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

Methods and compositions for treating brain diseases Download PDF

Info

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
Authority
AU
Australia
Prior art keywords
aav
subject
brain
mice
therapeutic agent
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
AU2015374043A
Other languages
English (en)
Other versions
AU2015374043A1 (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
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Iowa Research Foundation UIRF filed Critical University of Iowa Research Foundation UIRF
Publication of AU2015374043A1 publication Critical patent/AU2015374043A1/en
Application granted granted Critical
Publication of AU2015374043B2 publication Critical patent/AU2015374043B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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

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)
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 (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

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
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
CA2971687C (en) 2024-05-28
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
AU2015374043A1 (en) 2017-07-13

Similar Documents

Publication Publication Date Title
AU2015374043B2 (en) Methods and compositions for treating brain diseases
US20230295663A1 (en) Compositions and methods of treating amyotrophic lateral sclerosis (als)
US20240226203A9 (en) Compositions and methods of treating huntington's disease
AU2018352236B2 (en) Treatment of amyotrophic lateral sclerosis (ALS)
KR102599909B1 (ko) 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
US20200270635A1 (en) Modulatory polynucleotides
JP2018521014A (ja) ミスフォールドタンパク質の分解のための組成物及び方法
AU2017234929A1 (en) Therapeutic for treatment of diseases including the central nervous system
JP2025011130A (ja) ネフローゼ症候群を処置するためのａａｖ遺伝子療法
EP3962514A1 (en) Methods and compositions involving tert activating therapies
HK1245127B (en) Therapeutic agent that activates mtorc1 function for use in treating huntington's disease
WO2022174036A2 (en) Methods and materials for treating tdp-43 proteinopathies
CA3163034A1 (en) Methods for treating huntington's disease
US20250177573A1 (en) Materials & Methods for Treatment of Macular Degeneration
WO2025015328A2 (en) Increasing nfe2l1 activity or expression as therapy for eye disorders
HK40052708B (zh) 用於肌萎缩性侧索硬化疗法中的胆固醇24-羟化酶的表达载体
JP2022531177A (ja) 神経変性障害を治療するための方法

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)