JP2016500519A5 - - Google Patents

Download PDF

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
vector
subject
composition
plasma
composition according
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
JP2015538476A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016500519A (ja
JP6454643B2 (ja
Filing date
Publication date
Priority claimed from PCT/EP2012/071297 external-priority patent/WO2014063753A1/en
Application filed filed Critical
Priority claimed from PCT/EP2013/072450 external-priority patent/WO2014064277A1/en
Publication of JP2016500519A publication Critical patent/JP2016500519A/ja
Publication of JP2016500519A5 publication Critical patent/JP2016500519A5/ja
Application granted granted Critical
Publication of JP6454643B2 publication Critical patent/JP6454643B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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 (cg-RX-API-DMAC7.html) 2016-12-15
JP6454643B2 JP6454643B2 (ja) 2019-01-16

Family

ID=49554217

Family Applications (1)

Application Number Title Priority Date Filing Date
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

Country Status (7)

Country Link
US (2) US11419950B2 (cg-RX-API-DMAC7.html)
EP (1) EP2911687B1 (cg-RX-API-DMAC7.html)
JP (1) JP6454643B2 (cg-RX-API-DMAC7.html)
AU (1) AU2013336601B2 (cg-RX-API-DMAC7.html)
CA (1) CA2888931C (cg-RX-API-DMAC7.html)
DK (1) DK2911687T3 (cg-RX-API-DMAC7.html)
WO (1) WO2014064277A1 (cg-RX-API-DMAC7.html)

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3513802B1 (en) 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
EP2920319B1 (en) 2012-11-16 2020-02-19 Poseida Therapeutics, Inc. Site-specific enzymes and methods of use
HUE051628T2 (hu) 2013-12-09 2021-03-01 Sangamo Therapeutics Inc Eljárások és kompozíciók genom-manipulálására
AU2015277180A1 (en) * 2014-06-17 2017-01-12 Poseida Therapeutics, Inc. A method for directing proteins to specific loci in the genome and uses thereof
CN107427557B (zh) 2014-08-13 2022-01-04 费城儿童医院 用于包装和表达变体因子viii以治疗血友病的改良表达组件
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CA2975734C (en) 2015-02-06 2025-02-18 The University Of North Carolina At Chapel Hill OPTIMIZED GENE EXPRESSION CASSETTES FOR HUMAN COAGULATION FACTOR VIII AND THEIR USE
AU2016232146B2 (en) 2015-03-17 2021-11-04 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CN115074366A (zh) * 2015-04-16 2022-09-20 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
AU2016260401B2 (en) 2015-05-14 2022-05-19 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
ES2886599T3 (es) 2015-06-17 2021-12-20 Poseida Therapeutics Inc Composiciones y métodos para dirigir proteínas a loci específicos en el genoma
IL257939B2 (en) * 2015-09-24 2023-03-01 Biomarin Pharm Inc Adeno-associated virus factor 8 vectors, associated virus particles, and medicinal formulations comprising the same
EP3361869B1 (en) 2015-10-14 2025-12-03 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
KR102766526B1 (ko) 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
CN108884145B (zh) 2015-11-13 2023-08-22 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
US10189889B2 (en) 2015-11-13 2019-01-29 Baxalta Incorporated Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
PT3411478T (pt) * 2016-02-01 2022-09-13 Bioverativ Therapeutics Inc Genes do fator viii otimizados
MX2018012537A (es) * 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
WO2017180861A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsulvania Gene therapy for treating hemophilia b
KR102481704B1 (ko) * 2016-05-16 2022-12-27 다케다 파머수티컬 컴패니 리미티드 항-인자 ix 파두아 항체
CA3035534A1 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
CN110914419A (zh) 2017-03-10 2020-03-24 吉尼松公司 糖原贮积病iii的治疗
EP3600445A1 (en) 2017-03-27 2020-02-05 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
US11633504B2 (en) 2017-05-09 2023-04-25 Emory University Nucleic acids encoding clotting factor variants and their use
TWI753168B (zh) * 2017-05-22 2022-01-21 日商武田藥品工業股份有限公司 用於血友病ｂ基因療法之編碼具有增強表現的重組ｆｉｘ變異體之病毒載體
US11452749B2 (en) * 2017-07-10 2022-09-27 Uniqure Ip B.V. Means and methods for AAV gene therapy in humans
PE20212076A1 (es) 2018-02-01 2021-10-26 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas
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
BR112021000695A2 (pt) 2018-07-16 2021-04-20 Baxalta Incorporated métodos para tratar hemofilia a e para monitorar a eficácia da terapia gênica de fator viii de hemofilia a.
CA3105382A1 (en) 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
JP2022505876A (ja) * 2018-10-26 2022-01-14 フリーイェ・ユニヴェルシテイト・ブリュッセル 遺伝子治療を向上させる新しいツール及びその使用
WO2020084162A1 (en) 2018-10-26 2020-04-30 Vrije Universiteit Brussel Liver-specific nucleic acid regulatory elements and methods 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
WO2020160303A1 (en) 2019-02-01 2020-08-06 Baxalta Incorporated Gene therapy of hemophilia b using viral vectors encoding recombinant fix variants with increased expression
JP7659550B2 (ja) 2019-10-23 2025-04-09 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 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
JP2023506171A (ja) 2019-12-12 2023-02-15 武田薬品工業株式会社 発現が上昇した組換えｆｖｉｉｉバリアントをコードするウイルスベクターを使用する、血友病ａの遺伝子療法
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
AU2021342503A1 (en) * 2020-09-16 2023-04-06 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
WO2022187671A1 (en) * 2021-03-04 2022-09-09 Poseida Therapeutics, Inc. Compositions and methods for the treatment of hemophilia
US20250152748A1 (en) * 2021-03-25 2025-05-15 Johns Hopkins University Hydrodynamic gene delivery
CN117858895A (zh) 2021-06-14 2024-04-09 武田药品工业株式会社 使用表达增强的编码重组fviii变体的病毒载体的血友病a基因疗法
AR126660A1 (es) 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
EP4463475A2 (en) 2022-01-14 2024-11-20 Anjarium Biosciences AG Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
EP4598590A1 (en) * 2022-10-08 2025-08-13 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
JP2003511082A (ja) * 1999-10-12 2003-03-25 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 第ｖｉｉｉ因子をコードするアデノ随伴ウィルスベクターとその使用法
CN104087591A (zh) * 2006-06-19 2014-10-08 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子viii和因子ix基因和载体
WO2009071679A1 (en) * 2007-12-05 2009-06-11 Vib Vzw Novel aav vector and uses thereof
US9617548B2 (en) * 2008-04-22 2017-04-11 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
AU2010266065B2 (en) * 2009-06-25 2016-01-14 The University Of North Carolina At Chapel Hill Chimeric Factor VII molecules
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
TWI557135B (zh) 2010-11-03 2016-11-11 介控生化科技公司 經修飾之第九因子多胜肽及其用途
EP3513802B1 (en) * 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof

