CA2971687C - Methods and compositions for treating brain diseases - Google Patents

Methods and compositions for treating brain diseases Download PDF

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Publication number
CA2971687C
CA2971687C CA2971687A CA2971687A CA2971687C CA 2971687 C CA2971687 C CA 2971687C CA 2971687 A CA2971687 A CA 2971687A CA 2971687 A CA2971687 A CA 2971687A CA 2971687 C CA2971687 C CA 2971687C
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CA
Canada
Prior art keywords
aav
therapeutic agent
subject
brain
carheb
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Active
Application number
CA2971687A
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English (en)
French (fr)
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CA2971687A1 (en
Inventor
Beverly L. Davidson
John H. Lee
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Iowa Research Foundation UIRF
Original Assignee
University of Iowa Research Foundation UIRF
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Publication of CA2971687A1 publication Critical patent/CA2971687A1/en
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Classifications

    • 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
    • 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
    • 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

Landscapes

  • 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Orthopedic Medicine & Surgery (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)
  • Peptides Or Proteins (AREA)
CA2971687A 2014-12-30 2015-12-30 Methods and compositions for treating brain diseases Active CA2971687C (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
CA2971687A1 CA2971687A1 (en) 2016-07-07
CA2971687C true CA2971687C (en) 2024-05-28

Family

ID=56285031

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2971687A Active CA2971687C (en) 2014-12-30 2015-12-30 Methods and compositions for treating brain diseases

Country Status (12)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016109649A1 (en) 2014-12-30 2016-07-07 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
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
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法

Family Cites Families (10)

* Cited by examiner, † Cited by third party
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
WO2006116716A2 (en) 2005-04-27 2006-11-02 University Of Florida Materials and methods for enhanced degradation of mutant proteins associated with human disease
KR101286870B1 (ko) * 2009-11-30 2013-07-16 (주)아모레퍼시픽 황기 추출물을 포함하는 대사 촉진 조성물
CN104039790B (zh) 2011-10-28 2016-04-13 诺华股份有限公司 嘌呤衍生物及它们在治疗疾病中的应用
EP2773382A4 (en) 2011-11-01 2016-03-23 Childrens Medical Center SIMULTANEOUS ACTIVATION OF MTOR AND STAT3 PATHS TO PROMOTE NEURONAL SURVIVAL AND REGENERATION
CA2854926A1 (en) * 2011-11-08 2013-05-16 Intellikine, Llc Treatment regimens using multiple pharmaceutical agents
WO2013116691A1 (en) 2012-02-02 2013-08-08 The 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
WO2016109649A1 (en) 2014-12-30 2016-07-07 University Of Iowa Research Foundation Methods and compositions for treating brain diseases

Also Published As

Publication number Publication date
US11612641B2 (en) 2023-03-28
EP3240577A1 (en) 2017-11-08
EP3240577A4 (en) 2018-07-25
CN107405414A (zh) 2017-11-28
HK1245127A1 (en) 2018-08-24
CA2971687A1 (en) 2016-07-07
US20180000907A1 (en) 2018-01-04
WO2016109649A1 (en) 2016-07-07
NZ733014A (en) 2024-05-31
ZA201704327B (en) 2025-02-26
JP6782701B2 (ja) 2020-11-11
EP3240577B1 (en) 2023-07-05
JP2018502112A (ja) 2018-01-25
KR20170098931A (ko) 2017-08-30
ES2962439T3 (es) 2024-03-19
SG11201704829QA (en) 2017-07-28
BR112017013674A2 (pt) 2018-02-06
KR102618947B1 (ko) 2023-12-27
AU2015374043B2 (en) 2021-05-13
AU2015374043A1 (en) 2017-07-13

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