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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JP2022519750A (ja
JP2022519750A5 (https=
JPWO2020163761A5 (https=
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
Publication of JPWO2020163761A5 publication Critical patent/JPWO2020163761A5/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
Active legal-status Critical Current
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Classifications

    • 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

Landscapes

  • 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 (4)

Publication Number Publication Date
JP2022519750A JP2022519750A (ja) 2022-03-24
JP2022519750A5 JP2022519750A5 (https=) 2023-02-14
JPWO2020163761A5 JPWO2020163761A5 (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) CN119320772A (https=)
AU (1) AU2020218547A1 (https=)
CA (1) CA3129422A1 (https=)
WO (1) WO2020163761A1 (https=)

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US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
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
CA3159549A1 (en) * 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7807380B2 (ja) 2020-02-21 2026-01-27 アコーオス インコーポレイテッド ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
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
JP2025504263A (ja) 2021-12-23 2025-02-10 ユニバーシティ オブ ロチェスター 内耳への薬剤の送達のためのｃｓｆ輸送経路
JP2025503508A (ja) 2021-12-23 2025-02-04 ユニバーシティ オブ ロチェスター 内耳への薬剤の送達のための組成物及び方法
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
JP2025000750A (ja) 2025-01-07
CN114072179B (zh) 2024-11-01
JP2026020285A (ja) 2026-02-06
JP2022519750A (ja) 2022-03-24
AU2020218547A1 (en) 2021-08-26
JP7778879B2 (ja) 2025-12-02
CN119307494A (zh) 2025-01-14
EP3920974A1 (en) 2021-12-15
US20250304637A1 (en) 2025-10-02
CA3129422A1 (en) 2020-08-13
US20210388045A1 (en) 2021-12-16
CN114072179A (zh) 2022-02-18
US12252520B2 (en) 2025-03-18
WO2020163761A1 (en) 2020-08-13
EP3920974A4 (en) 2022-11-30
CN119320772A (zh) 2025-01-17

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