AU2013336601B2 - Vector for liver-directed gene therapy of hemophilia and methods and use thereof - Google Patents
Vector for liver-directed gene therapy of hemophilia and methods and use thereof Download PDFInfo
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- AU2013336601B2 AU2013336601B2 AU2013336601A AU2013336601A AU2013336601B2 AU 2013336601 B2 AU2013336601 B2 AU 2013336601B2 AU 2013336601 A AU2013336601 A AU 2013336601A AU 2013336601 A AU2013336601 A AU 2013336601A AU 2013336601 B2 AU2013336601 B2 AU 2013336601B2
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- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14311—Parvovirus, e.g. minute virus of mice
- C12N2750/14341—Use of virus, viral particle or viral elements as a vector
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- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
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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 |
| AUPCT/EP2012/071297 | 2012-10-26 | ||
| 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 (2)
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| AU2013336601A1 AU2013336601A1 (en) | 2015-05-28 |
| AU2013336601B2 true AU2013336601B2 (en) | 2018-01-25 |
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| AU2013336601A Ceased AU2013336601B2 (en) | 2012-10-26 | 2013-10-25 | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
Country Status (7)
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| 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)
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| 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 |
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| WO2009071679A1 (en) * | 2007-12-05 | 2009-06-11 | Vib Vzw | Novel aav vector and uses thereof |
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| 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 |
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Also Published As
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| WO2014064277A1 (en) | 2014-05-01 |
| US20190240350A1 (en) | 2019-08-08 |
| JP2016500519A (ja) | 2016-01-14 |
| EP2911687B1 (en) | 2019-02-13 |
| US11419950B2 (en) | 2022-08-23 |
| CA2888931A1 (en) | 2014-05-01 |
| AU2013336601A1 (en) | 2015-05-28 |
| CA2888931C (en) | 2023-09-05 |
| JP6454643B2 (ja) | 2019-01-16 |
| US20230022390A1 (en) | 2023-01-26 |
| DK2911687T3 (da) | 2019-05-13 |
| EP2911687A1 (en) | 2015-09-02 |
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