[Federal Register Volume 78, Number 235 (Friday, December 6, 2013)]
[Notices]
[Pages 73549-73551]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-29096]
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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 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.
Use of Antisense Oligodeoxynucleotides (ODNs) for Inhibiting JC Virus
(JCV)
Description of Technology: Progressive multifocal
leukoencephalopathy (PML) is a rare, fatal demyelinating disease of the
brain caused by the polyomavirus JC (JCV) under immunosuppressive
conditions. It is pathologically characterized by progressive damage of
white matter of the brain by destroying oligodendrocytes at multiple
locations. Clinically, PML symptoms include weakness or paralysis,
vision loss, impaired speech, and cognitive deterioration. The
prognosis of PML is generally poor. No effective therapy for PML has
been established. The current strategies to develop a PML therapy focus
on blocking viral infection or inhibiting JCV replication. Antisense
oligodeoxynucleotides (ODNs) that can block JCV replication and
multiplication have been identified and optimized. Use of the ODNs
provide a method of inhibiting JCV replication and thereby provide a
treatment for PML.
Potential Commercial Applications:
JCV/PML Therapeutics.
JCV Diagnostics.
JCV Kits.
Competitive Advantages:
Low cost PML therapeutics.
Lower cost JCV diagnostics.
Ease of synthesis.
Development Status:
Pre-clinical.
In vitro data available.
In vivo data available (animal).
Inventors: Laura B. Jaeger, Avindra Nath, Eugene O. Major (all of
NINDS).
Intellectual Property: HHS Reference No. E-547-2013/0--US
Provisional Application No. 61/879,833, filed 19 Sep 2013.
Licensing Contact: Peter Soukas, J.D.; 301-435-4646;
[email protected].
Collaborative Research Opportunity: The National Institute of
Neurological Disorders and Stroke is seeking statements of capability
or interest from parties interested in collaborative
[[Page 73550]]
research to further develop, evaluate or commercialize anti-JCV
antisense cocktails. For collaboration opportunities, please contact
Melissa Maderia, Ph.D. at [email protected] or 240-276-5533.
A Novel HIV-1 Anti-HIV and Anti-Retroviral Compound
Description of Technology: The subject invention describes the
thioether prodrug that targets the highly conserved nucleocapsid
protein 7 (NCp7) of HIV. In contrast to clinically approved anti-
retroviral drugs used to treat HIV, the virus is not able to develop
resistance to the drug in this invention. In addition, the prodrug is
stable at room temperature, crystalline, easily synthesized in two
steps on the kilogram scale from inexpensive starting materials, orally
bioavailable, and is non-toxic in all animal models investigated to
date. There is potential to use the molecule described in the invention
as an orally administered systemic drug for the treatment of HIV
infection either alone or in combination with other approved anti-
retroviral therapies.
Animal safety testing is in process as are efficacy studies.
Potential Commercial Applications:
HIV therapeutics.
Prophylactics.
Topical application.
Competitive Advantages:
Does not develop resistance due to the high sequence
conservation of the target.
More stable than thioesters.
Development Stage:
Early-stage.
Pre-clinical.
In vitro data available.
In vivo data available (animal).
Inventors: Daniel Appella, Pankaj Kumar, Nathaniel Shank, Matthew
Hassink (all of NIDDK).
Publications:
1. Goudreau N, et al. Discovery and structural characterization of
a new inhibitor series of HIV-1 nucleocapsid function: NMR solution
structure determination of a ternary complex involving a 2:1 inhibitor/
NC stoichiometry. J Mol Biol. 2013 Jun 12;425(11):1982-98. [PMID
23485336]
2. Ouyang W, et al. Probing the RNA Binding Surface of the HIV-1
Nucleocapsid Protein by Site-Directed Mutagenesis. Biochemistry
2013;52(19):3358-68. [PMID 23594178]
Intellectual Property: HHS Reference No. E-539-2013/0--US
Provisional Application No. 61/874,182 filed 05 September 2013.
Related Technologies: HHS Reference No. E-177-2010 family which is
abandoned. However, the subject compound was described in PCT
Application No. PCT/US2011/039909 (E-177-2010/0-PCT-02).
Licensing Contact: Sally H. Hu, Ph.D., M.B.A.; 301-435-5606;
[email protected].
