AU2018234695B2 - Polyploid adeno-associated virus vectors and methods of making and using the same - Google Patents

Polyploid adeno-associated virus vectors and methods of making and using the same Download PDF

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AU2018234695B2
AU2018234695B2 AU2018234695A AU2018234695A AU2018234695B2 AU 2018234695 B2 AU2018234695 B2 AU 2018234695B2 AU 2018234695 A AU2018234695 A AU 2018234695A AU 2018234695 A AU2018234695 A AU 2018234695A AU 2018234695 B2 AU2018234695 B2 AU 2018234695B2
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aav
capsid
virus
vector
transduction
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AU2018234695A1 (en
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Chengwen Li
Richard Jude Samulski
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University of North Carolina at Chapel Hill
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    • C12N15/09Recombinant DNA-technology
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    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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AU2018234695A 2017-03-15 2018-03-15 Polyploid adeno-associated virus vectors and methods of making and using the same Active AU2018234695B2 (en)

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US201762471762P 2017-03-15 2017-03-15
US62/471,762 2017-03-15
US201762520901P 2017-06-16 2017-06-16
US62/520,901 2017-06-16
US201862630558P 2018-02-14 2018-02-14
US62/630,558 2018-02-14
PCT/US2018/022725 WO2018170310A1 (en) 2017-03-15 2018-03-15 Polyploid adeno-associated virus vectors and methods of making and using the same

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JP (1) JP7132934B2 (https=)
CN (2) CN110770346B (https=)
AU (2) AU2018234695B2 (https=)
CA (1) CA3054711A1 (https=)
ES (1) ES2966692T3 (https=)
IL (3) IL316259A (https=)
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Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
WO2019216932A1 (en) * 2018-05-07 2019-11-14 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
US20200069819A1 (en) * 2018-05-14 2020-03-05 Biomarin Pharmaceutical Inc. Stable expression of aav vectors in juvenile subjects
MX2021005727A (es) * 2018-11-16 2021-09-10 Spark Therapeutics Inc Ensayo in vitro para detectar potenciadores e inhibidores de la transducción de vectores virales adenoasociados (aav) y/o para detectar o cuantificar anticuerpos de unión anti-aav.
CN113395975A (zh) * 2018-11-20 2021-09-14 宾夕法尼亚州大学信托人 用于靶向血脑屏障的组合物和方法
AU2019418750A1 (en) * 2019-01-02 2021-07-22 Intima Bioscience, Inc. Modified adeno-associated viral vectors for use in genetic engineering
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
EP3955970A1 (en) * 2019-04-15 2022-02-23 University Of Iowa Research Foundation Methods and compositions for transgene expression
PE20220014A1 (es) * 2019-04-29 2022-01-11 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
AR122013A1 (es) * 2020-05-05 2022-08-03 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso
CN116121274B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
JP2024515626A (ja) * 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル, インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン
CA3242989A1 (en) * 2021-12-21 2023-06-29 Research Institute At Nationwide Children's Hospital MATERIALS AND METHODS FOR THE TREATMENT OF MUSCULAR BELLY DYSTROPHY
EP4703468A1 (en) * 2024-08-29 2026-03-04 Sartorius Albumedix Limited Methods and uses

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
AU7966987A (en) 1986-09-08 1988-04-07 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
WO1990005142A1 (en) 1988-11-10 1990-05-17 Imperial Cancer Research Technology Ltd. Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
CA2223837C (en) 1995-06-07 2010-05-25 University Of North Carolina At Chapel Hill Aav transduction of myoblasts
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
DE69636937T3 (de) 1995-12-15 2011-01-05 Virxsys Corp. Durch trans-spaltung erhaltene therapeutische molekule
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
AU774706B2 (en) 1998-09-22 2004-07-08 Johns Hopkins University, The Methods for large-scale production of recombinant AAV vectors
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
CA2373110A1 (en) * 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
US6648075B2 (en) * 2001-07-13 2003-11-18 Weatherford/Lamb, Inc. Method and apparatus for expandable liner hanger with bypass
US7071172B2 (en) 2002-04-30 2006-07-04 The University Of North Carolina At Chapel Hill Secretion signal vectors
WO2004027019A2 (en) * 2002-05-01 2004-04-01 University Of Florida Research Foundation, Inc. Improved raav expression systems for genetic modification of specific capsid proteins
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
JP4346526B2 (ja) 2004-08-31 2009-10-21 株式会社東芝 半導体集積回路装置
CN101056539B (zh) 2004-09-09 2012-12-12 综合医院公司 调节心脏细胞中的磷酸酶活性
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
JP2008539698A (ja) 2005-04-29 2008-11-20 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 転写後レベルでの核酸発現調節のための方法および組成物
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US7989606B2 (en) 2006-02-10 2011-08-02 The University Of Cincinnati Phosphatase inhibitor protein-1 as a regulator of cardiac function
WO2008027084A2 (en) * 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にnNOSを回復させる合成ミニ/ミクロジストロフィン遺伝子
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
US20120093775A1 (en) * 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
ES2685611T3 (es) * 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
US9441206B2 (en) 2011-10-28 2016-09-13 The University Of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
KR20240090694A (ko) * 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
WO2016210170A1 (en) * 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues

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NZ756587A (en) 2025-12-19
AU2024227303A1 (en) 2024-10-31
IL268891B2 (en) 2024-09-01
EP3610023A4 (en) 2021-01-20
IL268891A (en) 2019-10-31
IL312266B2 (en) 2025-03-01
IL312266B1 (en) 2024-11-01
CN110770346A (zh) 2020-02-07
JP7132934B2 (ja) 2022-09-07
IL268891B1 (en) 2024-05-01
CA3054711A1 (en) 2018-09-20
EP4303225A2 (en) 2024-01-10
EP3610023A1 (en) 2020-02-19
CN110770346B (zh) 2024-01-12
EP3610023B1 (en) 2023-10-04
EP4303225A3 (en) 2024-04-10
AU2018234695A1 (en) 2019-09-12
EP4303225B1 (en) 2025-11-26
JP2020511127A (ja) 2020-04-16
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ES2966692T3 (es) 2024-04-23
IL316259A (en) 2024-12-01
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