JP2018513678A5 - - Google Patents
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- Publication number
- JP2018513678A5 JP2018513678A5 JP2017549256A JP2017549256A JP2018513678A5 JP 2018513678 A5 JP2018513678 A5 JP 2018513678A5 JP 2017549256 A JP2017549256 A JP 2017549256A JP 2017549256 A JP2017549256 A JP 2017549256A JP 2018513678 A5 JP2018513678 A5 JP 2018513678A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- promoter
- expression cassette
- acid expression
- seq
- 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
Links
- 150000007523 nucleic acids Chemical group 0.000 claims 34
- 108020004707 nucleic acids Proteins 0.000 claims 28
- 102000039446 nucleic acids Human genes 0.000 claims 28
- 108010054218 Factor VIII Proteins 0.000 claims 13
- 102000001690 Factor VIII Human genes 0.000 claims 13
- 108700019146 Transgenes Proteins 0.000 claims 13
- 108010076282 Factor IX Proteins 0.000 claims 11
- 102100022641 Coagulation factor IX Human genes 0.000 claims 10
- 239000013598 vector Substances 0.000 claims 10
- 229960004222 factor ix Drugs 0.000 claims 9
- 229960000301 factor viii Drugs 0.000 claims 9
- 210000004185 liver Anatomy 0.000 claims 6
- 238000010361 transduction Methods 0.000 claims 6
- 230000026683 transduction Effects 0.000 claims 6
- 239000013603 viral vector Substances 0.000 claims 6
- 230000008488 polyadenylation Effects 0.000 claims 5
- 230000001105 regulatory effect Effects 0.000 claims 5
- 230000001225 therapeutic effect Effects 0.000 claims 5
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims 4
- 108010071690 Prealbumin Proteins 0.000 claims 4
- 102000009190 Transthyretin Human genes 0.000 claims 4
- 229940105778 coagulation factor viii Drugs 0.000 claims 3
- 108010088751 Albumins Proteins 0.000 claims 2
- 102000009027 Albumins Human genes 0.000 claims 2
- 102000005666 Apolipoprotein A-I Human genes 0.000 claims 2
- 108010059886 Apolipoprotein A-I Proteins 0.000 claims 2
- 108010056301 Apolipoprotein C-III Proteins 0.000 claims 2
- 102100030970 Apolipoprotein C-III Human genes 0.000 claims 2
- 101710201076 Carboxylesterase 1 Proteins 0.000 claims 2
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 102000003712 Complement factor B Human genes 0.000 claims 2
- 108090000056 Complement factor B Proteins 0.000 claims 2
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 2
- 102000013271 Hemopexin Human genes 0.000 claims 2
- 108010026027 Hemopexin Proteins 0.000 claims 2
- 208000009292 Hemophilia A Diseases 0.000 claims 2
- 102100036284 Hepcidin Human genes 0.000 claims 2
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 2
- 101001021253 Homo sapiens Hepcidin Proteins 0.000 claims 2
- 102100023418 Ketohexokinase Human genes 0.000 claims 2
- 108010062852 Ketohexokinase Proteins 0.000 claims 2
- 102100030817 Liver carboxylesterase 1 Human genes 0.000 claims 2
- 102100026046 Mannan-binding lectin serine protease 2 Human genes 0.000 claims 2
- 101710117460 Mannan-binding lectin serine protease 2 Proteins 0.000 claims 2
- 108010088865 Nicotinamide N-Methyltransferase Proteins 0.000 claims 2
- 102000009063 Nicotinamide N-methyltransferase Human genes 0.000 claims 2
- 101800004937 Protein C Proteins 0.000 claims 2
- 102000017975 Protein C Human genes 0.000 claims 2
- 101800001700 Saposin-D Proteins 0.000 claims 2
- 239000004480 active ingredient Substances 0.000 claims 2
- 108010006025 bovine growth hormone Proteins 0.000 claims 2
- 208000009429 hemophilia B Diseases 0.000 claims 2
- 230000037417 hyperactivation Effects 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 229960000856 protein c Drugs 0.000 claims 2
- 102000015081 Blood Coagulation Factors Human genes 0.000 claims 1
- 108010039209 Blood Coagulation Factors Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108091092195 Intron Proteins 0.000 claims 1
- 241000702321 Microvirus Species 0.000 claims 1
- 229940122344 Peptidase inhibitor Drugs 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 150000001413 amino acids Chemical group 0.000 claims 1
- 239000004019 antithrombin Substances 0.000 claims 1
- 239000003114 blood coagulation factor Substances 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 230000005030 transcription termination Effects 0.000 claims 1
- 238000001890 transfection Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP15159395.1 | 2015-03-17 | ||
| EP15159395 | 2015-03-17 | ||
| PCT/EP2016/055825 WO2016146757A1 (en) | 2015-03-17 | 2016-03-17 | Optimized liver-specific expression systems for fviii and fix |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018513678A JP2018513678A (ja) | 2018-05-31 |
| JP2018513678A5 true JP2018513678A5 (enExample) | 2019-04-25 |
| JP6879486B2 JP6879486B2 (ja) | 2021-06-02 |
Family
ID=52686196
