JP2020508648A - 蝸牛および前庭細胞に核酸を送達するための物質および方法 - Google Patents

蝸牛および前庭細胞に核酸を送達するための物質および方法 Download PDF

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JP2020508648A
JP2020508648A JP2019542464A JP2019542464A JP2020508648A JP 2020508648 A JP2020508648 A JP 2020508648A JP 2019542464 A JP2019542464 A JP 2019542464A JP 2019542464 A JP2019542464 A JP 2019542464A JP 2020508648 A JP2020508648 A JP 2020508648A
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harmonin
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ジェフリー ホルト
ジェフリー ホルト
グウェナエル ゲレオク
グウェナエル ゲレオク
友香子 浅井
友香子 浅井
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Boston Children's Hospital
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    • 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
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    • 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
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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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • 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
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • 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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  • Veterinary Medicine (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2019542464A 2017-02-06 2018-02-06 蝸牛および前庭細胞に核酸を送達するための物質および方法 Withdrawn JP2020508648A (ja)

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JP2023000088A JP7601916B2 (ja) 2017-02-06 2023-01-04 蝸牛および前庭細胞に核酸を送達するための物質および方法

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US201762455197P 2017-02-06 2017-02-06
US62/455,197 2017-02-06
PCT/US2018/017104 WO2018145111A1 (en) 2017-02-06 2018-02-06 Materials and methods for delivering nucleic acids to cochlear and vestibular cells

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JP2023000088A Active JP7601916B2 (ja) 2017-02-06 2023-01-04 蝸牛および前庭細胞に核酸を送達するための物質および方法

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AU (2) AU2018215785B2 (enExample)
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KR20220124603A (ko) * 2021-03-03 2022-09-14 연세대학교 산학협력단 Kcnq4 유전자 교정용 가이드 rna 및 이를 포함하는 난청 치료용 조성물

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WO2018039375A1 (en) 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
IL325122A (en) 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
JP7430652B2 (ja) 2018-03-05 2024-02-13 ザ チルドレンズ メディカル センター コーポレーション 蝸牛および前庭細胞に核酸を送達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
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
US12491223B2 (en) 2018-09-27 2025-12-09 Akouos, Inc. Method for treating an auditory neuropathy spectrum disorder
US12534739B2 (en) 2018-10-11 2026-01-27 Decibel Therapeutics, Inc. AAV1 vectors and uses thereof for treatment of otic indications
CN113825521A (zh) * 2018-12-21 2021-12-21 美利坚合众国由健康及人类服务部部长代表 腺相关病毒及其用于内耳疗法的用途
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
WO2020163761A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3959324B1 (en) * 2019-04-26 2025-09-17 President and Fellows of Harvard College Aav vectors encoding mini-pcdh15 and uses thereof
CN114269926A (zh) 2019-04-26 2022-04-01 美商圣加莫治疗股份有限公司 Aav工程化
US12667628B2 (en) 2019-07-03 2026-06-30 Children's Medical Center Corporation Compositions and methods for gene replacement
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
TWI896570B (zh) 2019-10-30 2025-09-11 美商迪賽博治療公司 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法
EP4055175A4 (en) * 2019-11-04 2023-11-29 Decibel Therapeutics, Inc. Cochlear inner hair cell promoters and uses thereof
CN119391775A (zh) * 2019-11-04 2025-02-07 分贝治疗公司 前庭支持细胞启动子和其用途
WO2021150850A1 (en) * 2020-01-24 2021-07-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Svcs. Adeno-associated viruses and their uses for inner ear therapy
CA3168055A1 (en) 2020-02-21 2021-08-26 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2022129543A1 (en) * 2020-12-18 2022-06-23 Institut Pasteur Gene therapy for treating usher syndrome
CN114762733B (zh) * 2021-01-15 2025-03-18 中国科学院脑科学与智能技术卓越创新中心 耳蜗外毛细胞特异性顺式调节元件及其应用
WO2023147383A1 (en) 2022-01-25 2023-08-03 Bridgebio Services Inc. Gene therapy for genetic hearing loss
CN116606885A (zh) * 2023-02-21 2023-08-18 东南大学 一种Lhfpl5基因敲除鼠构建实验方法
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
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

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220124603A (ko) * 2021-03-03 2022-09-14 연세대학교 산학협력단 Kcnq4 유전자 교정용 가이드 rna 및 이를 포함하는 난청 치료용 조성물
KR102751019B1 (ko) 2021-03-03 2025-01-09 연세대학교 산학협력단 Kcnq4 유전자 교정용 가이드 rna 및 이를 포함하는 난청 치료용 조성물

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CN111698975A (zh) 2020-09-22
AU2018215785A9 (en) 2019-09-26
EP3576696A4 (en) 2020-09-16
US11730827B2 (en) 2023-08-22
AU2018215785B2 (en) 2023-08-31
JP2023036921A (ja) 2023-03-14
CA3052704A1 (en) 2018-08-09
AU2023214288A1 (en) 2023-11-30
AU2018215785A1 (en) 2019-08-22
EP3576696A1 (en) 2019-12-11
US20190351072A1 (en) 2019-11-21
JP7601916B2 (ja) 2024-12-17
WO2018145111A9 (en) 2019-08-29
WO2018145111A1 (en) 2018-08-09
US20230330268A1 (en) 2023-10-19

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