic fibrosis, graft-vs.-host, and diabetes, have gene therapy treatments being researched and developed. The total potential dollar value of these gene therapy markets are billions of dollars.

Patents:
US 5,993,800

Type of Offer: Licensing



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