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
EPPCT/EP2012/071297 2012-10-26
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/EP2013/064054 2013-07-03
EP2013064054 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 (https=) 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 (https=)
EP (1) EP2911687B1 (https=)
JP (1) JP6454643B2 (https=)
AU (1) AU2013336601B2 (https=)
CA (1) CA2888931C (https=)
DK (1) DK2911687T3 (https=)
WO (1) WO2014064277A1 (https=)

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
ES2778033T3 (es) 2012-11-16 2020-08-07 Poseida Therapeutics Inc Enzimas específicas de sitio y métodos de uso
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
EP3157328B1 (en) * 2014-06-17 2021-08-04 Poseida Therapeutics, Inc. A method for directing proteins to specific loci in the genome and uses thereof
CA3237630A1 (en) 2014-08-13 2016-02-18 The Children's Hospital Of Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
KR102554850B1 (ko) 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
EP3270944B1 (en) * 2015-03-17 2019-10-23 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
WO2016183422A1 (en) 2015-05-14 2016-11-17 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
EP3929286A1 (en) 2015-06-17 2021-12-29 Poseida Therapeutics, Inc. Compositions and methods for directing proteins to specific loci in the genome
RU2742352C2 (ru) * 2015-09-24 2021-02-05 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
US11230720B2 (en) 2015-10-14 2022-01-25 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
CA3002524A1 (en) * 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
EP3374387A1 (en) 2015-11-13 2018-09-19 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EA202092049A1 (ru) 2015-11-13 2020-11-10 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
WO2017136358A1 (en) * 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
KR102450833B1 (ko) * 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
EP3442597A1 (en) 2016-04-15 2019-02-20 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia b
CN109563154B (zh) 2016-05-16 2024-12-13 武田药品工业株式会社 抗帕多瓦因子ix抗体
WO2018049009A2 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
US11541131B2 (en) 2017-03-10 2023-01-03 Genethon Treatment of glycogen storage disease III
CA3057899A1 (en) 2017-03-27 2018-10-04 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
EP3622065A1 (en) * 2017-05-09 2020-03-18 Emory University Clotting factor variants and their use
CN110945127B (zh) * 2017-05-22 2024-07-12 武田药品工业株式会社 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体
PT3652326T (pt) * 2017-07-10 2024-12-26 Uniqure Ip Bv Meios e métodos para terapia génica de aav em humanos
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN111902164A (zh) 2018-02-01 2020-11-06 同源药物公司 用于恢复pah基因功能的腺相关病毒组合物及其使用方法
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
EP3823985A1 (en) 2018-07-16 2021-05-26 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
WO2020028830A1 (en) 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US20210379203A1 (en) 2018-10-26 2021-12-09 Vrije Universiteit Brussel New Tools for Improving Gene Therapy and Use Thereof
US20210403948A1 (en) 2018-10-26 2021-12-30 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
US11596671B2 (en) 2019-02-01 2023-03-07 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression
EP4048800A1 (en) 2019-10-23 2022-08-31 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
EP4073106A2 (en) 2019-12-12 2022-10-19 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
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
AU2022242921A1 (en) * 2021-03-25 2023-10-19 The Johns Hopkins University Hydrodynamic gene delivery
EP4355768A1 (en) 2021-06-14 2024-04-24 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
AR126660A1 (es) 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法
JP2025534900A (ja) * 2022-10-08 2025-10-21 リンイ バイオテック カンパニー リミテッド 遺伝子発現を増強するためのコンストラクト
TW202517669A (zh) 2023-07-05 2025-05-01 日商武田藥品工業股份有限公司 用於Ａ型血友病之基因療法的編碼表現增加之重組ＦＶｌｌｌ變異體的病毒載體
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
AU7879000A (en) * 1999-10-12 2001-04-23 University Of North Carolina At Chapel Hill, The Adeno-associated virus vectors encoding factor viii and methods of using the same
EP2848253A1 (en) * 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor VIII and factor IX genes and vectors for gene therapy
WO2009071679A1 (en) * 2007-12-05 2009-06-11 Vib Vzw Novel aav vector and uses thereof
EP4154903A1 (en) * 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
CN102596231B (zh) * 2009-06-25 2016-01-20 北卡罗来纳-查佩尔山大学 嵌合因子ⅶ分子
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
TWI595004B (zh) 2010-11-03 2017-08-11 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 (https=)
US20230022390A1 (en) Vectors for Liver-Directed Gene Therapy of Hemophilia and Methods and Use Thereof
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
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
US10398787B2 (en) Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
JP2018513678A5 (https=)
Spencer et al. State of the art: gene therapy of haemophilia
RU2018143411A (ru) Лечение комплемент-опосредуемых расстройств
Vance et al. AAV Biology, Infectivity and Therapeutic Use from
WO2014063753A1 (en) Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
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
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.
Zhou et al. Gene therapy for hemophilia A: where we stand
RU2018120484A (ru) Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии
Akula et al. Balancing Promise and Peril: Hemophilia Gene Therapy Insights
Pignani et al. CRISPR activation on coagulation F7 or F8 promoters potentiate trascriptional activity in the normal and mutated gene context
Brown et al. 682. Applied In Silico Engineering of Factor VIII-AAV Transgene Cassettes
Hanley The use of matrix attachment regions to enhance the in-vivo potency of rAAV vectors