JP2019503649A5 - - Google Patents
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- JP2019503649A5 JP2019503649A5 JP2018521281A JP2018521281A JP2019503649A5 JP 2019503649 A5 JP2019503649 A5 JP 2019503649A5 JP 2018521281 A JP2018521281 A JP 2018521281A JP 2018521281 A JP2018521281 A JP 2018521281A JP 2019503649 A5 JP2019503649 A5 JP 2019503649A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- aav
- sequence
- acid variant
- seq
- 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
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022093877A JP2022137029A (ja) | 2015-10-30 | 2022-06-09 | ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用 |
| JP2024131729A JP2024170416A (ja) | 2015-10-30 | 2024-08-08 | ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用 |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562249001P | 2015-10-30 | 2015-10-30 | |
| US62/249,001 | 2015-10-30 | ||
| US201662331872P | 2016-05-04 | 2016-05-04 | |
| US62/331,872 | 2016-05-04 | ||
| US201662349532P | 2016-06-13 | 2016-06-13 | |
| US62/349,532 | 2016-06-13 | ||
| US201662357874P | 2016-07-01 | 2016-07-01 | |
| US62/357,874 | 2016-07-01 | ||
| PCT/US2016/059793 WO2017075619A1 (en) | 2015-10-30 | 2016-10-31 | Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022093877A Division JP2022137029A (ja) | 2015-10-30 | 2022-06-09 | ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019503649A JP2019503649A (ja) | 2019-02-14 |
| JP2019503649A5 true JP2019503649A5 (https=) | 2019-12-05 |
| JP7088831B2 JP7088831B2 (ja) | 2022-06-21 |
Family
ID=58630834
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521281A Active JP7088831B2 (ja) | 2015-10-30 | 2016-10-31 | 核酸変種、アデノ随伴ウイルスベクター及び医薬組成物 |
| JP2022093877A Pending JP2022137029A (ja) | 2015-10-30 | 2022-06-09 | ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用 |
| JP2024131729A Pending JP2024170416A (ja) | 2015-10-30 | 2024-08-08 | ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022093877A Pending JP2022137029A (ja) | 2015-10-30 | 2022-06-09 | ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用 |
| JP2024131729A Pending JP2024170416A (ja) | 2015-10-30 | 2024-08-08 | ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用 |
Country Status (19)
| Country | Link |
|---|---|
| US (3) | US20180312571A1 (https=) |
| EP (1) | EP3368560B1 (https=) |
| JP (3) | JP7088831B2 (https=) |
| KR (1) | KR20180090795A (https=) |
| CN (1) | CN108368164A (https=) |
| AU (1) | AU2016344030B2 (https=) |
| BR (1) | BR112018008766A2 (https=) |
| CA (1) | CA3002689A1 (https=) |
| CL (1) | CL2018001107A1 (https=) |
| CO (1) | CO2018005306A2 (https=) |
| IL (1) | IL258959B2 (https=) |
| MX (2) | MX2018005490A (https=) |
| MY (1) | MY190926A (https=) |
| PE (2) | PE20231949A1 (https=) |
| PH (1) | PH12018501055A1 (https=) |
| RU (1) | RU2745506C2 (https=) |
| SA (1) | SA518391463B1 (https=) |
| SG (1) | SG11201802972UA (https=) |
| WO (1) | WO2017075619A1 (https=) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| MX390848B (es) | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| 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 |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| PE20200722A1 (es) * | 2017-08-01 | 2020-07-21 | Spark Therapeutics Inc | Metodos de terapia genica del factor viii (fviii) |
| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| KR101952102B1 (ko) * | 2017-12-07 | 2019-02-26 | 주식회사 지앤피바이오사이언스 | 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터 |
