JP2018537089A5 - - Google Patents

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Publication number
JP2018537089A5
JP2018537089A5 JP2018524806A JP2018524806A JP2018537089A5 JP 2018537089 A5 JP2018537089 A5 JP 2018537089A5 JP 2018524806 A JP2018524806 A JP 2018524806A JP 2018524806 A JP2018524806 A JP 2018524806A JP 2018537089 A5 JP2018537089 A5 JP 2018537089A5
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JP
Japan
Prior art keywords
polynucleotide
nucleotide sequence
seq
factor viii
aav
Prior art date
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JP2018524806A
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English (en)
Japanese (ja)
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JP6695426B2 (ja
JP2018537089A (ja
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Priority claimed from PCT/US2016/061688 external-priority patent/WO2017083764A1/en
Publication of JP2018537089A publication Critical patent/JP2018537089A/ja
Publication of JP2018537089A5 publication Critical patent/JP2018537089A5/ja
Application granted granted Critical
Publication of JP6695426B2 publication Critical patent/JP6695426B2/ja
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JP2018524806A 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 Active JP6695426B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562255323P 2015-11-13 2015-11-13
US62/255,323 2015-11-13
PCT/US2016/061688 WO2017083764A1 (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Publications (3)

Publication Number Publication Date
JP2018537089A JP2018537089A (ja) 2018-12-20
JP2018537089A5 true JP2018537089A5 (enExample) 2019-12-19
JP6695426B2 JP6695426B2 (ja) 2020-05-20

Family

ID=57539606

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018524806A Active JP6695426B2 (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

Country Status (18)

Country Link
US (1) US10189889B2 (enExample)
EP (1) EP3374388A1 (enExample)
JP (1) JP6695426B2 (enExample)
KR (1) KR102404550B1 (enExample)
CN (1) CN108602876B (enExample)
AU (1) AU2016354550B2 (enExample)
BR (1) BR112018009732A8 (enExample)
CA (1) CA3005565A1 (enExample)
CL (2) CL2018001301A1 (enExample)
CO (1) CO2018005380A2 (enExample)
EA (2) EA202092049A1 (enExample)
IL (1) IL259295B2 (enExample)
MX (1) MX2018005969A (enExample)
MY (1) MY205187A (enExample)
SG (1) SG11201804064WA (enExample)
TW (2) TWI823830B (enExample)
WO (1) WO2017083764A1 (enExample)
ZA (1) ZA201803442B (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3374388A1 (en) * 2015-11-13 2018-09-19 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
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
WO2020168362A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
CN109735547B (zh) * 2019-03-19 2022-05-31 青岛蔚蓝生物集团有限公司 一种提高毕赤酵母外源蛋白表达量的启动子及其应用
US20200405883A1 (en) * 2019-06-20 2020-12-31 Baxalta Incorporated Method of treatment with viral-based gene therapy
CN112575034B (zh) * 2019-09-29 2023-04-25 济南赛尔生物科技股份有限公司 一种治疗a型血友病的产品及应用

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JPH0788399B2 (ja) 1985-04-12 1995-09-27 ジェネティックス・インスチチュ−ト・インコ−ポレ−テッド 新規プロコアギュラント蛋白質
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US5171844A (en) 1987-06-12 1992-12-15 Gist-Brocades N.W. 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
ATE322547T1 (de) 1993-06-10 2006-04-15 Genetic Therapy Inc Adenovirale vektoren für die behandlung der hämophilie
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
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CA2461443C (en) 2001-10-05 2011-07-12 Emory University Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
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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
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GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
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BR112014017111A8 (pt) * 2012-01-12 2018-05-15 Biogen Idec Inc métodos de redução de imunogenicidade contra o fator viii em indivíduos submetidos à terapia de fator viii
CN111548418A (zh) * 2012-02-15 2020-08-18 比奥贝拉蒂治疗公司 因子viii组合物及其制备和使用方法
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SG11201804070XA (en) * 2015-11-13 2018-06-28 Baxalta Inc Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EP3374388A1 (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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