JP2020520662A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020520662A5 JP2020520662A5 JP2019564430A JP2019564430A JP2020520662A5 JP 2020520662 A5 JP2020520662 A5 JP 2020520662A5 JP 2019564430 A JP2019564430 A JP 2019564430A JP 2019564430 A JP2019564430 A JP 2019564430A JP 2020520662 A5 JP2020520662 A5 JP 2020520662A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- polynucleotide
- factor
- acid composition
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762509616P | 2017-05-22 | 2017-05-22 | |
| US62/509,616 | 2017-05-22 | ||
| PCT/US2018/033866 WO2018217731A1 (en) | 2017-05-22 | 2018-05-22 | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020520662A JP2020520662A (ja) | 2020-07-16 |
| JP2020520662A5 true JP2020520662A5 (enExample) | 2021-05-20 |
| JP6961171B2 JP6961171B2 (ja) | 2021-11-05 |
Family
ID=62621009
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019564430A Active JP6961171B2 (ja) | 2017-05-22 | 2018-05-22 | 血友病ｂの遺伝子治療のための、発現の増加を伴う組換えｆｉｘをコードするウイルスベクター |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US10842853B2 (enExample) |
| EP (1) | EP3630974A1 (enExample) |
| JP (1) | JP6961171B2 (enExample) |
| KR (2) | KR102386890B1 (enExample) |
| CN (2) | CN110945127B (enExample) |
| AR (1) | AR112057A1 (enExample) |
| AU (1) | AU2018272831B2 (enExample) |
| BR (1) | BR112019024256A2 (enExample) |
| CA (1) | CA3064730A1 (enExample) |
| CL (2) | CL2019003395A1 (enExample) |
| CO (1) | CO2019012885A2 (enExample) |
| IL (2) | IL280637B2 (enExample) |
| MX (2) | MX2019013836A (enExample) |
| NZ (1) | NZ759034A (enExample) |
| TW (1) | TWI753168B (enExample) |
| WO (1) | WO2018217731A1 (enExample) |
| ZA (1) | ZA201907716B (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| 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 |
| EP3867380A2 (en) | 2018-10-18 | 2021-08-25 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| 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 |
| EP3941509B1 (en) | 2019-03-19 | 2025-12-03 | CSL Innovation Pty Ltd | Factor ix variants and uses thereof in therapy |
| 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 |
| US20200405883A1 (en) | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
| CN110684798A (zh) * | 2019-09-03 | 2020-01-14 | 深圳新诺微环生物科技有限公司 | 肌肉靶向的微环dna基因治疗 |
| EP4087489B1 (en) * | 2020-01-08 | 2025-05-14 | Vitruvia Holdings Inc. | Methods and computing system for processing ultrasound image to determine health of subdermal tissue |
| KR20220130174A (ko) * | 2020-01-16 | 2022-09-26 | 다케다 야쿠힌 고교 가부시키가이샤 | 페닐케톤뇨증에 대한 아데노 연관 바이러스 기반 유전자 치료법 |
| 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 |
| AU2022298827A1 (en) * | 2021-06-23 | 2024-02-08 | Frontera Therapeutics, Inc. | Composition and method for treating hemophilia b |
| CN114277057B (zh) * | 2021-07-09 | 2023-10-13 | 上海天泽云泰生物医药有限公司 | 用于治疗或预防b型血友病的重组腺相关病毒载体和方法 |
| WO2023077012A1 (en) * | 2021-10-27 | 2023-05-04 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
| EP4509606A4 (en) * | 2022-04-19 | 2025-12-10 | Kanglin Biotech Hangzhou Co Ltd | CONSTRUCTION OF NUCLEIC ACID FOR THE TREATMENT OF AN INHERITED COAGULATION FACTOR DEFICIENCY AND ITS USE |
| WO2025143742A1 (ko) * | 2023-12-26 | 2025-07-03 | 삼성바이오에피스 주식회사 | 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드 |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| ATE322547T1 (de) | 1993-06-10 | 2006-04-15 | Genetic Therapy Inc | Adenovirale vektoren für die behandlung der hämophilie |
| US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
| EP0785803A4 (en) | 1994-09-23 | 2000-08-02 | Gen Hospital Corp | Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| US5858351A (en) | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
| US5866552A (en) | 1996-09-06 | 1999-02-02 | The Trustees Of The University Of Pennsylvania | Method for expressing a gene in the absence of an immune response |
| US6114148C1 (en) | 1996-09-20 | 2012-05-01 | Gen Hospital Corp | High level expression of proteins |
| ATE486614T1 (de) | 1997-03-14 | 2010-11-15 | Philadelphia Children Hospital | Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie |
| WO1999003496A1 (en) | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
