JP2018537087A5 - - Google Patents
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- JP2018537087A5 JP2018537087A5 JP2018524747A JP2018524747A JP2018537087A5 JP 2018537087 A5 JP2018537087 A5 JP 2018537087A5 JP 2018524747 A JP2018524747 A JP 2018524747A JP 2018524747 A JP2018524747 A JP 2018524747A JP 2018537087 A5 JP2018537087 A5 JP 2018537087A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide
- seq
- nucleotide sequence
- factor viii
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562255317P | 2015-11-13 | 2015-11-13 | |
| US62/255,317 | 2015-11-13 | ||
| PCT/US2016/061684 WO2017083762A1 (en) | 2015-11-13 | 2016-11-11 | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020132079A Division JP7069258B2 (ja) | 2015-11-13 | 2020-08-04 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018537087A JP2018537087A (ja) | 2018-12-20 |
| JP2018537087A5 true JP2018537087A5 (https=) | 2019-12-12 |
| JP6768798B2 JP6768798B2 (ja) | 2020-10-14 |
Family
ID=57539605
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018524747A Active JP6768798B2 (ja) | 2015-11-13 | 2016-11-11 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2020132079A Active JP7069258B2 (ja) | 2015-11-13 | 2020-08-04 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2022075864A Active JP7307836B2 (ja) | 2015-11-13 | 2022-05-02 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2023108132A Active JP7631425B2 (ja) | 2015-11-13 | 2023-06-30 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020132079A Active JP7069258B2 (ja) | 2015-11-13 | 2020-08-04 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2022075864A Active JP7307836B2 (ja) | 2015-11-13 | 2022-05-02 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2023108132A Active JP7631425B2 (ja) | 2015-11-13 | 2023-06-30 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
Country Status (18)
| Country | Link |
|---|---|
| US (4) | US10189888B2 (https=) |
| EP (1) | EP3374387A1 (https=) |
| JP (4) | JP6768798B2 (https=) |
| KR (2) | KR102484553B1 (https=) |
| CN (3) | CN116949052A (https=) |
| AU (4) | AU2016353353B2 (https=) |
| BR (1) | BR112018009717B1 (https=) |
| CA (1) | CA3005334A1 (https=) |
| CL (1) | CL2018001299A1 (https=) |
| CO (1) | CO2018005377A2 (https=) |
| EA (2) | EA038288B1 (https=) |
| IL (3) | IL281038B2 (https=) |
| MX (1) | MX2018005982A (https=) |
| MY (2) | MY198589A (https=) |
| SG (2) | SG10202106307UA (https=) |
| TW (3) | TWI777175B (https=) |
| WO (1) | WO2017083762A1 (https=) |
| ZA (3) | ZA201803443B (https=) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EA202092049A1 (ru) * | 2015-11-13 | 2020-11-10 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| EP3442597A1 (en) | 2016-04-15 | 2019-02-20 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia b |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| PT3534922T (pt) | 2016-11-04 | 2024-06-07 | Takeda Pharmaceuticals Co | Formulações de vírus adenoassociado |
| WO2019067766A1 (en) * | 2017-09-27 | 2019-04-04 | Sigilon Therapeutics, Inc. | METHODS, COMPOSITIONS AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS |
| AU2018345772B2 (en) * | 2017-10-02 | 2025-04-17 | Research Institute At Nationwide Children's Hospital | MiRNA detargeting system for tissue specific interference |
| KR102105145B1 (ko) * | 2017-10-13 | 2020-05-13 | 영남대학교 산학협력단 | 촬영 장치 및 용기 |
| KR101952102B1 (ko) * | 2017-12-07 | 2019-02-26 | 주식회사 지앤피바이오사이언스 | 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터 |
| EP3823985A1 (en) * | 2018-07-16 | 2021-05-26 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| WO2020028830A1 (en) * | 2018-08-03 | 2020-02-06 | Sangamo Therapeutics, Inc. | Improved clinical parameters by expression of factor viii |
| 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 |
| 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 |
| US11529427B2 (en) | 2019-02-15 | 2022-12-20 | Crispr Therapeutics Ag | Gene editing for hemophilia A with improved factor VIII expression |
| BR112021015776A2 (pt) * | 2019-06-05 | 2021-11-09 | Bayer Healthcare Llc | Edição de gene para hemofilia a com expressão de fator viii melhorada |
| US20200405883A1 (en) * | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
| EP4051704A2 (en) * | 2019-11-01 | 2022-09-07 | Freeline Therapeutics Limited | Factor viii construct |
| EP4073106A2 (en) * | 2019-12-12 | 2022-10-19 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| KR102817222B1 (ko) * | 2021-04-02 | 2025-06-11 | 연세대학교 산학협력단 | Fe-fviii 변이 유전자로 교정된 줄기세포, 이로부터 분화된 내피 세포 및 이를 포함하는 혈우병 예방 또는 치료용 약제학적 조성물 |
