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 PDF

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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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sod1
sirna
vector
aav
als
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JP2017535266A5 (enExample
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ウェン‐イー サー、ディナー
ウェン‐イー サー、ディナー
ホウ、ジンジャオ
イー． ノンネンマッチャー、マシュー
イー． ノンネンマッチャー、マシュー
ジョウ、ペンチェン
ホスバッハ、マルクス
デッカート、ヨヘン
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Voyager Therapeutics Inc
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Voyager Therapeutics Inc
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Publication of JP2017535266A publication Critical patent/JP2017535266A/ja
Publication of JP2017535266A5 publication Critical patent/JP2017535266A5/ja
Priority to JP2022030717A priority Critical patent/JP2022081546A/ja
Priority to JP2024050822A priority patent/JP2024079794A/ja
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JP2017525847A 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 Pending JP2017535266A (ja)

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)

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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

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JP2017535266A true JP2017535266A (ja) 2017-11-30
JP2017535266A5 JP2017535266A5 (enExample) 2018-10-25

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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

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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

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US (5) US10597660B2 (enExample)
EP (1) EP3218484A4 (enExample)
JP (4) JP2017535266A (enExample)
KR (2) KR20230169197A (enExample)
CN (2) CN107109407A (enExample)
AU (2) AU2015346162B2 (enExample)
CA (2) CA2967367C (enExample)
HK (1) HK1244299A1 (enExample)
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MX (2) MX2017006216A (enExample)
RU (2) RU2020108189A (enExample)
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Cited By (5)

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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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EP4137573A3 (en) 2014-04-01 2023-05-03 Biogen MA Inc. Compositions for modulating sod-1 expression
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
CN117904112A (zh) 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
GB201609597D0 (en) 2016-06-01 2016-07-13 Univ Sheffield Therapy
CN119491003A (zh) * 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CA3061652A1 (en) * 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
TWI897844B (zh) * 2017-05-05 2025-09-21 美商航海家醫療公司 調節多核苷酸
WO2019079242A1 (en) * 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CA3083582A1 (en) * 2017-10-23 2019-05-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
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
EP3807404A1 (en) 2018-06-13 2021-04-21 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
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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
JP2022512621A (ja) 2018-10-05 2022-02-07 ボイジャー セラピューティクス インコーポレイテッド Ａａｖ産生タンパク質をコードする操作された核酸コンストラクト
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
MA54383A (fr) * 2018-12-06 2021-10-13 Biogen Ma Inc Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique
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US20220168450A1 (en) * 2019-04-29 2022-06-02 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
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US20220389067A1 (en) * 2019-11-05 2022-12-08 The Board Of Trustees Of The Leland Stanford Junior University Methods of Treating Neurodegenerative Diseases Caused by G4C2 Expansion in C9ORF72
EP3892283A1 (en) * 2020-04-09 2021-10-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Nucleic acids encoding human fus protein and use in the treatment of amyotrophic lateral sclerosis (als)
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
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WO2022109368A1 (en) * 2020-11-20 2022-05-27 Texas Tech University System Methods of treating or preventing neurological disorders using zpr1
CA3210763A1 (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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AU2022348798A1 (en) 2021-09-16 2024-03-14 H. Lundbeck A/S Compositions and methods for treating synucleinopathies
CA3232577A1 (en) * 2021-09-20 2023-03-23 Alexander SHTILBANS Combination therapy for neurodegenerative diseases
EP4430191A2 (en) * 2021-11-08 2024-09-18 University Of Massachusetts Oligonucleotides for sod1 modulation
CN119730859A (zh) * 2022-06-15 2025-03-28 箭头药业股份有限公司 用于抑制超氧化物歧化酶1(SOD1)表达的RNAi剂、其组合物及使用方法
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
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