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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- 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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- 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
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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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- 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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- General Engineering & Computer Science (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (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 (cg-RX-API-DMAC7.html) | 2018-10-25 |
Family
ID=55955103
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 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3218484A4 (cg-RX-API-DMAC7.html) |
| JP (4) | JP2017535266A (cg-RX-API-DMAC7.html) |
| KR (2) | KR102599909B1 (cg-RX-API-DMAC7.html) |
| CN (2) | CN114717264A (cg-RX-API-DMAC7.html) |
| AU (2) | AU2015346162B2 (cg-RX-API-DMAC7.html) |
| CA (2) | CA3193811A1 (cg-RX-API-DMAC7.html) |
| HK (1) | HK1244299A1 (cg-RX-API-DMAC7.html) |
| IL (3) | IL292999A (cg-RX-API-DMAC7.html) |
| MX (2) | MX2017006216A (cg-RX-API-DMAC7.html) |
| RU (2) | RU2020108189A (cg-RX-API-DMAC7.html) |
| SG (1) | SG11201703281RA (cg-RX-API-DMAC7.html) |
| WO (1) | WO2016077687A1 (cg-RX-API-DMAC7.html) |
| ZA (1) | ZA201702991B (cg-RX-API-DMAC7.html) |
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) |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT3126499T (lt) | 2014-04-01 | 2020-07-27 | Biogen Ma Inc. | Kompozicijos, skirtos sod-1 ekspresijos moduliavimui |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| IL302748B1 (en) | 2016-05-18 | 2025-09-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| GB201609597D0 (en) | 2016-06-01 | 2016-07-13 | Univ Sheffield | Therapy |
| US11752181B2 (en) * | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| CA3061365A1 (en) * | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| 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) |
| US20210261981A1 (en) * | 2017-10-23 | 2021-08-26 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| MX2020012532A (es) * | 2018-05-31 | 2021-04-28 | Immunity Pharma Ltd | Composiciones y procedimientos de su uso para el tratamiento de esclerosis lateral amiotrofica (ela). |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (UTR) for AAV production |
| US20210361318A1 (en) | 2018-07-02 | 2021-11-25 | Voyager Therapeutics, Inc. | Cannula system and use thereof |
| CN112770812A (zh) | 2018-07-24 | 2021-05-07 | 沃雅戈治疗公司 | 产生基因治疗制剂的系统和方法 |
| TW202035689A (zh) | 2018-10-04 | 2020-10-01 | 美商航海家醫療公司 | 測量病毒載體粒子的效價及強度之方法 |
| AU2019354793A1 (en) | 2018-10-05 | 2021-05-13 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding AAV production proteins |
| JP2022505106A (ja) | 2018-10-15 | 2022-01-14 | ボイジャー セラピューティクス インコーポレイテッド | バキュロウイルス／Ｓｆ９システムにおけるｒＡＡＶの大規模産生のための発現ベクター |
| KR20210102294A (ko) * | 2018-12-06 | 2021-08-19 | 바이오젠 엠에이 인코포레이티드 | 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질 |
| WO2020150556A1 (en) | 2019-01-18 | 2020-07-23 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
| AU2020216480A1 (en) | 2019-01-31 | 2021-09-09 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of AAV capsids |
| US20220168450A1 (en) * | 2019-04-29 | 2022-06-02 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| EP3962536A1 (en) | 2019-04-29 | 2022-03-09 | Voyager Therapeutics, Inc. | Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors |
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| AU2022220704A1 (en) * | 2021-02-12 | 2022-08-18 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
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