Government-Owned Inventions; Availability for Licensing, 77884-77885 [2010-31320]

Agencies

• DEPARTMENT OF HEALTH AND HUMAN SERVICES
• National Institutes of Health

[Federal Register Volume 75, Number 239 (Tuesday, December 14, 2010)]
[Notices]
[Pages 77884-77885]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2010-31320]



[[Page 77884]]

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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, Public Health Service, 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. 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.

Pyruvate Kinase M2 Activators for the Treatment of Cancer

    Description of Invention: NIH investigators have discovered a 
series of small compounds with the potential to treat a variety of 
cancers as well as hemolytic anemia. Contrary to most cancer 
medications, these molecules can be non-toxic to normal cells because 
they target a protein specific to the metabolic pathways in tumors, 
thus representing a significant clinical advantage over less-specific 
chemotherapeutics.
    The invention described here is a series of small molecules that 
activate pyruvate kinase (PK) isoform M2. PK-M2 is a critical metabolic 
enzyme that is affected in all forms of cancer. Inactivation of PK-M2 
leads to a buildup of metabolic intermediates inside the cell. Tumor 
cells require a buildup of metabolic intermediates in order to undergo 
rapid cell growth and proliferation. Hence, activation of PK-M2 in 
tumor cells may prevent the buildup of metabolic intermediates and 
thereby stall tumor cell proliferation or destroy the tumor cells. 
Further, while in normal post-embryonic cells only PK isoforms R, L, or 
M1 are active, in all tumors only PK-M2 is active. So, PK-M2 activation 
would affect only tumor cells, and small-molecule PK-M2 activators may 
not be toxic to healthy cells.
    This invention discloses the use of two new small molecule 
pharmacophores that can activate PKM2 through the allosteric site: 3-
oxo-3,4-dihydro-2H-benzo [b] [1,4] oxazine-7-sulfonamides, and 2-oxo-
1,2,3,4-tetrahydroquinoline-6-sulfonamides.
    Applications:
     Therapeutic developments for various cancers.
     Diagnostic assays for various cancers.
     Regulation of embryonic stem cell proliferation.
    Advantages:
     Small molecule (series of analogs can be derived in search 
of improved performance).
     Target a select group of cells (Cancerous cells).
    Development Status:
     Pre-clinical; no animal data.
     In vitro data available.
    Market:
     Cancer-diagnostics.
     Cancer-therapeutics.
     Research tool-proliferation of embryonic stem cells and/or 
cancer cells.
    Inventors: Matthew Boxer (NHGRI-NCGC); Min Shen (NHGRI-NCGC); Doug 
Auld (NHGRI-NCGC); Craig Thomas (NHGRI-NCGC).
    Publications:
    1. Jiang JK et al. (2010) Bioorg Med Chem Lett 20:3387-93 [PubMed: 
20451379].
    2. Boxer MB et al. (2010) J Med Chem. 53:1048-55 [PubMed: 
20017496].
    Patent Status: U.S. Provisional Application No. 61/329,158 filled 
29 April 2010 (HHS Reference No. E-120-2010/0-US-01).
    Licensing Status: Available for licensing.
    Licensing Contact: Steven H. Standley, PhD; 301-435-4074; 
sstand@mail.nih.gov.
    Collaborative Research Opportunity: The NIH Chemical Genomics 
Center (NCGC), National Human Genome Research Institute, is seeking 
statements of capability or interest from parties interested in 
collaborative research to further develop, evaluate, or commercialize 
these pyruvate kinase M2 activators. Please contact Dr. Matthew Boxer 
at boxerm@mail.nih.gov for more information.

