CA2888931C - 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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- CA2888931C CA2888931C CA2888931A CA2888931A CA2888931C CA 2888931 C CA2888931 C CA 2888931C CA 2888931 A CA2888931 A CA 2888931A CA 2888931 A CA2888931 A CA 2888931A CA 2888931 C CA2888931 C CA 2888931C
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| EP2013064054 | 2013-07-03 | ||
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| PCT/EP2013/072450 WO2014064277A1 (en) | 2012-10-26 | 2013-10-25 | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
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| US10398787B2 (en) | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
| 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 |
| EP3757116A1 (en) | 2013-12-09 | 2020-12-30 | Sangamo Therapeutics, Inc. | Methods and compositions for genome engineering |
| 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 |
| WO2016025764A2 (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 |
| KR102698387B1 (ko) | 2015-02-06 | 2024-08-22 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도 |
| JP6879486B2 (ja) * | 2015-03-17 | 2021-06-02 | フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ | Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系 |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| US10058624B2 (en) | 2015-04-16 | 2018-08-28 | 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 |
| 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 |
| DK3310909T3 (da) | 2015-06-17 | 2021-09-13 | Poseida Therapeutics Inc | Sammensætninger og fremgangsmåder til at føre proteiner til specifikke loci i genomet |
| MA42934A (fr) * | 2015-09-24 | 2018-08-01 | Biomarin Pharm Inc | Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci |
| WO2017066579A1 (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 |
| EA036944B1 (ru) | 2015-11-13 | 2021-01-19 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| CN116949052A (zh) | 2015-11-13 | 2023-10-27 | 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 |
| PL3411478T3 (pl) * | 2016-02-01 | 2022-10-03 | Bioverativ Therapeutics Inc. | Geny zoptymalizowanego czynnika VIII |
| AU2017248659B2 (en) * | 2016-04-15 | 2022-08-11 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia A |
| WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
| CA3024485A1 (en) | 2016-05-16 | 2017-11-23 | Baxalta Incorporated | Anti-factor ix padua antibodies |
| US10960085B2 (en) | 2016-09-07 | 2021-03-30 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| EP3592848A1 (en) | 2017-03-10 | 2020-01-15 | Genethon | Treatment of glycogen storage disease iii |
| 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 |
| 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 |
| 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 |
| 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 |
| EP4048800A1 (en) | 2019-10-23 | 2022-08-31 | Shire Human Genetic Therapies, Inc. | Adeno-associated virus vectors based gene therapy for hereditary angioedema |
| CN114981299A (zh) | 2019-12-12 | 2022-08-30 | 武田药品工业株式会社 | 使用编码具有增加的表达的重组fviii变体的病毒载体的a型血友病的基因疗法 |
| 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 |
| 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 |
| US20240269241A1 (en) | 2021-06-14 | 2024-08-15 | 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 |
| 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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| US6200560B1 (en) * | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| EP1224312A1 (en) * | 1999-10-12 | 2002-07-24 | The University of North Carolina at Chapel Hill | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| 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 |
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| JP5797551B2 (ja) | 2008-04-22 | 2015-10-21 | フエー・イー・ベー・フエー・ゼツト・ウエー | 肝特異的核酸調節要素ならびにその方法および用途 |
| ES2524974T3 (es) * | 2009-06-25 | 2014-12-16 | The University Of North Carolina At Chapel Hill | Moléculas del Factor VII quimérico |
| 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 |
| US10398787B2 (en) * | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
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