JP2023509443A5 - - Google Patents

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Publication number
JP2023509443A5
JP2023509443A5 JP2022540917A JP2022540917A JP2023509443A5 JP 2023509443 A5 JP2023509443 A5 JP 2023509443A5 JP 2022540917 A JP2022540917 A JP 2022540917A JP 2022540917 A JP2022540917 A JP 2022540917A JP 2023509443 A5 JP2023509443 A5 JP 2023509443A5
Authority
JP
Japan
Prior art keywords
acid sequence
optionally
amino acid
promoter
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022540917A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023509443A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/067664 external-priority patent/WO2021138559A1/en
Publication of JP2023509443A publication Critical patent/JP2023509443A/ja
Publication of JP2023509443A5 publication Critical patent/JP2023509443A5/ja
Pending legal-status Critical Current

Links

JP2022540917A 2019-12-31 2020-12-31 改善されたａａｖ－ａｂｃｄ１コンストラクトならびに副腎白質ジストロフィー（ａｌｄ）および／または副腎脊髄ニューロパチー（ａｍｎ）の処置または予防のための使用 Pending JP2023509443A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962955667P 2019-12-31 2019-12-31
US62/955,667 2019-12-31
PCT/US2020/067664 WO2021138559A1 (en) 2019-12-31 2020-12-31 Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)

Publications (2)

Publication Number Publication Date
JP2023509443A JP2023509443A (ja) 2023-03-08
JP2023509443A5 true JP2023509443A5 (https=) 2023-07-05

Family

ID=76686949

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022540917A Pending JP2023509443A (ja) 2019-12-31 2020-12-31 改善されたａａｖ－ａｂｃｄ１コンストラクトならびに副腎白質ジストロフィー（ａｌｄ）および／または副腎脊髄ニューロパチー（ａｍｎ）の処置または予防のための使用

Country Status (11)

Country Link
US (2) US11779655B2 (https=)
EP (1) EP4085143B1 (https=)
JP (1) JP2023509443A (https=)
KR (1) KR20220128632A (https=)
CN (1) CN116096904A (https=)
AU (1) AU2020416291A1 (https=)
BR (1) BR112022013027A2 (https=)
CA (1) CA3166374A1 (https=)
IL (1) IL294278A (https=)
MX (1) MX2022008201A (https=)
WO (1) WO2021138559A1 (https=)

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AR126727A1 (es) * 2021-08-06 2023-11-08 Wuhan Neurophth Biotechnology Ltd Company Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4
US20240229067A9 (en) * 2022-05-12 2024-07-11 AAVnerGene Inc. Compositions and methods for recombinant parvovirus production
EP4522729A1 (en) * 2022-05-12 2025-03-19 Aavnergene Inc. Compositions and methods for recombinant parvovirus production
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
CN121693566A (zh) * 2023-07-28 2026-03-17 瑞泽恩制药公司 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达

Family Cites Families (23)

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Publication number Priority date Publication date Assignee Title
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EA201100225A1 (ru) * 2008-07-23 2011-08-30 Бёрингер Ингельхайм Фарма Гмбх Унд Ко. Кг Новые регуляторные элементы
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
KR20150029756A (ko) * 2011-06-10 2015-03-18 블루버드 바이오, 인코포레이티드. 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
US9938541B2 (en) * 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
WO2015153889A2 (en) * 2014-04-02 2015-10-08 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
WO2016154055A1 (en) * 2015-03-20 2016-09-29 Bluebird Bio, Inc. Vector formulations
EP3285788B1 (en) * 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
EP4066862A1 (en) 2015-11-05 2022-10-05 The General Hospital Corporation Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy
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WO2017137585A1 (en) * 2016-02-12 2017-08-17 Combigene Ab Vector
CN107287239B (zh) * 2016-04-11 2020-09-22 厦门继景生物技术有限责任公司 一种用于视网膜色素变性的基因治疗载体及药物
JP7165410B2 (ja) * 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
CA3050690A1 (en) * 2017-01-17 2018-07-26 Dana-Farber Cancer Institute, Inc. Compositions and methods for diagnosing and treating peroxisomal diseases
US12012602B2 (en) * 2017-03-10 2024-06-18 The Medical College Of Wisconsin, Inc. Riboswitch modulated gene therapy for retinal diseases
US20200377887A1 (en) * 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
MX2020004092A (es) * 2017-10-18 2021-01-08 Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas.
GB201817470D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy

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