Government-Owned Inventions; Availability for Licensing

AGENCY:

National Institutes of Health, Public Health Service, HHS.

ACTION:

Notice.

SUMMARY:

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.

ADDRESSES:

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.

Genetic Mutations Associated With Stuttering

Description of Technology: NIH investigators, for the first time, identified specific mutations associated with stuttering. These mutations are located within the genes encoding three enzymes, Glc-NAc phosphotransferase catalytic subunit [GNPTAB], Glc-NAc phosphotransferase recognition subunit [GNPTG], and N-acetylglucosamine-1-phosphodiester alpha-N-acetylglucosaminidase [NAGPA]. Together these constitute the pathway that targets lysosomal enzymes to their proper location. This pathway is associated with lysosomal storage disorders, and thereby this discovery provides potential novel therapeutic targets for amelioration of stuttering. This discovery has the potential to facilitate DNA-based (micro-array) testing among individuals who stutter, as well as enzyme-replacement therapy and small-molecule chaperone therapy for treatment of stuttering. The mutations described in this invention may account for up to 5-10% of this disorder in individuals who stutter, estimated to represent 60,000-120,000 individuals in the United States.

Applications: Genetic diagnosis of stuttering disorder; Therapeutics for stuttering disorder.

Development Status: Early stage.

Market: According to the Stuttering Foundation of America, stuttering affects over 3 million individuals in the United States.

Inventors: Dennis T. Drayna (NIDCD), Changsoo P. Kang (NIDCD), et al.

Patent Status: U.S. Provisional Application No. 61/150,954 filed 02 Feb 2009 (HHS Reference No. E-084-2009/0-US-01).

Licensing Status: Available for licensing.

Licensing Contact: Suryanarayana (Sury) Vepa, Ph.D., J.D.; 301-435-5020; vepas@mail.nih.gov.

Mast Cells Defective in the Syk Protein Tyrosine Kinase

Description of Technology: NIH investigators, through screening for variants of RBL-2H3 cells, have identified and developed TB1A2 mast cells that are defective in the expression of the Syk protein tyrosine kinase. These cells had no detectable Syk protein by immunoblotting or in vitro kinase reaction, and no detectable Syk mRNA by Northern hybridization. These TB1A2 cells failed to secrete or generate cytokines after high affinity receptor for immunoglobulin E (Fc epsilon RI) stimulation. In these Syk-deficient TB1A2 cells, aggregation of these receptors did not induce histamine release and there was no detectable increase in total cellular protein tyrosine phosphorylation. However, stimulation of these cells with the calcium ionophore did induce degranulation. These cells provide a useful experimental model to study the role of Syk tyrosine kinase in signal transduction pathways in immune cells.

Inventors: Juan Zhang, Elsa H. Berenstein, and Reuben P. Siraganian (NIDCR).

Publication: J Zhang, EH Berenstein, RL Evans, RP Siraganian. Transfection of Syk protein tyrosine kinase reconstitutes high affinity IgE receptor-mediated degranulation in a Syk-negative variant of rat basophilic leukemia RBL-2H3 cells. J Exp Med. 1996 July 1;184(1):71-79.

Patent Status: HHS Reference No. E-342-2008/0—Research Tool. Patent protection is not being pursued for this technology.

Licensing Status: Available for licensing under a Biological Materials License Agreement.

Licensing Contact: Suryanarayana (Sury) Vepa, Ph.D., J.D.; 301-435-5020; vepas@mail.nih.gov.

Collaborative Research Opportunity: The National Institute of Dental and Craniofacial Research, Oral Infection and Immunity Branch, Receptors and Signal Transduction Section, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize this technology. Please contact David W. Bradley, Ph.D. at 301-402-0540 or bradleyda@nidcr.nih.gov for more information.

Novel Means of Regulation of Gene Expression: Modular and Artificial Splicing Factors

Description of Technology: This discovery provides a new therapeutic approach for treatment of diseases caused by altered gene regulation resulting from defective alternative splicing of genes. This technology offers the following advantages over currently available methods for regulating splicing: (a) Delivery can be through standard gene therapy methods, such as viral vectors, (b) site of delivery of the artificial splicing factors can be controlled, which enables targeted expression and limited side effects, and (c) the artificial splicing factors described here can be readily adapted to a variety of splicing effector modules. This invention provides proteins that combine an RNA recognition module that can specifically target an endogenous pre-mRNA with splicing effector modules that alter splicing to favor a particular isoform of a mature mRNA.

The artificial splicing factors disclosed here can be used to treat conditions requiring directed alternative splicing. For example, the artificial splicing factors described here can be used in combination with other anti-tumor drugs as a cancer treatment. Other examples where this technology may find use include diabetes (insulin receptor), psoriasis (fibronectin), polycystic kidney disease (PKD2), and prostate cancer (fibroblast growth factor receptor 2).

Applications: Therapeutics for diabetes, psoriasis, polycystic kidney disease, and prostate cancer; Research Tools.

Development Status: Early stage.

Inventors: Traci M. T. Hall (NIEHS), et al.

Patent Status: U.S. Provisional Application No. 61/140,326 filed 23 Dec 2008 (HHS Reference No. E-334-2008/0-US-01).

Licensing Status: Available for licensing.

Licensing Contact: Suryanarayana (Sury) Vepa, Ph.D., J.D.; 301-435-5020; vepas@mail.nih.gov.

Collaborative Research Opportunity: The NIEHS Division of Intramural Research is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize Modular and Artificial Splicing Factors. Please contact Elizabeth M. Denholm, Ph.D. at 919-541-0981 or denholme@niehs.nih.gov or Traci Hall, Ph.D. at hall4@niehs.nih.gov for more information.