KR20250005425A - Fviii-bdd 및 이종의 신호 펩타이드에 기반한 융합 단백질을 인코딩하는 단리된 핵산 - Google Patents

Fviii-bdd 및 이종의 신호 펩타이드에 기반한 융합 단백질을 인코딩하는 단리된 핵산 Download PDF

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KR20250005425A
KR20250005425A KR1020247039725A KR20247039725A KR20250005425A KR 20250005425 A KR20250005425 A KR 20250005425A KR 1020247039725 A KR1020247039725 A KR 1020247039725A KR 20247039725 A KR20247039725 A KR 20247039725A KR 20250005425 A KR20250005425 A KR 20250005425A
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fviii
bdd
nucleic acid
aav
seq
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Korean (ko)
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마리야 파블로브나 페레펠키나
엘레나 베니아미노브나 블라소바
아나스타샤 블라디미로브나 포미나
파벨 미하일로비치 게르쇼비치
비탈리아 알렉산드로브나 마르코바
드미트리 발렌티노비치 모로조프
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조인트 스탁 컴퍼니 "바이오케드"
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Priority claimed from RU2022111695A external-priority patent/RU2818229C2/ru
Application filed by 조인트 스탁 컴퍼니 "바이오케드" filed Critical 조인트 스탁 컴퍼니 "바이오케드"
Publication of KR20250005425A publication Critical patent/KR20250005425A/ko
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
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    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/76Albumins
    • C07K14/765Serum albumin, e.g. HSA
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • C12N15/625DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector 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)
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  • 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)
KR1020247039725A 2022-04-28 2023-04-18 Fviii-bdd 및 이종의 신호 펩타이드에 기반한 융합 단백질을 인코딩하는 단리된 핵산 Pending KR20250005425A (ko)

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 (1)

Publication Number Publication Date
KR20250005425A true KR20250005425A (ko) 2025-01-09

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KR1020247039725A Pending KR20250005425A (ko) 2022-04-28 2023-04-18 Fviii-bdd 및 이종의 신호 펩타이드에 기반한 융합 단백질을 인코딩하는 단리된 핵산

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

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Publication number Publication date
PY2331055A (es) 2023-12-05
TW202342751A (zh) 2023-11-01
WO2023211315A1 (en) 2023-11-02
MA68435A1 (fr) 2025-04-30
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
JP2025515356A (ja) 2025-05-14
CR20240525A (es) 2025-03-05
EP4514975A1 (en) 2025-03-05

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