JP2017535266A - 筋萎縮性側索硬化症(als)を治療する組成物および方法 - Google Patents
筋萎縮性側索硬化症(als)を治療する組成物および方法 Download PDFInfo
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- JP2017535266A JP2017535266A JP2017525847A JP2017525847A JP2017535266A JP 2017535266 A JP2017535266 A JP 2017535266A JP 2017525847 A JP2017525847 A JP 2017525847A JP 2017525847 A JP2017525847 A JP 2017525847A JP 2017535266 A JP2017535266 A JP 2017535266A
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- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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- C12N15/09—Recombinant DNA-technology
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- 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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- A—HUMAN NECESSITIES
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2330/00—Production
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- C12N2330/51—Specially adapted vectors
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- 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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- Chemical & Material Sciences (AREA)
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- Bioinformatics & Cheminformatics (AREA)
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- General Chemical & Material Sciences (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Virology (AREA)
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- Psychiatry (AREA)
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- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pain & Pain Management (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022030717A JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2024050822A JP2024079794A (ja) | 2014-11-14 | 2024-03-27 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462079588P | 2014-11-14 | 2014-11-14 | |
| US62/079,588 | 2014-11-14 | ||
| US201562211992P | 2015-08-31 | 2015-08-31 | |
| US62/211,992 | 2015-08-31 | ||
| US201562234466P | 2015-09-29 | 2015-09-29 | |
| US62/234,466 | 2015-09-29 | ||
| PCT/US2015/060562 WO2016077687A1 (en) | 2014-11-14 | 2015-11-13 | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020159246A Division JP2021007394A (ja) | 2014-11-14 | 2020-09-24 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2022030717A Division JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017535266A true JP2017535266A (ja) | 2017-11-30 |
| JP2017535266A5 JP2017535266A5 (enExample) | 2018-10-25 |
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Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017525847A Pending JP2017535266A (ja) | 2014-11-14 | 2015-11-13 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2020159246A Pending JP2021007394A (ja) | 2014-11-14 | 2020-09-24 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2022030717A Pending JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2024050822A Pending JP2024079794A (ja) | 2014-11-14 | 2024-03-27 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020159246A Pending JP2021007394A (ja) | 2014-11-14 | 2020-09-24 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2022030717A Pending JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2024050822A Pending JP2024079794A (ja) | 2014-11-14 | 2024-03-27 | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
Country Status (14)
| Country | Link |
|---|---|
| US (5) | US10597660B2 (enExample) |
| EP (1) | EP3218484A4 (enExample) |
| JP (4) | JP2017535266A (enExample) |
| KR (2) | KR102599909B1 (enExample) |
| CN (2) | CN107109407A (enExample) |
| AU (2) | AU2015346162B2 (enExample) |
| CA (2) | CA3193811A1 (enExample) |
| HK (1) | HK1244299A1 (enExample) |
| IL (3) | IL292999A (enExample) |
| MX (2) | MX2017006216A (enExample) |
| RU (2) | RU2716422C2 (enExample) |
| SG (1) | SG11201703281RA (enExample) |
| WO (1) | WO2016077687A1 (enExample) |
| ZA (1) | ZA201702991B (enExample) |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019132624A1 (ko) * | 2017-12-29 | 2019-07-04 | 주식회사 헬릭스미스 | 하이브리드 hgf 유전자가 도입된 aav(아데노-연관 바이러스) 벡터 |
| KR20210131992A (ko) * | 2018-12-14 | 2021-11-03 | 바이오젠 엠에이 인코포레이티드 | 근위축 측삭 경화증의 치료 및 방지를 위한 조성물 및 방법 |
| JP2021529513A (ja) * | 2018-07-02 | 2021-11-04 | 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. | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
| JP2023537980A (ja) * | 2020-08-12 | 2023-09-06 | ユーシービー バイオファルマ エスアールエル | メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 |
| US12116589B2 (en) | 2017-10-16 | 2024-10-15 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| HUE059109T2 (hu) | 2014-04-01 | 2022-10-28 | Biogen Ma Inc | A SOD-1 expresszió modulálására szolgáló készítmények |
| IL284949B2 (en) | 2014-11-14 | 2024-06-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| GB201609597D0 (en) | 2016-06-01 | 2016-07-13 | Univ Sheffield | Therapy |
| CN111108198A (zh) * | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| CN118910166A (zh) * | 2017-05-05 | 2024-11-08 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2019079242A1 (en) * | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| KR20200075865A (ko) * | 2017-10-23 | 2020-06-26 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환에 대한 유전자 요법 |
| JP7350019B2 (ja) * | 2018-05-31 | 2023-09-25 | イミュニティ ファルマ リミテッド | 筋萎縮性側索硬化症(als)を治療するための組成物、及び治療のためにそれを使用する方法 |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| US20210361318A1 (en) | 2018-07-02 | 2021-11-25 | Voyager Therapeutics, Inc. | Cannula system and use thereof |
| JP2021530548A (ja) | 2018-07-24 | 2021-11-11 | 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. | 遺伝子治療製剤を生産するための系および方法 |
| EP3861113A1 (en) | 2018-10-04 | 2021-08-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| CA3115248A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| CN113365603B (zh) | 2018-10-11 | 2025-04-11 | 分贝治疗公司 | Aav1载体及其用于治疗耳部适应症的用途 |
| TW202028468A (zh) | 2018-10-15 | 2020-08-01 | 美商航海家醫療公司 | 用於桿狀病毒/Sf9系統中rAAV之大規模生產的表現載體 |
| EP3890752A1 (en) | 2018-12-06 | 2021-10-13 | Biogen MA Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
| SG11202107645RA (en) | 2019-01-18 | 2021-08-30 | Voyager Therapeutics Inc | Methods and systems for producing aav particles |
| EP3917566A4 (en) | 2019-01-31 | 2022-10-26 | Oregon Health & Science University | METHODS OF USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS |
| WO2020223274A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS |
| WO2020223296A1 (en) * | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
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| EP4430191A2 (en) * | 2021-11-08 | 2024-09-18 | University Of Massachusetts | Oligonucleotides for sod1 modulation |
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| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
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| CN116832050A (zh) * | 2023-07-06 | 2023-10-03 | 南京大学 | 一种用于治疗肌萎缩侧索硬化症的rna递送系统 |
Citations (2)
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| WO2007109097A2 (en) * | 2006-03-16 | 2007-09-27 | Alnylam Pharmaceuticals, Inc. | RNAi MODULATION OF TGF-BETA AND THERAPEUTIC USES THEREOF |
| JP2013143917A (ja) * | 2012-01-13 | 2013-07-25 | Mie Univ | 線維症予防又は治療剤 |
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| JP2023537980A (ja) * | 2020-08-12 | 2023-09-06 | ユーシービー バイオファルマ エスアールエル | メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 |
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