JP2011528913A5 - - Google Patents
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- Publication number
- JP2011528913A5 JP2011528913A5 JP2011520375A JP2011520375A JP2011528913A5 JP 2011528913 A5 JP2011528913 A5 JP 2011528913A5 JP 2011520375 A JP2011520375 A JP 2011520375A JP 2011520375 A JP2011520375 A JP 2011520375A JP 2011528913 A5 JP2011528913 A5 JP 2011528913A5
- Authority
- JP
- Japan
- Prior art keywords
- mutant
- factor
- amino acid
- acid substitution
- variant
- 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
- 125000003275 alpha amino acid group Chemical group 0.000 claims 24
- 238000006467 substitution reaction Methods 0.000 claims 24
- 102100022641 Coagulation factor IX Human genes 0.000 claims 18
- 108010076282 Factor IX Proteins 0.000 claims 17
- 229960004222 factor ix Drugs 0.000 claims 17
- 102000039446 nucleic acids Human genes 0.000 claims 5
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 208000031169 hemorrhagic disease Diseases 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 102220160314 rs886062844 Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102200147804 rs193929360 Human genes 0.000 claims 3
- 108010048049 Factor IXa Proteins 0.000 claims 2
- 108010054218 Factor VIII Proteins 0.000 claims 2
- 102000001690 Factor VIII Human genes 0.000 claims 2
- 108010061932 Factor VIIIa Proteins 0.000 claims 2
- 208000009292 Hemophilia A Diseases 0.000 claims 2
- 208000032843 Hemorrhage Diseases 0.000 claims 2
- 206010073391 Platelet dysfunction Diseases 0.000 claims 2
- 208000034158 bleeding Diseases 0.000 claims 2
- 230000000740 bleeding effect Effects 0.000 claims 2
- 239000008280 blood Substances 0.000 claims 2
- 210000004369 blood Anatomy 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 229960000301 factor viii Drugs 0.000 claims 2
- 238000001415 gene therapy Methods 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 102220048271 rs587784390 Human genes 0.000 claims 2
- 102220034170 rs75368761 Human genes 0.000 claims 2
- 206010008111 Cerebral haemorrhage Diseases 0.000 claims 1
- 102100023804 Coagulation factor VII Human genes 0.000 claims 1
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 206010053567 Coagulopathies Diseases 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 108010014172 Factor V Proteins 0.000 claims 1
- 108010023321 Factor VII Proteins 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 108010014173 Factor X Proteins 0.000 claims 1
- 108010074864 Factor XI Proteins 0.000 claims 1
- 208000031220 Hemophilia Diseases 0.000 claims 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 1
- 208000027276 Von Willebrand disease Diseases 0.000 claims 1
- 208000027418 Wounds and injury Diseases 0.000 claims 1
- 239000003146 anticoagulant agent Substances 0.000 claims 1
- 229940127219 anticoagulant drug Drugs 0.000 claims 1
- 230000010100 anticoagulation Effects 0.000 claims 1
- 208000015294 blood coagulation disease Diseases 0.000 claims 1
- 238000010322 bone marrow transplantation Methods 0.000 claims 1
- 238000002659 cell therapy Methods 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 230000035602 clotting Effects 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- 230000006378 damage Effects 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 238000003745 diagnosis Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000013613 expression plasmid Substances 0.000 claims 1
- 229940012413 factor vii Drugs 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 239000003112 inhibitor Substances 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 208000014674 injury Diseases 0.000 claims 1