| KR102421131B1 (ko) * | 2021-05-28 | 2022-07-15 | 알엔에이진(주) | 세포내에서 장시간동안 존재할 수 있는 외부 도입 mRNA를 선별하는 방법 |
| EP4211153A4 (en) * | 2021-11-25 | 2023-11-01 | Sichuan Real&Best Biotech Co., Ltd. | ENGINEERED HUMAN FVII1 WITH INCREASED SECRETION ABILITY AND COAGULATION ACTIVITY |
| IL316584A (en) * | 2022-04-28 | 2024-12-01 | Biocad Joint Stock Co | Codon-optimized nucleic acid encoding FVIII-BDD |
| 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 |
| TW202435846A (zh) | 2023-03-14 | 2024-09-16 | 美商希吉隆醫療公司 | 包含共價交聯多醣及胰島細胞之水膠膠囊 |
| TW202517669A (zh) * | 2023-07-05 | 2025-05-01 | 日商武田藥品工業股份有限公司 | 用於Ａ型血友病之基因療法的編碼表現增加之重組ＦＶｌｌｌ變異體的病毒載體 |
| 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 (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| ATE72838T1 (de) | 1985-04-12 | 1992-03-15 | Genetics Inst | Neue prokoagulierungsproteine. |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
| US5543502A (en) | 1986-06-24 | 1996-08-06 | Novo Nordisk A/S | Process for producing a coagulation active complex of factor VIII fragments |
| US6060447A (en) | 1987-05-19 | 2000-05-09 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
| US6346513B1 (en) | 1987-06-12 | 2002-02-12 | Baxter Trading Gmbh | Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them |
| IL86693A (en) | 1987-06-12 | 1994-06-24 | Stichting Centraal Lab | Proteins with factor VIII activity, process for their preparation using genetically engineered cells and pharmaceutical compositions containing them |
| FR2619314B1 (fr) | 1987-08-11 | 1990-06-15 | Transgene Sa | Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant |
| CA2162497A1 (en) | 1993-06-10 | 1994-12-22 | Sheila Connelly | Adenoviral vectors for treatment of hemophilia |
| SE504074C2 (sv) | 1993-07-05 | 1996-11-04 | Pharmacia Ab | Proteinberedning för subkutan, intramuskulär eller intradermal administrering |
| 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 |
| US5952198A (en) * | 1995-05-04 | 1999-09-14 | Bayer Corporation | Production of recombinant Factor VIII in the presence of liposome-like substances of mixed composition |
| SE9503380D0 (sv) | 1995-09-29 | 1995-09-29 | Pharmacia Ab | Protein derivatives |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| US6458563B1 (en) | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
| US6114148C1 (en) | 1996-09-20 | 2012-05-01 | Gen Hospital Corp | High level expression of proteins |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6221349B1 (en) * | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US20060099685A1 (en) | 1999-04-15 | 2006-05-11 | Yallop Christopher A | Recombinant expression of factor VIII in human cells |
| CN1179976C (zh) * | 2000-12-29 | 2004-12-15 | 中国科学院上海生物化学研究所 | 产生凝血因子ⅷ的生产方法和宿主细胞 |
| WO2003031598A2 (en) | 2001-10-05 | 2003-04-17 | Emory University | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US7041635B2 (en) | 2003-01-28 | 2006-05-09 | In2Gen Co., Ltd. | Factor VIII polypeptide |
| WO2005017149A1 (en) * | 2003-06-03 | 2005-02-24 | Cell Genesys, Inc. | Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site |
| US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| EP2848253A1 (en) | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor VIII and factor IX genes and vectors for gene therapy |
| AU2007338298B2 (en) | 2006-12-22 | 2013-02-07 | Csl Behring Gmbh | Modified coagulation factors with prolonged in vivo half-life |
| KR100959454B1 (ko) | 2007-12-10 | 2010-05-25 | 주식회사 동부하이텍 | 반도체 소자 및 그 제조 방법 |
| EP4154903A1 (en) | 2008-04-22 | 2023-03-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| JP5770161B2 (ja) | 2009-04-06 | 2015-08-26 | ノヴォ ノルディスク アー／エス | 血小板への第ｖｉｉｉ因子タンパク質の標的送達 |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| SG10201505217WA (en) | 2010-07-09 | 2015-08-28 | Biogen Hemophilia Inc | Chimeric clotting factors |
| US20130017997A1 (en) * | 2010-08-19 | 2013-01-17 | Amunix Operating Inc. | Factor VIII Compositions and Methods of Making and Using Same |
| WO2013016454A1 (en) | 2011-07-25 | 2013-01-31 | Biogen Idec Hemophilia Inc. | Assays to monitor bleeding disorders |
| EP3970737A1 (en) * | 2012-01-12 | 2022-03-23 | Bioverativ Therapeutics Inc. | Reducing immunogenicity against factor viii in individuals undergoing factor viii therapy |
| DK2804623T3 (da) | 2012-01-12 | 2019-11-11 | Bioverativ Therapeutics Inc | Kimære faktor viii-polypeptider og anvendelser deraf |
