[Federal Register Volume 79, Number 181 (Thursday, September 18, 2014)]
[Notices]
[Pages 56080-56081]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-22210]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Prospective Grant of Exclusive License: Therapeutics for the 
Treatment of Lysosomal Storage Disorders

AGENCY: National Institutes of Health, HHS.

ACTION: Notice.

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SUMMARY: This is notice, in accordance with 35 U.S.C. 209 and 37 CFR 
part 404, that the National Institutes of Health (NIH), Department of 
Health and Human Services (HHS), is contemplating the grant of an 
exclusive license to practice the following invention as embodied in 
the following patent applications:

1. U.S. Provisional Patent Application No. 61/365,712, filed July 19, 
2010 HHS Ref. No. E-294-2009/0-US-01
    Titled: Use of Delta-Tocopherol for the Treatment of Lysosomal 
Storage Disorders
2. PCT Application No. PCT/US2011/044590, filed July 19, 2011 HHS Ref. 
No. E-294-2009/0-PCT-02
    Titled: Use of Delta-Tocopherol for the Treatment of Lysosomal 
Storage Disorders
3. European Patent Application No. 11741023.3, filed July 19, 2011 HHS 
Ref. No. E-294-2009/0-EP-03
    Titled: Use of Delta-Tocopherol for the Treatment of Lysosomal 
Storage Disorders
4. U.S. Patent Application No. 13/810,774, filed January 17, 2013 HHS 
Ref. No. E-294-2009/0-US-04
    Titled: Use of Delta-Tocopherol for the Treatment of Lysosomal 
Storage Disorders
5. U.S. Provisional Patent Application No. 61/679,668, filed on August 
3, 2012 HHS Ref. No. E-050-2012/0-US-01
    Titled: Cyclodextrin for the Treatment of Lysosomal Storage 
Diseases
6. PCT Patent Application No. PCT/US2013/053527, filed on August 3, 
2013 HHS Ref. No. E-050-2012/0-PCT-02
    Titled: Cyclodextrin for the Treatment of Lysosomal Storage 
Diseases
7. U.S. Provisional Patent Application No. 61/727,296, filed November 
16, 2012 HHS Ref. No. E-148-2012/0-US-01
    Titled: Tocopherol and Tocopheryl Quinone Derivatives as Correctors 
of Lysosomal Storage Disorders
8. PCT Application No. PCT/US2013/070156, November 14, 2013 HHS Ref. 
No. E-148-2012/0-PCT-02
    Titled: Tocopherol and Tocopheryl Quinone Derivatives as Correctors 
of Lysosomal Storage Disorders,

to Vtesse, Inc., having a place of business in Cambridge, 
Massachusetts, United States of America. The patent rights in these 
inventions have been assigned to the United States of America.

DATES: Only written comments and/or application for a license which are 
received by the NIH Office of Technology Transfer on or before October 
3, 2014 will be considered.

ADDRESSES: Requests for a copy of the patent application, inquiries, 
comments and other materials relating to the contemplated license 
should be directed to: Suryanarayana Vepa, Office of Technology 
Transfer, National Institutes of Health, 6011 Executive Boulevard, 
Suite 325, Rockville, MD 20852-3804; Email: [email protected]; 
Telephone: (301) 435-5020; Facsimile: (301) 402-0220.

SUPPLEMENTARY INFORMATION: These technologies relate to the use of 
cyclodextrin (CD), delta-tocopherol and their derivatives for the 
treatment of lysosomal storage disorders (LSDs). LSDs are inherited 
metabolic disorders caused by a deficiency in lysosomal enzymes, of 
which approximately fifty (50) have been described to date. These 
diseases usually affect children, many of whom die within several years 
of birth and some following years of dealing with symptoms of the 
disease that may include developmental delay, movement disorders, 
seizures, dementia, deafness and blindness. LSDs affect a significant 
number of individuals and some can be treated with enzyme-replacement 
therapies. However, because enzymes cannot cross the blood-brain 
barrier, replacement therapeutics are unable to address the central 
nervous system manifestations of the disorders. The inventors have 
identified an unexpected and previously unrecognized use for delta-
tocopherol, which is a form of vitamin E, in the treatment of diseases 
and conditions related to LSDs. Further, the inventors showed that CD 
(alpha-, beta- and gamma-CDs) in combination with delta-tocopherol 
synergistically/additively reduced cholesterol accumulation in cells 
derived from patients suffering from Niemann Pick Type C disease (NPC) 
and Wolman diseases. The inventors have also discovered that tocopherol 
and tocopheryl quinone derivatives with side chain modifications (such 
as terminal tri-halogenated methyl groups) exhibit improved 
pharmacokinetics, modulation of mitochondrial potential

[[Page 56081]]

and restoration of some LSDs phenotypes. These technologies can be used 
to develop novel therapeutics for LSDs including NPC, Wolman, Niemann 
Pick Type A, Farber, TaySachs, MSIIIB and CLN2 (Batten) diseases.
    The prospective exclusive license will be royalty bearing and will 
comply with the terms and conditions of 35 U.S.C. 209 and 37 CFR 404. 
The prospective exclusive license may be granted unless, within fifteen 
(15) days from the date of this published Notice, NIH receives written 
evidence and argument that establishes that the grant of the license 
would not be consistent with the requirements of 35 U.S.C. 209 and 37 
CFR 404.
    The fields of use may be limited to ``Use of cyclodextrin, delta-
tocopherol, or derivatives thereof, alone or in combination, for the 
treatment of lysosomal storage disorders in humans.''
    Properly filed competing applications for a license filed in 
response to this notice will be treated as objections to the 
contemplated license. Comments and objections submitted in response to 
this notice will not be made available for public inspection, and, to 
the extent permitted by law, will not be released under the Freedom of 
Information Act, 5 U.S.C. 552.

    Dated: September 12, 2014.
Richard U. Rodriguez,
Director, Division of Technology Development and Transfer, Office of 
Technology Transfer, National Institutes of Health.
[FR Doc. 2014-22210 Filed 9-17-14; 8:45 am]
BILLING CODE 4140-01-P