CN114667349A - 用于调节腺相关病毒(aav)和aav受体(aavr)之间的相互作用以改变aav生物分布的方法和组合物 - Google Patents

用于调节腺相关病毒(aav)和aav受体(aavr)之间的相互作用以改变aav生物分布的方法和组合物 Download PDF

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CN114667349A
CN114667349A CN202080077968.8A CN202080077968A CN114667349A CN 114667349 A CN114667349 A CN 114667349A CN 202080077968 A CN202080077968 A CN 202080077968A CN 114667349 A CN114667349 A CN 114667349A
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modified
raav
capsid protein
aav
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E.津恩
C.蒂珀
L.H.范登贝格
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University of California San Diego UCSD
Massachusetts Eye and Ear
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Massachusetts Eye and Ear Infirmary
University of California San Diego UCSD
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CN202080077968.8A 2019-09-09 2020-09-09 用于调节腺相关病毒(aav)和aav受体(aavr)之间的相互作用以改变aav生物分布的方法和组合物 Pending CN114667349A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962897973P 2019-09-09 2019-09-09
US62/897,973 2019-09-09
US201962934996P 2019-11-13 2019-11-13
US62/934,996 2019-11-13
PCT/US2020/050027 WO2021050614A2 (en) 2019-09-09 2020-09-09 Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav

Publications (1)

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CN114667349A true CN114667349A (zh) 2022-06-24

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US (1) US12570998B2 (https=)
EP (1) EP4028531A2 (https=)
JP (1) JP2022547197A (https=)
KR (1) KR20220054435A (https=)
CN (1) CN114667349A (https=)
AU (1) AU2020347165A1 (https=)
BR (1) BR112022004183A2 (https=)
CA (1) CA3153972A1 (https=)
IL (1) IL291136A (https=)
WO (1) WO2021050614A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3100006A1 (en) 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Altering tissue tropism of adeno-associated viruses
CA3153972A1 (en) 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
WO2022150634A2 (en) * 2021-01-08 2022-07-14 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
US20240352077A1 (en) * 2021-08-25 2024-10-24 Children's Medical Research Institute Modified aav capsids and vectors
US20240425549A1 (en) * 2021-08-25 2024-12-26 Children's Medical Research Institute Modified aav capsid polypeptides and vectors
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2023095034A1 (en) * 2021-11-24 2023-06-01 Novartis Ag Modulators of adeno-associated virus transduction and uses thereof
WO2023106256A1 (ja) * 2021-12-06 2023-06-15 学校法人順天堂 改変型アデノ随伴ウイルスベクター
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025179124A1 (en) * 2024-02-23 2025-08-28 The Board Of Regents Of The University Of Texas System Enhanced aav9 capsid protein
WO2025194042A1 (en) 2024-03-14 2025-09-18 Affinia Therapeutics Inc. Plasmid system for production of recombinant adenoassociated virus
WO2025235491A1 (en) 2024-05-07 2025-11-13 Affinia Therapeutics Inc. Recombinant aav for treatment of cardiac diseases

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018152333A1 (en) * 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
WO2019028306A2 (en) * 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
WO2019104279A1 (en) * 2017-11-27 2019-05-31 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
JP5915467B2 (ja) 2012-08-30 2016-05-11 株式会社島津製作所 液体クロマトグラフ検出器用送液管及び液体クロマトグラフ
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
US9474181B2 (en) 2013-12-23 2016-10-18 Dell Products, L.P. Combined power and management module for use in a rack-configured information handling system
WO2015164757A1 (en) * 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
JP6475998B2 (ja) 2015-02-10 2019-02-27 デクセリアルズ株式会社 タッチパッド用アンテナ装置、及び電子機器
CN108603235A (zh) 2015-07-30 2018-09-28 马萨诸塞眼科耳科诊所 祖先病毒序列及其用途
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
SG10201913266UA (en) 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP6947739B2 (ja) 2016-02-16 2021-10-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド
JP6936788B2 (ja) 2016-04-01 2021-09-22 株式会社カネカ 軽量化及びリブ設計が可能な樹脂組成物による表面性が良好な射出発泡成形体
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CA3061968A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses
US11247659B2 (en) 2017-08-08 2022-02-15 Aisin Seiki Kabushiki Kaisha Vehicle control apparatus
CA3100006A1 (en) 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Altering tissue tropism of adeno-associated viruses
WO2020041498A1 (en) 2018-08-21 2020-02-27 Massachusetts Eye And Ear Infirmary Compositions and methods for modulating transduction efficiency of adeno-associated viruses
CA3153972A1 (en) 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
WO2022150634A2 (en) 2021-01-08 2022-07-14 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018152333A1 (en) * 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
WO2019028306A2 (en) * 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
WO2019104279A1 (en) * 2017-11-27 2019-05-31 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LOCHRIE MA 等: "Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization", J VIROL, vol. 80, no. 2, 31 January 2006 (2006-01-31), pages 1 - 2, XP055549257, DOI: 10.1128/JVI.80.2.821-834.2006 *

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CA3153972A1 (en) 2021-03-18
IL291136A (en) 2022-05-01
US12570998B2 (en) 2026-03-10
AU2020347165A1 (en) 2022-03-31
WO2021050614A3 (en) 2021-05-20
EP4028531A2 (en) 2022-07-20
KR20220054435A (ko) 2022-05-02
WO2021050614A2 (en) 2021-03-18
US20220411820A1 (en) 2022-12-29
BR112022004183A2 (pt) 2022-05-31
JP2022547197A (ja) 2022-11-10

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