JP2021533098A5 - - Google Patents

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Publication number
JP2021533098A5
JP2021533098A5 JP2021504376A JP2021504376A JP2021533098A5 JP 2021533098 A5 JP2021533098 A5 JP 2021533098A5 JP 2021504376 A JP2021504376 A JP 2021504376A JP 2021504376 A JP2021504376 A JP 2021504376A JP 2021533098 A5 JP2021533098 A5 JP 2021533098A5
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JP
Japan
Prior art keywords
composition according
aav vector
seq
aav
administered
Prior art date
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JP2021504376A
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English (en)
Japanese (ja)
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JP7684212B2 (ja
JP2021533098A (ja
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Priority claimed from PCT/US2019/044946 external-priority patent/WO2020028830A1/en
Publication of JP2021533098A publication Critical patent/JP2021533098A/ja
Publication of JP2021533098A5 publication Critical patent/JP2021533098A5/ja
Application granted granted Critical
Publication of JP7684212B2 publication Critical patent/JP7684212B2/ja
Active legal-status Critical Current
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JP2021504376A 2018-08-03 2019-08-02 第viii因子の発現による改善された臨床パラメーター Active JP7684212B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862714553P 2018-08-03 2018-08-03
US62/714,553 2018-08-03
US201962826887P 2019-03-29 2019-03-29
US62/826,887 2019-03-29
US201962869445P 2019-07-01 2019-07-01
US62/869,445 2019-07-01
PCT/US2019/044946 WO2020028830A1 (en) 2018-08-03 2019-08-02 Improved clinical parameters by expression of factor viii

Publications (3)

Publication Number Publication Date
JP2021533098A JP2021533098A (ja) 2021-12-02
JP2021533098A5 true JP2021533098A5 (enExample) 2022-08-09
JP7684212B2 JP7684212B2 (ja) 2025-05-27

Family

ID=69230742

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021504376A Active JP7684212B2 (ja) 2018-08-03 2019-08-02 第viii因子の発現による改善された臨床パラメーター

Country Status (10)

Country Link
US (1) US12497632B2 (enExample)
EP (1) EP3829650A4 (enExample)
JP (1) JP7684212B2 (enExample)
KR (1) KR20210039428A (enExample)
CN (1) CN112533645B (enExample)
BR (1) BR112021001315A2 (enExample)
CA (1) CA3105382A1 (enExample)
IL (1) IL280402B1 (enExample)
MX (1) MX2021001375A (enExample)
WO (1) WO2020028830A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240066146A1 (en) 2020-12-18 2024-02-29 Sangamo Therapeutics, Inc. Improved pharmaceutical compositions containing adeno-associated viral vector

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AU5588298A (en) 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US7238346B2 (en) 2002-07-08 2007-07-03 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw High capacity recombinant adenoviral vector for treatment of hemophilia A
PT1675956E (pt) 2003-10-21 2010-11-04 Merck Serono Sa Sequência mínima de adn que actua como um isolador de cromatina e sua utilização na expressão de proteína
JPWO2006043354A1 (ja) 2004-10-20 2008-05-22 独立行政法人放射線医学総合研究所 組込型の低線量放射線誘導性ベクター
JP2008543283A (ja) 2005-06-10 2008-12-04 ジェネンテック・インコーポレーテッド インスレーターエレメントを含むベクターを使用する抗体発現の改良
CN101965409A (zh) 2007-11-01 2011-02-02 罗切斯特大学 具有增加的稳定性的重组因子viii
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2009134681A2 (en) * 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA3018046A1 (en) * 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CN104271150A (zh) 2012-01-12 2015-01-07 比奥根艾迪克Ma公司 嵌合因子viii多肽及其用途
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
JP2015529466A (ja) 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
AU2014246731A1 (en) 2013-03-30 2015-11-19 Usha Biotech Limited Methods and constructs for expressing biologically active proteins in mammalian cells
EP3044231B1 (en) 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
CN105940013B (zh) 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
AU2015204646B2 (en) 2014-01-10 2020-08-27 Bioverativ Therapeutics Inc. Factor VIII chimeric proteins and uses thereof
MX388739B (es) 2015-02-06 2025-03-20 Univ North Carolina Chapel Hill Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
EP3294064A1 (en) 2015-05-11 2018-03-21 Bayer CropScience AG Herbicide combinations comprising l-glufosinate and indaziflam
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
TW202446416A (zh) 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
EA202190827A1 (ru) * 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
PE20200722A1 (es) * 2017-08-01 2020-07-21 Spark Therapeutics Inc Metodos de terapia genica del factor viii (fviii)

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