CN115066264A - 耳蜗外毛细胞启动子及其用途 - Google Patents

耳蜗外毛细胞启动子及其用途 Download PDF

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Publication number
CN115066264A
CN115066264A CN202080091076.3A CN202080091076A CN115066264A CN 115066264 A CN115066264 A CN 115066264A CN 202080091076 A CN202080091076 A CN 202080091076A CN 115066264 A CN115066264 A CN 115066264A
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Prior art keywords
protein
nucleic acid
ohc
hearing loss
acid vector
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Pending
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CN202080091076.3A
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English (en)
Chinese (zh)
Inventor
J·伯恩斯
M·施万德
X·吴
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Decibel Therapeutics Inc
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Decibel Therapeutics Inc
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Publication of CN115066264A publication Critical patent/CN115066264A/zh
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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)
CN202080091076.3A 2019-11-04 2020-11-04 耳蜗外毛细胞启动子及其用途 Pending CN115066264A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962930486P 2019-11-04 2019-11-04
US62/930486 2019-11-04
PCT/US2020/058780 WO2021091938A1 (en) 2019-11-04 2020-11-04 Cochlear outer hair cell promoters and uses thereof

Publications (1)

Publication Number Publication Date
CN115066264A true CN115066264A (zh) 2022-09-16

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080091076.3A Pending CN115066264A (zh) 2019-11-04 2020-11-04 耳蜗外毛细胞启动子及其用途

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=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024239226A1 (zh) * 2023-05-23 2024-11-28 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
WO2024245357A1 (zh) * 2023-06-02 2024-12-05 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

Families Citing this family (6)

* 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

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050281786A1 (en) * 2004-06-18 2005-12-22 David Poulsen AAV mediated gene delivery to cochlear cells
US20130280368A1 (en) * 2011-01-12 2013-10-24 Morinaga Milk Industry Co., Ltd. Method for screening for diet providing production of milk having immunoregulatory action
WO2018148071A1 (en) * 2017-02-10 2018-08-16 St. Jude Children's Research Hospital Combination therapy for treating disorders of the ear
US20190142969A1 (en) * 2016-04-26 2019-05-16 Massachusetts Eye And Ear Infirmary ISL1-Based Gene Therapy to Treat Hearing Loss
CA3093490A1 (en) * 2018-03-05 2019-09-12 Children's Medical Center Corporation Compositions and methods for delivering nucleic acids to cochlear and vestibular cells
WO2019210181A1 (en) * 2018-04-27 2019-10-31 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008073303A2 (en) * 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050281786A1 (en) * 2004-06-18 2005-12-22 David Poulsen AAV mediated gene delivery to cochlear cells
US20130280368A1 (en) * 2011-01-12 2013-10-24 Morinaga Milk Industry Co., Ltd. Method for screening for diet providing production of milk having immunoregulatory action
US20190142969A1 (en) * 2016-04-26 2019-05-16 Massachusetts Eye And Ear Infirmary ISL1-Based Gene Therapy to Treat Hearing Loss
WO2018148071A1 (en) * 2017-02-10 2018-08-16 St. Jude Children's Research Hospital Combination therapy for treating disorders of the ear
CA3093490A1 (en) * 2018-03-05 2019-09-12 Children's Medical Center Corporation Compositions and methods for delivering nucleic acids to cochlear and vestibular cells
WO2019210181A1 (en) * 2018-04-27 2019-10-31 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
ATHAS GB 等: "In Vitro Analysis of the Oncomodulin Promoter for Targeting Gene Expression to the Ear", 《OTOLARYNGOLOGY-HEAD AND NECK SURGERY》, vol. 121, no. 2, pages 6 - 7 *
ISHIKAWA Y等: "Structure of the core promoter of human and mouse ribosomal RNA gene Asymmetry of species-specific transcription", J MOL BIOL, vol. 218, no. 1, 5 March 1991 (1991-03-05), pages 55 - 67, XP024021492, DOI: 10.1016/0022-2836(91)90873-5 *
RENTSCH JM 等: "The LTR promoter of the rat oncomodulin gene is regulated by cell-line specific accessibility in the LTR U3 region", 《ARCH BIOCHEM BIOPHYS》, vol. 447, no. 1, pages 68 - 79, XP024943005, DOI: 10.1016/j.abb.2006.01.006 *
无: "Accession number: JN959272.1，Mus musculus targeted KO-first, conditional ready, lacZ-tagged mutant allele Ocm:tm1a(EUCOMM) Wtsi;transgenic", 《GENBANK》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024239226A1 (zh) * 2023-05-23 2024-11-28 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
WO2024245357A1 (zh) * 2023-06-02 2024-12-05 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

Also Published As

Publication number Publication date
WO2021091938A8 (en) 2021-07-15
WO2021091938A1 (en) 2021-05-14
US20220288236A1 (en) 2022-09-15
EP4054653A1 (en) 2022-09-14
JP2023500672A (ja) 2023-01-10
EP4054653A4 (en) 2023-11-29

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