CN108884145B - 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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Publication number
CN108884145B
CN108884145B CN201680067846.4A CN201680067846A CN108884145B CN 108884145 B CN108884145 B CN 108884145B CN 201680067846 A CN201680067846 A CN 201680067846A CN 108884145 B CN108884145 B CN 108884145B
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factor viii
seq
sequence
amino acid
nucleotide sequence
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CN108884145A (zh
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J·伦勒
F·法尔科纳
F·霍林
H·罗滕施泰纳
F·沙伊夫灵格
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Takeda Pharmaceutical Co Ltd
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Baxalta GmbH
Takeda Pharmaceutical Co Ltd
Baishen Co
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
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    • C12N15/09—Recombinant DNA-technology
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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
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    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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CN201680067846.4A 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 CN108884145B (zh)

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Application Number Priority Date Filing Date Title
CN202310490697.4A CN116479001A (zh) 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
CN202310964403.7A CN116949052A (zh) 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

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

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CN202310964403.7A Division CN116949052A (zh) 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
CN202310490697.4A Division CN116479001A (zh) 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

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CN108884145B true CN108884145B (zh) 2023-08-22

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CN202310964403.7A Pending CN116949052A (zh) 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
CN202310490697.4A Pending CN116479001A (zh) 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

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CN202310490697.4A Pending CN116479001A (zh) 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

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US (4) US10189888B2 (enExample)
EP (1) EP3374387A1 (enExample)
JP (4) JP6768798B2 (enExample)
KR (2) KR20230008256A (enExample)
CN (3) CN108884145B (enExample)
AU (4) AU2016353353B2 (enExample)
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CA (1) CA3005334A1 (enExample)
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US10143760B2 (en) 2015-10-28 2018-12-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
CN108884145B (zh) 2015-11-13 2023-08-22 武田药品工业株式会社 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
KR102404550B1 (ko) * 2015-11-13 2022-05-31 다케다 야쿠힌 고교 가부시키가이샤 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터
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US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
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KR20200057051A (ko) * 2017-09-27 2020-05-25 시질론 테라퓨틱스, 인크. 활성 세포를 포함하는 방법, 조성물 및 이식 가능한 요소
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CN114981299A (zh) * 2019-12-12 2022-08-30 武田药品工业株式会社 使用编码具有增加的表达的重组fviii变体的病毒载体的a型血友病的基因疗法
WO2022211604A1 (ko) * 2021-04-02 2022-10-06 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
CN118638211A (zh) * 2021-11-25 2024-09-13 四川至善唯新生物科技有限公司 基因工程改造获得的分泌能力和凝血活性增强的人类八因子
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