81 FR 32761 - Prospective Grant of Exclusive Patent License: Development of Adeno-Associated Virus Vectors for the Treatment of Glycogen Storage Disease Type Ia

DEPARTMENT OF HEALTH AND HUMAN SERVICES
National Institutes of Health

Federal Register Volume 81, Issue 100 (May 24, 2016)

This notice, in accordance with 35 U.S.C. 209(c)(1) and 37 CFR 404.7(a)(1)(i), that the National Institutes of Health, Department of Health and Human Services (HHS), is contemplating the grant of an exclusive license to practice the inventions embodied in the following Patent Applications to Dimension Therapeutics, Inc. (``Dimension'') located in Cambridge, Massachusetts, USA:

[Federal Register Volume 81, Number 100 (Tuesday, May 24, 2016)]
[Notices]
[Pages 32761-32762]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2016-12169]


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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 Vectors for the Treatment of Glycogen Storage 
Disease Type Ia

AGENCY: National Institutes of Health, HHS.

ACTION: Notice.

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SUMMARY: This notice, in accordance with 35 U.S.C. 209(c)(1) and 37 CFR 
404.7(a)(1)(i), that the National Institutes of Health, Department of 
Health and Human Services (HHS), is contemplating the grant of an 
exclusive license to practice the inventions embodied in the following 
Patent Applications to Dimension Therapeutics, Inc. (``Dimension'') 
located in Cambridge, Massachusetts, USA:

Intellectual Property

    United States Provisional Patent Application No. 61/908,861, filed 
November 26, 2013, titled ``Adeno-Associated Virus Vectors for the 
Treatment of Glycogen Storage Disease'' [HHS Reference No. E-552-2013/
0-US-01]; International Patent Application No. PCT/US2014/067415 filed 
November 25, 2014 titled ``Adeno-Associated Virus Vectors for the 
Treatment of Glycogen Storage Disease'' [HHS Reference No. E-552-2013/
0-PCT-02] and continuation applications, divisional applications and 
foreign counterpart applications claiming priority to the US 
provisional application No. 61/908,861.
    With respect to persons who have an obligation to assign their 
right, title and interest to the Government of the United States of 
America, the patent rights in these inventions have been assigned to 
the Government of the United States of America.
    The prospective exclusive licensed territory may be worldwide and 
the field of use may be limited to: ``Development and commercialization 
of gene therapy using adeno-associated viral vectors for the treatment 
of Glycogen Storage Disease Type Ia.''

DATES: Only written comments and/or applications for a license which 
are received by the NIH Office of Technology Transfer on or before June 
8, 2016 will be considered.

ADDRESSES: Requests for copies of the patent application, inquiries, 
comments, and other materials relating to the contemplated exclusive 
license should be directed to: Surekha Vathyam, Ph.D., Senior Licensing 
and Patenting Manager, National Cancer Institute Technology Transfer 
Center, 9609 Medical Center Drive, Rm. 1E-530, MSC9702, Rockville, MD 
20850-9702, Email: [email protected].

SUPPLEMENTARY INFORMATION: The subject technologies disclose novel 
adeno-associated virus (AAV) vectors expressing human glucose-6-
phosphatase-alpha (G6Pase-alpha or G6PC) for the treatment of glycogen 
storage disease, particularly glycogen storage disease type Ia (GSD-
Ia). GSD-Ia is an inherited disorder of metabolism associated with 
life-threatening hypoglycemia, hepatic malignancy, and renal failure 
caused by the deficiency of G6Pase-alpha, a key enzyme in maintaining 
blood glucose homeostasis between meals. The two novel gene therapy 
vectors of the invention, rAAV-GPE-G6PC and rAAV-GPE-co-G6PC are 
recombinant AAV vectors expressing

[[Page 32762]]

wild-type G6Pase-alpha and codon-optimized (co) G6Pase-alpha, 
respectively. G6Pase-alpha in both vectors is directed by nucleotides -
2864 to -1 of the G6PC gene 5'-flanking promoter/enhancer region (GPE). 
The vectors also contain an intron and stuffer sequences. The rAAV-GPE-
G6PC vector not only corrects metabolic abnormalities in murine GSD-Ia 
(G6pc-/- mice) but also prevents long-term risk of hepatocellular 
adenoma. The results also showed that the enhancer elements upstream 
the human G6PC minimal promoter at nucleotides -382 to -1 contained 
within the rAAV-GPE-G6PC vector are responsible for the increased 
efficacy in treating GSD-Ia mice.
    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 404.7. 
The prospective exclusive license may be granted unless within fifteen 
(15) days from the date of this published notice, the 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 404.7.
    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 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: May 17, 2016.
Richard U. Rodriguez,
Associate Director, NCI, National Institutes of Health.
[FR Doc. 2016-12169 Filed 5-23-16; 8:45 am]
 BILLING CODE 4140-01-P