CN108602876B - 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体 - Google Patents

用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体 Download PDF

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CN108602876B
CN108602876B CN201680067847.9A CN201680067847A CN108602876B CN 108602876 B CN108602876 B CN 108602876B CN 201680067847 A CN201680067847 A CN 201680067847A CN 108602876 B CN108602876 B CN 108602876B
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factor viii
nucleotide sequence
sequence
identity
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CN108602876A (zh
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F-G·法尔科纳
F·霍林
J·伦勒
H·罗腾施泰纳
F·沙伊夫灵格
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Takeda Pharmaceutical Co Ltd
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201680067847.9A 2015-11-13 2016-11-11 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体 Active CN108602876B (zh)

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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

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CN108602876B true CN108602876B (zh) 2022-07-05

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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
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc 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
EP3986481A2 (en) * 2019-06-20 2022-04-27 Takeda Pharmaceutical Company Limited 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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CN1361178A (zh) * 2000-12-29 2002-07-31 中国科学院上海生物化学研究所 新的重组凝血因子ⅷ及其生产方法和药物组合物
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MX2018005969A (es) 2018-11-29
KR20180070700A (ko) 2018-06-26
JP6695426B2 (ja) 2020-05-20
TW202229555A (zh) 2022-08-01
AU2016354550A1 (en) 2018-06-07
EA201891138A1 (ru) 2018-12-28
CA3005565A1 (en) 2017-05-18
IL259295A (en) 2018-07-31
IL259295B1 (en) 2024-06-01
TWI823830B (zh) 2023-12-01
CL2018001301A1 (es) 2018-12-28
EA036944B1 (ru) 2021-01-19
BR112018009732A8 (pt) 2019-02-26
ZA201803442B (en) 2019-07-31
SG11201804064WA (en) 2018-06-28
US10189889B2 (en) 2019-01-29
TW201723180A (zh) 2017-07-01
JP2018537089A (ja) 2018-12-20
MY205187A (en) 2024-10-04
CN108602876A (zh) 2018-09-28
NZ742555A (en) 2023-12-22
WO2017083764A1 (en) 2017-05-18
CL2020001611A1 (es) 2020-10-30
KR102404550B1 (ko) 2022-05-31
EP3374388A1 (en) 2018-09-19
TWI850587B (zh) 2024-08-01
AU2016354550B2 (en) 2020-04-16
IL259295B2 (en) 2024-10-01
US20170226188A1 (en) 2017-08-10
BR112018009732A2 (pt) 2018-11-21
EA202092049A1 (ru) 2020-11-10
CO2018005380A2 (es) 2018-05-31

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