TW202323274A - 優化因子viii基因 - Google Patents

優化因子viii基因 Download PDF

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Publication number
TW202323274A
TW202323274A TW111131533A TW111131533A TW202323274A TW 202323274 A TW202323274 A TW 202323274A TW 111131533 A TW111131533 A TW 111131533A TW 111131533 A TW111131533 A TW 111131533A TW 202323274 A TW202323274 A TW 202323274A
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nucleic acid
seq
acid molecule
nucleotide sequence
sequence
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TW111131533A
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Chinese (zh)
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亞傑 瑪格迪亞
彤瑤 劉
菲利普 札卡斯
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美商百歐維拉提夫治療公司
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    • C07ORGANIC CHEMISTRY
    • 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
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0066Manipulation 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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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
    • C12N15/63Introduction 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
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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
    • C12N15/63Introduction 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14311Parvovirus, e.g. minute virus of mice
    • C12N2750/14341Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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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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  • Pharmacology & Pharmacy (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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TW111131533A 2021-08-23 2022-08-22 優化因子viii基因 TW202323274A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163236225P 2021-08-23 2021-08-23
US63/236,225 2021-08-23

Publications (1)

Publication Number Publication Date
TW202323274A true TW202323274A (zh) 2023-06-16

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TW111131533A TW202323274A (zh) 2021-08-23 2022-08-22 優化因子viii基因

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US (1) US20230084036A1 (https=)
EP (1) EP4392443A1 (https=)
JP (1) JP2024532262A (https=)
KR (1) KR20240049332A (https=)
CN (1) CN117836319A (https=)
AR (1) AR126846A1 (https=)
AU (1) AU2022332276A1 (https=)
CA (1) CA3229323A1 (https=)
CO (1) CO2024001464A2 (https=)
IL (1) IL310997A (https=)
MX (1) MX2024002334A (https=)
TW (1) TW202323274A (https=)
WO (1) WO2023028456A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
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NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
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

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CA3229323A1 (en) 2023-03-02
MX2024002334A (es) 2024-05-23
EP4392443A1 (en) 2024-07-03
AU2022332276A1 (en) 2024-04-04
WO2023028456A1 (en) 2023-03-02
AR126846A1 (es) 2023-11-22
US20230084036A1 (en) 2023-03-16
CN117836319A (zh) 2024-04-05
KR20240049332A (ko) 2024-04-16
JP2024532262A (ja) 2024-09-05
IL310997A (en) 2024-04-01
CO2024001464A2 (es) 2024-03-07

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