JP2018506261A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018506261A5 JP2018506261A5 JP2017507431A JP2017507431A JP2018506261A5 JP 2018506261 A5 JP2018506261 A5 JP 2018506261A5 JP 2017507431 A JP2017507431 A JP 2017507431A JP 2017507431 A JP2017507431 A JP 2017507431A JP 2018506261 A5 JP2018506261 A5 JP 2018506261A5
- Authority
- JP
- Japan
- Prior art keywords
- fviii
- nucleic acid
- variant
- aav
- acid 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.)
- Pending
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462036936P | 2014-08-13 | 2014-08-13 | |
| US62/036,936 | 2014-08-13 | ||
| PCT/US2015/045142 WO2016025764A2 (en) | 2014-08-13 | 2015-08-13 | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020153781A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018506261A JP2018506261A (ja) | 2018-03-08 |
| JP2018506261A5 true JP2018506261A5 (enExample) | 2018-09-20 |
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 After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
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) | CA2958141C (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 (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EA201691111A1 (ru) | 2014-01-10 | 2017-01-30 | Байоджен Ма Инк. | Химерные белки фактора viii и их применение |
| CN115094062A (zh) * | 2015-04-16 | 2022-09-23 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| AU2016260401B2 (en) * | 2015-05-14 | 2022-05-19 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
| WO2017066579A1 (en) | 2015-10-14 | 2017-04-20 | 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 |
| KR102404550B1 (ko) | 2015-11-13 | 2022-05-31 | 다케다 야쿠힌 고교 가부시키가이샤 | A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터 |
| CN108884145B (zh) | 2015-11-13 | 2023-08-22 | 武田药品工业株式会社 | 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体 |
| KR20190090827A (ko) | 2016-12-02 | 2019-08-02 | 바이오버라티브 테라퓨틱스 인크. | 키메라 응고 인자를 사용해 혈우병성 관절증을 치료하는 방법 |
| US20200237930A1 (en) * | 2017-08-01 | 2020-07-30 | Spark Therapeutics, Inc. | Factor viii (fviii) gene therapy methods |
| US20210093735A1 (en) * | 2018-04-26 | 2021-04-01 | The University Of North Carolina At Chapel Hill | Methods and compositions for treatment of hemophilia |
| KR102914074B1 (ko) | 2018-05-18 | 2026-01-16 | 바이오버라티브 테라퓨틱스 인크. | A형 혈우병의 치료 방법 |
| 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 |
| US12091675B2 (en) | 2018-07-16 | 2024-09-17 | Takeda Pharmaceutical Company Limited | 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 |
| CN119233998A (zh) * | 2022-07-07 | 2024-12-31 | 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 |
| EP4598590A4 (en) * | 2022-10-08 | 2026-05-27 | Lingyi Biotech Co Ltd | CONSTRUCTION TO IMPROVE 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 |
| DE23193606T1 (de) * | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| CN101511373B (zh) * | 2006-06-19 | 2015-12-09 | 阿斯克肋匹奥生物制药公司 | 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体 |
| 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 |
| US10023628B2 (en) * | 2012-07-06 | 2018-07-17 | Bioverativ Therapeutics 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 |
| RS61387B2 (sr) * | 2013-02-15 | 2024-06-28 | Bioverativ Therapeutics Inc | Gen optimizovanog faktora viii |
-
2015
- 2015-08-13 SG SG11201701033QA patent/SG11201701033QA/en unknown
- 2015-08-13 KR KR1020177006818A patent/KR102665348B1/ko active Active
- 2015-08-13 JP JP2017507431A patent/JP2018506261A/ja active Pending
- 2015-08-13 EP EP15832128.1A patent/EP3180022B1/en active Active
- 2015-08-13 CN CN201580055574.1A patent/CN107427557B/zh active Active
- 2015-08-13 AU AU2015301598A patent/AU2015301598B2/en active Active
- 2015-08-13 US US15/503,120 patent/US11014975B2/en active Active
- 2015-08-13 ES ES15832128T patent/ES2993915T3/es active Active
- 2015-08-13 CA CA2958141A patent/CA2958141C/en active Active
- 2015-08-13 WO PCT/US2015/045142 patent/WO2016025764A2/en not_active Ceased
- 2015-08-13 CA CA3237630A patent/CA3237630A1/en active Pending
-
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
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021003120A5 (enExample) | ||
| US20220016264A1 (en) | Aav virions with decreased immunoreactivity and uses therefor | |
| US7427396B2 (en) | AAV vectors for gene delivery to the lung | |
| US7943374B2 (en) | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb | |
| JP2020522269A5 (enExample) | ||
| RU2018119710A (ru) | Кодирующие фактор viii варианты с пониженным содержанием cpg, композиции и способы, и применение в лечении нарушений гемостаза | |
| JP2020533276A5 (enExample) | ||
| RU2018116076A (ru) | Модифицированные гены атаксии фридрейха и векторы для генной терапии | |
| JP2019503649A5 (enExample) | ||
| JP2018531609A5 (enExample) | ||
| JP2021523718A5 (enExample) | ||
| IL296255A (en) | Gene therapy | |
| WO2022126189A1 (en) | Adeno-associated virus capsids and vectors | |
| CN118660969A (zh) | 使用aav载体的亲联蛋白-2（jph2）基因疗法 | |
| JP2020510433A5 (enExample) | ||
| RU2018124448A (ru) | Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii | |
| JP2024536223A (ja) | Ｓｌｃ１３ａ５遺伝子療法ベクターおよびその使用 | |
| NZ729107B2 (en) | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders | |
| NZ729107A (en) | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders | |
| US20240181082A1 (en) | Methods and means for the prevention and/or treatment of hemophilic arthropathy in hemophilia | |
| WO2024163335A2 (en) | Pten gene therapy vectors and uses thereof | |
| RU2022111695A (ru) | Выделенная нуклеиновая кислота, которая кодирует слитый белок на основе FVIII-BDD и гетерологичного сигнального пептида, и ее применение | |
| JP2025508743A5 (enExample) | ||
| JP2023543356A (ja) | 組成物およびその使用 | |
| CN119569833A (zh) | 靶向骨骼肌的aav衣壳蛋白变体及其用途 |