- 208000019423 liver disease Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- 238000012216 screening Methods 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- 238000001356 surgical procedure Methods 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 206010043554 thrombocytopenia Diseases 0.000 claims 1
- 230000005030 transcription termination Effects 0.000 claims 1
- 230000000472 traumatic effect Effects 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
- 108010047303 von Willebrand Factor Proteins 0.000 claims 1
- 208000012137 von Willebrand disease (hereditary or acquired) Diseases 0.000 claims 1
- 102100036537 von Willebrand factor Human genes 0.000 claims 1
- 229960001134 von willebrand factor Drugs 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP08013561.9 | 2008-07-28 | ||
| EP08013561A EP2149603A1 (en) | 2008-07-28 | 2008-07-28 | Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders |
| PCT/EP2009/005465 WO2010012451A1 (en) | 2008-07-28 | 2009-07-28 | Factor ix variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2011528913A JP2011528913A (ja) | 2011-12-01 |
| JP2011528913A5 true JP2011528913A5 (enExample) | 2012-09-20 |
| JP6029826B2 JP6029826B2 (ja) | 2016-11-24 |
Family
ID=40032573
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011520375A Active JP6029826B2 (ja) | 2008-07-28 | 2009-07-28 | 補因子の非存在下において凝固活性を有する第ｉｘ因子変異体および出血性疾患の処置のためのその使用 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US10125357B2 (enExample) |
| EP (6) | EP2149603A1 (enExample) |
| JP (1) | JP6029826B2 (enExample) |
| KR (2) | KR101947424B1 (enExample) |
| CN (1) | CN102112608B (enExample) |
| AU (1) | AU2009275391B2 (enExample) |
| CA (2) | CA2729501C (enExample) |
| ES (3) | ES2441167T3 (enExample) |
| WO (1) | WO2010012451A1 (enExample) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUE067487T2 (hu) | 2008-09-15 | 2024-10-28 | Uniqure Biopharma B V | IX-es faktor polipeptid mutánsa, alkalmazásai és eljárás az elõállítására |
| 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 |
| AU2012222366A1 (en) | 2011-03-02 | 2013-08-01 | Novo Nordisk A/S | Coagulation factor-targeting to TLT-1 on activated platelets |
| US10656167B2 (en) | 2011-07-25 | 2020-05-19 | Bioverativ Therapeutics Inc. | Assays to monitor bleeding disorders |
| CN103173482A (zh) * | 2011-12-22 | 2013-06-26 | 武汉科技大学 | 野生型和突变体hFⅨ毕赤酵母表达载体及构建方法和应用 |
| EP2666782A1 (en) * | 2012-05-22 | 2013-11-27 | Imnate Sarl | Coagulation factor VIII with reduced immunogenicity. |
| US20150337284A1 (en) * | 2012-10-29 | 2015-11-26 | Regents Of The University Of Minnesota | Factor ix variants |
| CN103060366A (zh) * | 2012-12-25 | 2013-04-24 | 武汉科技大学 | 人凝血因子ⅸ突变体毕赤酵母表达载体及构建方法和应用 |
| LT2956477T (lt) | 2013-02-15 | 2021-02-10 | Bioverativ Therapeutics Inc. | Optimizuotas faktoriaus viii genas |
| US9651567B2 (en) * | 2013-05-14 | 2017-05-16 | Cgt Enterprises, Llc | Test strips for determining coagulation factor activities |
| EP2881463A1 (en) | 2013-12-09 | 2015-06-10 | DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH | Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders |
| EP3160478A4 (en) * | 2014-06-30 | 2018-05-16 | Bioverativ Therapeutics Inc. | Optimized factor ix gene |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| EP3325010B1 (en) | 2015-07-23 | 2023-06-21 | The Regents of The University of California | Antibodies to coagulation factor xia and uses thereof |
| PL3411478T3 (pl) | 2016-02-01 | 2022-10-03 | Bioverativ Therapeutics Inc. | Geny zoptymalizowanego czynnika VIII |
| 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 |
| 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 |
| GB201817810D0 (en) * | 2018-10-31 | 2018-12-19 | Ucl Business Plc | Modified factor IX polypeptides |
