JP7378417B2 - 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 - Google Patents

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 Download PDF

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JP7378417B2
JP7378417B2 JP2020553490A JP2020553490A JP7378417B2 JP 7378417 B2 JP7378417 B2 JP 7378417B2 JP 2020553490 A JP2020553490 A JP 2020553490A JP 2020553490 A JP2020553490 A JP 2020553490A JP 7378417 B2 JP7378417 B2 JP 7378417B2
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aav
mutant
capsid polypeptide
capsid
human
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カーティア ペクルン，
マーク， エー． ケイ，
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Leland Stanford Junior University
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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

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

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

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EP3774853A1 (en) * 2018-03-30 2021-02-17 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
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
BR112022016965A2 (pt) * 2020-02-25 2022-12-06 Childrens Medical Res Institute Polipeptídeos e vetores de capsídeo do vírus adenoassociado
US20230235353A1 (en) * 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
US20250188126A1 (en) * 2021-04-26 2025-06-12 Alexion Pharma International Operations Limited Adeno-associated viral vector capsids with improved tissue tropism
WO2022232267A1 (en) * 2021-04-27 2022-11-03 The Trustees Of The University Of Pennsylvania Porcine-derived adeno-associated virus capsids and uses thereof
US20240374755A1 (en) * 2021-06-02 2024-11-14 Uniqure Biopharma B.V. Adeno-associated virus vectors modified to bind High-Density Lipoprotein
JP2024522230A (ja) * 2021-06-18 2024-06-11 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
EP4355889A4 (en) * 2021-06-18 2025-07-09 Dyno Therapeutics Inc CAPSID VARIANTS AND METHODS OF USE
WO2023034996A1 (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
WO2023034990A1 (en) * 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
JP2024533174A (ja) * 2021-09-03 2024-09-12 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法
JP2024534888A (ja) * 2021-09-03 2024-09-26 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
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
AU2022414072A1 (en) * 2021-12-15 2024-07-04 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
CN121013858A (zh) * 2023-04-27 2025-11-25 俄勒冈健康与科学大学 靶向肾脏的aav衣壳及其使用方法
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

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