JP2025515356A - 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 - Google Patents

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 Download PDF

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JP2025515356A
JP2025515356A JP2024563591A JP2024563591A JP2025515356A JP 2025515356 A JP2025515356 A JP 2025515356A JP 2024563591 A JP2024563591 A JP 2024563591A JP 2024563591 A JP2024563591 A JP 2024563591A JP 2025515356 A JP2025515356 A JP 2025515356A
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fviii
bdd
aav
seq
nucleic acid
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JP2024563591A
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JP2025515356A5 (https=
Inventor
ペレペルキナ，マリヤ・パブロブナ
ブラソバ，エレナ・ベニアモノブナ
フォミナ，アナスタシーア・ブラディミロブナ
ゲルショビッチ，パベル・ミクハリオビッチ
マルコバ，ビタリーア・アレクサンドロブナ
モロゾフ，ドミトリー・バレンチノビッチ
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ジョイント・ストック・カンパニー “バイオキャド”
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Priority claimed from RU2022111695A external-priority patent/RU2818229C2/ru
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Publication of JP2025515356A publication Critical patent/JP2025515356A/ja
Publication of JP2025515356A5 publication Critical patent/JP2025515356A5/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • 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
    • 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
    • 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
    • C07K14/76—Albumins
    • C07K14/765—Serum albumin, e.g. HSA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2024563591A 2022-04-28 2023-04-18 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 Pending JP2025515356A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
RU2022111695 2022-04-28
RU2022111695A RU2818229C2 (ru) 2022-04-28 Выделенная нуклеиновая кислота, которая кодирует слитый белок на основе FVIII-BDD и гетерологичного сигнального пептида, и ее применение
PCT/RU2023/050093 WO2023211315A1 (en) 2022-04-28 2023-04-18 Isolated nucleic acid that encodes fusion protein based on fviii-bdd and on heterologous signal peptide

Publications (2)

Publication Number Publication Date
JP2025515356A true JP2025515356A (ja) 2025-05-14
JP2025515356A5 JP2025515356A5 (https=) 2026-04-28

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JP2024563591A Pending JP2025515356A (ja) 2022-04-28 2023-04-18 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

Country Status (19)

Country Link
EP (1) EP4514975A1 (https=)
JP (1) JP2025515356A (https=)
KR (1) KR20250005425A (https=)
CN (1) CN117402901A (https=)
AR (1) AR129164A1 (https=)
AU (1) AU2023261711A1 (https=)
CA (1) CA3250663A1 (https=)
CL (1) CL2024003295A1 (https=)
CO (1) CO2024014703A2 (https=)
CR (1) CR20240525A (https=)
IL (1) IL316578A (https=)
JO (1) JOP20240240A1 (https=)
MA (1) MA68435A1 (https=)
MX (1) MX2024013281A (https=)
PE (1) PE20251064A1 (https=)
PY (1) PY2331055A (https=)
TW (1) TW202342751A (https=)
UY (1) UY40243A (https=)
WO (1) WO2023211315A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120365434A (zh) * 2024-01-23 2025-07-25 上海苹谱医疗科技有限公司 一种提高凝血因子ⅷ分泌水平的双信号肽及其应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3326643T3 (pl) * 2009-12-06 2021-10-25 Bioverativ Therapeutics Inc. Chimeryczne i hybrydowe polipeptydy czynnika VIII-FC oraz sposoby ich zastosowania
JP2016519110A (ja) * 2013-04-22 2016-06-30 キャタレント ファーマ ソリューションズ リミテッド ライアビリティ カンパニー 獣医学的デコリン組成物及びそれらの使用
RU2647769C2 (ru) * 2015-12-30 2018-03-19 Закрытое Акционерное Общество "Биокад" Химерный улучшенный рекомбинантный фактор VIII
WO2017136358A1 (en) * 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
CN114651065B (zh) * 2019-09-03 2024-08-20 五松尖端医疗产业振兴财团 导入用于提高蛋白质生产性的单个条形码系统的信号肽超高速筛选方法
EP4051704A2 (en) * 2019-11-01 2022-09-07 Freeline Therapeutics Limited Factor viii construct

Also Published As

Publication number Publication date
PY2331055A (es) 2023-12-05
TW202342751A (zh) 2023-11-01
WO2023211315A1 (en) 2023-11-02
MA68435A1 (fr) 2025-04-30
KR20250005425A (ko) 2025-01-09
MX2024013281A (es) 2024-12-06
AR129164A1 (es) 2024-07-24
CO2024014703A2 (es) 2024-12-30
CL2024003295A1 (es) 2025-02-21
CN117402901A (zh) 2024-01-16
CA3250663A1 (en) 2023-11-02
IL316578A (en) 2024-12-01
PE20251064A1 (es) 2025-04-09
JOP20240240A1 (ar) 2024-10-27
AU2023261711A1 (en) 2024-11-21
UY40243A (es) 2023-11-15
CR20240525A (es) 2025-03-05
EP4514975A1 (en) 2025-03-05

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