Nitisinone for Treatment of Oculocutaneous/Ocular Albinism and for 
Increasing Pigmentation

    Description of Invention: Albinism (also called achromia, 
achromasia, or achromatosis) is a congenital disorder characterized by 
the complete or partial absence of pigment in the skin, hair and eyes 
due to absence or defect in any one of a number of proteins involved in 
the production of melanin. Certain forms of albinism are known to be 
due to mutations in tyrosine metabolism. In oculocutaneous albinism 
(OCA), pigment is lacking in the eyes, skin and hair. In ocular 
albinism, only the eyes lack pigment. Patients with albinism experience 
varying degrees of vision loss associated with foveal hypoplasia, 
nystagmus, photophobia and/or glare sensitivity, refractive errors, and 
abnormal decussation of ganglion cell axons at the optic chiasm. 
Current treatment options for vision problems caused by albinism are 
limited to correction of refractive errors and amblyopia, low vision 
aids, and (in some cases) extraocular muscle surgery.
    Nitisinone (NTBC) is an FDA-approved drug used in the treatment of 
tyrosinemia, type 1. The drug blocks the normal degradation pathway of 
tyrosine thus allowing greater circulating plasma levels of tyrosine. 
NIH investigators have identified that administration of NTBC to 
subjects (e.g., mice or humans) with certain forms of albinism, can 
result in increased circulating tyrosine levels, an increase in 
tyrosinase activity, and, subsequently, increased pigmentation.
    This technology provides methods for increasing tyrosine plasma 
concentrations in patients suffering from oculocutaneous albinism or 
ocular albinism by administering a pharmaceutically acceptable 
composition of NTBC. Specifically, this technology can be useful in 
treating patients with type OCA1a albinism, who possess no measurable 
tyrosinase activity, or type OCA1b albinism, who exhibit greatly 
diminished tyrosinase activity.
    Applications for this technology include treatment of impaired 
vision in patients suffering from oculocutaneous albinism, or ocular 
albinism, and as a treatment for increasing pigmentation in the eyes, 
hair and/or skin of patients.
    Inventors: Brian P. Brooks (NEI), David R. Adams (NHGRI), William 
A. Gahl (NHGRI).
    Patent Status: U.S. Provisional Application No. 61/308,771 filed 26 
Feb 2010 (HHS Reference No. E-113-2010/0-US-01).
    Licensing Status: Available for licensing.
    Licensing Contact: Suryanarayana (Sury) Vepa, PhD, J.D.; 301-435-
5020; vepas@mail.nih.gov.
    Collaborative Research Opportunity: The National Eye Institute, 
Ophthalmic

[[Page 77885]]

Genetics and Visual Function Branch, is seeking statements of 
capability or interest from parties interested in collaborative 
research to further develop, evaluate, or commercialize the use of 
nitisinone (NTBC) for oculocutaneous albinism or as a treatment for 
increasing pigmentation in the eyes, hair and/or skin of patients. 
Please contact Alan Hubbs, PhD at 301-594-4263 or hubbsa@mail.nih.gov 
for more information.

Modulators of Survival Motor Neuron Production

    Description of Invention: This technology discloses compounds that 
modulate the amount of Survival Motor Neuron protein (SMN). Low levels 
of SMN protein are associated with Spinal Muscular Atrophy (SMA), which 
constitutes a group of inherited diseases that cause progressive muscle 
degeneration leading to death. Consequently, therapeutic inventions 
have focused on increasing SMN protein levels. This invention discloses 
novel arylthiazolyl piperidines which are shown to be modulators of SMN 
production. This invention also discloses methods of treating SMA by 
administering SMN protein modulators.
    Applications: Therapeutic developments for Spinal Muscular Atrophy.
    Advantages: Small molecule (series of analogs can be derived in 
search of improved performance).
    Development Status:
     Pre-clinical; no animal data.
     In vitro data available.
    Market: Muscular dystrophy.
    Inventors: Juan Jose Marugan (NHGRI-NCGC); Wei Zheng (NHGRI-NCGC); 
Noel Southall (NHGRI-NCGC); Jingbo Xiao (NHGRI-NCGC); Steve Titus 
(NHGRI-NCGC); Elliot Androphy (University of Massachusetts Medical 
School); Jonathan Cherry (University of Massachusetts Medical School).
    Patent Status: U.S. Provisional Application No. 61/323,963 filed 14 
April 2010 (HHS Reference No. E-109-2010/0-US-01).
    Licensing Status: Available for licensing.
    Licensing Contact: Steven H. Standley, PhD; 301-435-4074; 
sstand@mail.nih.gov.
    Collaborative Research Opportunity: The NIH Chemical Genomics 
Center (NCGC), National Human Genome Research Institute, is seeking 
statements of capability or interest from parties interested in 
collaborative research to further develop, evaluate, or commercialize 
these SMN modulator compounds. Please contact Dr. Juan Marugan at 
maruganj@mail.nih.gov for more information.

