JP2020048571A5 - - Google Patents

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JP2020048571A5
JP2020048571A5 JP2019214352A JP2019214352A JP2020048571A5 JP 2020048571 A5 JP2020048571 A5 JP 2020048571A5 JP 2019214352 A JP2019214352 A JP 2019214352A JP 2019214352 A JP2019214352 A JP 2019214352A JP 2020048571 A5 JP2020048571 A5 JP 2020048571A5
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seq
cells
raav
raav particles
particles
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JP2019214352A
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JP2020048571A (ja
JP7021786B2 (ja
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JP2019214352A 2013-05-21 2019-11-27 キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 Active JP7021786B2 (ja)

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JP2022011418A JP7344595B2 (ja) 2013-05-21 2022-01-28 キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法

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Application Number Priority Date Filing Date Title
US13/899,481 US9920097B2 (en) 2007-04-09 2013-05-21 Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use
US13/899,481 2013-05-21

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JP2016515056A Division JP6628286B2 (ja) 2013-05-21 2014-05-21 キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法

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JP2020048571A JP2020048571A (ja) 2020-04-02
JP2020048571A5 true JP2020048571A5 (https=) 2020-06-25
JP7021786B2 JP7021786B2 (ja) 2022-02-17

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JP2016515056A Active JP6628286B2 (ja) 2013-05-21 2014-05-21 キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
JP2019214352A Active JP7021786B2 (ja) 2013-05-21 2019-11-27 キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
JP2022011418A Active JP7344595B2 (ja) 2013-05-21 2022-01-28 キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法

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JP2016515056A Active JP6628286B2 (ja) 2013-05-21 2014-05-21 キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法

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Country Status (9)

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EP (2) EP3492597A3 (https=)
JP (3) JP6628286B2 (https=)
KR (4) KR102649543B1 (https=)
CN (3) CN111763690A (https=)
AU (3) AU2014274457B2 (https=)
CA (1) CA2917018A1 (https=)
DK (1) DK2999791T3 (https=)
SG (2) SG11201510757PA (https=)
WO (1) WO2014193716A2 (https=)

Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5410017A (en) * 1993-05-21 1995-04-25 Rohm And Haas Company Continuous thermal polycondensation process for preparing polypeptide polymers
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
JP7406783B2 (ja) * 2015-12-14 2023-12-28 ザ 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 パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
IL315358A (en) * 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
PE20200488A1 (es) * 2017-06-27 2020-03-03 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas
KR102696307B1 (ko) * 2017-09-08 2024-08-16 제너레이션 바이오 컴퍼니 비-바이러스성 캡시드-비함유 dna 벡터의 지질 나노입자 제형
AU2019209770B2 (en) * 2018-01-16 2025-07-31 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
SI3740222T1 (sl) * 2018-01-17 2023-11-30 Meiragtx Uk Ii Limited Modificiran kapsidni protein raav za gensko terapijo
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
CN111902539B (zh) * 2018-02-07 2025-05-16 吉尼松公司 杂合调控元件
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド F8遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
JP2021519612A (ja) * 2018-03-29 2021-08-12 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 中和を回避する肝臓向性組換えaav6ベクター
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
EP3784697A4 (en) * 2018-04-27 2022-07-06 Spark Therapeutics, Inc. GMO AAV CAPSIDS WITH INCREASED TROPISM AND AAV VECTORS CONTAINING THE GMO CAPSIDS, AND METHODS FOR THEIR MANUFACTURE AND USE
JP7142815B2 (ja) * 2018-06-21 2022-09-28 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2020014471A1 (en) 2018-07-11 2020-01-16 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
WO2020079256A1 (en) * 2018-10-19 2020-04-23 Genethon Modulation, monitoring and prediction of the immune response directed against aav gene therapy vectors
EP4295913A3 (en) * 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
EP3736330A1 (en) * 2019-05-08 2020-11-11 European Molecular Biology Laboratory Modified adeno-associated virus (aav) particles for gene therapy
AU2020299026A1 (en) * 2019-07-04 2022-01-20 Children's Medical Research Institute Methods and AAV vectors for in vivo transduction
EP3997214B1 (en) * 2019-07-11 2023-08-02 Centre National de la Recherche Scientifique Chemically-modified adeno-associated virus
EP4028064A1 (en) * 2019-09-12 2022-07-20 The Broad Institute, Inc. Engineered adeno-associated virus capsids
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
US20230340526A1 (en) * 2019-11-25 2023-10-26 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target hepatocytes and evade the humoral immune response
WO2021108468A1 (en) * 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target human hepatocytes in the liver of humanized mice
CN115279400B (zh) * 2020-01-10 2026-04-28 布里格姆妇女医院 用于跨血脑屏障递送免疫治疗剂以治疗脑癌的方法和组合物
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
JP2023524577A (ja) * 2020-05-05 2023-06-12 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 改変アデノ随伴ウイルス5カプシドおよびその使用
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
AU2021378075B2 (en) 2020-11-11 2025-03-27 Borea Therapeutics S.R.L. Modified viral particles for gene therapy
CN113647359A (zh) * 2021-08-15 2021-11-16 芜湖职业技术学院 注射衣霉素诱导急性肝损伤小鼠模型的构建方法
CN118201947B (zh) * 2021-10-28 2025-03-21 上海天泽云泰生物医药有限公司 腺相关病毒衣壳
EP4508065A2 (en) * 2022-04-15 2025-02-19 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
GB202407863D0 (en) 2024-06-03 2024-07-17 Purespring Therapeutics Ltd Methods
WO2026001958A1 (zh) * 2024-06-24 2026-01-02 黄菁 Aav载体、aav载体的组合、构建aml pdx和mm pdx非人动物的方法及构建免疫系统人源化非人动物的方法
KR20260014488A (ko) * 2024-07-22 2026-01-30 한국생명공학연구원 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도
CN119380820B (zh) * 2024-10-10 2025-06-17 四川大学华西医院 一种aav2衣壳蛋白变体设计方法、系统和存储介质

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
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
ES2714007T3 (es) * 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2010011404A2 (en) * 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
WO2012109570A1 (en) * 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CN103060331B (zh) * 2012-12-05 2014-10-08 南京医科大学第一附属医院 介导膜稳定cd40l基因的衣壳蛋白突变的双链重组腺相关病毒制备方法与应用

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