Government-Owned Inventions; Availability for Licensing, 73549-73551 [2013-29096]
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emcdonald on DSK67QTVN1PROD with NOTICES
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[FR Doc. 2013–29185 Filed 12–5–13; 8:45 am]
BILLING CODE 4120–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
Government-Owned Inventions;
Availability for Licensing
AGENCY:
National Institutes of Health,
HHS.
ACTION:
Notice.
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
SUMMARY:
PO 00000
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73549
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; ps193c@nih.gov.
Collaborative Research Opportunity:
The National Institute of Neurological
Disorders and Stroke is seeking
statements of capability or interest from
parties interested in collaborative
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Federal Register / Vol. 78, No. 235 / Friday, December 6, 2013 / Notices
research to further develop, evaluate or
commercialize anti-JCV antisense
cocktails. For collaboration
opportunities, please contact Melissa
Maderia, Ph.D. at maderiam@
mail.nih.gov or 240–276–5533.
emcdonald on DSK67QTVN1PROD with NOTICES
A Novel HIV–1 Anti-HIV and AntiRetroviral 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
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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; hus@
mail.nih.gov.
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
Marguerite.Miller@nih.gov 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 byproducts 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 liverspecific 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 noninvasive means of assessing the efficacy
of renal-targeted therapies of all classes
PO 00000
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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; vince.contreras@
nih.gov.
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 venditti@mail.nih.gov 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
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Federal Register / Vol. 78, No. 235 / Friday, December 6, 2013 / Notices
emcdonald on DSK67QTVN1PROD with NOTICES
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-oforigin 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;
eggerton.campbell@nih.gov.
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 hewesj@
mail.nih.gov.
AAV-Aquaporin-1 Gene Therapy for
¨
Sjogren’s Syndrome
¨
Description of Technology: Sjogren’s
syndrome is a chronic inflammatory
disease affecting over 2 million
Americans, whereby moistureproducing 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 Sjogren’s syndrome. This
technology consists of local delivery of
adeno-associated virus (AAV) mediated
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aquaporin-1 (AQP1) fusion protein to
salivary glands. Using a murine model
¨
that mimics Sjogren’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 Sjogren’s
syndrome.
Potential Commercial Applications:
Prevention of dry mouth (xerostomia)
associated with salivary gland
¨
dysfunction in patients with Sjogren’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; vince.contreras@
nih.gov.
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 Sjogren’s. For collaboration
opportunities, please contact David
Bradley at bradleyda@nidcr.nih.gov.
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73551
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]
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HUMAN SERVICES
National Institutes of Health
National Institute of Diabetes and
Digestive and Kidney Diseases; Notice
of Closed Meeting
Pursuant to section 10(d) of the
Federal Advisory Committee Act, as
amended (5 U.S.C. App.), notice is
hereby given of the following meeting.
The meeting will be closed to the
public in accordance with the
provisions set forth in sections
552b(c)(4) and 552b(c)(6), Title 5 U.S.C.,
as amended. The grant applications and
the discussions could disclose
confidential trade secrets or commercial
property such as patentable material,
and personal information concerning
individuals associated with the grant
applications, the disclosure of which
would constitute a clearly unwarranted
invasion of personal privacy.
Name of Committee: National Institute of
Diabetes and Digestive and Kidney Diseases
Special Emphasis Panel; Digestive Diseases
Ancillary Study.
Date: December 17, 2013
Time: 11:00 a.m. to 12:00 p.m.
Agenda: To review and evaluate grant
applications.
Place: National Institutes of Health, Two
Democracy Plaza, 6707 Democracy
Boulevard, Bethesda, MD 20892, (Telephone
Conference Call).
Contact Person: Thomas A. Tatham, Ph.D.,
Scientific Review Officer, Review Branch,
DEA, NIDDK, National Institutes of Health,
Room 760, 6707 Democracy Boulevard,
Bethesda, MD 20892–5452, (301) 594–3993,
tathamt@mail.nih.gov.
This notice is being published less than 15
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(Catalogue of Federal Domestic Assistance
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Research; 93.849, Kidney Diseases, Urology
and Hematology Research, National Institutes
of Health, HHS)
Dated: November 29, 2013.
David Clary,
Program Analyst, Office of Federal Advisory
Committee Policy.
[FR Doc. 2013–29098 Filed 12–5–13; 8:45 am]
BILLING CODE 4140–01–P
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]]>Agencies
[Federal Register Volume 78, Number 235 (Friday, December 6, 2013)]
[Notices]
[Pages 73549-73551]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-29096]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
National Institutes of Health
Government-Owned Inventions; Availability for Licensing
AGENCY: National Institutes of Health, 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. 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;
ps193c@nih.gov.
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 maderiam@mail.nih.gov 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;
hus@mail.nih.gov.
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 Marguerite.Miller@nih.gov 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;
vince.contreras@nih.gov.
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 venditti@mail.nih.gov 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;
eggerton.campbell@nih.gov.
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
hewesj@mail.nih.gov.
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;
vince.contreras@nih.gov.
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 bradleyda@nidcr.nih.gov.
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]
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