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.)
- Granted
Links
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2021533098A JP2021533098A (ja) | 2021-12-02 |
| JPWO2020028830A5 true JPWO2020028830A5 (enExample) | 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 (11)
| Country | Link |
|---|---|
| US (1) | US12497632B2 (enExample) |
| EP (1) | EP3829650A4 (enExample) |
| JP (1) | JP7684212B2 (enExample) |
| KR (1) | KR20210039428A (enExample) |
| CN (1) | CN112533645A (enExample) |
| AU (1) | AU2019314513A1 (enExample) |
| BR (1) | BR112021001315A2 (enExample) |
| CA (1) | CA3105382A1 (enExample) |
| IL (1) | IL280402A (enExample) |
| MX (1) | MX2021001375A (enExample) |
| WO (1) | WO2020028830A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL303737A (en) | 2020-12-18 | 2023-08-01 | 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 |
| EP1675956B1 (en) | 2003-10-21 | 2010-10-13 | Merck Serono SA | Minimal dna sequence acting as a chromatin insulator and its use in protein expression |
| 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 |
| CA2611465A1 (en) | 2005-06-10 | 2007-02-22 | Genentech, Inc. | Improving antibody expression using vectors containing insulator elements |
| KR20100113478A (ko) | 2007-11-01 | 2010-10-21 | 유니버시티 오브 로체스터 | 증가된 안정성을 갖는 재조합 인자 ⅴⅰⅰⅰ |
| DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| 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 |
| KR102061557B1 (ko) | 2011-09-21 | 2020-01-03 | 상가모 테라퓨틱스, 인코포레이티드 | 이식 유전자 발현의 조절을 위한 방법 및 조성물 |
| EP4144378A1 (en) * | 2011-12-16 | 2023-03-08 | ModernaTX, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| RS59670B1 (sr) | 2012-01-12 | 2020-01-31 | Bioverativ Therapeutics Inc | Himerni polipeptidi faktora viii i njihove upotrebe |
| EP3816281A1 (en) | 2012-07-11 | 2021-05-05 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| WO2014043131A1 (en) | 2012-09-14 | 2014-03-20 | The Regents Of The University Of California | Lentiviral vector for stem cell gene therapy of sickle cell disease |
| DK2911687T3 (da) | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| AU2014246731A1 (en) | 2013-03-30 | 2015-11-19 | Usha Biotech Limited | Methods and constructs for expressing biologically active proteins in mammalian cells |
| SI3044231T1 (sl) | 2013-09-12 | 2020-12-31 | Biomarin Pharmaceutical Inc. | Vektorji AAV, ki vsebujejo gen, ki kodira faktor VIII |
| CN105940013B (zh) | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
| CN114736305B (zh) | 2014-01-10 | 2023-05-05 | 比奥贝拉蒂治疗公司 | 因子viii嵌合蛋白及其用途 |
| MX388739B (es) * | 2015-02-06 | 2025-03-20 | Univ North Carolina Chapel Hill | Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso. |
| ES3009565T3 (en) * | 2015-04-16 | 2025-03-27 | Univ Emory | Recombinant promoters and vectors for protein expression in liver and use thereof |
| CU20170141A7 (es) | 2015-05-11 | 2018-04-03 | Bayer Cropscience Ag | Combinaciones herbicidas que comprenden l-glufosinato e indaziflam |
| AU2016291778B2 (en) | 2015-07-13 | 2021-05-06 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| RU2020126034A (ru) * | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3368664A4 (en) * | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| AU2016353353B2 (en) * | 2015-11-13 | 2020-07-09 | Takeda Pharmaceutical Company Limited | Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A |
| MY208862A (en) * | 2017-08-01 | 2025-06-04 | Spark Therapeutics Inc | Factor viii (fviii) gene therapy methods |
-
2019
- 2019-08-02 MX MX2021001375A patent/MX2021001375A/es unknown
- 2019-08-02 EP EP19843874.9A patent/EP3829650A4/en active Pending
- 2019-08-02 JP JP2021504376A patent/JP7684212B2/ja active Active
- 2019-08-02 WO PCT/US2019/044946 patent/WO2020028830A1/en not_active Ceased
- 2019-08-02 KR KR1020217006266A patent/KR20210039428A/ko active Pending
- 2019-08-02 AU AU2019314513A patent/AU2019314513A1/en active Pending
- 2019-08-02 CN CN201980051349.9A patent/CN112533645A/zh active Pending
- 2019-08-02 BR BR112021001315-6A patent/BR112021001315A2/pt unknown
- 2019-08-02 CA CA3105382A patent/CA3105382A1/en active Pending
- 2019-08-02 US US17/265,750 patent/US12497632B2/en active Active
-
2021
- 2021-01-26 IL IL280402A patent/IL280402A/en unknown
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