Similar Documents

Publication Publication Date Title
JP2016500519A5 (cg-RX-API-DMAC7.html)
JP6831779B2 (ja) 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
JP6373763B2 (ja) Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法
JP6454643B2 (ja) 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
KR102380265B1 (ko) 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
US11007280B2 (en) Optimized liver-specific expression systems for FVIII and FIX
US20200390866A1 (en) Factor viii sequences
Murphy et al. Gene therapy for haemophilia
RU2018136611A (ru) Генная терапия для лечения гемофилии a
JP2018513678A5 (cg-RX-API-DMAC7.html)
US20150283267A1 (en) Vectors for Liver-Directed Gene Therapy of Hemophilia and Methods and Use Thereof
Spencer et al. State of the art: gene therapy of haemophilia
TW201629225A (zh) 第九因子基因療法
RU2018143411A (ru) Лечение комплемент-опосредуемых расстройств
Vance et al. AAV Biology, Infectivity and Therapeutic Use from
CA3169529A1 (en) Compositions and methods for the treatment of metabolic liver disorders
WO2014063753A1 (en) Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
Hollinger et al. Viral vector-mediated gene therapies
BR112021013874A2 (pt) Composição de ácido nucleico, vetor de terapia de gene de mamífero, partícula de vírus adeno-associado, métodos para tratar hemofilia a e para produzir uma partícula de vírus adeno-associado, e, uso de uma composição de ácido nucleico e de um vetor de terapia de gene de mamífero
WO2014016580A1 (en) Transgene expression
WO2021042944A1 (zh) 肌肉靶向的微环dna基因治疗
Chai et al. Dexamethasone transiently enhances transgene expression in the liver when administered at late-phase post long-term adeno-associated virus transduction
Khan et al. Hemophilia healing with AAV: navigating the frontier of gene therapy
ES2907741T3 (es) Polipéptidos de elabela (ELA) para uso en el tratamiento del cáncer de riñón
BR112021000695A2 (pt) métodos para tratar hemofilia a e para monitorar a eficácia da terapia gênica de fator viii de hemofilia a.