| TW202016314A (zh) | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| TW202016298A (zh) | 2018-05-14 | 2020-05-01 | 美商拜奧馬林製藥公司 | Aav 載體於幼年受試者中之穩定表現 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| EP3833766A1 (en) | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| 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 |
| BR112021003399A2 (pt) * | 2018-08-24 | 2021-05-18 | Spark Therapeutics, Inc. | sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso |
| CA3119349A1 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | In vitro assay for detecting enhancers and inhibitors of adeno associated virus (aav) vector transduction and/or detecting or quantitating anti-aav binding antibodies |
| GB201900702D0 (en) | 2019-01-18 | 2019-03-06 | Univ Bristol | Therapy |
| SG11202108357PA (en) | 2019-02-15 | 2021-08-30 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| SG11202109850SA (en) | 2019-03-13 | 2021-10-28 | Generation Bio Co | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| CN114929735A (zh) * | 2019-11-01 | 2022-08-19 | 自由行疗法有限公司 | 因子viii构建体 |
| JP2023510392A (ja) * | 2020-01-16 | 2023-03-13 | 武田薬品工業株式会社 | フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療 |
| GB202009039D0 (en) | 2020-06-15 | 2020-07-29 | Univ Bristol | Therapy |
| WO2023036054A2 (en) * | 2021-09-08 | 2023-03-16 | Inspirar Limited | Composition and method for treating hemophilia |
| KR20250007615A (ko) * | 2022-04-28 | 2025-01-14 | 조인트 스탁 컴퍼니 "바이오케드" | Fviii-bdd를 인코딩하는 코돈 최적화된 핵산 |
| JP2025529195A (ja) * | 2022-08-31 | 2025-09-04 | ハイドロジーン セラピューティクス，インコーポレイテッド | 流体力学的遺伝子送達のための方法および組成物 |
| 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 |
| WO2025164792A1 (ja) * | 2024-02-02 | 2025-08-07 | 学校法人自治医科大学 | 高比活性／高分泌発現型の第ｖｉｉｉ因子改変体、高発現型の第ｖｉｉｉ因子コード核酸及びそれらの用途 |
| WO2025180508A1 (en) * | 2024-02-29 | 2025-09-04 | Shanghai Vitalgen Biopharma Co., Ltd. | Human factor viii variant expression cassettes and uses thereof for treating hemophilia a |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6093699A (en) | 1987-07-09 | 2000-07-25 | The University Of Manitoba | Method for gene therapy involving suppression of an immune response |
| WO1994017810A1 (en) | 1993-02-12 | 1994-08-18 | The Wistar Institute Of Anatomy And Biology | Recombinant cytomegalovirus vaccine |
| EP0708658A4 (en) | 1993-04-16 | 1997-05-21 | Wistar Inst | RECOMBINANT CYTOMEGALOVIRUS VACCINE |
| WO1995005864A1 (en) | 1993-08-27 | 1995-03-02 | Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services | Convection-enhanced drug delivery |
| AT401270B (de) | 1994-09-26 | 1996-07-25 | Immuno Ag | Verfahren zur quantifizierung von genomischer dna |
| US5998205A (en) | 1994-11-28 | 1999-12-07 | Genetic Therapy, Inc. | Vectors for tissue-specific replication |
| US6818439B1 (en) | 1994-12-30 | 2004-11-16 | Chiron Corporation | Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders |
| EA001616B1 (ru) | 1995-02-28 | 2001-06-25 | Зе Риджентс Оф Зи Юнивесити Оф Кэлифоньэ | Способ лечения болезни сердца, способ лечения недостаточности периферических сосудов и способ ограничения доставки и экспрессии трансгенной конструкции в определенном органе или структуре |
| AU2319497A (en) | 1996-03-07 | 1997-09-22 | Regents Of The University Of California, The | Helper-free, totally defective adenovirus for gene therapy |
| US6458563B1 (en) * | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US20020065236A1 (en) | 1998-09-09 | 2002-05-30 | Yew Nelson S. | CpG reduced plasmids and viral vectors |
| US20060099685A1 (en) | 1999-04-15 | 2006-05-11 | Yallop Christopher A | Recombinant expression of factor VIII in human cells |