| AU5470900A (en) | 1999-06-08 | 2000-12-28 | Children's Hospital Of Philadelphia, The | Methods for preventing formation of inhibitory antibodies in the setting of genetherapy |
| CN1148228C (zh) | 2000-08-30 | 2004-05-05 | 夏家辉 | 治疗血友病b的基因药物及其制备方法 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| ES2436741T3 (es) | 2004-09-22 | 2014-01-07 | St. Jude Children's Research Hospital | Expresión mejorada de Factor IX en vectores de terapia génica |
| US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US8383388B2 (en) * | 2006-06-19 | 2013-02-26 | Catalyst Biosciences, Inc. | Modified coagulation factor IX polypeptides and use thereof for treatment |
| DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| EP2337849B1 (en) | 2008-09-15 | 2018-06-13 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
| US9663778B2 (en) * | 2009-07-09 | 2017-05-30 | OPKO Biologies Ltd. | Long-acting coagulation factors and methods of producing same |
| 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 |
| HK1202434A1 (en) | 2011-11-22 | 2015-10-02 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
| KR20140102759A (ko) * | 2011-12-16 | 2014-08-22 | 모더나 세라퓨틱스, 인코포레이티드 | 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물 |
| WO2013123503A1 (en) | 2012-02-17 | 2013-08-22 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
| 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 |
| 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 |
| 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 |
| CN116732066A (zh) * | 2016-10-14 | 2023-09-12 | 四川至善唯新生物科技有限公司 | 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用 |
-
2018
- 2018-05-22 KR KR1020197037828A patent/KR102386890B1/ko active Active
- 2018-05-22 WO PCT/US2018/033866 patent/WO2018217731A1/en not_active Ceased
- 2018-05-22 IL IL280637A patent/IL280637B2/en unknown
- 2018-05-22 CA CA3064730A patent/CA3064730A1/en active Pending
- 2018-05-22 EP EP18731636.9A patent/EP3630974A1/en not_active Withdrawn
- 2018-05-22 AR ARP180101356A patent/AR112057A1/es unknown
- 2018-05-22 US US15/986,350 patent/US10842853B2/en active Active
- 2018-05-22 CN CN201880042428.9A patent/CN110945127B/zh active Active
- 2018-05-22 AU AU2018272831A patent/AU2018272831B2/en active Active
- 2018-05-22 BR BR112019024256A patent/BR112019024256A2/pt not_active Application Discontinuation
- 2018-05-22 JP JP2019564430A patent/JP6961171B2/ja active Active
- 2018-05-22 NZ NZ759034A patent/NZ759034A/en unknown
- 2018-05-22 CN CN202410819836.8A patent/CN118581121A/zh active Pending
- 2018-05-22 KR KR1020227012028A patent/KR102551733B1/ko active Active
- 2018-05-22 MX MX2019013836A patent/MX2019013836A/es unknown
- 2018-05-22 TW TW107117388A patent/TWI753168B/zh active
-
2019
- 2019-11-11 IL IL270567A patent/IL270567B/en active IP Right Grant
- 2019-11-18 CO CONC2019/0012885A patent/CO2019012885A2/es unknown
- 2019-11-20 MX MX2024009571A patent/MX2024009571A/es unknown
- 2019-11-21 ZA ZA2019/07716A patent/ZA201907716B/en unknown
- 2019-11-21 CL CL2019003395A patent/CL2019003395A1/es unknown
-
2020
- 2020-09-23 CL CL2020002445A patent/CL2020002445A1/es unknown
- 2020-10-20 US US17/075,556 patent/US20210128700A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020520662A5 (enExample) | ||
| JP2018537087A5 (enExample) | ||
| JP2023115348A5 (enExample) | ||
| JP2018537089A5 (enExample) | ||
| JP2020518268A5 (enExample) | ||
| JP2014012022A5 (enExample) | ||
| JP2018138049A5 (enExample) | ||
| FI3313991T3 (fi) | Muunnettu tekijä IX sekä koostumukset, menetelmät ja käytöt geeninsiirtoa varten soluihin, elimiin ja kudoksiin | |
| JP2014239686A5 (enExample) | ||
| JP2017018125A5 (enExample) | ||
| JP2018520997A5 (enExample) | ||
| JP2019530463A5 (enExample) | ||
| JP2019122392A5 (enExample) | ||
| JP2004512020A5 (enExample) | ||
| RU2005136233A (ru) | Выделенный рекомбинантный вирус гриппа и способы его получения | |
| JP2019506166A5 (enExample) | ||
| JP2019513794A5 (enExample) | ||
| JP2010227101A5 (enExample) | ||
| JPWO2019193119A5 (enExample) | ||
| JP2018531624A5 (enExample) | ||
| JPWO2020069461A5 (enExample) | ||
| JP2021507712A5 (enExample) | ||
| JP2020535804A5 (enExample) | ||
| JPWO2019183150A5 (enExample) | ||
| JP2019524090A5 (enExample) |