| WO2020104480A1 (en) * | 2018-11-19 | 2020-05-28 | Uniqure Biopharma B.V. | Adeno-associated virus vectors for expressing fviii mimetics and uses thereof |
| WO2020187969A1 (en) * | 2019-03-19 | 2020-09-24 | CSL Behring Lengnau AG | Factor ix variants and uses thereof in 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 |
| CN115279896A (zh) | 2019-09-30 | 2022-11-01 | 比奥维拉迪维治疗股份有限公司 | 慢病毒载体配制品 |
| MX2023000156A (es) | 2020-06-24 | 2023-02-16 | Bioverativ Therapeutics Inc | Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina. |
| 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 |
| EP4423119A1 (en) | 2021-10-27 | 2024-09-04 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
| US20250242056A1 (en) | 2024-01-26 | 2025-07-31 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing |
| WO2025184567A1 (en) | 2024-03-01 | 2025-09-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6458563B1 (en) | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
| US6531298B2 (en) * | 1997-07-21 | 2003-03-11 | The University Of North Carolina At Chapel Hill | Factor IX antihemophilic factor with increased clotting activity |
| WO2001085198A1 (en) | 2000-05-10 | 2001-11-15 | Novo Nordisk A/S | tHARMACEUTICAL COMPOSITION COMPRISING A FACTOR VIIA AND A FACTOR XIII |
| US7176288B2 (en) * | 2000-09-13 | 2007-02-13 | Novo Nordisk Healthcare A/G | Human coagulation factor VII variants |
| AU2002226028A1 (en) | 2000-11-14 | 2002-05-27 | Board Of Regents, Unversity Of Texas Systems | Mutant human factor ix with an increased resistance to inhibition by heparin |
| US20060040856A1 (en) * | 2003-12-03 | 2006-02-23 | Neose Technologies, Inc. | Glycopegylated factor IX |
| JP2008509688A (ja) * | 2004-08-17 | 2008-04-03 | ツェー・エス・エル・ベーリング・ゲー・エム・ベー・ハー | 改変ビタミンｋ依存性ポリペプチド |
| TW200804416A (en) * | 2006-06-19 | 2008-01-16 | Nautilus Technology Llc | Modified coagulation factor IX polypeptides and use thereof for treatment |
| US7700734B2 (en) * | 2007-01-09 | 2010-04-20 | Shu-Wha Lin | Recombinant human factor IX and use thereof |
| EP2108045B1 (en) * | 2007-02-01 | 2013-12-11 | Baxter International Inc. | Improved fix-mutant proteins for hemophilia b treatment |
| CA2674881A1 (en) * | 2007-02-01 | 2008-08-07 | Baxter International Inc. | Fviii-independent fix-mutant proteins for hemophilia a treatment |
| DE102008034724A1 (de) * | 2008-07-25 | 2010-01-28 | Alexander Koslow | Verkugelungs- / Zerkleinerungsmaschine |
-
2008
- 2008-07-28 EP EP08013561A patent/EP2149603A1/en not_active Withdrawn
-
2009
- 2009-07-28 EP EP11005215.6A patent/EP2390323B1/en active Active
- 2009-07-28 EP EP09777493A patent/EP2318526A1/en not_active Ceased
- 2009-07-28 ES ES11005215.6T patent/ES2441167T3/es active Active
- 2009-07-28 ES ES17164245T patent/ES2753638T3/es active Active
- 2009-07-28 CA CA2729501A patent/CA2729501C/en active Active
- 2009-07-28 EP EP19191719.4A patent/EP3623470A3/en not_active Withdrawn
- 2009-07-28 JP JP2011520375A patent/JP6029826B2/ja active Active
- 2009-07-28 WO PCT/EP2009/005465 patent/WO2010012451A1/en not_active Ceased
- 2009-07-28 ES ES14165396.4T patent/ES2628688T3/es active Active
- 2009-07-28 KR KR1020177022245A patent/KR101947424B1/ko active Active
- 2009-07-28 CN CN2009801296698A patent/CN102112608B/zh active Active
- 2009-07-28 CA CA3033578A patent/CA3033578C/en active Active
- 2009-07-28 EP EP17164245.7A patent/EP3241899B1/en active Active
- 2009-07-28 US US13/001,187 patent/US10125357B2/en active Active
- 2009-07-28 KR KR1020117001765A patent/KR101768896B1/ko active Active
- 2009-07-28 EP EP14165396.4A patent/EP2767587B1/en active Active
- 2009-07-28 AU AU2009275391A patent/AU2009275391B2/en active Active
-
2018
- 2018-10-01 US US16/148,819 patent/US10883097B2/en active Active
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