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017549256A Active JP6879486B2 (ja) | 2015-03-17 | 2016-03-17 | Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US11007280B2 (enExample) |
| EP (1) | EP3270944B1 (enExample) |
| JP (1) | JP6879486B2 (enExample) |
| AU (1) | AU2016232146B2 (enExample) |
| CA (1) | CA2979495A1 (enExample) |
| DK (1) | DK3270944T3 (enExample) |
| ES (1) | ES2764453T3 (enExample) |
| WO (1) | WO2016146757A1 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2337849B1 (en) | 2008-09-15 | 2018-06-13 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
| ES2704677T3 (es) | 2013-04-18 | 2019-03-19 | Fond Telethon | Suministro eficaz de genes grandes por vectores de AAV duales |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| HUE068603T2 (hu) | 2015-06-23 | 2025-01-28 | Childrens Hospital Philadelphia | Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
| EP3374388A1 (en) | 2015-11-13 | 2018-09-19 | Baxalta Incorporated | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| SG11201804070XA (en) | 2015-11-13 | 2018-06-28 | Baxalta Inc | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| WO2018178067A1 (en) | 2017-03-27 | 2018-10-04 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
| US11633504B2 (en) | 2017-05-09 | 2023-04-25 | Emory University | Nucleic acids encoding clotting factor variants and their use |
| 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 |
| DK3652326T3 (da) * | 2017-07-10 | 2024-10-28 | Uniqure Ip Bv | Midler og fremgangsmåder til AAV-genterapi i mennesker |
| 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 |
| 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 |
| AU2019306194A1 (en) | 2018-07-16 | 2021-02-04 | Baxalta GmbH | Gene therapy of hemophilia A using viral vectors encoding recombinant FVIII variants with increased expression |
| WO2020020475A1 (en) * | 2018-07-27 | 2020-01-30 | Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh | Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| BR112021007229A2 (pt) | 2018-10-16 | 2021-08-10 | Blueallele, Llc | métodos para inserção dirigida de dna em genes |
| WO2020084162A1 (en) | 2018-10-26 | 2020-04-30 | Vrije Universiteit Brussel | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| WO2020084161A1 (en) | 2018-10-26 | 2020-04-30 | Vrije Universiteit Brussel | New tools for improving gene therapy and use thereof |
| WO2020102723A1 (en) * | 2018-11-16 | 2020-05-22 | 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 |
| CN115176022A (zh) | 2019-10-23 | 2022-10-11 | 夏尔人类遗传性治疗公司 | 用于遗传性血管性水肿的基于腺相关病毒载体的基因疗法 |
| US20220396610A1 (en) * | 2019-11-01 | 2022-12-15 | Freeline Therapeutics Limited | Factor viii polypeptide |
| 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 |
| KR20230038425A (ko) * | 2020-07-17 | 2023-03-20 | 프로바이오겐 아게 | 과활성 트랜스포손 및 트랜스포사제 |
| IL301196A (en) * | 2020-09-16 | 2023-05-01 | Generation Bio Co | Closed-end DNA vectors and their uses for phenylalanine hydroxylase (pah) expression |
| 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 |
| KR20240005950A (ko) * | 2021-05-12 | 2024-01-12 | 폰다지오네 텔레톤 이티에스 | 벡터 시스템 |
| AR126660A1 (es) | 2021-07-28 | 2023-11-01 | Univ Brussel Vrije | Mejorando la eficacia de la terapia génica dirigida a músculos |
| EP4402267A4 (en) * | 2021-09-16 | 2025-11-26 | Generation Bio Co | Liver-specific expression cassettes, vectors, and their uses 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 |
| WO2025239324A1 (ja) * | 2024-05-13 | 2025-11-20 | 中外製薬株式会社 | 遺伝子改変非ヒト動物 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US20040185555A1 (en) * | 2003-03-17 | 2004-09-23 | Emini Emilio A. | 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 |
| EP1986661B1 (en) * | 2006-02-08 | 2018-08-29 | Genzyme Corporation | Gene therapy for niemann-pick disease type a |
| WO2007120533A2 (en) * | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| EP2037892B1 (en) | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
| DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| 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 |
| ES2813698T3 (es) | 2013-09-12 | 2021-03-24 | Biomarin Pharm Inc | Vectores de AAV que comprenden un gen que codifica el factor VIII |
-
2016
- 2016-03-17 JP JP2017549256A patent/JP6879486B2/ja active Active
- 2016-03-17 CA CA2979495A patent/CA2979495A1/en active Pending
- 2016-03-17 WO PCT/EP2016/055825 patent/WO2016146757A1/en not_active Ceased
- 2016-03-17 AU AU2016232146A patent/AU2016232146B2/en not_active Ceased
- 2016-03-17 EP EP16713774.4A patent/EP3270944B1/en active Active
- 2016-03-17 US US15/558,725 patent/US11007280B2/en active Active
- 2016-03-17 DK DK16713774.4T patent/DK3270944T3/da active
- 2016-03-17 ES ES16713774T patent/ES2764453T3/es active Active
-
2021
- 2021-04-26 US US17/240,845 patent/US20210369868A1/en not_active Abandoned
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