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 (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 |
|---|---|---|---|---|
| EP4219547A3 (en) | 2008-09-15 | 2023-10-18 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
| CA2909733C (en) | 2013-04-18 | 2023-10-17 | 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 |
| WO2016210170A1 (en) | 2015-06-23 | 2016-12-29 | The Children's Hospital Of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| JP7460643B2 (ja) | 2018-10-16 | 2024-04-02 | ブルーアレル， エルエルシー | 遺伝子へのｄｎａの標的化挿入のための方法 |
| US20210403948A1 (en) | 2018-10-26 | 2021-12-30 | 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 |
| TW202039854A (zh) * | 2018-11-16 | 2020-11-01 | 美商編碼製藥公司 | 治療威爾遜氏病的組合物和方法 |
| 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 |
| US20230038502A1 (en) * | 2019-06-11 | 2023-02-09 | Shire Human Genetic Therapies, Inc. | Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein |
| EP4048800A1 (en) | 2019-10-23 | 2022-08-31 | Shire Human Genetic Therapies, Inc. | Adeno-associated virus vectors based gene therapy for hereditary angioedema |
| EP4051704A2 (en) * | 2019-11-01 | 2022-09-07 | 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 |
| CA3173696A1 (en) * | 2020-07-17 | 2022-01-20 | Volker Sandig | Hyperactive transposons and transposases |
| US20240026374A1 (en) * | 2020-09-16 | 2024-01-25 | Generation Bio Co. | Closed-ended dna vectors and uses thereof 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 |
| MX2023013393A (es) * | 2021-05-12 | 2023-12-08 | Fond Telethon | Sistema de vectores. |
| AR126660A1 (es) | 2021-07-28 | 2023-11-01 | Univ Brussel Vrije | Mejorando la eficacia de la terapia génica dirigida a músculos |
| IL311240A (en) * | 2021-09-16 | 2024-05-01 | Generation Bio Co | Liver-specific expression cassettes, vectors and their uses for the expression of therapeutic proteins |
| KR20230166039A (ko) | 2022-05-27 | 2023-12-06 | 주식회사 엔세이지 | 항응고인자 9 분비 유도만능줄기세포를 이용한 b형 혈우병 치료제 개발 |
| 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 |
| 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 |
| 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 |
| JP5706602B2 (ja) * | 2006-02-08 | 2015-04-22 | ジェンザイム・コーポレーション | ニーマンピック病ａ型に対する遺伝子治療 |
| WO2007120533A2 (en) * | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| EP2848253A1 (en) | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor VIII and factor IX genes and vectors for gene therapy |
| EP4154903A1 (en) | 2008-04-22 | 2023-03-29 | Vib 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 |
| 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 |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| RS61039B1 (sr) * | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
-
2016
- 2016-03-17 EP EP16713774.4A patent/EP3270944B1/en active 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 DK DK16713774.4T patent/DK3270944T3/da active
- 2016-03-17 WO PCT/EP2016/055825 patent/WO2016146757A1/en not_active Ceased
- 2016-03-17 US US15/558,725 patent/US11007280B2/en active Active
-
2021
- 2021-04-26 US US17/240,845 patent/US20210369868A1/en not_active Abandoned
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