JP7713130B2 - 遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム - Google Patents

遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム

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Publication number
JP7713130B2
JP7713130B2 JP2022520059A JP2022520059A JP7713130B2 JP 7713130 B2 JP7713130 B2 JP 7713130B2 JP 2022520059 A JP2022520059 A JP 2022520059A JP 2022520059 A JP2022520059 A JP 2022520059A JP 7713130 B2 JP7713130 B2 JP 7713130B2
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gjb2
promoter
aav
nucleic acid
cell
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JP2022549380A5 (https=
JPWO2021067448A5 (https=
JP2022549380A (ja
Inventor
バルトロメ，クリス
デイヴィッド ハラミージョ，ルイス
ティマース，エイドリアン
ペノック，スティーブン
シャーマン，マーク
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イーライ リリー アンド カンパニー
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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • 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
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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)
JP2022520059A 2019-09-30 2020-09-30 遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム Active JP7713130B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025035714A JP2025110904A (ja) 2019-09-30 2025-03-06 遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962907834P 2019-09-30 2019-09-30
US62/907,834 2019-09-30
PCT/US2020/053561 WO2021067448A1 (en) 2019-09-30 2020-09-30 Adeno-associated virus (aav) systems for treatment of genetic hearing loss

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JP2025035714A Division JP2025110904A (ja) 2019-09-30 2025-03-06 遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム

Publications (4)

Publication Number Publication Date
JP2022549380A JP2022549380A (ja) 2022-11-24
JP2022549380A5 JP2022549380A5 (https=) 2023-10-10
JPWO2021067448A5 JPWO2021067448A5 (https=) 2023-10-10
JP7713130B2 true JP7713130B2 (ja) 2025-07-25

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JP2022520059A Active JP7713130B2 (ja) 2019-09-30 2020-09-30 遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム
JP2025035714A Pending JP2025110904A (ja) 2019-09-30 2025-03-06 遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム

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Country Status (9)

Country Link
US (2) US12241077B2 (https=)
EP (1) EP4037771A4 (https=)
JP (2) JP7713130B2 (https=)
KR (1) KR20220133854A (https=)
CN (1) CN115103710A (https=)
AU (1) AU2020357740A1 (https=)
CA (1) CA3153133A1 (https=)
IL (1) IL291789A (https=)
WO (1) WO2021067448A1 (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
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 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
EP4164695A4 (en) * 2020-05-13 2024-06-05 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
US20230340038A1 (en) * 2020-09-14 2023-10-26 President And Fellows Of Harvard College Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
JP2023541443A (ja) * 2020-09-14 2023-10-02 プレジデント アンド フェローズ オブ ハーバード カレッジ ＧＪＢ２をコードする組換えアデノ随伴ウイルス（ｒＡＡＶ）およびその使用。
AU2021376225A1 (en) * 2020-11-06 2023-06-22 Eli Lilly And Company Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss
EP4278002A2 (en) * 2021-01-18 2023-11-22 Life Technologies Corporation Compositions, kits and methods for direct amplification from crude biological samples
US20240197920A1 (en) * 2021-04-27 2024-06-20 The Children's Hospital Of Philadelphia Adeno-associated viral vectors for transduction of cochlea
US20250339560A1 (en) * 2021-05-13 2025-11-06 Akouos, Inc. Gene therapy delivery compositions and methods for treating hearing loss
WO2023106256A1 (ja) * 2021-12-06 2023-06-15 学校法人順天堂 改変型アデノ随伴ウイルスベクター
US20240252685A1 (en) * 2022-05-09 2024-08-01 Akouos, Inc. Cell specific gene therapy delivery compositions and methods for treating hearing loss
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
US20260103728A1 (en) * 2022-10-12 2026-04-16 The Johns Hopkins University Method of treating lbsl by enhancing dars2 expression
CN115717166B (zh) * 2022-12-15 2026-04-14 东南大学 遗传性耳聋检测系统、标志物或探针、药物及应用
EP4658759A2 (en) * 2023-02-02 2025-12-10 Agathos Biologics Baby hamster kidney (bhk) cells transformed with the adenoviral e1 gene for production of recombinant adeno-associated virus
EP4713463A1 (en) * 2023-05-18 2026-03-25 Decibel Therapeutics, Inc. Compositions and methods for treating gjb2-related hearing loss
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
CN119510774A (zh) * 2024-11-26 2025-02-25 华中科技大学同济医学院附属协和医院 Sptbn1作为靶点在治疗gjb2相关感音神经性听力损失中的应用

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IL291789A (en) 2022-06-01
US20250236887A1 (en) 2025-07-24
US12241077B2 (en) 2025-03-04
US20210095313A1 (en) 2021-04-01
AU2020357740A1 (en) 2022-04-28
WO2021067448A1 (en) 2021-04-08
EP4037771A1 (en) 2022-08-10
CN115103710A (zh) 2022-09-23
JP2022549380A (ja) 2022-11-24
EP4037771A4 (en) 2023-10-25
JP2025110904A (ja) 2025-07-29
KR20220133854A (ko) 2022-10-05
CA3153133A1 (en) 2021-04-08

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