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
JP6454643B2 (ja
JP2016500519A (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 (enExample) 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 (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)

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10398787B2 (en) 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
CA2891510C (en) 2012-11-16 2022-10-18 Transposagen Biopharmaceuticals, Inc. Site-specific enzymes and methods of use
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
US20170107541A1 (en) * 2014-06-17 2017-04-20 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
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.
US11007280B2 (en) 2015-03-17 2021-05-18 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
EP3283126B1 (en) * 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3294309A4 (en) * 2015-05-14 2019-01-16 St. Jude Children's Research Hospital, Inc. NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF
US11473082B2 (en) 2015-06-17 2022-10-18 Poseida Therapeutics, Inc. Compositions and methods for directing proteins to specific loci in the genome
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
CA3001594A1 (en) 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
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
CA3005565A1 (en) 2015-11-13 2017-05-18 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
CA3012695A1 (en) * 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
MX2018012537A (es) * 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
KR102625416B1 (ko) * 2016-05-16 2024-01-15 다케다 파머수티컬 컴패니 리미티드 항-인자 ix 파두아 항체
CA3035534A1 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
WO2018178067A1 (en) 2017-03-27 2018-10-04 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
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
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
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
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
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
US12497632B2 (en) 2018-08-03 2025-12-16 Sangamo Therapeutics, Inc. 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
JP7529286B2 (ja) * 2018-10-26 2024-08-06 フリーイェ・ユニヴェルシテイト・ブリュッセル 肝特異的核酸調節エレメント並びにその方法及び使用
CN113164560A (zh) 2018-10-26 2021-07-23 Vrije布鲁塞尔大学 改善基因疗法的新工具及其用途
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
WO2021081280A1 (en) 2019-10-23 2021-04-29 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
WO2021119357A2 (en) 2019-12-12 2021-06-17 Baxalta Incorporated 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
CA3191743A1 (en) * 2020-09-16 2022-03-24 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
EP4313169A4 (en) * 2021-03-25 2025-01-08 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
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
ES2535877T3 (es) * 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
WO2009071679A1 (en) * 2007-12-05 2009-06-11 Vib Vzw Novel aav vector and uses thereof
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
JP5909180B2 (ja) * 2009-06-25 2016-04-26 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 キメラ第vii因子分子
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
US10398787B2 (en) * 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof

Similar Documents

Publication Publication Date Title
JP2016500519A5 (enExample)
US20230022390A1 (en) Vectors for Liver-Directed Gene Therapy of Hemophilia and Methods and Use Thereof
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 (enExample)
Spencer et al. State of the art: gene therapy of haemophilia
RU2018143411A (ru) Лечение комплемент-опосредуемых расстройств
TW201629225A (zh) 第九因子基因療法
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
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
JP2022550954A5 (enExample)
WO2023150702A2 (en) Compositions and methods for treatment of connective tissue disorders
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
TW202503063A (zh) 用於表現il-1ra和il-18bp的組合物和方法