BR112017013674A2 - métodos e composições para tratamento de doenças cerebrais. - Google Patents

métodos e composições para tratamento de doenças cerebrais.

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Publication number
BR112017013674A2
BR112017013674A2 BR112017013674A BR112017013674A BR112017013674A2 BR 112017013674 A2 BR112017013674 A2 BR 112017013674A2 BR 112017013674 A BR112017013674 A BR 112017013674A BR 112017013674 A BR112017013674 A BR 112017013674A BR 112017013674 A2 BR112017013674 A2 BR 112017013674A2
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BR
Brazil
Prior art keywords
methods
level
function
subject
compositions
Prior art date
Application number
BR112017013674A
Other languages
English (en)
Inventor
L Davidson Beverly
H Lee John
Original Assignee
Univ Iowa Res Found
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 Univ Iowa Res Found filed Critical Univ Iowa Res Found
Publication of BR112017013674A2 publication Critical patent/BR112017013674A2/pt

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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

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)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Microbiology (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)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

a presente invenção refere-se a métodos para tratamento ou prevenção de doença de huntington (hd) em um indivíduo com ne-cessidade dos mesmos compreendendo administração de um agente terapêutico que ativa função de mtorc1 e/ou aumenta nível de ho-mólogo de ras enriquecido no estriado (rhes) no cérebro do indivíduo comparado com a função ou nível no indivíduo antes do tratamento e métodos para modulação de fenótipos metabólicos associados a mhtt e/ou reversão de atrofia estriatal através da administração de um agente terapêutico que ativa função de mtorc1 e/ou aumenta o nível de homólogo de ras enriquecido no estriado (rhes) no cérebro do indivíduo comparado com a função ou nível no indivíduo antes do tratamento.
BR112017013674A 2014-12-30 2015-12-30 métodos e composições para tratamento de doenças cerebrais. BR112017013674A2 (pt)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
BR112017013674A2 true BR112017013674A2 (pt) 2018-02-06

Family

ID=56285031

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112017013674A BR112017013674A2 (pt) 2014-12-30 2015-12-30 métodos e composições para tratamento de doenças cerebrais.

Country Status (12)

Country Link
US (1) US11612641B2 (pt)
EP (1) EP3240577B1 (pt)
JP (1) JP6782701B2 (pt)
KR (1) KR102618947B1 (pt)
CN (1) CN107405414A (pt)
AU (1) AU2015374043B2 (pt)
BR (1) BR112017013674A2 (pt)
CA (1) CA2971687A1 (pt)
ES (1) ES2962439T3 (pt)
HK (1) HK1245127A1 (pt)
SG (1) SG11201704829QA (pt)
WO (1) WO2016109649A1 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
BR112017013674A2 (pt) 2014-12-30 2018-02-06 Univ Iowa Res Found métodos e composições para tratamento de doenças cerebrais.
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
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
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease

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
CA2606226A1 (en) 2005-04-27 2006-11-02 University Of Florida Research Foundation, Inc. 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
EP2771342B1 (en) 2011-10-28 2016-05-18 Novartis AG Purine derivatives and their use in the treatment of disease
CA2854225A1 (en) 2011-11-01 2013-05-10 Children's Medical Center Corporation Co-activation of mtor and stat3 pathways 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
BR112017013674A2 (pt) 2014-12-30 2018-02-06 Univ Iowa Res Found métodos e composições para tratamento de doenças cerebrais.

Also Published As

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

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Legal Events

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B11B Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements