Government-Owned Inventions; Availability for Licensing, 73549-73551 [2013-29096]

Download as PDF Federal Register / Vol. 78, No. 235 / Friday, December 6, 2013 / Notices • Presenters must clearly explain the actions that they are requesting CMS to take in the appropriate section of the form. A presenter’s relationship with the organization that they represent must also be clearly listed. • The form is now available through the CMS Forms Web site. The Uniform Resource Locator (URL) for linking to this form is as follows: http:// www.cms.hhs.gov/cmsforms/ downloads/cms20017.pdf IV. Oral Comments In addition to formal oral presentations, which are limited to 5 minutes total per presentation, there will be an opportunity during the meeting for public oral comments, which will be limited to 1 minute for each individual and a total of 3 minutes per organization. V. Meeting Attendance The meeting is open to the public; however, attendance is limited to space available. Priority will be given to those who pre-register, and attendance may be limited based on the number of registrants and the space available. Persons wishing to attend this meeting, which is located on Federal property, must register by following the instructions in the ‘‘Meeting Registration Timeframe’’ section of this notice. A confirmation email will be sent to the registrants shortly after completing the registration process. emcdonald on DSK67QTVN1PROD with NOTICES VI. Security, Building, and Parking Guidelines The following are the security, building, and parking guidelines: • Persons attending the meeting, including presenters, must be preregistered and on the attendance list by the prescribed date. • Individuals who are not preregistered in advance may not be permitted to enter the building and may be unable to attend the meeting. • Attendees must present valid photo identification to the Federal Protective Service or Guard Service personnel before entering the building. Without a current, valid photo ID, persons may not be permitted entry to the building. • Security measures include inspection of vehicles, inside and out, at the entrance to the grounds. • All persons entering the building must pass through a metal detector. • All items brought into CMS including personal items, for example, laptops and cell phones are subject to physical inspection. • The public may enter the building 30 to 45 minutes before the meeting convenes each day. VerDate Mar<15>2010 17:28 Dec 05, 2013 Jkt 232001 • All visitors must be escorted in areas other than the lower and first-floor levels in the Central Building. • The main-entrance guards will issue parking permits and instructions upon arrival at the building. VII. Special Accommodations Individuals requiring sign-language interpretation or other special accommodations must include the request for these services during registration. VIII. Panel Recommendations and Discussions The Panel’s recommendations at any Panel meeting generally are not final until they have been reviewed and approved by the Panel on the last day of the meeting, before the final adjournment. These recommendations will be posted to our Web site after the meeting. IX. Collection of Information Requirements This document does not impose information collection and recordkeeping requirements. Consequently, it need not be reviewed by the Office of Management and Budget under the authority of the Paperwork Reduction Act of 1995 (44 U.S.C. 35). (Catalog of Federal Domestic Assistance Program; No. 93.773 Medicare—Hospital Insurance Program; and No. 93.774, Medicare—Supplementary Medical Insurance Program) Dated: November 29, 2013. Marilyn Tavenner, Administrator, Centers for Medicare & Medicaid Services. [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 Frm 00052 Fmt 4703 Sfmt 4703 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 E:\FR\FM\06DEN1.SGM 06DEN1 73550 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 VerDate Mar<15>2010 17:28 Dec 05, 2013 Jkt 232001 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 Frm 00053 Fmt 4703 Sfmt 4703 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 E:\FR\FM\06DEN1.SGM 06DEN1 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 VerDate Mar<15>2010 17:28 Dec 05, 2013 Jkt 232001 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. PO 00000 Frm 00054 Fmt 4703 Sfmt 9990 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] BILLING CODE 4140–01–P DEPARTMENT OF HEALTH AND 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 days prior to the meeting due to the timing limitations imposed by the review and funding cycle. (Catalogue of Federal Domestic Assistance Program Nos. 93.847, Diabetes, Endocrinology and Metabolic Research; 93.848, Digestive Diseases and Nutrition 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 E:\FR\FM\06DEN1.SGM 06DEN1

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]
BILLING CODE 4140-01-P