JP7381494B2 - 転写調節要素及びその使用 - Google Patents

転写調節要素及びその使用 Download PDF

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JP7381494B2
JP7381494B2 JP2020564045A JP2020564045A JP7381494B2 JP 7381494 B2 JP7381494 B2 JP 7381494B2 JP 2020564045 A JP2020564045 A JP 2020564045A JP 2020564045 A JP2020564045 A JP 2020564045A JP 7381494 B2 JP7381494 B2 JP 7381494B2
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nucleic acid
acid sequence
vector
seq
segment
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JP2021512649A (ja
JP2021512649A5 (enExample
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ティー． グレイ，ジョン
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オーデンツ セラピューティクス，インコーポレーテッド
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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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2020564045A 2018-02-05 2019-02-05 転写調節要素及びその使用 Active JP7381494B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023188066A JP7776477B2 (ja) 2018-02-05 2023-11-02 転写調節要素及びその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862626561P 2018-02-05 2018-02-05
US62/626,561 2018-02-05
PCT/US2019/016692 WO2019153009A1 (en) 2018-02-05 2019-02-05 Transcription regulatory elements and uses thereof

Related Child Applications (1)

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JP2023188066A Division JP7776477B2 (ja) 2018-02-05 2023-11-02 転写調節要素及びその使用

Publications (3)

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JP2021512649A JP2021512649A (ja) 2021-05-20
JP2021512649A5 JP2021512649A5 (enExample) 2022-02-09
JP7381494B2 true JP7381494B2 (ja) 2023-11-15

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JP2020564045A Active JP7381494B2 (ja) 2018-02-05 2019-02-05 転写調節要素及びその使用
JP2023188066A Active JP7776477B2 (ja) 2018-02-05 2023-11-02 転写調節要素及びその使用

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US (1) US12296026B2 (enExample)
EP (1) EP3749756A4 (enExample)
JP (2) JP7381494B2 (enExample)
CN (1) CN111936621B (enExample)
MA (1) MA51753A (enExample)
WO (1) WO2019153009A1 (enExample)

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AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CA3086942A1 (en) * 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
KR20210148273A (ko) * 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
AU2020372429A1 (en) * 2019-10-25 2022-04-28 Audentes Therapeutics, Inc. Compositions and methods for treating glycogen storage disorders
WO2021102107A1 (en) * 2019-11-19 2021-05-27 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders
EP4107259A1 (en) * 2020-02-18 2022-12-28 Vrije Universiteit Brussel Novel combination of nucleic acid regulatory elements and methods and uses thereof
GB202013194D0 (en) * 2020-08-24 2020-10-07 Combigene Ab Gene therapy for lipodystrophy
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
AR126660A1 (es) * 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
WO2023012313A1 (en) * 2021-08-04 2023-02-09 Genethon Hybrid promoters for gene expression in muscles and in the cns
TW202400796A (zh) * 2022-03-11 2024-01-01 美商同源醫藥公司 雙向雙重啟動子表現載體及其用途
TW202409290A (zh) * 2022-06-27 2024-03-01 美商安斯泰來基因治療股份有限公司 用於治療肌強直性營養不良之組合物及方法
EP4598590A4 (en) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd CONSTRUCTION TO IMPROVE GENE EXPRESSION
WO2025004002A2 (en) 2023-06-30 2025-01-02 Takeda Pharmaceutical Company Limited Treatment of pompe disease
CN118064436B (zh) * 2024-04-18 2024-07-26 上海凌医生物科技有限公司 一种增强基因表达的嵌合启动子

Citations (4)

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JP2015523998A (ja) 2012-06-19 2015-08-20 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 疾患を治療するための組成物および方法
JP2017505126A (ja) 2014-01-21 2017-02-16 フレイエ ユニヴェルシテイト ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｔｅｉｔ Ｂｒｕｓｓｅｌ 筋特異的核酸調節エレメント並びにその方法及び使用
US20170218395A1 (en) 2014-06-20 2017-08-03 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
US20180311290A1 (en) 2015-04-23 2018-11-01 University Of Massachusetts Modulation of aav vector transgene expression

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EP1390490B1 (en) * 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
US8021875B2 (en) 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
US20070161031A1 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
CN105229144A (zh) * 2013-02-22 2016-01-06 细胞动力学国际有限公司 通过组合的遗传工程和化学工程经由正向编程产生肝细胞
JP2018535929A (ja) * 2015-09-24 2018-12-06 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤

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JP2015523998A (ja) 2012-06-19 2015-08-20 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 疾患を治療するための組成物および方法
JP2017505126A (ja) 2014-01-21 2017-02-16 フレイエ ユニヴェルシテイト ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｔｅｉｔ Ｂｒｕｓｓｅｌ 筋特異的核酸調節エレメント並びにその方法及び使用
US20170218395A1 (en) 2014-06-20 2017-08-03 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
US20180311290A1 (en) 2015-04-23 2018-11-01 University Of Massachusetts Modulation of aav vector transgene expression

Also Published As

Publication number Publication date
CN111936621B (zh) 2025-05-13
EP3749756A4 (en) 2021-12-08
JP2021512649A (ja) 2021-05-20
US20210162073A1 (en) 2021-06-03
JP2024012479A (ja) 2024-01-30
EP3749756A1 (en) 2020-12-16
CN111936621A (zh) 2020-11-13
JP7776477B2 (ja) 2025-11-26
WO2019153009A1 (en) 2019-08-08
US12296026B2 (en) 2025-05-13
MA51753A (fr) 2020-12-16

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