JP7873074B2 - 眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための組成物及び方法 - Google Patents

眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための組成物及び方法

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JP7873074B2
JP7873074B2 JP2021549908A JP2021549908A JP7873074B2 JP 7873074 B2 JP7873074 B2 JP 7873074B2 JP 2021549908 A JP2021549908 A JP 2021549908A JP 2021549908 A JP2021549908 A JP 2021549908A JP 7873074 B2 JP7873074 B2 JP 7873074B2
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JP2022522166A (ja
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ストリングス－ウフォムバー，ヴァネッサ
スーイ，デイヴィッド
カオ，シン－チュー
ダブリュ． ロエルヴィンク，ペトルス
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ベニテック アイピー ホールディングス インコーポレーテッド
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JP2021549908A 2019-02-28 2020-02-28 眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための組成物及び方法 Active JP7873074B2 (ja)

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US201962812187P 2019-02-28 2019-02-28
US62/812,187 2019-02-28
PCT/AU2020/050182 WO2020172720A1 (en) 2019-02-28 2020-02-28 Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)

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CN (2) CN113631706A (enExample)
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Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3866859A4 (en) * 2018-10-17 2023-08-09 Benitec IP Holdings Inc. METHODS OF TREATMENT FOR OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD)
EP4493703A4 (en) * 2022-03-18 2026-08-05 Univ Florida METHODS AND COMPOSITIONS FOR TREATING TMEM43-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
KR20260030147A (ko) * 2023-06-28 2026-03-05 베니텍 아이피 홀딩스 아이엔씨. 인두 근육에 치료 조성물을 투여하기 위한 장치 및 방법
WO2026011009A1 (en) * 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018139634A1 (ja) 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
US7208315B2 (en) 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
US20060166363A1 (en) * 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
WO2007120533A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
EP2242840B1 (en) 2008-01-29 2019-07-24 Applied Genetic Technologies Corporation Recombinant adeno-associated virus production using bhk cells in suspension
RU2664471C2 (ru) * 2013-07-26 2018-08-17 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
CN104815338A (zh) * 2015-05-01 2015-08-05 许瑞安 一种携带kal基因重组腺相关病毒载体的基因治疗药物
CA3012699C (en) 2016-04-21 2023-11-21 Virovek, Inc Aav production in insect cells, methods and compositions therefor
KR102495222B1 (ko) * 2016-12-14 2023-02-09 베니텍 아이피 홀딩스 아이엔씨. 안구인두근위축증 (opmd)의 치료제 및 이의 사용
UY37863A (es) 2017-08-31 2019-03-29 Benitec Biopharma Ltd Virus adenoasociado (aav) con dominio de fosfolipasa modificado

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018139634A1 (ja) 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Molecular Therapy，2018年，Vol. 26, No. 5S1，p. 165，354

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AU2020229886B2 (en) 2025-09-18
JP2022522166A (ja) 2022-04-14
CN121160804A (zh) 2025-12-19
BR112021017054A2 (pt) 2021-11-09
KR20210138030A (ko) 2021-11-18
CN113631706A (zh) 2021-11-09
IL285841A (en) 2021-10-31
EP3931317A4 (en) 2023-01-04
MX2021010412A (es) 2022-01-24
KR102959609B1 (ko) 2026-04-29
EP3931317A1 (en) 2022-01-05
US20220098614A1 (en) 2022-03-31
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