JP2021003120A5 - - Google Patents
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- Publication number
- JP2021003120A5 JP2021003120A5 JP2020153781A JP2020153781A JP2021003120A5 JP 2021003120 A5 JP2021003120 A5 JP 2021003120A5 JP 2020153781 A JP2020153781 A JP 2020153781A JP 2020153781 A JP2020153781 A JP 2020153781A JP 2021003120 A5 JP2021003120 A5 JP 2021003120A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- fviii
- aav
- acid variant
- vector
- 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 40
- 108020004707 nucleic acids Proteins 0.000 claims 39
- 102000039446 nucleic acids Human genes 0.000 claims 39
- 108010054218 Factor VIII Proteins 0.000 claims 30
- 102000001690 Factor VIII Human genes 0.000 claims 30
- 239000013604 expression vector Substances 0.000 claims 29
- 229960000301 factor viii Drugs 0.000 claims 28
- 239000013607 AAV vector Substances 0.000 claims 23
- 241000700605 Viruses Species 0.000 claims 17
- 108020004705 Codon Proteins 0.000 claims 9
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 5
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims 5
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims 5
- 241000124008 Mammalia Species 0.000 claims 5
- 239000013598 vector Substances 0.000 claims 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 4
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 4
- 230000007812 deficiency Effects 0.000 claims 4
- 102000057593 human F8 Human genes 0.000 claims 4
- 210000004962 mammalian cell Anatomy 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 229940079593 drug Drugs 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 238000004519 manufacturing process Methods 0.000 claims 3
- 102100035233 Furin Human genes 0.000 claims 2
- 108090001126 Furin Proteins 0.000 claims 2
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 2
- 239000004472 Lysine Substances 0.000 claims 2
- 230000004071 biological effect Effects 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- 210000002889 endothelial cell Anatomy 0.000 claims 2
- 239000000945 filler Substances 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 210000000056 organ Anatomy 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 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
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 102000003712 Complement factor B Human genes 0.000 claims 1
- 108090000056 Complement factor B Proteins 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 208000032843 Hemorrhage Diseases 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 230000002159 abnormal effect Effects 0.000 claims 1
- 238000004458 analytical method Methods 0.000 claims 1
- 210000004102 animal cell Anatomy 0.000 claims 1
- 238000003556 assay Methods 0.000 claims 1
- 230000000740 bleeding effect Effects 0.000 claims 1
- 210000001772 blood platelet Anatomy 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 230000015271 coagulation Effects 0.000 claims 1
- 238000005345 coagulation Methods 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 210000003890 endocrine cell Anatomy 0.000 claims 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 230000010354 integration Effects 0.000 claims 1
- 210000003593 megakaryocyte Anatomy 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 230000007017 scission Effects 0.000 claims 1
- 210000002955 secretory cell Anatomy 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 238000002627 tracheal intubation Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462036936P | 2014-08-13 | 2014-08-13 | |
| US62/036,936 | 2014-08-13 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017507431A Division JP2018506261A (ja) | 2014-08-13 | 2015-08-13 | 止血疾患を治療するための変異因子ｖｉｉｉのゲノム組込及び発現のための改良された発現カセット |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021003120A JP2021003120A (ja) | 2021-01-14 |
| JP2021003120A5 true JP2021003120A5 (enExample) | 2021-11-11 |
| JP7237903B2 JP7237903B2 (ja) | 2023-03-13 |
Family
ID=55304765
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017507431A Pending JP2018506261A (ja) | 2014-08-13 | 2015-08-13 | 止血疾患を治療するための変異因子ｖｉｉｉのゲノム組込及び発現のための改良された発現カセット |
| JP2020153781A Active JP7237903B2 (ja) | 2014-08-13 | 2020-09-14 | 止血疾患を治療するための変異因子ｖｉｉｉのゲノム組込及び発現のための改良された発現カセット |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017507431A Pending JP2018506261A (ja) | 2014-08-13 | 2015-08-13 | 止血疾患を治療するための変異因子ｖｉｉｉのゲノム組込及び発現のための改良された発現カセット |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US11014975B2 (enExample) |
| EP (1) | EP3180022B1 (enExample) |
| JP (2) | JP2018506261A (enExample) |
| KR (1) | KR102665348B1 (enExample) |
| CN (1) | CN107427557B (enExample) |
| AU (1) | AU2015301598B2 (enExample) |
| CA (2) | CA3237630A1 (enExample) |
| ES (1) | ES2993915T3 (enExample) |