| BR112014020694A2 (pt) | 2012-02-15 | 2018-05-08 | Amunix Operating Inc. | proteína de fusão do fator viii compreendendo polipeptídeo do fator viii fusionado a polipeptí-deo recombinante estendido (xten) e seu método de fabricação, ácido nucleico, vetores, célula hospedeira, bem como composição farmacêutica e seu uso no tratamento de coagulopatia, episódio de hemorragia e hemofilia a |
| AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| CA2868391A1 (en) * | 2012-04-02 | 2013-10-10 | Stephane Bancel | Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same |
| GB201210357D0 (en) * | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| WO2014008172A2 (en) | 2012-07-03 | 2014-01-09 | Expression Therapeutics, Llc | High yield suspension cell line, system and method for making same |
| EP3513802B1 (en) | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| SI3889173T1 (sl) | 2013-02-15 | 2023-11-30 | Bioverativ Therapeutics Inc. | Optimiran gen dejavnika VIII |
| US20160030524A1 (en) | 2013-03-15 | 2016-02-04 | Bayer Healthcare Llc | Recombinant factor viii formulations |
| EP3904376B1 (en) | 2013-06-24 | 2024-11-13 | Xiao, Weidong | Mutant factor viii compositions and methods |
| RS61039B1 (sr) * | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| CA3237630A1 (en) | 2014-08-13 | 2016-02-18 | The Children's Hospital Of Philadelphia | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders |
| 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 |
| EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
| 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 |
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2016
- 2016-11-11 EP EP16809573.5A patent/EP3374387A1/en active Pending
- 2016-11-11 MY MYPI2020005892A patent/MY198589A/en unknown
- 2016-11-11 TW TW109118505A patent/TWI777175B/zh active
- 2016-11-11 CN CN202310964403.7A patent/CN116949052A/zh active Pending
- 2016-11-11 KR KR1020187015189A patent/KR102484553B1/ko active Active
- 2016-11-11 CA CA3005334A patent/CA3005334A1/en active Pending
- 2016-11-11 KR KR1020227046419A patent/KR20230008256A/ko not_active Ceased
- 2016-11-11 MY MYPI2018000735A patent/MY190067A/en unknown
- 2016-11-11 IL IL281038A patent/IL281038B2/en unknown
- 2016-11-11 MX MX2018005982A patent/MX2018005982A/es unknown
- 2016-11-11 CN CN202310490697.4A patent/CN116479001A/zh active Pending
- 2016-11-11 BR BR112018009717-9A patent/BR112018009717B1/pt active IP Right Grant
- 2016-11-11 SG SG10202106307UA patent/SG10202106307UA/en unknown
- 2016-11-11 AU AU2016353353A patent/AU2016353353B2/en active Active
- 2016-11-11 TW TW111137316A patent/TW202302627A/zh unknown
- 2016-11-11 WO PCT/US2016/061684 patent/WO2017083762A1/en not_active Ceased
- 2016-11-11 TW TW105136965A patent/TWI695069B/zh active
- 2016-11-11 JP JP2018524747A patent/JP6768798B2/ja active Active
- 2016-11-11 EA EA201891137A patent/EA038288B1/ru unknown
- 2016-11-11 IL IL305526A patent/IL305526A/en unknown
- 2016-11-11 SG SG11201804070XA patent/SG11201804070XA/en unknown
- 2016-11-11 EA EA202190827A patent/EA202190827A1/ru unknown
- 2016-11-11 US US15/349,930 patent/US10189888B2/en active Active
- 2016-11-11 CN CN201680067846.4A patent/CN108884145B/zh active Active
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2018
- 2018-05-13 IL IL259302A patent/IL259302B/en active IP Right Grant
- 2018-05-14 CL CL2018001299A patent/CL2018001299A1/es unknown
- 2018-05-23 CO CONC2018/0005377A patent/CO2018005377A2/es unknown
- 2018-05-24 ZA ZA2018/03443A patent/ZA201803443B/en unknown
- 2018-12-05 US US16/211,201 patent/US11492388B2/en active Active
- 2018-12-05 US US16/211,202 patent/US11254731B2/en not_active Expired - Fee Related
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2019
- 2019-07-23 ZA ZA2019/04814A patent/ZA201904814B/en unknown
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2020
- 2020-06-05 AU AU2020203761A patent/AU2020203761B2/en active Active
- 2020-06-05 AU AU2020203760A patent/AU2020203760B2/en active Active
- 2020-08-04 JP JP2020132079A patent/JP7069258B2/ja active Active
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2022
- 2022-01-06 US US17/570,187 patent/US20220213172A1/en not_active Abandoned
- 2022-02-25 AU AU2022201319A patent/AU2022201319B2/en active Active
- 2022-05-02 JP JP2022075864A patent/JP7307836B2/ja active Active
- 2022-12-19 ZA ZA2022/13661A patent/ZA202213661B/en unknown
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2023
- 2023-06-30 JP JP2023108132A patent/JP7631425B2/ja active Active