JP7562544B2 - ミオシン１５プロモーター及びその使用 - Google Patents

ミオシン１５プロモーター及びその使用 Download PDF

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Publication number
JP7562544B2
JP7562544B2 JP2021546388A JP2021546388A JP7562544B2 JP 7562544 B2 JP7562544 B2 JP 7562544B2 JP 2021546388 A JP2021546388 A JP 2021546388A JP 2021546388 A JP2021546388 A JP 2021546388A JP 7562544 B2 JP7562544 B2 JP 7562544B2
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JP
Japan
Prior art keywords
seq
sequence
nucleic acid
item
polynucleotide
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JP2021546388A
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English (en)
Japanese (ja)
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JP2022519750A5 (https=
JP2022519750A (ja
Inventor
バーンズ、ジョセフ
エリス、キャサリン
パレルモ、アダム
シュワンダー、マーティン
ホイットン、ジョナサン
サビン、レア
キラツォス、クリストス
サミュエルソン、メーガン ドラモンド
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Regeneron Pharmaceuticals Inc
Decibel Therapeutics Inc
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Regeneron Pharmaceuticals Inc
Decibel Therapeutics Inc
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Application filed by Regeneron Pharmaceuticals Inc, Decibel Therapeutics Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of JP2022519750A publication Critical patent/JP2022519750A/ja
Publication of JP2022519750A5 publication Critical patent/JP2022519750A5/ja
Priority to JP2024165838A priority Critical patent/JP7778879B2/ja
Application granted granted Critical
Publication of JP7562544B2 publication Critical patent/JP7562544B2/ja
Priority to JP2025199231A priority patent/JP2026020285A/ja
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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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2021546388A 2019-02-08 2020-02-07 ミオシン１５プロモーター及びその使用 Active JP7562544B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024165838A JP7778879B2 (ja) 2019-02-08 2024-09-25 ミオシン１５プロモーター及びその使用
JP2025199231A JP2026020285A (ja) 2019-02-08 2025-11-19 ミオシン１５プロモーター及びその使用

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962802874P 2019-02-08 2019-02-08
US62/802,874 2019-02-08
US201962928311P 2019-10-30 2019-10-30
US62/928,311 2019-10-30
US202062965773P 2020-01-24 2020-01-24
US62/965,773 2020-01-24
PCT/US2020/017292 WO2020163761A1 (en) 2019-02-08 2020-02-07 Myosin 15 promoters and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024165838A Division JP7778879B2 (ja) 2019-02-08 2024-09-25 ミオシン１５プロモーター及びその使用

Publications (3)

Publication Number Publication Date
JP2022519750A JP2022519750A (ja) 2022-03-24
JP2022519750A5 JP2022519750A5 (https=) 2023-02-14
JP7562544B2 true JP7562544B2 (ja) 2024-10-07

Family

ID=71947522

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2021546388A Active JP7562544B2 (ja) 2019-02-08 2020-02-07 ミオシン１５プロモーター及びその使用
JP2024165838A Active JP7778879B2 (ja) 2019-02-08 2024-09-25 ミオシン１５プロモーター及びその使用
JP2025199231A Pending JP2026020285A (ja) 2019-02-08 2025-11-19 ミオシン１５プロモーター及びその使用

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2024165838A Active JP7778879B2 (ja) 2019-02-08 2024-09-25 ミオシン１５プロモーター及びその使用
JP2025199231A Pending JP2026020285A (ja) 2019-02-08 2025-11-19 ミオシン１５プロモーター及びその使用

Country Status (7)

Country Link
US (2) US12252520B2 (https=)
EP (1) EP3920974A4 (https=)
JP (3) JP7562544B2 (https=)
CN (3) CN119307494A (https=)
AU (1) AU2020218547A1 (https=)
CA (1) CA3129422A1 (https=)
WO (1) WO2020163761A1 (https=)

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CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
KR20220130093A (ko) * 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN112194717B (zh) * 2020-09-30 2021-12-14 东南大学 一种促进耳蜗毛细胞再生的肽段Tr及其应用
US20250073354A1 (en) 2021-12-23 2025-03-06 University Of Rochester Compositions and methods for delivery of agents to inner ear
EP4452336A2 (en) 2021-12-23 2024-10-30 University of Rochester Csf transport pathway for delivery of agents to inner ear
CN116836975A (zh) * 2022-03-25 2023-10-03 上海玮美基因科技有限责任公司 一种耳蜗和/或前庭细胞特异性启动子及其应用
WO2024250774A1 (zh) * 2023-06-07 2024-12-12 苏州星奥拓维生物技术有限公司 耳蜗外毛细胞特异性启动子及其应用
CN117486995A (zh) * 2023-11-03 2024-02-02 首都医科大学附属北京胸科医院 Kncn蛋白在结核诊断试剂中的应用

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Publication number Publication date
JP7778879B2 (ja) 2025-12-02
CN119307494A (zh) 2025-01-14
CN114072179B (zh) 2024-11-01
JP2025000750A (ja) 2025-01-07
CN114072179A (zh) 2022-02-18
CA3129422A1 (en) 2020-08-13
US12252520B2 (en) 2025-03-18
JP2026020285A (ja) 2026-02-06
EP3920974A4 (en) 2022-11-30
JP2022519750A (ja) 2022-03-24
US20250304637A1 (en) 2025-10-02
US20210388045A1 (en) 2021-12-16
CN119320772A (zh) 2025-01-17
WO2020163761A1 (en) 2020-08-13
EP3920974A1 (en) 2021-12-15
AU2020218547A1 (en) 2021-08-26

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