JP2023500672A - 蝸牛外有毛細胞プロモーター及びその使用 - Google Patents

蝸牛外有毛細胞プロモーター及びその使用 Download PDF

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Publication number
JP2023500672A
JP2023500672A JP2022525649A JP2022525649A JP2023500672A JP 2023500672 A JP2023500672 A JP 2023500672A JP 2022525649 A JP2022525649 A JP 2022525649A JP 2022525649 A JP2022525649 A JP 2022525649A JP 2023500672 A JP2023500672 A JP 2023500672A
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Prior art keywords
nucleic acid
protein
ohc
acid vector
polynucleotide
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JP2022525649A
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Japanese (ja)
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JP2023500672A5 (https=
Inventor
バーンズ、ジョセフ
シュワンダー、マーティン
ウー、シュドン
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Decibel Therapeutics Inc
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Decibel Therapeutics Inc
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Publication of JP2023500672A publication Critical patent/JP2023500672A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184—Virus capsids or envelopes enclosing drugs
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0046—Ear
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005—Wall or coating material
    • A61K9/5063—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5068—Cell membranes or bacterial membranes enclosing drugs
    • 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
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (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)
JP2022525649A 2019-11-04 2020-11-04 蝸牛外有毛細胞プロモーター及びその使用 Pending JP2023500672A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962930486P 2019-11-04 2019-11-04
US62/930,486 2019-11-04
PCT/US2020/058780 WO2021091938A1 (en) 2019-11-04 2020-11-04 Cochlear outer hair cell promoters and uses thereof

Publications (2)

Publication Number Publication Date
JP2023500672A true JP2023500672A (ja) 2023-01-10
JP2023500672A5 JP2023500672A5 (https=) 2023-11-10

Family

ID=75848059

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022525649A Pending JP2023500672A (ja) 2019-11-04 2020-11-04 蝸牛外有毛細胞プロモーター及びその使用

Country Status (5)

Country Link
US (1) US20220288236A1 (https=)
EP (1) EP4054653A4 (https=)
JP (1) JP2023500672A (https=)
CN (1) CN115066264A (https=)
WO (1) WO2021091938A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
WO2020014625A1 (en) 2018-07-13 2020-01-16 Akouos, Inc. Methods of treating non-syndromic sensorineural hearing loss
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
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
EP4409009A4 (en) * 2021-09-30 2025-12-17 Akouos Inc Compositions for administering gene therapy and methods for treating hearing loss
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
CN121263526A (zh) * 2023-05-23 2026-01-02 苏州星奥拓维生物技术有限公司 耳蜗毛细胞特异性启动子及其用途
CN119061007A (zh) * 2023-06-02 2024-12-03 苏州星奥拓维生物技术有限公司 耳蜗支持细胞基因治疗和再生的方法

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1755400A2 (en) * 2004-06-18 2007-02-28 The University Of Montana Aav mediated gene delivery to cochlear cells
WO2008073303A2 (en) * 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
CN103119180A (zh) * 2011-01-12 2013-05-22 森永乳业株式会社 产生具有免疫调节作用的乳的食物的筛选方法
US20190142969A1 (en) * 2016-04-26 2019-05-16 Massachusetts Eye And Ear Infirmary ISL1-Based Gene Therapy to Treat Hearing Loss
US11331373B2 (en) * 2017-02-10 2022-05-17 St. Jude Children's Research Hospital Combination therapy for treating disorders of the ear
EP3762040A4 (en) * 2018-03-05 2022-01-05 Children's Medical Center Corporation COMPOSITIONS AND METHODS OF ADMINISTRATION OF NUCLEIC ACIDS TO COCHLEAR AND VESTIBULAR CELLS
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

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
"Accession No.AC004983.3", DATABASE GENBANK, JPN6024039947, 15 October 2003 (2003-10-15), ISSN: 0005432748 *
"Accession No.AC121917.2", DATABASE GENBANK, JPN6024039948, 5 November 2003 (2003-11-05), ISSN: 0005432747 *
GRACE B ATHAS ET AL.: "In Vitro Analysis of the Oncomodulin Promoter for Targeting Gene Expression to the Ear", OTOLARYNGOLOGY-HEAD AND NECK SURGERY, vol. XP005450538, JPN6024039949, August 1999 (1999-08-01), pages 64 - 65, ISSN: 0005432746 *

Also Published As

Publication number Publication date
WO2021091938A8 (en) 2021-07-15
WO2021091938A1 (en) 2021-05-14
CN115066264A (zh) 2022-09-16
US20220288236A1 (en) 2022-09-15
EP4054653A1 (en) 2022-09-14
EP4054653A4 (en) 2023-11-29

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