ES2764453T3 - Sistemas de expresión específica de hígado optimizados para FVIII y FIX - Google Patents
Sistemas de expresión específica de hígado optimizados para FVIII y FIX Download PDFInfo
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| HUE067487T2 (hu) | 2008-09-15 | 2024-10-28 | Uniqure Biopharma B V | IX-es faktor polipeptid mutánsa, alkalmazásai és eljárás az elõállítására |
| US10494645B2 (en) | 2013-04-18 | 2019-12-03 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| PT3313991T (pt) | 2015-06-23 | 2024-09-27 | Childrens Hospital Philadelphia | Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos |
| 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 |
| EA036944B1 (ru) | 2015-11-13 | 2021-01-19 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| 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 |
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| 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 |
| EP3799569A1 (en) * | 2018-07-27 | 2021-04-07 | Zentrum für Forschungsförderung in der Pädiatrie GmbH | Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles |
| 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 |
| GB2611929B (en) | 2018-10-16 | 2023-11-22 | Blueallele Corp | Methods for targeted insertion of DNA in genes |
| 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 |
| US20210369870A1 (en) * | 2018-11-16 | 2021-12-02 | Encoded Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| GB201818816D0 (en) * | 2018-11-19 | 2019-01-02 | Synpromics Ltd | Regulatory nucleic acid sequences |
| 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 |
| GB201907493D0 (en) | 2019-05-28 | 2019-07-10 | Ospedale San Raffaele | Agents and methods for treating viral infections |
| JP2022536692A (ja) * | 2019-06-11 | 2022-08-18 | 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 | 調節不全の血漿カリクレインによって媒介される疾患の治療のための抗体のアデノ随伴ウイルスベクター送達 |
| EP4048800A1 (en) | 2019-10-23 | 2022-08-31 | Shire Human Genetic Therapies, Inc. | Adeno-associated virus vectors based gene therapy for hereditary angioedema |
| WO2021084276A2 (en) * | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Factor viii construct |
| CN111218446B (zh) * | 2019-12-25 | 2023-03-28 | 劲帆生物医药科技（武汉）有限公司 | 一种肝脏特异性启动子及其应用 |
| 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 |
| WO2022012758A1 (en) * | 2020-07-17 | 2022-01-20 | Probiogen Ag | Hyperactive transposons and transposases |
| KR20230066615A (ko) * | 2020-09-16 | 2023-05-16 | 제너레이션 바이오 컴퍼니 | 폐쇄형 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 발현을 위한 이의 용도 |
| CA3094859A1 (en) * | 2020-10-01 | 2022-04-01 | Entos Pharmaceuticals Inc. | Proteolipid vesicles formulated with fusion associated small transmembrane proteins |
| GB202018657D0 (en) | 2020-11-26 | 2021-01-13 | Ospedale San Raffaele Srl | Agents and methods for increasing liver immune response |
| CN112608921B (zh) * | 2020-12-23 | 2022-04-08 | 武汉枢密脑科学技术有限公司 | 用于肝脏中蛋白质表达的重组启动子、载体及其应用 |
| TW202246505A (zh) * | 2021-03-05 | 2022-12-01 | 俄羅斯聯邦商亞那拜恩有限公司 | 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途 |
| AU2022274162A1 (en) * | 2021-05-12 | 2023-11-30 | Fondazione Telethon | Vector system |
| AR126660A1 (es) | 2021-07-28 | 2023-11-01 | Univ Brussel Vrije | Mejorando la eficacia de la terapia génica dirigida a músculos |
| CA3232641A1 (en) * | 2021-09-16 | 2023-03-23 | Generation Bio Co. | Liver-specific expression cassettes, vectors and uses thereof for expressing therapeutic proteins |
| KR20230166039A (ko) | 2022-05-27 | 2023-12-06 | 주식회사 엔세이지 | 항응고인자 9 분비 유도만능줄기세포를 이용한 b형 혈우병 치료제 개발 |
| 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 |
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| US7351813B2 (en) * | 2000-06-20 | 2008-04-01 | The Board Of Trustees Of The Leland Stanford Junior University | Liver-specific gene expression cassettes, and methods of use |
| US8071740B2 (en) * | 2000-11-17 | 2011-12-06 | Vascular Biogenics Ltd. | Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis |
| WO2004083418A1 (en) * | 2003-03-17 | 2004-09-30 | Merck & Co., Inc. | Adenovirus serotype 24 vectors, nucleic acids and virus produced thereby |
| US8008468B2 (en) * | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
| AR059371A1 (es) * | 2006-02-08 | 2008-03-26 | Genzyme Corp | Terapia genica para la enfermedad de niemann-pick tipo a |
| US8129510B2 (en) * | 2006-03-30 | 2012-03-06 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| 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 |
| 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 |
| GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| CA2888931C (en) | 2012-10-26 | 2023-09-05 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| WO2014063753A1 (en) | 2012-10-26 | 2014-05-01 | Vrije Universiteit Brussel | Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof |
| EP3044231B1 (en) * | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
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