CN119410636A - 肌球蛋白15启动子及其用途 - Google Patents

肌球蛋白15启动子及其用途 Download PDF

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Publication number
CN119410636A
CN119410636A CN202411557776.3A CN202411557776A CN119410636A CN 119410636 A CN119410636 A CN 119410636A CN 202411557776 A CN202411557776 A CN 202411557776A CN 119410636 A CN119410636 A CN 119410636A
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China
Prior art keywords
seq
sequence
polynucleotide
nucleic acid
functional portion
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Pending
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CN202411557776.3A
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English (en)
Chinese (zh)
Inventor
J·伯恩斯
K·埃利斯
A·帕勒莫
M·施万德
J·怀顿
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Decibel Therapeutics Inc
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Decibel Therapeutics Inc
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Application filed by Decibel Therapeutics Inc filed Critical Decibel Therapeutics Inc
Publication of CN119410636A publication Critical patent/CN119410636A/zh
Pending legal-status Critical Current

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0625—Epidermal cells, skin cells; Cells of the oral mucosa
    • C12N5/0627—Hair cells
    • 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
    • C12N2510/00—Genetically modified cells
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Virology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN202411557776.3A 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途 Pending CN119410636A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862663679P 2018-04-27 2018-04-27
US62/663679 2018-04-27
PCT/US2019/029366 WO2019210181A1 (en) 2018-04-27 2019-04-26 Myosin 15 promoters and uses thereof
CN201980042739.XA CN112703016B (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201980042739.XA Division CN112703016B (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途

Publications (1)

Publication Number Publication Date
CN119410636A true CN119410636A (zh) 2025-02-11

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Family Applications (3)

Application Number Title Priority Date Filing Date
CN202411557776.3A Pending CN119410636A (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途
CN201980042739.XA Active CN112703016B (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途
CN202411557780.XA Pending CN119410637A (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途

Family Applications After (2)

Application Number Title Priority Date Filing Date
CN201980042739.XA Active CN112703016B (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途
CN202411557780.XA Pending CN119410637A (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途

Country Status (6)

Country Link
US (2) US12233136B2 (https=)
JP (3) JP7331011B2 (https=)
CN (3) CN119410636A (https=)
AU (1) AU2019257782B2 (https=)
CA (1) CA3098198A1 (https=)
WO (1) WO2019210181A1 (https=)

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JP2019530737A (ja) 2016-08-23 2019-10-24 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性聴力障害を治療するための組成物および方法
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2020123713A1 (en) * 2018-12-11 2020-06-18 Decibel Therapeutics, Inc. Compositions and methods for the delivery of therapeutic agents across the round window membrane
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
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP4054653A4 (en) * 2019-11-04 2023-11-29 Decibel Therapeutics, Inc. PROMOTERS OF COCHLEAR EXTERNAL HAIR CELLS AND THEIR USES
EP4055175A4 (en) 2019-11-04 2023-11-29 Decibel Therapeutics, Inc. Cochlear inner hair cell promoters and uses thereof
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
WO2021222682A1 (en) * 2020-04-30 2021-11-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Application of aav44.9 vector in gene therapy for the inner ear
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Also Published As

Publication number Publication date
JP2021521842A (ja) 2021-08-30
CN112703016A (zh) 2021-04-23
CN119410637A (zh) 2025-02-11
JP2025122095A (ja) 2025-08-20
US20210236654A1 (en) 2021-08-05
AU2019257782B2 (en) 2025-11-20
CA3098198A1 (en) 2019-10-31
US20250319205A1 (en) 2025-10-16
EP3784289A1 (en) 2021-03-03
JP2023138741A (ja) 2023-10-02
CN112703016B (zh) 2024-11-22
JP7331011B2 (ja) 2023-08-22
US12233136B2 (en) 2025-02-25
WO2019210181A1 (en) 2019-10-31
AU2019257782A1 (en) 2020-11-19
JP7686709B2 (ja) 2025-06-02

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