JP2021519791A5 - - Google Patents

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Publication number
JP2021519791A5
JP2021519791A5 JP2020553490A JP2020553490A JP2021519791A5 JP 2021519791 A5 JP2021519791 A5 JP 2021519791A5 JP 2020553490 A JP2020553490 A JP 2020553490A JP 2020553490 A JP2020553490 A JP 2020553490A JP 2021519791 A5 JP2021519791 A5 JP 2021519791A5
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JP
Japan
Prior art keywords
aav
mutant
capsid polypeptide
aav capsid
vector
Prior art date
Application number
JP2020553490A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2019191701A5 (https=
JP7378417B2 (ja
JP2021519791A (ja
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Priority claimed from PCT/US2019/025026 external-priority patent/WO2019191701A1/en
Publication of JP2021519791A publication Critical patent/JP2021519791A/ja
Publication of JPWO2019191701A5 publication Critical patent/JPWO2019191701A5/ja
Publication of JP2021519791A5 publication Critical patent/JP2021519791A5/ja
Application granted granted Critical
Publication of JP7378417B2 publication Critical patent/JP7378417B2/ja
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JP2020553490A 2018-03-30 2019-03-29 増強されたヒト膵臓トロピズムを有する新規な組換えアデノ随伴ウイルスキャプシド Active JP7378417B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862651010P 2018-03-30 2018-03-30
US62/651,010 2018-03-30
US201862745226P 2018-10-12 2018-10-12
US62/745,226 2018-10-12
PCT/US2019/025026 WO2019191701A1 (en) 2018-03-30 2019-03-29 Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism

Publications (4)

Publication Number Publication Date
JP2021519791A JP2021519791A (ja) 2021-08-12
JPWO2019191701A5 JPWO2019191701A5 (https=) 2022-02-21
JP2021519791A5 true JP2021519791A5 (https=) 2022-02-21
JP7378417B2 JP7378417B2 (ja) 2023-11-13

Family

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JP2020553490A Active JP7378417B2 (ja) 2018-03-30 2019-03-29 増強されたヒト膵臓トロピズムを有する新規な組換えアデノ随伴ウイルスキャプシド

Country Status (5)

Country Link
US (2) US11608510B2 (https=)
EP (1) EP3774853A1 (https=)
JP (1) JP7378417B2 (https=)
CN (1) CN112004820B (https=)
WO (1) WO2019191701A1 (https=)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11608510B2 (en) * 2018-03-30 2023-03-21 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2021154414A2 (en) 2020-01-29 2021-08-05 Catalyst Biosciences, Inc. Gene therapy for hemophilia b with a chimeric aav capsid vector encoding modified factor ix polypeptides
TW202142554A (zh) * 2020-02-25 2021-11-16 澳洲兒童醫學研究所 腺相關病毒蛋白殼多肽及載體
JP2023518415A (ja) * 2020-03-19 2023-05-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えaavにおいてcapおよびrep配列の逆パッケージングを低減するための組成物および方法
CN117377500A (zh) * 2021-04-26 2024-01-09 阿雷克森国际制药有限公司 具有改善的组织向性的腺相关病毒载体衣壳
TW202309066A (zh) * 2021-04-27 2023-03-01 賓州大學委員會 衍生自豬的腺相關病毒衣殼及其用途
CA3219847A1 (en) * 2021-06-02 2022-12-08 Sander Jan Hendrik Van Deventer Adeno-associated virus vectors modified to bind high-density lipoprotein
CA3222911A1 (en) * 2021-06-18 2022-12-22 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
JP2024527496A (ja) * 2021-06-18 2024-07-25 ダイノ セラピューティクス インコーポレイテッド カプシドバリアント及びそれを使用する方法
WO2023034990A1 (en) * 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034980A1 (en) * 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240376496A1 (en) * 2021-09-03 2024-11-14 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240384242A1 (en) * 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034994A1 (en) * 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
CN114107231B (zh) * 2021-12-13 2023-08-18 重庆大学 实现全脑突触后神经元胞体标记的重组腺相关病毒及其应用
WO2023114835A1 (en) * 2021-12-15 2023-06-22 Fractyl Health, Inc. Gene therapies for metabolic disorders
CN116686050B (zh) * 2021-12-30 2026-01-23 京东方科技集团股份有限公司 预测大分子间相互作用的负样本集的生成方法、预测大分子间相互作用的方法、模型的训练方法
CN114561337B (zh) * 2022-03-09 2023-10-03 广州源井生物科技有限公司 一种提高HepG2细胞克隆形成率的单克隆增强培养基和方法
GB202304767D0 (en) * 2023-03-30 2023-05-17 Ucl Business Ltd Synthetic AAV Capsid
AU2024262827A1 (en) * 2023-04-27 2025-10-16 Oregon Health & Science University Kidney-targeting aav capsids and methods of use thereof
WO2024233135A1 (en) * 2023-05-05 2024-11-14 The Regents Of The University Of California Long-read sequencing of diverse dna libraries through barcode-labeled short reads
WO2025019295A2 (en) * 2023-07-17 2025-01-23 Oregon Health & Science University Renal pelvic injection for drug delivery to the kidney
GB202407863D0 (en) 2024-06-03 2024-07-17 Purespring Therapeutics Ltd Methods

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
JP3534749B2 (ja) 1991-08-20 2004-06-07 アメリカ合衆国 アデノウイルスが介在する胃腸管への遺伝子の輸送
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
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
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
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
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
SI3235827T1 (sl) * 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US8106180B2 (en) 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
US20050266552A1 (en) 2003-12-05 2005-12-01 Doench John G Reagents and methods for identification of RNAi pathway genes and chemical modulators of RNAi
KR100614827B1 (ko) 2004-05-06 2006-08-25 재단법인서울대학교산학협력재단 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
ES2857773T5 (es) * 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
US11136557B2 (en) * 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
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
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
US10179176B2 (en) 2016-02-16 2019-01-15 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
US11608510B2 (en) * 2018-03-30 2023-03-21 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism

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