AU2015374043B2 - Methods and compositions for treating brain diseases - Google Patents

Methods and compositions for treating brain diseases Download PDF

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Publication number
AU2015374043B2
AU2015374043B2 AU2015374043A AU2015374043A AU2015374043B2 AU 2015374043 B2 AU2015374043 B2 AU 2015374043B2 AU 2015374043 A AU2015374043 A AU 2015374043A AU 2015374043 A AU2015374043 A AU 2015374043A AU 2015374043 B2 AU2015374043 B2 AU 2015374043B2
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AU
Australia
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aav
subject
brain
mice
therapeutic agent
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AU2015374043A
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English (en)
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AU2015374043A1 (en
Inventor
Beverly L. Davidson
John H. Lee
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University of Iowa Research Foundation UIRF
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University of Iowa Research Foundation UIRF
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
    • C12Y306/05002—Small monomeric GTPase (3.6.5.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2015374043A 2014-12-30 2015-12-30 Methods and compositions for treating brain diseases Active AU2015374043B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462098085P 2014-12-30 2014-12-30
US62/098,085 2014-12-30
PCT/US2015/068034 WO2016109649A1 (en) 2014-12-30 2015-12-30 Methods and compositions for treating brain diseases

Publications (2)

Publication Number Publication Date
AU2015374043A1 AU2015374043A1 (en) 2017-07-13
AU2015374043B2 true AU2015374043B2 (en) 2021-05-13

Family

ID=56285031

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2015374043A Active AU2015374043B2 (en) 2014-12-30 2015-12-30 Methods and compositions for treating brain diseases

Country Status (12)

Country Link
US (1) US11612641B2 (enExample)
EP (1) EP3240577B1 (enExample)
JP (1) JP6782701B2 (enExample)
KR (1) KR102618947B1 (enExample)
CN (1) CN107405414A (enExample)
AU (1) AU2015374043B2 (enExample)
BR (1) BR112017013674A2 (enExample)
CA (1) CA2971687C (enExample)
ES (1) ES2962439T3 (enExample)
SG (1) SG11201704829QA (enExample)
WO (1) WO2016109649A1 (enExample)
ZA (1) ZA201704327B (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3240577B1 (en) 2014-12-30 2023-07-05 University of Iowa Research Foundation Therapeutic agent that activates mtorc1 function for use in treating huntington's disease
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
CL2016003282A1 (es) * 2016-12-21 2017-08-18 Univ Chile Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington
WO2018132791A1 (en) * 2017-01-16 2018-07-19 The Regents Of The University Of California Treatment of neurodegenerative conditions by disruption of rhes
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease

Family Cites Families (10)

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Publication number Priority date Publication date Assignee Title
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050009112A1 (en) 2003-03-07 2005-01-13 Fred Hutchinson Cancer Research Center, Office Of Technology Transfer Methods for identifying Rheb effectors as lead compounds for drug development for diabetes and diseases associated with abnormal cell growth
KR20080018874A (ko) 2005-04-27 2008-02-28 유니버시티 오브 플로리다 리서치 파운데이션, 아이엔씨. 인간 질병과 관련된 돌연변이 단백질의 분해 촉진을 위한물질 및 방법
KR101286870B1 (ko) * 2009-11-30 2013-07-16 (주)아모레퍼시픽 황기 추출물을 포함하는 대사 촉진 조성물
WO2013061305A1 (en) * 2011-10-28 2013-05-02 Novartis Ag Novel purine derivatives and their use in the treatment of disease
US9511036B2 (en) * 2011-11-01 2016-12-06 Children's Medical Center Corporation Co-activation of mTOR and STAT3 pathways to promote neuronal survival and regeneration
HK1201828A1 (en) * 2011-11-08 2015-09-11 Intellikine, Llc Treatment regimens using multiple pharmaceutical agents
US9095521B2 (en) * 2012-02-02 2015-08-04 Washington University Methods for improving muscle strength
KR101626526B1 (ko) 2014-10-30 2016-06-02 경북대학교 산학협력단 ｈＲｈｅｂ를 유효성분으로 포함하는 허혈성 뇌질환의 예방 또는 치료용 조성물
EP3240577B1 (en) 2014-12-30 2023-07-05 University of Iowa Research Foundation Therapeutic agent that activates mtorc1 function for use in treating huntington's disease

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
C. N. CEARLEY ET AL, THE JOURNAL OF NEUROSCIENCE, US, (2007-09-12), vol. 27, no. 37, doi:10.1523/JNEUROSCI.2185-07.2007, ISSN 0270-6474, pages 9928 - 9940 *
J H LEE ET AL, "Rhes modulation reveals a critical role for mTOR activity in Huntington's Disease", 43RD ANNUAL MEETING OF THE SOCIETY-FOR-NEUROSCIENCE; SAN DIEGO, CA, USA; NOVEMBER 09 -13, 2013, (2013-11-11) *
LIJUN YAN ET AL, "Hyperactivation of Mammalian Target of Rapamycin (mTOR) Signaling by a Gain-of-Function Mutant of the Rheb GTPase", JOURNAL OF BIOLOGICAL CHEMISTRY, US, (2006-05-25), vol. 281, no. 29, pages 19793 - 19797 *
SANG RYONG KIM et al. Molecular Therapy: The Journal of the American Society of Gene Therapy, Vol. 20, No. 2, page 275-286. 01/02/2012. *

Also Published As

Publication number Publication date
HK1245127A1 (en) 2018-08-24
KR20170098931A (ko) 2017-08-30
AU2015374043A1 (en) 2017-07-13
EP3240577A1 (en) 2017-11-08
ES2962439T3 (es) 2024-03-19
WO2016109649A1 (en) 2016-07-07
CA2971687C (en) 2024-05-28
US11612641B2 (en) 2023-03-28
KR102618947B1 (ko) 2023-12-27
CN107405414A (zh) 2017-11-28
CA2971687A1 (en) 2016-07-07
BR112017013674A2 (pt) 2018-02-06
SG11201704829QA (en) 2017-07-28
JP6782701B2 (ja) 2020-11-11
ZA201704327B (en) 2025-02-26
EP3240577A4 (en) 2018-07-25
JP2018502112A (ja) 2018-01-25
EP3240577B1 (en) 2023-07-05
NZ733014A (en) 2024-05-31
US20180000907A1 (en) 2018-01-04

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