[Federal Register Volume 79, Number 133 (Friday, July 11, 2014)]
[Notices]
[Pages 40120-40122]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-16265]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Government-Owned Inventions; Availability for Licensing

AGENCY: National Institutes of Health, HHS.

ACTION: Notice.

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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. 209 and 37 CFR part 404 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.

FOR FURTHER INFORMATION CONTACT: 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

[[Page 40121]]

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.

SUPPLEMENTARY INFORMATION: Technology descriptions follow.

Delta Tocopherol for the Treatment of Lysosomal Storage Disorders

    Description of Technology: Delta Tocopherol is identified as a 
novel therapeutic to treat lysosomal disorders characterized by 
defective cellular cholesterol and other lipid trafficking and storage. 
Currently, there is no treatment for many of Lysosomal Storage 
Disorders. In some cases, such as Gaucher disease, enzyme replacement 
therapy and substrate deduction treatment are available with very high 
cost (over $100,000 per patient per year). NIH investigators have 
identified an unexpected and previously unrecognized use for delta 
tocopherol, which is a form of vitamin E, in the treatment of diseases 
and conditions related to lysosomal storage disorders. Scientists at 
the National Center for Advancing Translational Sciences, NIH 
discovered a clear difference between the effects of delta-tocopherol 
and alpha tocopherol on the cell-based disease models of Niemann Pick C 
(NPC) disease. They found that while delta-tocopherol significantly 
reduced the cholesterol accumulation in NPC cells and reduced the size 
of enlarged lysosomes, alpha-tocopherol only showed weak effects in the 
same cells.
    The present invention can be used to develop new therapies 
involving delta-tocopherol to treat lysosomal disorders, such as 
Niemann-Pick type C disease, Mucopolysaccharidoses disorder, and 
Neuronal Ceroid Lipofuscinoses. This invention provides potential novel 
methods for the modulation of cholesterol and other lipids' recycling. 
It may be also possible to use delta-tocopherol for the reduction of 
the size of enlarged lysosomes caused by accumulation of lipids and 
macromolecules.
    Potential Commercial Applications:
     Therapeutics for lysosomal disorders
     Therapeutics for Niemann-Pick type C disease
    Competitive Advantages: delta-tocopherol is a novel lead compound 
for drug development to treat a variety of lysosomal storage diseases 
characterized by lipid/macromolecule accumulation and defective lipid 
trafficking.
    Development Stage:
     Early-stage
     In vitro data available
    Inventors: Wei Zheng et al. (NCATS).
    Publications:

1. Xu M, et al. delta-Tocopherol reduces lipid accumulation in 
Niemann-Pick type C1 and Wolman cholesterol storage disorders. J 
Biol Chem. 2012 Nov 16;287(47):39349-60. [PMID 23035117]
2. Yu D, et al. Niemann-Pick Disease Type C: Induced Pluripotent 
Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and 
Evaluating Drug Efficacy. J Biomol Screen. 2014 Jun 6. pii: 
1087057114537378. [PMID 24907126]

    Intellectual Property: HHS Reference No. E-294-2009/0--
     US Patent Application No. 13/810,774 filed 17 Jan 2013
     EP Patent Application No. 11741023.3 filed 19 July 2011
    Related Technology: HHS Reference No. E-148-2011/0--PCT Patent 
Application No. PCT/US2013070156 filed 14 Nov 2013, entitled 
``Tocopherol and Tocopheryl Quinone Derivatives as Correctors of 
Lysosomal Storage Disorders.''
    Licensing Contact: Suryanarayana Vepa, Ph.D., J.D.; 301-435-5020; 
[email protected].
    Collaborative Research Opportunity: The National Center for 
Advancing Translational Sciences is seeking statements of capability or 
interest from parties interested in collaborative research to further 
develop, evaluate, or commercialize particular therapeutic uses of 
delta tocopherol. Please contact Dr. Wei Zheng at [email protected] 
for more information.

\18\F-Labeled Calcofluor Derivatives for PET Imaging and Diagnosis of 
Aspergillus Infection

    Description of Technology: Aspergillus is a common fungal lung 
infection with high mortality rates in immune compromised patients. The 
inability to diagnose this infection impedes treatment. Blood based 
diagnostic tests for this infection lack sensitivity and specificity 
due to cross reactivity. Other methods of diagnosis are invasive and 
labor intensive. The ability to accurately and non-invasively diagnose 
infection in Aspergillus immune compromised populations may greatly 
improve treatment and lower mortality rates. This technology uses 
\18\F-labeled calcofluor derivatives for positron emission tomography 
(PET) imaging of filamentous fungal infections. \18\F-labeled 
calcofluor derivatives have low toxicity, high binding specificity to 
Aspergillus species due to uptake by Aspergillus-specific siderphore 
system, and low binding affinity to patient tissue. These compounds may 
be used for rapid and accurate PET diagnostic imaging of infection by 
species of Aspergillus.
    Potential Commercial Applications: Diagnosis of Aspergillus 
infection.
    Competitive Advantages: Non-invasive, low toxicity, specific for 
Aspergillus.
    Development Stage: In vivo data available (animal).
    Inventors: Peter Williamson (NIAID), John Panepinto (Univ. 
Buffalo), Dale Kiesewetter (NIBIB), Jin Qui (NIAID).
    Publications:

