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
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2021504376A
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English (en)
Japanese (ja)
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JP2021533098A (ja
JP7684212B2 (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
Anticipated expiration legal-status Critical

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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 (OSRAM) 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 (11)

Country Link
US (1) US12497632B2 (OSRAM)
EP (1) EP3829650A4 (OSRAM)
JP (1) JP7684212B2 (OSRAM)
KR (1) KR20210039428A (OSRAM)
CN (1) CN112533645B (OSRAM)
AU (1) AU2019314513B2 (OSRAM)
BR (1) BR112021001315A2 (OSRAM)
CA (1) CA3105382A1 (OSRAM)
IL (1) IL280402B2 (OSRAM)
MX (1) MX2021001375A (OSRAM)
WO (1) WO2020028830A1 (OSRAM)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2023006471A (es) * 2020-12-18 2023-08-11 Sangamo Therapeutics Inc Composiciones farmaceuticas mejoradas que contienen vector viral adenoasociado.

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WO1998024479A1 (en) 1996-12-02 1998-06-11 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
ATE484591T1 (de) 2003-10-21 2010-10-15 Merck Serono Sa Minimale dna sequenz, die als chromatin-isolator wirkt, und deren verwendung für die protein- expression
WO2006043354A1 (ja) 2004-10-20 2006-04-27 National Institute Of Radiological Sciences 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
JP2011502478A (ja) 2007-11-01 2011-01-27 ユニバーシティー オブ ロチェスター 安定性が増大した組換え型第viii因子
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
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
US9150847B2 (en) 2011-09-21 2015-10-06 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
JP2015501844A (ja) * 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
EP2804623B1 (en) 2012-01-12 2019-08-07 Bioverativ Therapeutics Inc. Chimeric factor viii polypeptides and uses thereof
CA2875618C (en) 2012-07-11 2021-04-27 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage 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
EP3770169A1 (en) 2013-09-12 2021-01-27 BioMarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
ES2813367T3 (es) 2013-12-09 2021-03-23 Sangamo Therapeutics Inc Métodos y composiciones para ingeniería genómica
SG11201605242YA (en) 2014-01-10 2016-07-28 Biogen Ma Inc Factor viii chimeric proteins and uses thereof
WO2016127057A1 (en) 2015-02-06 2016-08-11 The University Of North Carolina At Chapel Hill Optimized human clotting factor viii gene expression cassettes and their use
EP3283126B1 (en) * 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
AU2016259710B2 (en) 2015-05-11 2020-08-27 Basf Se Herbicide combinations comprising L-glufosinate and indaziflam
CN108024544B (zh) 2015-07-13 2022-04-29 桑格摩生物治疗股份有限公司 用于核酸酶介导的基因组工程化的递送方法及组合物
PE20181338A1 (es) * 2015-09-24 2018-08-21 Biomarin Pharm Inc Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
CN115141836B (zh) * 2015-10-28 2024-08-27 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法
CN116479001A (zh) * 2015-11-13 2023-07-25 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
KR20240160648A (ko) * 2017-08-01 2024-11-11 스파크 테라퓨틱스, 인코포레이티드 인자 viii (fviii) 유전자 요법 방법

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