81_FR_32861 81 FR 32761 - Prospective Grant of Exclusive Patent License: Development of Adeno-Associated Virus Vectors for the Treatment of Glycogen Storage Disease Type Ia

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)

Page Range32761-32762
FR Document2016-12169

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 Issue 100 (Tuesday, May 24, 2016) 
[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

Federal Register / Vol. 81, No. 100 / Tuesday, May 24, 2016 / Notices 32761 TOTAL ESTIMATED ANNUALIZED BURDEN—HOURS Average Number of Total Number of Total burden per Form name responses per burden respondents responses response respondent hours (in hours) Corps Community Month Event Planning Form .................. 300 1 300 .066 19.8 Corps Community Month Event Satisfaction ....................... 300 1 300 .033 9.9 Total .............................................................................. * 300 ........................ * 300 ........................ 29.7 * The same individuals complete both of the forms for a total of 300 respondents and responses. Jason E. Bennett, Contact Person: Ken D. Nakamura, Ph.D., Treatment of Glycogen Storage Disease’’ Director, Division of the Executive Secretariat. Scientific Review Officer, Scientific Review [HHS Reference No. E–552–2013/0– Branch, National Human Genome Research PCT–02] and continuation applications, [FR Doc. 2016–12146 Filed 5–23–16; 8:45 am] Institute, National Institutes of Health, 5635 BILLING CODE 4165–15–P divisional applications and foreign Fishers Lane, Suite 4076, MSC 9306, Rockville, MD 20852, 301–402–0838, counterpart applications claiming nakamurk@mail.nih.gov. priority to the US provisional DEPARTMENT OF HEALTH AND (Catalogue of Federal Domestic Assistance application No. 61/908,861. HUMAN SERVICES Program Nos. 93.172, Human Genome With respect to persons who have an Research, National Institutes of Health, HHS). obligation to assign their right, title and National Institutes of Health interest to the Government of the United Dated: May 17, 2016. States of America, the patent rights in National Human Genome Research Sylvia L. Neal, these inventions have been assigned to Institute; Notice of Closed Meetings Program Analyst, Office of Federal Advisory the Government of the United States of Committee Policy. Pursuant to section 10(d) of the America. [FR Doc. 2016–12139 Filed 5–23–16; 8:45 am] The prospective exclusive licensed Federal Advisory Committee Act, as amended (5 U.S.C. App.), notice is BILLING CODE 4140–01–P territory may be worldwide and the hereby given of the following meetings. field of use may be limited to: ‘‘Development and commercialization of The meetings will be closed to the DEPARTMENT OF HEALTH AND gene therapy using adeno-associated public in accordance with the HUMAN SERVICES viral vectors for the treatment of provisions set forth in sections Glycogen Storage Disease Type Ia.’’ 552b(c)(4) and 552b(c)(6), title 5 U.S.C., National Institutes of Health as amended. The grant applications and DATES: Only written comments and/or the discussions could disclose Prospective Grant of Exclusive Patent applications for a license which are confidential trade secrets or commercial License: Development of Adeno- received by the NIH Office of property such as patentable material, Associated Virus Vectors for the Technology Transfer on or before June and personal information concerning Treatment of Glycogen Storage 8, 2016 will be considered. individuals associated with the grant Disease Type Ia ADDRESSES: Requests for copies of the applications, the disclosure of which patent application, inquiries, comments, AGENCY: National Institutes of Health, would constitute a clearly unwarranted and other materials relating to the HHS. invasion of personal privacy. contemplated exclusive license should ACTION: Notice. be directed to: Surekha Vathyam, Ph.D., Name of Committee: National Human Genome Research Institute Special Emphasis SUMMARY: This notice, in accordance Senior Licensing and Patenting Panel; Gabriella Miller Kids First Sequencing with 35 U.S.C. 209(c)(1) and 37 CFR Manager, National Cancer Institute Center. 404.7(a)(1)(i), that the National Technology Transfer Center, 9609 Date: June 14, 2016. Institutes of Health, Department of Medical Center Drive, Rm. 1E–530, Time: 1:00 p.m. to 4:00 p.m. Health and Human Services (HHS), is MSC9702, Rockville, MD 20850–9702, Agenda: To review and evaluate grant contemplating the grant of an exclusive Email: vathyams@mail.nih.gov. applications. SUPPLEMENTARY INFORMATION: The Place: NHGRI, 5635FL, Twinbrook 4th license to practice the inventions embodied in the following Patent subject technologies disclose novel Floor Conf. Rm., Twinbrook, Rockville, MD 20852 (Telephone Conference Call). Applications to Dimension adeno-associated virus (AAV) vectors Contact Person: Rudy O. Pozzatti, Ph.D., Therapeutics, Inc. (‘‘Dimension’’) expressing human glucose-6- Scientific Review Officer, Scientific Review located in Cambridge, Massachusetts, phosphatase-alpha (G6Pase-alpha or Branch, National Human Genome Research USA: G6PC) for the treatment of glycogen Institute, 5635 Fishers Lane, Suite 4076, MSC storage disease, particularly glycogen 9306, Rockville, MD 20852, (301) 402–0838, Intellectual Property storage disease type Ia (GSD-Ia). GSD-Ia pozzattr@mail.nih.gov. United States Provisional Patent is an inherited disorder of metabolism Name of Committee: National Human Application No. 61/908,861, filed associated with life-threatening Genome Research Institute Special Emphasis November 26, 2013, titled ‘‘Adeno- hypoglycemia, hepatic malignancy, and sradovich on DSK3TPTVN1PROD with NOTICES Panel; ENCODE MAPPING. Associated Virus Vectors for the renal failure caused by the deficiency of Date: June 21, 2016. Treatment of Glycogen Storage Disease’’ G6Pase-alpha, a key enzyme in Time: 8:00 a.m. to 5:00 p.m. Agenda: To review and evaluate grant [HHS Reference No. E–552–2013/0–US– maintaining blood glucose homeostasis applications. 01]; International Patent Application between meals. The two novel gene Place: Washington Dulles Airport Marriott, No. PCT/US2014/067415 filed therapy vectors of the invention, rAAV- 45020, Dulles View, Aviation Drive, Dulles, November 25, 2014 titled ‘‘Adeno- GPE-G6PC and rAAV-GPE-co-G6PC are VA 20166. Associated Virus Vectors for the recombinant AAV vectors expressing VerDate Sep<11>2014 17:24 May 23, 2016 Jkt 238001 PO 00000 Frm 00044 Fmt 4703 Sfmt 4703 E:\FR\FM\24MYN1.SGM 24MYN1

