JP2022553083A5 - - Google Patents

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Publication number
JP2022553083A5
JP2022553083A5 JP2022523915A JP2022523915A JP2022553083A5 JP 2022553083 A5 JP2022553083 A5 JP 2022553083A5 JP 2022523915 A JP2022523915 A JP 2022523915A JP 2022523915 A JP2022523915 A JP 2022523915A JP 2022553083 A5 JP2022553083 A5 JP 2022553083A5
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JP
Japan
Prior art keywords
raav
composition
sequence
vector
inh
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Granted
Application number
JP2022523915A
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English (en)
Japanese (ja)
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JP7659550B2 (ja
JP2022553083A (ja
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Priority claimed from PCT/US2020/056984 external-priority patent/WO2021081280A1/en
Publication of JP2022553083A publication Critical patent/JP2022553083A/ja
Publication of JP2022553083A5 publication Critical patent/JP2022553083A5/ja
Priority to JP2025055764A priority Critical patent/JP2025098221A/ja
Application granted granted Critical
Publication of JP7659550B2 publication Critical patent/JP7659550B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2022523915A 2019-10-23 2020-10-23 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療 Active JP7659550B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025055764A JP2025098221A (ja) 2019-10-23 2025-03-28 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924877P 2019-10-23 2019-10-23
US62/924,877 2019-10-23
PCT/US2020/056984 WO2021081280A1 (en) 2019-10-23 2020-10-23 Adeno-associated virus vectors based gene therapy for hereditary angioedema

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025055764A Division JP2025098221A (ja) 2019-10-23 2025-03-28 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療

Publications (3)

Publication Number Publication Date
JP2022553083A JP2022553083A (ja) 2022-12-21
JP2022553083A5 true JP2022553083A5 (https=) 2023-10-30
JP7659550B2 JP7659550B2 (ja) 2025-04-09

Family

ID=73498274

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022523915A Active JP7659550B2 (ja) 2019-10-23 2020-10-23 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療
JP2025055764A Ceased JP2025098221A (ja) 2019-10-23 2025-03-28 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025055764A Ceased JP2025098221A (ja) 2019-10-23 2025-03-28 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療

Country Status (5)

Country Link
US (1) US20230043051A1 (https=)
EP (1) EP4048800A1 (https=)
JP (2) JP7659550B2 (https=)
CN (1) CN115176022A (https=)
WO (1) WO2021081280A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023503850A (ja) * 2019-11-14 2023-02-01 バイオマリン ファーマシューティカル インコーポレイテッド 肝臓特異的遺伝子療法ベクターによる遺伝性血管浮腫の治療
WO2022130014A1 (en) * 2020-12-16 2022-06-23 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
CA3207268A1 (en) * 2021-01-27 2022-08-04 Spark Therapeutics, Inc. Compositions and methods for treating hereditary angioedema
US20240408240A1 (en) * 2021-10-01 2024-12-12 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations
CN117947040A (zh) 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用

Family Cites Families (26)

* Cited by examiner, † Cited by third party
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US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US7419829B2 (en) 2000-10-06 2008-09-02 Oxford Biomedica (Uk) Limited Vector system
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
RU2305708C2 (ru) 2001-10-02 2007-09-10 Энститю Клейтон Де Ля Решерш Рекомбинантный лентивирусный вектор, клетка-хозяин, трансдуцированная лентивирусным вектором, способ ее трансдукции и применение
DK1737966T3 (da) 2004-04-02 2012-07-09 Univ Texas Cancerspecifik promotorer
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
GB201206455D0 (en) 2012-04-12 2012-05-30 Royal Holloway & Bedford New College Gene expression
WO2014063753A1 (en) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
CA2906770A1 (en) 2013-03-15 2014-09-18 Vlp Biotech, Inc. Palivizumab epitope-based virus-like particles
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
CL2014003590A1 (es) 2014-12-30 2015-07-10 Univ Chile Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje.
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
US11007280B2 (en) 2015-03-17 2021-05-18 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
CN113999875A (zh) * 2015-05-28 2022-02-01 康奈尔大学 腺相关病毒介导的c1ei递送作为用于血管性水肿的疗法
US10214731B2 (en) * 2015-05-28 2019-02-26 Cornell University Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
EP3368672B1 (en) 2015-10-27 2020-11-25 Celltheon Corporation Chimeric post-transcriptional regulatory element
CN108463243B (zh) * 2015-11-19 2022-06-14 夏尔人类遗传性治疗公司 重组人c1酯酶抑制剂及其用途
WO2017139381A1 (en) * 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US20190161530A1 (en) 2016-04-07 2019-05-30 Bluebird Bio, Inc. Chimeric antigen receptor t cell compositions
MX2018014256A (es) 2016-05-20 2019-08-16 Harvard College Metodos de terapia genica para enfermedades y condiciones relacionadas con la edad.
CA3053534A1 (en) 2017-02-17 2018-08-23 Purdue Research Foundation Targeted ligand-payload based drug delivery for cell therapy
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

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