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 (https=
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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)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Obesity (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)
  • 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)

Application Number Title Priority Date Filing Date
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 (https=) 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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Country Link
US (1) US12296026B2 (https=)
EP (1) EP3749756A4 (https=)
JP (2) JP7381494B2 (https=)
CN (1) CN111936621B (https=)
MA (1) MA51753A (https=)
WO (1) WO2019153009A1 (https=)

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DK3635009T3 (da) 2017-06-07 2026-03-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
CN111902539B (zh) * 2018-02-07 2025-05-16 吉尼松公司 杂合调控元件
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
KR20210148273A (ko) * 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
JP2022554141A (ja) * 2019-10-25 2022-12-28 オーデンツ セラピューティクス，インコーポレーテッド 糖原病を治療するための組成物及び方法
KR20220098384A (ko) * 2019-11-19 2022-07-12 아스클레피오스 바이오파마슈티컬, 인크. 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스
JP7744027B2 (ja) * 2020-02-18 2025-09-25 フレイエ ユニヴェルシテイト ブリュッセル 核酸調節エレメントの新規な組合せ並びにその方法及び使用
GB202013194D0 (en) * 2020-08-24 2020-10-07 Combigene Ab Gene therapy for lipodystrophy
AR126660A1 (es) * 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
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
WO2024006775A2 (en) * 2022-06-27 2024-01-04 Astellas Gene Therapies, Inc. Compositions and methods for the treatment of myotonic dystrophies
EP4598590A1 (en) * 2022-10-08 2025-08-13 Lingyi Biotech Co., Ltd. Construct for enhancing 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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US8021875B2 (en) * 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
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Patent 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

Also Published As

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

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