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
- 150000007523 nucleic acids Chemical class 0.000 claims description 110
- 239000000203 mixture Substances 0.000 claims description 72
- 102000039446 nucleic acids Human genes 0.000 claims description 72
- 108020004707 nucleic acids Proteins 0.000 claims description 72
- 108091033319 polynucleotide Proteins 0.000 claims description 66
- 102000040430 polynucleotide Human genes 0.000 claims description 66
- 239000002157 polynucleotide Substances 0.000 claims description 66
- 102100022641 Coagulation factor IX Human genes 0.000 claims description 64
- 108010076282 Factor IX Proteins 0.000 claims description 61
- 229960004222 factor ix Drugs 0.000 claims description 47
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 38
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 10
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 9
- 241000702421 Dependoparvovirus Species 0.000 claims description 8
- 238000001415 gene therapy Methods 0.000 claims description 6
- 210000004185 liver Anatomy 0.000 claims description 6
- 239000002773 nucleotide Substances 0.000 claims description 4
- 125000003729 nucleotide group Chemical group 0.000 claims description 4
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims description 3
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims description 3
- 229920001184 polypeptide Polymers 0.000 claims description 3
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 3
- 238000006467 substitution reaction Methods 0.000 claims description 3
- 230000014621 translational initiation Effects 0.000 claims description 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 description 3
- 238000004519 manufacturing process Methods 0.000 description 3
- 238000000034 method Methods 0.000 description 3
- 239000002245 particle Substances 0.000 description 2
- 230000003362 replicative effect Effects 0.000 description 2
- 108091026890 Coding region Proteins 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
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) | CN118581121A (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 |
| 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 |
| CN113260701A (zh) | 2018-10-18 | 2021-08-13 | 英特利亚治疗股份有限公司 | 用于表达因子ix的组合物和方法 |
| 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 |
| EP3941509B1 (en) * | 2019-03-19 | 2025-12-03 | CSL Innovation Pty Ltd | Factor ix variants and uses thereof in therapy |
| AR119271A1 (es) * | 2019-06-11 | 2021-12-09 | Shire Human Genetic Therapies | Administración del vector viral adenoasociado para el tratamiento de enfermedades mediadas por la calicreína plasmática desregulada |
| 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 |
| EP4090382A2 (en) * | 2020-01-16 | 2022-11-23 | Takeda Pharmaceutical Company Limited | Adeno associated virus based gene therapy for phenylketonuria |
| 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 |
| AR126407A1 (es) * | 2021-07-08 | 2023-10-11 | Tenaya Therapeutics Inc | Casetes de expresión optimizada para genoterapia |
| 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 |
| MX2024005124A (es) * | 2021-10-27 | 2024-05-16 | Regeneron Pharma | Composiciones y metodos para expresar el factor ix para terapia para la hemofilia b. |
| 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 |
| JP4289687B2 (ja) | 1997-03-14 | 2009-07-01 | ザ チルドレンズ ホスピタル オブ フィラデルフィア | 血友病の治療のための遺伝子治療で使用する方法と組成物 |
| 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 |
| US8063022B1 (en) | 1999-06-08 | 2011-11-22 | The Children's Hospital Of Philadelphia | Methods for preventing formation of inhibitory antibodies in the setting of gene therapy |
| 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 |
| PT2359869T (pt) | 2001-12-17 | 2019-04-16 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| ES2381796T3 (es) * | 2004-09-22 | 2012-05-31 | 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 |
| EP2423305A1 (en) * | 2006-06-19 | 2012-02-29 | Catalyst Biosciences, Inc. | Modified coagulation factor IX polypeptides and use thereof for treatment |
| WO2009130208A1 (en) | 2008-04-22 | 2009-10-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| ES2985035T3 (es) | 2008-09-15 | 2024-11-04 | Uniqure Biopharma B V | Mutante de polipéptido de factor IX, sus usos y su procedimiento de producción |
| 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 |
| CA2856137A1 (en) | 2011-11-22 | 2013-05-30 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
| DE12858350T1 (de) * | 2011-12-16 | 2021-10-07 | Modernatx, Inc. | Modifizierte mrna zusammensetzungen |
| CA2864879C (en) | 2012-02-17 | 2021-07-20 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
| 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 |
| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| EP3270944B1 (en) * | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
| IL256517B2 (en) * | 2015-06-23 | 2024-06-01 | Childrens Hospital Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| CN116355883A (zh) * | 2016-10-14 | 2023-06-30 | 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 AR ARP180101356A patent/AR112057A1/es unknown
- 2018-05-22 US US15/986,350 patent/US10842853B2/en active Active
- 2018-05-22 CN CN202410819836.8A patent/CN118581121A/zh active Pending
- 2018-05-22 JP JP2019564430A patent/JP6961171B2/ja active Active
- 2018-05-22 AU AU2018272831A patent/AU2018272831B2/en active Active
- 2018-05-22 NZ NZ759034A patent/NZ759034A/en unknown
- 2018-05-22 TW TW107117388A patent/TWI753168B/zh active
- 2018-05-22 KR KR1020197037828A patent/KR102386890B1/ko active Active
- 2018-05-22 BR BR112019024256A patent/BR112019024256A2/pt not_active Application Discontinuation
- 2018-05-22 MX MX2019013836A patent/MX2019013836A/es unknown
- 2018-05-22 CN CN201880042428.9A patent/CN110945127B/zh active Active
- 2018-05-22 WO PCT/US2018/033866 patent/WO2018217731A1/en not_active Ceased
- 2018-05-22 EP EP18731636.9A patent/EP3630974A1/en not_active Withdrawn
- 2018-05-22 KR KR1020227012028A patent/KR102551733B1/ko active Active
- 2018-05-22 IL IL280637A patent/IL280637B2/en unknown
- 2018-05-22 CA CA3064730A patent/CA3064730A1/en active Pending
-
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) | ||
| JP2017532966A5 (enExample) | ||
| JP2020522259A5 (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) | ||
| JP2004512020A5 (enExample) | ||
| RU2005136233A (ru) | Выделенный рекомбинантный вирус гриппа и способы его получения | |
| JP2019506166A5 (enExample) | ||
| JP2019513794A5 (enExample) | ||
| JP2010227101A5 (enExample) | ||
| JPWO2019193119A5 (enExample) | ||
| JP2018531624A5 (enExample) | ||
| JP2020510426A5 (enExample) | ||
| JP2017507644A5 (enExample) | ||
| JP2021507712A5 (enExample) | ||
| JP2020535804A5 (enExample) |