JP2022522166A5 - - Google Patents

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Publication number
JP2022522166A5
JP2022522166A5 JP2021549908A JP2021549908A JP2022522166A5 JP 2022522166 A5 JP2022522166 A5 JP 2022522166A5 JP 2021549908 A JP2021549908 A JP 2021549908A JP 2021549908 A JP2021549908 A JP 2021549908A JP 2022522166 A5 JP2022522166 A5 JP 2022522166A5
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JP
Japan
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JP2021549908A
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Japanese (ja)
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JP2022522166A (ja
JPWO2020172720A5 (https=
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Publication date
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Priority claimed from PCT/AU2020/050182 external-priority patent/WO2020172720A1/en
Publication of JP2022522166A publication Critical patent/JP2022522166A/ja
Publication of JP2022522166A5 publication Critical patent/JP2022522166A5/ja
Publication of JPWO2020172720A5 publication Critical patent/JPWO2020172720A5/ja
Pending legal-status Critical Current

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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 Pending JP2022522166A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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)

Publications (3)

Publication Number Publication Date
JP2022522166A JP2022522166A (ja) 2022-04-14
JP2022522166A5 true JP2022522166A5 (https=) 2023-03-03
JPWO2020172720A5 JPWO2020172720A5 (https=) 2023-03-03

Family

ID=72238736

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021549908A Pending JP2022522166A (ja) 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

Country Status (11)

Country Link
US (1) US20220098614A1 (https=)
EP (1) EP3931317A4 (https=)
JP (1) JP2022522166A (https=)
CN (2) CN121160804A (https=)
AU (1) AU2020229886B2 (https=)
BR (1) BR112021017054A2 (https=)
CA (1) CA3130221A1 (https=)
IL (1) IL285841A (https=)
MX (1) MX2021010412A (https=)
SG (1) SG11202108469XA (https=)
WO (1) WO2020172720A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112867511A (zh) * 2018-10-17 2021-05-28 贝尼泰克生物制药有限公司 用于治疗眼咽肌营养不良(opmd)的方法
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

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US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
US7208315B2 (en) 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
EP2017338A1 (en) 2001-05-24 2009-01-21 Genzyme Corporation Muscle-specific expression vectors
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
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
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A 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
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
WO2007120533A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 aav의 생산에 유용한 aav-rep78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
EP3024497B1 (en) * 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
EP3039129B1 (en) 2013-08-30 2018-06-27 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
KR20230079511A (ko) 2016-04-21 2023-06-07 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물
EP3555294A4 (en) * 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE
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
UY37863A (es) 2017-08-31 2019-03-29 Benitec Biopharma Ltd Virus adenoasociado (aav) con dominio de fosfolipasa modificado

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