TW202227476A - 編碼GJB2之重組腺相關病毒(rAAV)及其用途 - Google Patents

編碼GJB2之重組腺相關病毒(rAAV)及其用途 Download PDF

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Publication number
TW202227476A
TW202227476A TW110134291A TW110134291A TW202227476A TW 202227476 A TW202227476 A TW 202227476A TW 110134291 A TW110134291 A TW 110134291A TW 110134291 A TW110134291 A TW 110134291A TW 202227476 A TW202227476 A TW 202227476A
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gjb2
nucleic acid
isolated nucleic
cells
seq
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TW110134291A
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Chinese (zh)
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大衛 Ｐ 柯瑞
凱文 Ｔ 布斯
柯爾 Ｗ Ｄ 彼得斯
馬里納 Ｖ 伊凡奇安科
麥克 Ｅ 葛林柏格
席尼薩 哈法汀
馬克 奧瑞爾 納吉
艾瑞克 Ｃ 葛里菲斯
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哈佛大學校長及研究員協會
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Publication of TW202227476A publication Critical patent/TW202227476A/zh

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    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (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)
TW110134291A 2020-09-14 2021-09-14 編碼GJB2之重組腺相關病毒(rAAV)及其用途 TW202227476A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063078233P 2020-09-14 2020-09-14
US63/078,233 2020-09-14
US202163161619P 2021-03-16 2021-03-16
US63/161,619 2021-03-16

Publications (1)

Publication Number Publication Date
TW202227476A true TW202227476A (zh) 2022-07-16

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TW110134291A TW202227476A (zh) 2020-09-14 2021-09-14 編碼GJB2之重組腺相關病毒(rAAV)及其用途

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Country Link
EP (1) EP4211151A4 (https=)
JP (1) JP2023541443A (https=)
KR (1) KR20230069157A (https=)
AU (1) AU2021339843A1 (https=)
BR (1) BR112023004605A2 (https=)
CA (1) CA3191533A1 (https=)
IL (1) IL301057A (https=)
MX (1) MX2023002978A (https=)
TW (1) TW202227476A (https=)
WO (1) WO2022056444A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021067448A1 (en) * 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss
WO2024011224A2 (en) * 2022-07-08 2024-01-11 The Trustees Of Columbia University In The City Of New York Regulatory element for cell type specific expression of genes in spinal motor neurons
WO2024218192A1 (en) * 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
EP4713463A1 (en) * 2023-05-18 2026-03-25 Decibel Therapeutics, Inc. Compositions and methods for treating gjb2-related hearing loss

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1308458A1 (en) * 2001-10-30 2003-05-07 GenOdyssee New polynucleotides and polypeptides of the CX26 gene
DE602005019174D1 (de) * 2004-06-17 2010-03-18 Epigenomics Ag Zusammensetzungen und verfahren zur verhinderung des mitschleppens einer kontamination in nukleinsäure-amplifikationsreaktionen
CN101389769A (zh) * 2005-12-28 2009-03-18 森托科尔公司 用于评价和治疗银屑病及相关疾病的标记与方法
WO2019200016A1 (en) * 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
WO2020077295A1 (en) * 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
EP4063510A1 (en) * 2018-11-07 2022-09-28 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
WO2021067448A1 (en) * 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss
WO2021231808A2 (en) * 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating gjb2-associated hearing loss
MX2023013394A (es) * 2021-05-13 2023-12-14 Akouos Inc Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva.

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CA3191533A1 (en) 2022-03-17
EP4211151A1 (en) 2023-07-19
MX2023002978A (es) 2023-06-01
KR20230069157A (ko) 2023-05-18
AU2021339843A1 (en) 2023-04-06
JP2023541443A (ja) 2023-10-02
EP4211151A4 (en) 2025-03-26
IL301057A (en) 2023-05-01
BR112023004605A2 (pt) 2023-04-11
WO2022056444A1 (en) 2022-03-17

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