JPWO2020028830A5 - - Google Patents
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- JPWO2020028830A5 JPWO2020028830A5 JP2021504376A JP2021504376A JPWO2020028830A5 JP WO2020028830 A5 JPWO2020028830 A5 JP WO2020028830A5 JP 2021504376 A JP2021504376 A JP 2021504376A JP 2021504376 A JP2021504376 A JP 2021504376A JP WO2020028830 A5 JPWO2020028830 A5 JP WO2020028830A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- human subject
- aav vector
- seq
- aav
- 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.)
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Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862714553P | 2018-08-03 | 2018-08-03 | |
| US62/714,553 | 2018-08-03 | ||
| US201962826887P | 2019-03-29 | 2019-03-29 | |
| US62/826,887 | 2019-03-29 | ||
| US201962869445P | 2019-07-01 | 2019-07-01 | |
| US62/869,445 | 2019-07-01 | ||
| PCT/US2019/044946 WO2020028830A1 (en) | 2018-08-03 | 2019-08-02 | Improved clinical parameters by expression of factor viii |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021533098A JP2021533098A (ja) | 2021-12-02 |
| JP2021533098A5 JP2021533098A5 (https=) | 2022-08-09 |
| JPWO2020028830A5 true JPWO2020028830A5 (https=) | 2022-08-09 |
| JP7684212B2 JP7684212B2 (ja) | 2025-05-27 |
Family
ID=69230742
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021504376A Active JP7684212B2 (ja) | 2018-08-03 | 2019-08-02 | 第ｖｉｉｉ因子の発現による改善された臨床パラメーター |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12497632B2 (https=) |
| EP (1) | EP3829650A4 (https=) |
| JP (1) | JP7684212B2 (https=) |
| KR (1) | KR20210039428A (https=) |
| CN (1) | CN112533645B (https=) |
| BR (1) | BR112021001315A2 (https=) |
| CA (1) | CA3105382A1 (https=) |
| IL (1) | IL280402B1 (https=) |
| MX (1) | MX2021001375A (https=) |
| WO (1) | WO2020028830A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240066146A1 (en) | 2020-12-18 | 2024-02-29 | Sangamo Therapeutics, Inc. | Improved pharmaceutical compositions containing adeno-associated viral vector |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU5588298A (en) | 1996-12-02 | 1998-06-29 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| CA2392863A1 (en) | 2002-07-08 | 2004-01-08 | Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw | A high capacity recombinant adenoviral vector for treatment of hemophilia a |
| PL1675956T3 (pl) | 2003-10-21 | 2011-06-30 | Merck Serono Sa | Minimalna sekwencja DNA działająca jako insulator chromatyny i jej zastosowanie do wyrażania białek |
| JPWO2006043354A1 (ja) | 2004-10-20 | 2008-05-22 | 独立行政法人放射線医学総合研究所 | 組込型の低線量放射線誘導性ベクター |
| MX2007015540A (es) | 2005-06-10 | 2008-03-07 | Genentech Inc | Mejora de la expresion de anticuerpos utilizando vectores que contienen elementos aislantes. |
| CN101965409A (zh) | 2007-11-01 | 2011-02-02 | 罗切斯特大学 | 具有增加的稳定性的重组因子viii |
| EP4154903A1 (en) | 2008-04-22 | 2023-03-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| WO2009134681A2 (en) * | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| AU2012312260B2 (en) * | 2011-09-21 | 2017-08-31 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| CN104114572A (zh) * | 2011-12-16 | 2014-10-22 | 现代治疗公司 | 经修饰的核苷、核苷酸和核酸组合物 |
| DK2804623T3 (da) | 2012-01-12 | 2019-11-11 | Bioverativ Therapeutics Inc | Kimære faktor viii-polypeptider og anvendelser deraf |
| DK3444342T3 (da) | 2012-07-11 | 2020-08-24 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme |
| JP2015529466A (ja) | 2012-09-14 | 2015-10-08 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| BR112015025041A2 (pt) | 2013-03-30 | 2017-09-12 | Usha Biotech Ltd | métodos e conceitos para expressar proteínas biologicamente ativas em células de mamíferos |
| RS61039B1 (sr) | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| DK3080274T3 (da) | 2013-12-09 | 2020-08-31 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til genom-manipulation |
| AU2015204646B2 (en) | 2014-01-10 | 2020-08-27 | Bioverativ Therapeutics Inc. | Factor VIII chimeric proteins and uses thereof |
| KR102554850B1 (ko) | 2015-02-06 | 2023-07-13 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도 |
| CN115074366A (zh) * | 2015-04-16 | 2022-09-20 | 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 |
| WO2016180755A1 (en) | 2015-05-11 | 2016-11-17 | Bayer Cropscience Aktiengesellschaft | Herbicide combinations comprising l-glufosinate and indaziflam |
| EP3322297B1 (en) | 2015-07-13 | 2024-12-04 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| RU2742352C2 (ru) | 2015-09-24 | 2021-02-05 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| CA3002524A1 (en) | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| EP3374387A1 (en) * | 2015-11-13 | 2018-09-19 | Baxalta Incorporated | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| AU2018312565A1 (en) * | 2017-08-01 | 2020-02-27 | Spark Therapeutics, Inc. | Factor VIII (FVIII) gene therapy methods |
-
2019
- 2019-08-02 WO PCT/US2019/044946 patent/WO2020028830A1/en not_active Ceased
- 2019-08-02 EP EP19843874.9A patent/EP3829650A4/en active Pending
- 2019-08-02 KR KR1020217006266A patent/KR20210039428A/ko active Pending
- 2019-08-02 CA CA3105382A patent/CA3105382A1/en active Pending
- 2019-08-02 BR BR112021001315-6A patent/BR112021001315A2/pt unknown
- 2019-08-02 US US17/265,750 patent/US12497632B2/en active Active
- 2019-08-02 MX MX2021001375A patent/MX2021001375A/es unknown
- 2019-08-02 JP JP2021504376A patent/JP7684212B2/ja active Active
- 2019-08-02 CN CN201980051349.9A patent/CN112533645B/zh active Active
-
2021
- 2021-01-26 IL IL280402A patent/IL280402B1/en unknown
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