32762 Federal Register / Vol. 81, No. 100 / Tuesday, May 24, 2016 / Notices wild-type G6Pase-alpha and codon- and personal information concerning Date: June 22–23, 2016. optimized (co) G6Pase-alpha, individuals associated with the grant Time: 8:00 a.m. to 5:00 p.m. respectively. G6Pase-alpha in both applications, the disclosure of which Agenda: To review and evaluate grant applications. vectors is directed by nucleotides -2864 would constitute a clearly unwarranted Place: Washington Plaza Hotel, 10 Thomas to -1 of the G6PC gene 5’-flanking invasion of personal privacy. Circle NW., Washington, DC 20005. promoter/enhancer region (GPE). The Name of Committee: Cardiovascular and Contact Person: Michael L. Bloom, Ph.D., vectors also contain an intron and Respiratory Sciences Integrated Review Scientific Review Officer, Center for stuffer sequences. The rAAV-GPE-G6PC Group; Cardiac Contractility, Hypertrophy, Scientific Review, National Institutes of vector not only corrects metabolic and Failure Study Section. Health, 6701 Rockledge Drive, Room 6187, abnormalities in murine GSD-Ia (G6pc- Date: June 13–14, 2016. MSC 7804, Bethesda, MD 20892, 301–451– /- mice) but also prevents long-term risk Time: 8:00 a.m. to 2:00 p.m. 0132, bloomm2@mail.nih.gov. of hepatocellular adenoma. The results Agenda: To review and evaluate grant Name of Committee: Brain Disorders and applications. Clinical Neuroscience Integrated Review also showed that the enhancer elements Place: Embassy Suites Hotel O’Hare— Group; Acute Neural Injury and Epilepsy upstream the human G6PC minimal Rosemont, 5500 North River Road, Rosemont, Study Section. promoter at nucleotides -382 to -1 IL 60018. Date: June 22–23, 2016. contained within the rAAV-GPE-G6PC Contact Person: Olga A. Tjurmina, Ph.D., Time: 8:30 a.m. to 5:00 p.m. vector are responsible for the increased Scientific Review Officer, Center for Agenda: To review and evaluate grant efficacy in treating GSD-Ia mice. Scientific Review, National Institutes of applications. The prospective exclusive license will Health, 6701 Rockledge Drive, Room 4030B, Place: Embassy Suites at the Chevy Chase be royalty bearing and will comply with MSC 7814, Bethesda, MD 20892, (301) 451– Pavilion, 4300 Military Road NW., the terms and conditions of 35 U.S.C. 1375, ot3d@nih.gov. Washington, DC 20015. 209 and 37 CFR 404.7. The prospective Name of Committee: Center for Scientific Contact Person: Seetha Bhagavan, Ph.D., Review Special Emphasis Panel; Nursing and Scientific Review Officer, Center for exclusive license may be granted unless Scientific Review, National Institutes of Related Clinical Sciences Overflow. within fifteen (15) days from the date of Health, 6701 Rockledge Drive, Room 5194, Date: June 14–15, 2016. this published notice, the NIH receives Time: 8:00 a.m. to 5:00 p.m. MSC 7846, Bethesda, MD 20892, (301) 237– written evidence and argument that Agenda: To review and evaluate grant 9838, bhagavas@csr.nih.gov. establishes that the grant of the license applications. Name of Committee: Center for Scientific would not be consistent with the Place: Doubletree Hotel Bethesda Review Special Emphasis Panel; PAR14–022: requirements of 35 U.S.C. 209 and 37 (Formerly Holiday Inn Select), 8120 Juvenile Protective Factors and Aging. CFR 404.7. Wisconsin Avenue, Bethesda, MD 20814. Date: June 22, 2016. Complete applications for a license in Contact Person: Martha L. Hare, Ph.D., RN, Time: 1:00 p.m. to 4:00 p.m. the prospective field of use that are filed Scientific Review Officer, Center for Agenda: To review and evaluate grant Scientific Review, National Institutes of applications. in response to this notice will be treated Health, 6701 Rockledge Drive, Room 3154, Place: National Institutes of Health, 6701 as objections to the grant of the