[Federal Register Volume 81, Number 186 (Monday, September 26, 2016)]
[Notices]
[Pages 66047-66048]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-23134]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Prospective Grant of Exclusive Patent License: Development of 
Adeno-Associated Virus-Based Vectors for the Treatment of Menkes 
Disease and Related Copper Transport Disorders

AGENCY: National Institutes of Health, HHS.

ACTION: Notice.

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SUMMARY: The Eunice Kennedy Shriver National Institute of Child Health 
and Human Development, an institute of the National Institutes of 
Health, Department of Health and Human Services, is contemplating the 
grant of an Exclusive Commercialization Patent License to practice the 
inventions embodied in the U.S. Patents and Patent Applications listed 
in the Supplementary Information section of this notice to Cyprium 
Therapeutics, Inc. (``Cyprium'') located in New York, NY, USA.

DATES: Only written comments and/or applications for a license which 
are received by the National Cancer Institute's Technology Transfer 
Center on or before October 11, 2016 will be considered.

ADDRESSES: Requests for copies of the patent application, inquiries, 
and comments relating to the contemplated Exclusive Commercialization 
Patent License should be directed to: Surekha Vathyam, Ph.D., Senior 
Licensing and Patenting Manager, NCI Technology Transfer Center, 9609 
Medical Center Drive, RM 1E530 MSC 9702, Bethesda, MD 20892-9702 (for 
business mail), Rockville, MD 20850-9702; Telephone: (240) 276-5530; 
Facsimile: (240) 276-5504; Email: [email protected].

SUPPLEMENTARY INFORMATION:

Intellectual Property

    United States Provisional Patent Application No. 62/244,594, filed 
October 21, 2015 and entitled ``Codon-optimized Reduced-size ATP7A cDNA 
and Uses for Treatment of Copper Transport Disorders'' [HHS Reference 
No. E-062-2015/0-US-01].
    The patent rights in these inventions have been assigned and/or 
exclusively licensed to the government of the United States of America.
    The prospective exclusive license territory may be worldwide and 
the field of use may be limited to the use of Licensed Patent Rights 
for the following: ``Development and commercialization of adeno-
associated virus-based vectors for the treatment of Menkes Disease and 
related copper transport disorders.''
    This technology discloses a codon-optimized reduced-size Adenosine 
Triphosphate 7A (ATP7-alpha or ATP7A) cDNA, vectors, and recombinant 
adeno-associated viruses (AAVs) and uses thereof for treatment of 
copper transport disorders. Such uses, include the administration of 
copper in addition to ATP7A in order to maximize the advantage of the 
gene therapy.
    Human P-type ATPase copper-transporting ATPase 1 (ATP7A) transports 
copper from enterocytes (where it is taken up from dietary copper) into 
the blood. ATP7A also mediates passage of copper across the blood-
cerebrospinal fluid barrier and the blood-brain barrier. In Menkes 
disease and occipital horn syndrome (OHS), ATP7A activity is reduced or 
absent and copper export from the enterocytes is impaired. As a result, 
copper accumulates in intestinal cells and less copper is delivered to 
the blood, resulting in restricted copper supply to other tissues, 
particularly the brain. If successfully developed, this invention would 
be a first of its kind therapy for treating copper transport disorders, 
such as Menkes disease, OHS, or ATP7A-related distal motor neuropathy, 
by administering the disclosed nucleic acid, vector, or recombinant 
virus to a subject with a copper transport disorder.
    This notice is made in accordance with 35 U.S.C. 209 and 37 CFR 
part 404. The prospective Exclusive Commercialization Patent License 
will be royalty bearing and may be granted unless within fifteen (15) 
days from the date of this published notice, the National Cancer 
Institute receives written evidence and argument that establishes that 
the grant of the license would not be consistent with the

[[Page 66048]]

requirements of 35 U.S.C. 209 and 37 CFR part 404.
    Complete applications for a license in the prospective field of use 
that are filed in response to this notice will be treated as objections 
to the grant of the contemplated Exclusive Commercialization Patent 
License Agreement. Comments and objections submitted 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 21, 2016.
Richard U. Rodriguez,
Associate Director, Technology Transfer Center, National Cancer 
Institute.
[FR Doc. 2016-23134 Filed 9-23-16; 8:45 am]
 BILLING CODE 4140-01-P