CN116970629A - 密码子优化的编码b-结构域缺失的凝血因子viii蛋白的核酸及其用途 - Google Patents

密码子优化的编码b-结构域缺失的凝血因子viii蛋白的核酸及其用途 Download PDF

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Publication number
CN116970629A
CN116970629A CN202310468975.6A CN202310468975A CN116970629A CN 116970629 A CN116970629 A CN 116970629A CN 202310468975 A CN202310468975 A CN 202310468975A CN 116970629 A CN116970629 A CN 116970629A
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Prior art keywords
fviii
aav
bdd
seq
nucleic acid
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Pending
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CN202310468975.6A
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English (en)
Chinese (zh)
Inventor
T·E·舒加娃
E·V·弗拉索娃
A·V·佛米纳
M·P·佩雷佩尔金纳
A·N·斯特雷科娃
P·M·格绍维奇
P·A·亚科夫列夫
D·V·莫洛佐夫
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Biocard Jsc
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Biocard Jsc
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Priority claimed from RU2022111734A external-priority patent/RU2808564C2/ru
Application filed by Biocard Jsc filed Critical Biocard Jsc
Publication of CN116970629A publication Critical patent/CN116970629A/zh
Pending legal-status Critical Current

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    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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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
    • 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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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • 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
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    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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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
    • C12N15/09—Recombinant DNA-technology
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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
    • 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
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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
    • 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
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/00—Fusion polypeptide
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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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    • C12N2800/00—Nucleic acids vectors
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    • C—CHEMISTRY; METALLURGY
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    • 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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  • Animal Behavior & Ethology (AREA)
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  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202310468975.6A 2022-04-28 2023-04-27 密码子优化的编码b-结构域缺失的凝血因子viii蛋白的核酸及其用途 Pending CN116970629A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2022111734A RU2808564C2 (ru) 2022-04-28 Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови VIII c делетированным B доменом, и ее применение
RU2022111734 2022-04-28

Publications (1)

Publication Number Publication Date
CN116970629A true CN116970629A (zh) 2023-10-31

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CN202310468975.6A Pending CN116970629A (zh) 2022-04-28 2023-04-27 密码子优化的编码b-结构域缺失的凝血因子viii蛋白的核酸及其用途

Country Status (20)

Country Link
US (1) US20250282847A1 (https=)
EP (1) EP4514836A1 (https=)
JP (1) JP2025514313A (https=)
KR (1) KR20250007615A (https=)
CN (1) CN116970629A (https=)
AR (1) AR129163A1 (https=)
AU (1) AU2023261338A1 (https=)
CA (1) CA3250665A1 (https=)
CL (1) CL2024003296A1 (https=)
CO (1) CO2024014690A2 (https=)
CR (1) CR20240524A (https=)
IL (1) IL316584A (https=)
JO (1) JOP20240241A1 (https=)
MA (1) MA68586A1 (https=)
MX (1) MX2024013280A (https=)
PE (1) PE20251838A1 (https=)
PY (1) PY2331315A (https=)
TW (1) TW202346601A (https=)
UY (1) UY40241A (https=)
WO (1) WO2023211316A1 (https=)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
EA202190827A1 (ru) * 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
BR112021003399A2 (pt) * 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso

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CR20240524A (es) 2025-05-05
KR20250007615A (ko) 2025-01-14
CA3250665A1 (en) 2023-11-02
IL316584A (en) 2024-12-01
PY2331315A (es) 2023-10-31
CL2024003296A1 (es) 2025-02-21
AU2023261338A1 (en) 2024-11-21
TW202346601A (zh) 2023-12-01
PE20251838A1 (es) 2025-07-17
MX2024013280A (es) 2024-12-06
CO2024014690A2 (es) 2025-03-06
UY40241A (es) 2023-10-13
JP2025514313A (ja) 2025-05-02
MA68586A1 (fr) 2025-04-30
AR129163A1 (es) 2024-07-24
WO2023211316A1 (en) 2023-11-02
JOP20240241A1 (ar) 2024-10-27
US20250282847A1 (en) 2025-09-11
EP4514836A1 (en) 2025-03-05

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