JP2021533805A - 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 - Google Patents

最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 Download PDF

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JP2021533805A
JP2021533805A JP2021510015A JP2021510015A JP2021533805A JP 2021533805 A JP2021533805 A JP 2021533805A JP 2021510015 A JP2021510015 A JP 2021510015A JP 2021510015 A JP2021510015 A JP 2021510015A JP 2021533805 A JP2021533805 A JP 2021533805A
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nucleic acid
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acid sequence
modified
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シャビエル アングエラ，
リーロン エルコビー，
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Spark Therapeutics Inc
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    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
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    • 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)
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775—Apolipopeptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • C07K14/8125—Alpha-1-antitrypsin
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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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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2021510015A 2018-08-24 2019-08-23 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 Pending JP2021533805A (ja)

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JP2024060919A JP7874125B2 (ja) 2018-08-24 2024-04-04 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法

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US201862722547P 2018-08-24 2018-08-24
US62/722,547 2018-08-24
US201862725096P 2018-08-30 2018-08-30
US62/725,096 2018-08-30
US201862784116P 2018-12-21 2018-12-21
US62/784,116 2018-12-21
PCT/US2019/048032 WO2020041773A1 (en) 2018-08-24 2019-08-23 Optimized promoter sequences, intron-free expression constructs and methods of use

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JP2024060919A Active JP7874125B2 (ja) 2018-08-24 2024-04-04 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法

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CN (1) CN113226352B (enExample)
AU (2) AU2019325688B2 (enExample)
BR (1) BR112021003399A2 (enExample)
CA (1) CA3111047A1 (enExample)
MX (1) MX2021002198A (enExample)
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EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
EP4333886A4 (en) * 2021-05-07 2025-11-12 Generation Bio Co NON-VIRAL DNA VECTORS FOR VACCINE ADMINISTRATION
WO2022236016A1 (en) * 2021-05-07 2022-11-10 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
AU2023261338A1 (en) * 2022-04-28 2024-11-21 Joint Stock Company "Biocad" Codon-optimized nucleic acid encoding the fviii-bdd
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
EP4598590A4 (en) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd CONSTRUCTION TO IMPROVE GENE EXPRESSION
WO2025264490A1 (en) * 2024-06-17 2025-12-26 Spark Therapeutics, Inc. Dna promoters
CN119242712A (zh) * 2024-09-27 2025-01-03 南京师范大学 特异性靶向骨骼肌细胞的携带miRNA-486基因的腺相关病毒载体、药物组合物

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WO2020041773A1 (en) 2020-02-27
JP7874125B2 (ja) 2026-06-15
AU2019325688A1 (en) 2021-03-18
EP3840775A1 (en) 2021-06-30
AU2019325688B2 (en) 2025-03-13
TW202012425A (zh) 2020-04-01
US12290574B2 (en) 2025-05-06
BR112021003399A2 (pt) 2021-05-18
CA3111047A1 (en) 2020-02-27
US20220362408A1 (en) 2022-11-17
CN113226352A (zh) 2021-08-06
MX2021002198A (es) 2021-07-15
JP2024102065A (ja) 2024-07-30
EP3840775A4 (en) 2022-07-06
CN113226352B (zh) 2025-08-26
AU2025204161A1 (en) 2025-06-26

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