CA2971687C - Methods and compositions for treating brain diseases - Google Patents
Methods and compositions for treating brain diseases Download PDFInfo
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- 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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- 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
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
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- 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
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- 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
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- 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
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- 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)
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- 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
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- 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
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| Application Number | Priority Date | Filing Date | Title |
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| 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 |
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| CA2971687A1 CA2971687A1 (en) | 2016-07-07 |
| CA2971687C true CA2971687C (en) | 2024-05-28 |
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| EP (1) | EP3240577B1 (enExample) |
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| 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 |
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| 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 |
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Also Published As
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|---|---|
| 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 |
| US11612641B2 (en) | 2023-03-28 |
| KR102618947B1 (ko) | 2023-12-27 |
| AU2015374043B2 (en) | 2021-05-13 |
| 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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