Rockledge Drive, Bethesda, MD 20892 Bethesda, MD 20892, (301) 451–8504, contemplated Exclusive Patent License harem@mail.nih.gov. (Virtual Meeting). Agreement. Comments and objections Contact Person: Wallace Ip, Ph.D., Name of Committee: Center for Scientific submitted to this notice will not be Review Special Emphasis Panel; Member Scientific Review Officer, Center for made available for public inspection Conflict: Auditory Neuroscience. Scientific Review, National Institutes of and, to the extent permitted by law, will Date: June 14–15, 2016. Health, 6701 Rockledge Drive, Room 5128, not be released under the Freedom of Time: 8:00 a.m. to 6:00 p.m. MSC 7840, Bethesda, MD 20892, 301–435– Information Act, 5 U.S.C. 552. Agenda: To review and evaluate grant 1191, ipws@mail.nih.gov. applications. (Catalogue of Federal Domestic Assistance Dated: May 17, 2016. Program Nos. 93.306, Comparative Medicine; Place: National Institutes of Health, 6701 Richard U. Rodriguez, Rockledge Drive, Bethesda, MD 20892 93.333, Clinical Research, 93.306, 93.333, Associate Director, NCI, National Institutes (Virtual Meeting). 93.337, 93.393–93.396, 93.837–93.844, of Health. Contact Person: John Bishop, Ph.D., 93.846–93.878, 93.892, 93.893, National [FR Doc. 2016–12169 Filed 5–23–16; 8:45 am] Scientific Review Officer, Center for Institutes of Health, HHS). BILLING CODE 4140–01–P Scientific Review, National Institutes of Dated: May 17, 2016. Health, 6701 Rockledge Drive, Room 5182, Sylvia L. Neal, MSC 7844, Bethesda, MD 20892, (301) 408– 9664, bishopj@csr.nih.gov. Program Analyst, Office of Federal Advisory DEPARTMENT OF HEALTH AND Committee Policy. HUMAN SERVICES Name of Committee: Cardiovascular and Respiratory Sciences Integrated Review [FR Doc. 2016–12137 Filed 5–23–16; 8:45 am] National Institutes of Health Group; Clinical and Integrative BILLING CODE 4140–01–P Cardiovascular Sciences Study Section. Center for Scientific Review; Notice of Date: June 16–17, 2016. Closed Meetings Time: 8:00 a.m. to 2:00 p.m. DEPARTMENT OF HEALTH AND Agenda: To review and evaluate grant HUMAN SERVICES Pursuant to section 10(d) of the applications. Federal Advisory Committee Act, as Place: Beacon Hotel and Corporate National Institutes of Health amended (5 U.S.C. App.), notice is Quarters, 1615 Rhode Island Avenue NW., hereby given of the following meetings. Washington, DC 20036. Prospective Grant of Exclusive Patent Contact Person: Yuanna Cheng, MD, Ph.D., License: Development of Adeno- The meetings will be closed to the sradovich on DSK3TPTVN1PROD with NOTICES Scientific Review Officer, Center for Associated Virus Vectors for the public in accordance with the Scientific Review, National Institutes of provisions set forth in sections Treatment of Glycogen Storage Health, 6701 Rockledge Drive, Room 4138, 552b(c)(4) and 552b(c)(6), Title 5 U.S.C., MSC 7814, Bethesda, MD 20892, (301) 435– Disease Type Ia as amended. The grant applications and 1195, Chengy5@csr.nih.gov. AGENCY: National Institutes of Health, the discussions could disclose Name of Committee: Oncology 1—Basic HHS. confidential trade secrets or commercial Translational Integrated Review Group; ACTION: Notice. property such as patentable material, Cancer Genetics Study Section. VerDate Sep<11>2014 17:24 May 23, 2016 Jkt 238001 PO 00000 Frm 00045 Fmt 4703 Sfmt 4703 E:\FR\FM\24MYN1.SGM 24MYN1


Document Created: 2016-05-24 05:21:32
Document Modified: 2016-05-24 05:21:32
CategoryRegulatory Information
CollectionFederal Register
sudoc ClassAE 2.7:
GS 4.107:
AE 2.106:
PublisherOffice of the Federal Register, National Archives and Records Administration
SectionNotices
ActionNotice.
DatesOnly 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.
FR Citation81 FR 32761 
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