| EP1240900B1 (en) * | 1999-12-24 | 2009-05-06 | Juridical Foundation, The Chemo-Sero-Therapeutic Research Institute | Factor viii or von willebrand factor for the treatment of hemorrhagic diseases associating thrombopathy |
| PL206105B1 (pl) * | 2000-09-19 | 2010-07-30 | Emory Universityemory University | Zmodyfikowany ludzki czynnik VIII oraz sposób modyfikacji ludzkiego czynnika VIII ze zmniejszeniem reaktywności na przeciwciało hamujące i zachowaniem aktywności prokoagulacyjnej |
| DE102004037611B4 (de) * | 2004-08-03 | 2013-10-02 | Geneart Ag | Induzierbare Genexpression |
| GB0908515D0 (en) | 2009-05-18 | 2009-06-24 | Apitope Technology Bristol Ltd | Peptide |
| GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| PT3513804T (pt) | 2011-07-08 | 2022-06-02 | Bioverativ Therapeutics Inc | Polipéptidos fator viii híbridos e quiméricos, e métodos de utilização dos mesmos |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| EP2822577B1 (en) * | 2012-02-15 | 2019-02-06 | Bioverativ Therapeutics Inc. | Recombinant factor viii proteins |
| NZ701693A (en) | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| US20140271550A1 (en) | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| EP3044231B1 (en) | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| WO2015054439A2 (en) * | 2013-10-08 | 2015-04-16 | Haplomics, Inc. | Hybrid factor viii polypeptides for use to treat hemophilia a |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| CN107427557B (zh) * | 2014-08-13 | 2022-01-04 | 费城儿童医院 | 用于包装和表达变体因子viii以治疗血友病的改良表达组件 |
| MY189674A (en) | 2015-10-28 | 2022-02-24 | Sangamo Therapeutics Inc | Liver-specific constructs, factor viii expression cassettes 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 |
| PE20200722A1 (es) | 2017-08-01 | 2020-07-21 | Spark Therapeutics Inc | Metodos de terapia genica del factor viii (fviii) |
-
2016
- 2016-10-30 PE PE2023001716A patent/PE20231949A1/es unknown
- 2016-10-31 EP EP16861065.7A patent/EP3368560B1/en active Active
- 2016-10-31 PE PE2018000713A patent/PE20181168A1/es unknown
- 2016-10-31 MX MX2018005490A patent/MX2018005490A/es unknown
- 2016-10-31 MY MYPI2018000612A patent/MY190926A/en unknown
- 2016-10-31 BR BR112018008766-1A patent/BR112018008766A2/en active IP Right Grant
- 2016-10-31 IL IL258959A patent/IL258959B2/en unknown
- 2016-10-31 RU RU2018119710A patent/RU2745506C2/ru active
- 2016-10-31 KR KR1020187015213A patent/KR20180090795A/ko active Pending
- 2016-10-31 AU AU2016344030A patent/AU2016344030B2/en active Active
- 2016-10-31 JP JP2018521281A patent/JP7088831B2/ja active Active
- 2016-10-31 WO PCT/US2016/059793 patent/WO2017075619A1/en not_active Ceased
- 2016-10-31 SG SG11201802972UA patent/SG11201802972UA/en unknown
- 2016-10-31 US US15/772,025 patent/US20180312571A1/en not_active Abandoned
- 2016-10-31 CN CN201680063183.9A patent/CN108368164A/zh active Pending
- 2016-10-31 CA CA3002689A patent/CA3002689A1/en active Pending
-
2017
- 2017-03-17 US US15/462,660 patent/US11168124B2/en active Active
-
2018
- 2018-04-26 CL CL2018001107A patent/CL2018001107A1/es unknown
- 2018-04-29 SA SA518391463A patent/SA518391463B1/ar unknown
- 2018-04-30 PH PH12018501055A patent/PH12018501055A1/en unknown
- 2018-04-30 MX MX2023001600A patent/MX2023001600A/es unknown
- 2018-05-21 CO CONC2018/0005306A patent/CO2018005306A2/es unknown
-
2022
- 2022-06-09 JP JP2022093877A patent/JP2022137029A/ja active Pending
-
2024
- 2024-08-08 JP JP2024131729A patent/JP2024170416A/ja active Pending
- 2024-11-26 US US18/960,905 patent/US20250171521A1/en active Pending
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