CN112703016B - 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
CN112703016B
CN112703016B CN201980042739.XA CN201980042739A CN112703016B CN 112703016 B CN112703016 B CN 112703016B CN 201980042739 A CN201980042739 A CN 201980042739A CN 112703016 B CN112703016 B CN 112703016B
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nucleic acid
seq
cells
vector
hair
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CN112703016A (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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Priority to CN202411557780.XA priority patent/CN119410637A/zh
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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)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Cell Biology (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201980042739.XA 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途 Active CN112703016B (zh)

Priority Applications (2)

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

Applications Claiming Priority (3)

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

Related Child Applications (2)

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CN202411557780.XA Division CN119410637A (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途
CN202411557776.3A Division CN119410636A (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途

Publications (2)

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CN112703016A CN112703016A (zh) 2021-04-23
CN112703016B true CN112703016B (zh) 2024-11-22

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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
CN202411557776.3A Pending CN119410636A (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途

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CN202411557780.XA Pending CN119410637A (zh) 2018-04-27 2019-04-26 肌球蛋白15启动子及其用途
CN202411557776.3A Pending CN119410636A (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) CN112703016B (https=)
AU (1) AU2019257782B2 (https=)
CA (1) CA3098198A1 (https=)
WO (1) WO2019210181A1 (https=)

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US20220054640A1 (en) * 2018-12-11 2022-02-24 Decibel Therapeutics, Inc. Compositions and methods for the delivery of therapeutic agents across the round window membrane
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
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
WO2021091938A1 (en) * 2019-11-04 2021-05-14 Decibel Therapeutics, Inc. Cochlear outer hair cell promoters and uses thereof
EP4055175A4 (en) 2019-11-04 2023-11-29 Decibel Therapeutics, Inc. Cochlear inner hair cell promoters and uses thereof
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
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
WO2023147383A1 (en) * 2022-01-25 2023-08-03 Bridgebio Services Inc. Gene therapy for genetic hearing loss

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JP2025122095A (ja) 2025-08-20
WO2019210181A1 (en) 2019-10-31
US20250319205A1 (en) 2025-10-16
EP3784289A1 (en) 2021-03-03
AU2019257782A1 (en) 2020-11-19
CN119410637A (zh) 2025-02-11
US12233136B2 (en) 2025-02-25
CN119410636A (zh) 2025-02-11
JP2021521842A (ja) 2021-08-30
US20210236654A1 (en) 2021-08-05
AU2019257782B2 (en) 2025-11-20
JP2023138741A (ja) 2023-10-02
CA3098198A1 (en) 2019-10-31
JP7686709B2 (ja) 2025-06-02
JP7331011B2 (ja) 2023-08-22
CN112703016A (zh) 2021-04-23

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