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
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- 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 (https=) | 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 (https=) |
| EP (1) | EP3270944B1 (https=) |
| JP (1) | JP6879486B2 (https=) |
| AU (1) | AU2016232146B2 (https=) |
| CA (1) | CA2979495A1 (https=) |
| DK (1) | DK3270944T3 (https=) |
| ES (1) | ES2764453T3 (https=) |
| WO (1) | WO2016146757A1 (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TR201813067T4 (tr) | 2008-09-15 | 2018-09-21 | Uniqure Biopharma B V | Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem. |
| AU2014255665B2 (en) | 2013-04-18 | 2018-08-02 | 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 |
| MX390848B (es) | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| 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 |
| 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 |
| 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 |
| 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 |
| SG11202103917VA (en) | 2018-10-16 | 2021-05-28 | Blueallele Llc | Methods for targeted insertion of dna in genes |
| 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 |
| 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 |
| EP3880809A4 (en) * | 2018-11-16 | 2023-01-11 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF 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 |
| 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 |
| GB201907493D0 (en) | 2019-05-28 | 2019-07-10 | Ospedale San Raffaele | Agents and methods for treating viral infections |
| CN114207135A (zh) * | 2019-06-11 | 2022-03-18 | 夏尔人类遗传性治疗公司 | 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送 |
| WO2021081280A1 (en) | 2019-10-23 | 2021-04-29 | Shire Human Genetic Therapies, Inc. | Adeno-associated virus vectors based gene therapy for hereditary angioedema |
| CN114929735A (zh) * | 2019-11-01 | 2022-08-19 | 自由行疗法有限公司 | 因子viii构建体 |
| 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 |
| CA3173696A1 (en) * | 2020-07-17 | 2022-01-20 | Volker Sandig | Hyperactive transposons and transposases |
| EP4214309A4 (en) * | 2020-09-16 | 2025-07-16 | Generation Bio Co | CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) |
| 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 |
| IL308356A (en) * | 2021-05-12 | 2024-01-01 | Fond Telethon | Vector system |
| AR126660A1 (es) | 2021-07-28 | 2023-11-01 | Univ Brussel Vrije | Mejorando la eficacia de la terapia génica dirigida a músculos |
| MX2024003186A (es) * | 2021-09-16 | 2024-04-23 | Generation Bio Co | Casetes de expresión específicos del hígado, vectores y usos de estos para expresar proteínas terapéuticas. |
| 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 |
| AR059371A1 (es) * | 2006-02-08 | 2008-03-26 | Genzyme Corp | Terapia genica para la enfermedad de niemann-pick tipo a |
| WO2007120533A2 (en) * | 2006-03-30 | 2007-10-25 | 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 |
| CA2722238C (en) * | 2008-04-22 | 2017-11-28 | Life Sciences Research Partners Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| AU2013336601B2 (en) * | 2012-10-26 | 2018-01-25 | 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 |
-
2016
- 2016-03-17 US US15/558,725 patent/US11007280B2/en active Active
- 2016-03-17 WO PCT/EP2016/055825 patent/WO2016146757A1/en not_active Ceased
- 2016-03-17 DK DK16713774.4T patent/DK3270944T3/da active
- 2016-03-17 AU AU2016232146A patent/AU2016232146B2/en not_active Ceased
- 2016-03-17 JP JP2017549256A patent/JP6879486B2/ja active Active
- 2016-03-17 CA CA2979495A patent/CA2979495A1/en active Pending
- 2016-03-17 ES ES16713774T patent/ES2764453T3/es active Active
- 2016-03-17 EP EP16713774.4A patent/EP3270944B1/en active Active
-
2021
- 2021-04-26 US US17/240,845 patent/US20210369868A1/en not_active Abandoned
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