| SA (1) | SA517380883B1 (enExample) |
| SG (1) | SG11201701033QA (enExample) |
| WO (1) | WO2016025764A2 (enExample) |
| ZA (1) | ZA201701039B (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3626274B1 (en) * | 2015-04-16 | 2025-01-22 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| US11103597B2 (en) * | 2015-05-14 | 2021-08-31 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers outside ITR |
| US11230720B2 (en) | 2015-10-14 | 2022-01-25 | Audentes Therapeutics, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| SG11201804070XA (en) | 2015-11-13 | 2018-06-28 | Baxalta Inc | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| EP3374388A1 (en) | 2015-11-13 | 2018-09-19 | Baxalta Incorporated | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| WO2018102743A1 (en) | 2016-12-02 | 2018-06-07 | Bioverativ Therapeutics Inc. | Methods of treating hemophilic arthropathy using chimeric clotting factors |
| BR112020001979A2 (pt) * | 2017-08-01 | 2020-08-18 | Spark Therapeutics, Inc. | métodos de terapia genética do fator viii (fviii) |
| WO2019210187A1 (en) * | 2018-04-26 | 2019-10-31 | The University Of North Carolina At Chapel Hill | Methods and compositions for treatment of hemophilia |
| JP7763589B2 (ja) | 2018-05-18 | 2025-11-04 | バイオベラティブ セラピューティクス インコーポレイテッド | 血友病ａを処置する方法 |
| CN108795986A (zh) * | 2018-05-31 | 2018-11-13 | 深圳市免疫基因治疗研究院 | 一种a型血友病慢病毒载体、慢病毒及其制备方法和应用 |
| AU2019306194A1 (en) | 2018-07-16 | 2021-02-04 | Baxalta GmbH | Gene therapy of hemophilia A using viral vectors encoding recombinant FVIII variants with increased expression |
| 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 |
| EP3736286A1 (en) * | 2019-05-09 | 2020-11-11 | Biotest AG | Single chain factor viii molecule |
| 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 |
| WO2022241041A2 (en) * | 2021-05-11 | 2022-11-17 | City Of Hope | Adeno-associated virus compositions and methods of use thereof |
| WO2024007978A1 (zh) * | 2022-07-07 | 2024-01-11 | 深圳新诺微环生物科技有限公司 | 一种连接肽、包含连接肽的凝血八因子蛋白或其变体及其用途 |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| WO2024074143A1 (en) * | 2022-10-08 | 2024-04-11 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
| CN115873099B (zh) * | 2022-12-30 | 2023-10-20 | 福因医药科技(武汉)有限公司 | 一种改造的重组凝血因子viii及其应用 |
| CN116715752B (zh) * | 2023-06-16 | 2024-11-15 | 苏州诺洁贝生物技术有限公司 | 凝血因子fviii蛋白变体、表达载体和用途 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6114148C1 (en) | 1996-09-20 | 2012-05-01 | Gen Hospital Corp | High level expression of proteins |
| US6924365B1 (en) * | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
| US6221349B1 (en) * | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US8999678B2 (en) * | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| EP2037892B1 (en) * | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
| US20120028900A1 (en) | 2006-06-30 | 2012-02-02 | Kaufman Randal J | Method of producing factor viii proteins by recombinant methods |
| CN101597616A (zh) * | 2008-06-04 | 2009-12-09 | 上海同科生物科技有限公司 | 一种提高基因重组人凝血因子ⅷ表达量的方法 |
| AU2013202564B2 (en) | 2008-06-24 | 2015-09-17 | Csl Behring Gmbh | Factor VIII, von Willebrand factor or complexes thereof with prolonged in vivo half-life |
| GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| DK2482841T4 (da) | 2009-10-02 | 2022-10-24 | Childrens Hospital Philadelphia | Sammensætninger og fremgangsmåder til at forbedre koagulationsfaktor-viii-funktion |
| WO2014008480A2 (en) * | 2012-07-06 | 2014-01-09 | Biogen Idec Ma Inc. | Cell line expressing single chain factor viii polypeptides and uses thereof |
| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| DK2956477T4 (da) * | 2013-02-15 | 2024-04-15 | Bioverativ Therapeutics Inc | Optimeret faktor viii-gen |
-
2015
- 2015-08-13 AU AU2015301598A patent/AU2015301598B2/en active Active
- 2015-08-13 ES ES15832128T patent/ES2993915T3/es active Active
- 2015-08-13 JP JP2017507431A patent/JP2018506261A/ja active Pending
- 2015-08-13 SG SG11201701033QA patent/SG11201701033QA/en unknown
- 2015-08-13 EP EP15832128.1A patent/EP3180022B1/en active Active
- 2015-08-13 CA CA3237630A patent/CA3237630A1/en active Pending
- 2015-08-13 CA CA2958141A patent/CA2958141C/en active Active
- 2015-08-13 KR KR1020177006818A patent/KR102665348B1/ko active Active
- 2015-08-13 CN CN201580055574.1A patent/CN107427557B/zh active Active
- 2015-08-13 WO PCT/US2015/045142 patent/WO2016025764A2/en not_active Ceased
- 2015-08-13 US US15/503,120 patent/US11014975B2/en active Active
-
2017
- 2017-02-10 ZA ZA2017/01039A patent/ZA201701039B/en unknown
- 2017-02-12 SA SA517380883A patent/SA517380883B1/ar unknown
-
2020
- 2020-09-14 JP JP2020153781A patent/JP7237903B2/ja active Active
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