JP2016500519A5 - - Google Patents

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JP2016500519A5
JP2016500519A5 JP2015538476A JP2015538476A JP2016500519A5 JP 2016500519 A5 JP2016500519 A5 JP 2016500519A5 JP 2015538476 A JP2015538476 A JP 2015538476A JP 2015538476 A JP2015538476 A JP 2015538476A JP 2016500519 A5 JP2016500519 A5 JP 2016500519A5
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Japan
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vector
subject
composition
plasma
composition according
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JP2015538476A
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JP2016500519A (ja
JP6454643B2 (ja
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Priority claimed from PCT/EP2012/071297 external-priority patent/WO2014063753A1/en
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Priority claimed from PCT/EP2013/072450 external-priority patent/WO2014064277A1/en
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JP2015538476A 2012-10-26 2013-10-25 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用 Active JP6454643B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
PCT/EP2012/071297 WO2014063753A1 (en) 2012-10-26 2012-10-26 Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
EPPCT/EP2012/071297 2012-10-26
EP2013064054 2013-07-03
EPPCT/EP2013/064054 2013-07-03
PCT/EP2013/072450 WO2014064277A1 (en) 2012-10-26 2013-10-25 Vector for liver-directed gene therapy of hemophilia and methods and use thereof

Publications (3)

Publication Number Publication Date
JP2016500519A JP2016500519A (ja) 2016-01-14
JP2016500519A5 true JP2016500519A5 (enExample) 2016-12-15
JP6454643B2 JP6454643B2 (ja) 2019-01-16

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JP2015538476A Active JP6454643B2 (ja) 2012-10-26 2013-10-25 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用

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US (2) US11419950B2 (enExample)
EP (1) EP2911687B1 (enExample)
JP (1) JP6454643B2 (enExample)
AU (1) AU2013336601B2 (enExample)
CA (1) CA2888931C (enExample)
DK (1) DK2911687T3 (enExample)
WO (1) WO2014064277A1 (enExample)

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CN105051204B (zh) 2012-11-16 2023-07-21 波赛伊达治疗学股份有限公司 位点特异性酶和使用方法
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US10960085B2 (en) 2016-09-07 2021-03-30 Sangamo Therapeutics, Inc. Modulation of liver genes
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US11920149B2 (en) 2017-03-27 2024-03-05 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
JP2020519272A (ja) * 2017-05-09 2020-07-02 エモリー ユニバーシティー 凝固因子変異体及びその使用
KR102386890B1 (ko) * 2017-05-22 2022-04-15 다케다 야쿠힌 고교 가부시키가이샤 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
JP7764129B2 (ja) * 2017-07-10 2025-11-05 ユニキュアー アイピー ビー．ブイ． ヒトにおけるａａｖ遺伝子療法のための手段及び方法
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
KR20210034013A (ko) 2018-07-16 2021-03-29 박스알타 인코퍼레이티드 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법
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EP3870237A1 (en) * 2018-10-26 2021-09-01 Vrije Universiteit Brussel Liver-specific nucleic acid regulatory elements and methods and use thereof
US20210379203A1 (en) * 2018-10-26 2021-12-09 Vrije Universiteit Brussel New Tools for Improving Gene Therapy and Use Thereof
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
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TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
US20230383311A1 (en) * 2020-09-16 2023-11-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
CN111926041B (zh) * 2020-10-16 2020-12-25 广州吉妮欧生物科技有限公司 一种SARS-CoV-2假病毒小鼠体内包装系统及其制备方法
EP4301863A1 (en) * 2021-03-04 2024-01-10 Poseida Therapeutics, Inc. Compositions and methods for the treatment of hemophilia
CA3213123A1 (en) * 2021-03-25 2022-09-29 Robert L. Kruse Hydrodynamic gene delivery
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WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
CN118715025A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于增强基因表达的构建体
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
WO2025226943A1 (en) 2024-04-25 2025-10-30 Masonic Medical Research Laboratory Polynucleotide for hepatocyte expression of vestigial like protein 4 and method of use thereof

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