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Government-Owned Inventions; Availability for Licensing

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National Institutes of Health, Public Health Service, HHS.




The inventions listed below are owned by an agency of the U.S. Government and are available for licensing in the U.S. in accordance with 35 U.S.C. 207 to achieve expeditious commercialization of results of federally-funded research and development. Foreign patent applications are filed on selected inventions to extend market coverage for companies and may also be available for licensing.


Licensing information and copies of the U.S. patent applications listed below may be obtained by writing to the indicated licensing contact at the Office of Technology Transfer, National Institutes of Health, 6011 Executive Boulevard, Suite 325, Rockville, Maryland 20852-3804; telephone: 301/496-7057; fax: 301/402-0220. A signed Confidential Disclosure Agreement will be required to receive copies of the patent applications.

Treatment for Cystic Fibrosis Cells and Other Reduced Cl−Conductance Cells

Description of Technology: Cystic fibrosis is the most common fatal genetic disease among Caucasians. It is caused by a defect in the cystic fibrosis transmembrane regulator (CFTR) protein. A normal CFTR transports chloride ions across the membrane of epithelial cells lining several organs in the body such as the lungs and the pancreas. The most debilitating consequence of the defective CFTR protein occurs in the lungs of cystic fibrosis patients, where insufficient chloride transport prevents water from exiting epithelial cells. This causes the lungs to produce abnormally thick, sticky mucus that clogs the airways and leads to fatal lung infections. Currently there is no cure for the disease. Present treatments result in undesired side effects such as cardiac, renal, and/or central nervous system tissue.

The NIH has developed a method of identifying cystic fibrosis transmembrane regulator binding compounds for treating cells having a reduced Cl−conductance, such as cystic fibrosis cells. It has also identified a compound, 1,3-Diallyl-8-cyclohexylxanthine (DAX), for potential treatment of cystic fibrosis. Because DAX has specificity in target areas of activity, treatment with this compound can potentially prevent all of the complications of cystic fibrosis including the production of abnormal mucus and without undesired side effects. DAX is active in extremely low concentrations.

Applications: Diagnostic; Therapeutic agent for the treatment of cells having a reduced Cl−conductance.

Market: This is intended for cystic fibrosis or other reduced Cl−conductance cells; Approximately 70,000 children and young adults worldwide, including 30,000 in the U.S. and 30,000 in Europe.

Development Status: Dr. Pollard has performed pre-clinical testing.

Inventors: Dr. Harvey B. Pollard and Dr. Kenneth A. Jacobson (NIDDK).


1. N Arispe et al. “Direct activation of cystic fibrosis transmembrane conductance regulator channels by 8-cyclopentyl-1,3-dipropylxanthine (CPX) and 1,3-diallyl-8-cyclohexylxanthine (DAX),” J Biol Chem. 1998 Mar 6;273(10):5727-5734.

2. KA Jacobson, C Guay-Broder, PJM van Galen, C Gallo-Rodriguez, N Melman, O Eidelman, HB Pollard. “Stimulation by alkylxanthines of chloride efflux in CFPAC-cells does not involve A1-adenosine receptors,” Biochemistry, 1995 Jul 18;34(28):9088-9094.

Patent Status:

U.S. Patent No. 5,877,179 issued 02 Mar 1999 (HHS Reference No. E-138-1992/1-US-01). This patent is for identifying binding compounds and composition of matter.

U.S. Patent No. 6,083,954 issued 04 Jul 2000 (HHS Reference No. E-138-1992/1-US-02). This patent is for treating CF.

Foreign patent rights available.

Licensing Status: Available for exclusive or non-exclusive licensing.

Licensing Contact: Catherine A. Wendelken; 301/435-5282;

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Dated: August 3, 2007.

Steven M. Ferguson,

Director, Division of Technology Development and Transfer, Office of Technology Transfer, National Institutes of Health.

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[FR Doc. E7-15622 Filed 8-9-07; 8:45 am]