Use of Sterculic Acid To Treat Choroidal Neovascularization

    Description of Invention: Sterculic acid is a naturally occurring 
cyclopropene acid present in kapok seed oil, cottonseed oil, and in the 
seeds of the Sterculia foetida tree. Sterculic acid has been reported 
to be a non-specific inhibitor of stearoyl-Co desaturase (SCD), which 
has been implicated in several disease states, including cardiovascular 
disease, obesity, non-insulin-dependent diabetes mellitus, skin 
disease, hypertension, neurological diseases, immune disorders and 
cancer (Ntambi JM, J. Lipid Res., 1999, 40(9):1549-1558). NIH 
investigators have recently discovered that sterculic acid inhibits the 
neovascularization of the chick chorioallantonic membrane demonstrating 
that this compound exhibits a potent anti-angiogenic activity. Further, 
the NIH investigators have shown that sterculic acid inhibits the 
formation of choroidal neovascularization in the retina of laser 
treated rats. These results suggest that sterculic acid possesses anti-
angiogenic effect likely through regulating genes involved in the 
angiogenic process.
    The present invention is directed to methods of using sterculic 
acid for the treatment of inflammation, in particular, 7-
ketocholesterol mediated inflammation, 7-ketocholesterol cytotoxicity, 
or unregulated angiogenesis. Diseases mediated by 7-ketocholesterol-
induced inflammation and 7-ketocholesterol cytotoxicity include 
atherosclerosis age-related macular degeneration, and Alzheimer's 
disease. Diseases mediated by unregulated angiogenesis include certain 
cancers and age-related macular degeneration. Also disclosed are 
methods of treating atherosclerosis or Alzheimer's disease using 
sterculic acid.
    Applications: Therapeutics for inflammation, in particular, 
atherosclerosis, age-related macular degeneration, and Alzheimer's 
disease
    Development Status: Early stage in vitro and animal model data.
    Inventors: Ignacio R. Rodriguez et al. (NEI).
    Patent Status: U.S. Provisional Application No. 61/358,485 filed 25 
Jun 2010 (HHS Reference No. E-092-2010/0-US-01).
    Licensing Status: Available for licensing.
    Licensing Contact: Suryanarayana Vepa, PhD, J.D.; 301-435-5020; 
vepas@mail.nih.gov.
    Collaborative Research Opportunity: The National Eye Institute 
(NEI), Laboratory of Retinal Cell and Molecular Biology, is seeking 
statements of capability or interest from parties interested in 
collaborative research to further develop, evaluate, or commercialize 
sterculic acid, and its derivatives for the treatment of diseases 
related to angiogenesis or mediated by 7-ketocholesterol-induced 
inflammation. Please contact David L. Whitmer, Technology Development 
Coordinator, NEI, at 301-496-4876 or whitmerd@mail.nih.gov for more 
information.

    Dated: December 8, 2010.
Richard U. Rodriguez,
Director, Division of Technology Development and Transfer, Office of 
Technology Transfer, National Institutes of Health.
[FR Doc. 2010-31320 Filed 12-13-10; 8:45 am]
BILLING CODE 4140-01-P