JP7217630B2 - 最適化第ｖｉｉｉ因子遺伝子 - Google Patents

最適化第ｖｉｉｉ因子遺伝子 Download PDF

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JP7217630B2
JP7217630B2 JP2018539962A JP2018539962A JP7217630B2 JP 7217630 B2 JP7217630 B2 JP 7217630B2 JP 2018539962 A JP2018539962 A JP 2018539962A JP 2018539962 A JP2018539962 A JP 2018539962A JP 7217630 B2 JP7217630 B2 JP 7217630B2
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seq
nucleic acid
fviii
nucleotides
nucleotide sequence
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JP2019506166A (ja
JP2019506166A5 (OSRAM
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シユアン タン，
トンヤオ リウ，
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バイオベラティブ セラピューティクス インコーポレイテッド
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K48/0008—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
    • A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2018539962A 2016-02-01 2017-01-31 最適化第ｖｉｉｉ因子遺伝子 Active JP7217630B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022068361A JP2022089920A (ja) 2016-02-01 2022-04-18 最適化第ｖｉｉｉ因子遺伝子
JP2024102527A JP2024114805A (ja) 2016-02-01 2024-06-26 最適化第ｖｉｉｉ因子遺伝子

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US201662289696P 2016-02-01 2016-02-01
US62/289,696 2016-02-01
US201662409739P 2016-10-18 2016-10-18
US62/409,739 2016-10-18
PCT/US2017/015879 WO2017136358A1 (en) 2016-02-01 2017-01-31 Optimized factor viii genes

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JP2022068361A Division JP2022089920A (ja) 2016-02-01 2022-04-18 最適化第ｖｉｉｉ因子遺伝子

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JP2019506166A JP2019506166A (ja) 2019-03-07
JP2019506166A5 JP2019506166A5 (OSRAM) 2020-01-30
JP7217630B2 true JP7217630B2 (ja) 2023-02-03

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JP2018539962A Active JP7217630B2 (ja) 2016-02-01 2017-01-31 最適化第ｖｉｉｉ因子遺伝子
JP2022068361A Pending JP2022089920A (ja) 2016-02-01 2022-04-18 最適化第ｖｉｉｉ因子遺伝子
JP2024102527A Pending JP2024114805A (ja) 2016-02-01 2024-06-26 最適化第ｖｉｉｉ因子遺伝子

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JP2024102527A Pending JP2024114805A (ja) 2016-02-01 2024-06-26 最適化第ｖｉｉｉ因子遺伝子

Country Status (24)

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US (2) US11753461B2 (OSRAM)
EP (2) EP3411478B1 (OSRAM)
JP (3) JP7217630B2 (OSRAM)
KR (1) KR20180118659A (OSRAM)
CN (2) CN116218863A (OSRAM)
AU (2) AU2017214378B2 (OSRAM)
CA (1) CA3012695A1 (OSRAM)
CL (1) CL2018002054A1 (OSRAM)
CO (1) CO2018009120A2 (OSRAM)
DK (1) DK3411478T3 (OSRAM)
ES (1) ES2926585T3 (OSRAM)
HR (1) HRP20221089T1 (OSRAM)
HU (1) HUE059941T2 (OSRAM)
IL (2) IL320404A (OSRAM)
LT (1) LT3411478T (OSRAM)
MX (2) MX2018009375A (OSRAM)
NZ (1) NZ745831A (OSRAM)
PH (1) PH12018501628B1 (OSRAM)
PL (1) PL3411478T3 (OSRAM)
PT (1) PT3411478T (OSRAM)
RS (1) RS63548B1 (OSRAM)
SG (2) SG11201806398YA (OSRAM)
SI (1) SI3411478T1 (OSRAM)
WO (1) WO2017136358A1 (OSRAM)

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DK3889173T5 (da) 2013-02-15 2024-08-05 Bioverativ Therapeutics Inc Optimeret faktor viii-gen
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
EP3411478B1 (en) 2016-02-01 2022-06-08 Bioverativ Therapeutics Inc. Optimized factor viii genes
CN111247251B (zh) 2017-08-09 2025-06-27 比奥维拉迪维治疗股份有限公司 核酸分子及其用途
JP2021512126A (ja) * 2018-02-01 2021-05-13 バイオベラティブ セラピューティクス インコーポレイテッド 第ｖｉｉｉ因子を発現するレンチウイルスベクターの使用
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CN113396223A (zh) * 2018-12-06 2021-09-14 比奥维拉迪维治疗股份有限公司 表达因子ix的慢病毒载体的用途
US10654911B1 (en) * 2019-04-02 2020-05-19 Beijing Neoletix Biological Technology Co., Ltd. Vector co-expressing truncated von Willebrand factor and factor VIII
CN114008069A (zh) 2019-04-17 2022-02-01 科迪亚克生物科学公司 外来体和aav的组合物
CN115279896A (zh) * 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
CN111647625A (zh) * 2019-12-25 2020-09-11 深圳三智医学科技有限公司 一种提高人凝血因子ix表达水平的方法
CN111808863B (zh) * 2020-06-23 2021-05-28 康霖生物科技（杭州）有限公司 一种密码子优化的凝血因子viii基因及其构建体
KR20230074703A (ko) 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법
EP4392444A1 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
AU2022332276A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
AU2022334711A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Baculovirus expression system
IL311725A (en) * 2021-09-30 2024-05-01 Bioverativ Therapeutics Inc Nucleic acids encoding factor VIII polypeptides with reduced immunogenicity
EP4511055A2 (en) * 2022-04-20 2025-02-26 AskBio Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
JP2025515356A (ja) * 2022-04-28 2025-05-14 ジョイント・ストック・カンパニー “バイオキャド” Ｆｖｉｉｉ－ｂｄｄおよび異種シグナルペプチドに基づく融合タンパク質をコードする単離された核酸
WO2025219525A1 (en) 2024-04-19 2025-10-23 Fondazione Telethon Combinations, inhibitors, viral vectors, methods, and uses for in vivo gene therapy and gene editing

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