Collaborative Research Opportunity: The NIDDK Technology
Advancement Office is seeking statements of capability or interest from
parties interested in collaborative research to further develop,
evaluate or commercialize this anti-retroviral drug that targets the
nucleocapsid protein 7 (NCp7). For collaboration opportunities, please
contact Marguerite J. Miller at [email protected] or 301-496-
9003.
Mouse Model for Methylmalonic Acidemia, an Inherited Metabolic Disorder
Description of Technology: Methylmalonic Acidemia (MMA) is a
metabolic disorder affecting 1 in 25,000 to 48,000 individuals
globally. MMA is characterized by increased acidity in the blood and
tissues due to toxic accumulation of protein and fat by-products
resulting in seizures, strokes, and chronic kidney failure. About 60%
of MMA cases stem from mutations in the methylmalonyl CoA mutase (MUT)
gene encoding a key enzyme required to break down amino acids and
lipids. Previous efforts to develop mice with null mutations in MUT
have been unsuccessful, as such mutations result in neonatal death.
The inventors have developed the first transgenic mouse model
available for the long-term study of Mut deficiency, in which low level
liver-specific expression of the MUT enzyme confers rescue from
neonatal lethality and replicates induction of the severe renal
symptoms consistent with human MMA. This model could serve as a
valuable research tool for designing treatments for MMA renal disease
or a platform for pre-clinical toxicology screening of compounds with
potential renal side effects.
Potential Commercial Applications:
Model for examining renoprotective antioxidants or
treatments for kidney failure resulting from drug toxicity,
mitochondrial dysfunction, environmental exposure, or aging.
Used in investigating renoprotective effects of
nutritional supplements from drugs known to cause kidney damage.
Used in discovery of MMA biomarkers.
Competitive Advantages: The model system provides a relatively non-
invasive means of assessing the efficacy of renal-targeted therapies of
all classes and biological types (gene therapy, small molecules,
nutritional supplements, repurposed drugs).
Development Stage:
Early-stage.
Pre-clinical.
In vitro data available.
In vivo data available (animal).
Inventors: Charles P. Venditti and Eirini Manoli (NHGRI).
Publication: Manoli I, et al. Targeting proximal tubule
mitochondrial dysfunction attenuates the renal disease of methylmalonic
acidemia. Proc Natl Acad Sci U S A. 2013 Aug 13;110(33):13552-7. [PMID
23898205]
Intellectual Property: HHS Reference No. E-285-2011/1--Research
Material. Patent protection is not being pursued for this technology.
Licensing Contact: Vince Contreras, Ph.D.; 301-435-4711;
[email protected].
Collaborative Research Opportunity: The National Human Genome
Research Institute, Organic Acid Research Section, is seeking
statements of capability or interest from parties interested in
collaborative research to further develop, evaluate or commercialize
renotherapeutic or renoprotective small molecules, gene and/or cell
therapies to treat MMA. For collaboration opportunities, please contact
Charles P. Venditti, M.D., Ph.D. at [email protected] or 301-496-
6213.
Reporter Plasmid To Identify Cancer Stem Cells
Description of Technology: Scientists at the NIH have developed a
research tool, an efficient lentiviral plasmid to visualize and purify
cancer stem cells, which is useful for screening compounds that
specifically kill or inhibit cancer stem cells. Cancer stem cells are a
minority population of cells that initiate and sustain tumors. These
cells are resistant to therapy and may cause tumors to recur after
curative treatment. Current therapies generally do not target cancer
stem cells. The key feature of the plasmid is a reporter system that
only detects cells expressing the core stem cell transcription factors
Sox2 and Oct4. The plasmid can identify the putative cancer stem cell
population through the expression of fluorescent or luminescent
proteins and has the potential to advance new therapies.
Potential Commercial Applications:
Laboratory tool to visualize, quantify and purify cancer
stem cells.
Research tool to monitor cancer stem cells in transplanted
tumors in vivo.
Research tool to identify cancer stem cells in high
through-put screening
[[Page 73551]]
of libraries for compounds that specifically inhibit or kill cancer
stem cells.
Research tool to optimize therapeutic regimens in
preclinical models.