1. Palmer GE, et al. The diverse roles of autophagy in medically 
important fungi. Autophagy. 2008 Nov;4(8):982-8. [PMID 18927489]
2. Panepinto JC, et al. Deletion of the Aspergillus fumigatus gene 
encoding the Ras-related protein RhbA reduces virulence in a model 
of invasive pulmonary aspergillosis. Infect Immun. 2003 
May;71(5):2819-26. [PMID 12704156]
3. Desoubeaux D, et al., Diagnosis of invasive pulmonary 
aspergillosis: Updates and recommendations, Med Mal Infect. 2014 
Mar; 44(3):89-101. [PMID 24548415]

    Intellectual Property: HHS Reference No. E-449-201/0--US 
Provisional Application No. 61/894,754 filed 23 Oct 2013.
    Licensing Contact: Edward (Tedd) Fenn; 424-297-0336; 
[email protected].
    Collaborative Research Opportunity: The National Institute of 
Allergy and Infectious Diseases is seeking statements of capability or 
interest from parties interested in collaborative research to further 
develop, evaluate, or commercialize this technology. For collaboration 
opportunities, please contact Nadine Chien at 301-827-0258.

Multifunctional RNA Nanoparticles as Therapeutic Agents

    Description of Technology: The promise of RNA interference based 
therapeutics is made evident by the recent surge of biotechnological 
drug companies that pursue such therapies and their progression into 
human clinical trials. The present invention discloses novel RNA and 
RNA/DNA nanoparticles including multiple siRNAs, RNA aptamers, 
fluorescent dyes, and proteins. These RNA nanoparticles are useful for 
various nanotechnological applications. This technology has a higher 
detection sensitivity and higher silencing efficiencies of targeted 
genes than conventional siRNAs. This technology has significant 
therapeutic potential against multiple disease types, including cancer 
and viral infections.
    Potential Commercial Applications:
     Treatment for various diseases

[[Page 40122]]

     Clinical research
     Basic research
    Competitive Advantages:
     More sensitivity
     Higher efficiency
     Low cytotoxicity
     Multiple functionality
     Multiple targets
     Visualization
     Controlled activation
    Development Stage:
     In vitro data available
     In vivo data available
    Inventors: Bruce A. Shapiro, Kirill A. Afonin, Angelica N. Martins, 
Mathias D. Viard (all of NCI)
    Intellectual Property: HHS Reference No. E-765-2013/0--US 
Provisional Application No. 61/878,758 filed 17 Sep 2013.
    Related Technologies:
     HHS Reference No. E-039-2012
     HHS Reference No. E-156-2014
    Licensing Contact: John Stansberry, Ph.D.; 301-435-5236; 
[email protected]
    Collaborative Research Opportunity: The National Cancer Institute 
is seeking statements of capability or interest from parties interested 
in collaborative research to further develop, evaluate or commercialize 
scaling up, animal models, multiple targets, delivery. For 
collaboration opportunities, please contact John D. Hewes, Ph.D. at 
[email protected].

Nucleic Acid Nanoparticles for Triggering RNA Interference

    Description of Technology: RNA interference (RNAi) is a naturally 
occurring cellular post-transcriptional gene regulation process that 
utilizes small double-stranded RNAs to trigger and guide gene 
silencing. By introducing synthetic RNA duplexes called small-
interfering RNAs (siRNAs), we can harness the RNAi machinery for 
therapeutic gene control and the treatment of various diseases.
    The present invention discloses RNA, RNA-DNA, DNA-RNA, hybrid 
nanocubes consisting of a DNA or RNA core (composed of six strands) 
with attached RNA or DNA hybrid duplexes. The nanocubes can induce the 
reassociation of the RNA duplexes, which can then be processed by the 
human recombinant Dicer enzyme, thus activating RNAi. This technology 
opens a new route for the development of ``smart'' nucleic acid based 
nanoparticles for a wide range of biomedical applications. Immune 
responses can be controlled by altering the composition of the 
particles.
    Potential Commercial Applications:
     Treatment for various diseases
     Clinical research
     Basic research
    Competitive Advantages:
     Low cytotoxicity
     Chemical stability
     More specificity
     Controlled activation
     Multiple targets
     Visualization
    Development Stage: In vitro data available
    Inventors: Bruce A. Shapiro, Kirill A. Afonin, Mathias D. Viard 
(all of NCI)
    Publications:
    1. Afonin KA, et al. Computational and experimental 
characterization of RNA cubic nanoscaffolds. Methods. 2014 May 
15;67(2):256-65. [PMID 24189588]
    2. Afonin KA, et al. In vitro assembly of cubic RNA-based 
scaffolds designed in silico. Nat Nanotechnol. 2010 Sep;5(9):676-82. 
[PMID 20802494]

    Intellectual Property: HHS Reference No. E-156-2014/0--US 
Provisional Application 61/989,520 filed 06 May 2014
    Related Technologies:
     HHS Reference No. E-765-2013
     HHS Reference No. E-039-2012
    Licensing Contact: John Stansberry, Ph.D.; 301-435-5236; 
[email protected]
    Collaborative Research Opportunity: The National Cancer Institute 
is seeking statements of capability or interest from parties interested 
in collaborative research to further develop, evaluate or commercialize 
scaling up, animal models, multiple targets, delivery. For 
collaboration opportunities, please contact John D. Hewes, Ph.D. at 
[email protected].

    Dated: July 10, 2014.
Richard U. Rodriguez,
Director, Division of Technology Development and Transfer, Office of 
Technology Transfer, National Institutes of Health.
[FR Doc. 2014-16265 Filed 7-10-14; 8:45 am]
BILLING CODE 4140-01-P