JP7451497B2 - アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法 - Google Patents

アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法 Download PDF

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JP7451497B2
JP7451497B2 JP2021509223A JP2021509223A JP7451497B2 JP 7451497 B2 JP7451497 B2 JP 7451497B2 JP 2021509223 A JP2021509223 A JP 2021509223A JP 2021509223 A JP2021509223 A JP 2021509223A JP 7451497 B2 JP7451497 B2 JP 7451497B2
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gpr108
cells
aav
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JPWO2020041498A5 (enExample
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エイチ． ヴァンデンベルゲ，ルク
ドゥデク，アマンダ
ザバレタ ラサルテ，ネレア
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マサチューセッツ アイ アンド イヤー インファーマリー
ザ スケペンス アイ リサーチ インスティテュート，インコーポレイテッド
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    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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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JP2021509223A 2018-08-21 2019-08-21 アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法 Active JP7451497B2 (ja)

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US201862720859P 2018-08-21 2018-08-21
US62/720,859 2018-08-21
PCT/US2019/047546 WO2020041498A1 (en) 2018-08-21 2019-08-21 Compositions and methods for modulating transduction efficiency of adeno-associated viruses

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JP2021533795A JP2021533795A (ja) 2021-12-09
JPWO2020041498A5 JPWO2020041498A5 (enExample) 2022-07-25
JP7451497B2 true JP7451497B2 (ja) 2024-03-18

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US (1) US12195745B2 (enExample)
EP (1) EP3841109A4 (enExample)
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AU (1) AU2019325332B2 (enExample)
WO (1) WO2020041498A1 (enExample)

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CN112368391A (zh) 2018-05-11 2021-02-12 马萨诸塞眼科耳科诊所 腺相关病毒的肝脏特异性嗜性
WO2021205028A1 (en) 2020-04-09 2021-10-14 University Of Sheffield Gene therapy treatment
US20230295658A1 (en) * 2020-11-10 2023-09-21 The Children's Hospital Of Philadelphia Compositions and methods for in vivo gene transfer
GB202215198D0 (en) 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
WO2025114699A1 (en) 2023-11-30 2025-06-05 Cytomos Limited Cell selection
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2

Citations (4)

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WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
US20150376240A1 (en) 2013-02-08 2015-12-31 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
WO2017083423A1 (en) 2015-11-10 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating aav infection
WO2018022608A2 (en) 2016-07-26 2018-02-01 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins

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US6855314B1 (en) * 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
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
ATE405295T1 (de) * 2002-05-01 2008-09-15 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
EP2383346B1 (en) * 2005-04-07 2014-10-08 The Trustees of the University of Pennsylvania Modified AAVrh.64 capsids, compositions containing same and uses thereof
EP3209311B1 (en) * 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
WO2017189959A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11364308B2 (en) * 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
EP3497207B1 (en) * 2016-08-15 2021-01-06 Genzyme Corporation Methods for detecting aav
WO2018119330A2 (en) * 2016-12-22 2018-06-28 Oregon Health & Science University Adeno associated viral vectors
US20210292373A1 (en) * 2018-07-10 2021-09-23 University Of Florida Research Foundation, Incorporated Aav vp1u chimeras
TWI862583B (zh) * 2019-04-26 2024-11-21 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼

Patent Citations (4)

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Publication number Priority date Publication date Assignee Title
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
US20150376240A1 (en) 2013-02-08 2015-12-31 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
WO2017083423A1 (en) 2015-11-10 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating aav infection
WO2018022608A2 (en) 2016-07-26 2018-02-01 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins

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US20210317478A1 (en) 2021-10-14
US12195745B2 (en) 2025-01-14
WO2020041498A1 (en) 2020-02-27
AU2019325332A1 (en) 2021-03-18
EP3841109A4 (en) 2022-09-21
AU2019325332B2 (en) 2025-11-13
JP2021533795A (ja) 2021-12-09
EP3841109A1 (en) 2021-06-30

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