Potential to support precision medicine approach by
screening therapeutics for efficacy against cancer stem cells in
patient-derived xenografts.
Competitive Advantages:
Efficient visualization of cancer stem cells by functional
property rather than by use of highly variable cell surface markers.
Flexible modular Gateway cloning technology allows
constructs with alternative reporters to be readily generated.
Approach is independent of cell-of-origin of tumor.
Cancer stem cell behavior can be monitored in real-time.
Development Stage:
Pre-clinical.
In vitro data available.
In vivo data available (animal).
Inventors: Lalage Wakefield and Binwu Tang (NCI).
Publication: Manuscript under review. Text available on request.
Intellectual Property: HHS Reference No. E-141-2011/0--Research
Tool. Patent protection is not being pursued for this technology.
Licensing Contact: Eggerton Campbell, Ph.D.; 301-435-5282;
[email protected].
Collaborative Research Opportunity: The National Cancer Institute,
Laboratory of Cancer Biology and Genetics, is seeking statements of
capability or interest from parties interested in collaborative
research to further develop, evaluate or commercialize a cancer stem
cell reporter construct for use in drug screens and therapy selection.
For collaboration opportunities, please contact John D. Hewes, Ph.D. at
[email protected].
AAV-Aquaporin-1 Gene Therapy for Sj[ouml]gren's Syndrome
Description of Technology: Sj[ouml]gren's syndrome is a chronic
inflammatory disease affecting over 2 million Americans, whereby
moisture-producing glands are attacked by the body's immune system. The
disease is marked by disabling dryness of the mouth and eyes as well as
fatigue and pain. Researchers at the National Institute of Dental and
Craniofacial Research have developed a therapy that alleviates
xerostomia in an animal model of Sj[ouml]gren's syndrome. This
technology consists of local delivery of adeno-associated virus (AAV)
mediated aquaporin-1 (AQP1) fusion protein to salivary glands. Using a
murine model that mimics Sj[ouml]gren's dry mouth symptoms, it was
discovered that treatment restored salivary fluid movement upon
expression of AQP1. Targeted delivery of the AAV-AQP1 system makes this
invention a novel and potential long-term therapeutic for restoration
of exocrine gland function and prevention of xerostomia-associated pain
associated with Sj[ouml]gren's syndrome.
Potential Commercial Applications: Prevention of dry mouth
(xerostomia) associated with salivary gland dysfunction in patients
with Sj[ouml]gren's syndrome.
Competitive Advantages:
AAV gene transfer to salivary glands is highly efficient.
AAV-AQP1 promotes de novo salivary flow.
Development Stage:
Pre-clinical.
In vitro data available.
In vivo data available (animal).
Inventor: John (Jay) Chiorini (NIDCR).
Intellectual Property: HHS Reference No. E-139-2011/1--US
Provisional Application No. 61/695,753 filed 31 August 2012; PCT
Application No. PCT/US13/57632 filed 30 August 2013.
Related Technologies:
HHS Reference No. E-179-2005/0--US Patent No. 8,283,151
issued 09 October 2012.
HHS Reference No. E-087-2011/0--US Provisional Application
No. 61/476,168 filed 15 April 2011.
HHS Reference No. E-127-1998/0--US Provisional Application
No. 60/087,029 filed 28 May 1998; US Patent No. 7,479,554 issued 20
January 2009; US Patent No. 6,984,517 issued 10 January 2006.
HHS Reference No. E-142-2011/0--US Provisional Application
No. 61/477,523 filed 20 April 2011.
Licensing Contact: Vince Contreras, Ph.D.; 301-435-4711;
[email protected].
Collaborative Research Opportunity: The National Institute of
Dental and Craniofacial Research, AAV Biology Section, is seeking
statements of capability or interest from parties interested in
collaborative research to further develop, evaluate or commercialize
AAV-Aquaporin-1 Gene Therapy for Sj[ouml]gren's. For collaboration
opportunities, please contact David Bradley at [email protected].
Dated: December 2, 2013.
Richard U. Rodriguez,
Director, Division of Technology Development and Transfer, Office of
Technology Transfer, National Institutes of Health.
[FR Doc. 2013-29096 Filed 12-5-13; 8:45 am]
BILLING CODE 4140-01-P