CA3129422A1 - Myosin 15 promoters and uses thereof - Google Patents

Myosin 15 promoters and uses thereof Download PDF

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Publication number
CA3129422A1
CA3129422A1 CA3129422A CA3129422A CA3129422A1 CA 3129422 A1 CA3129422 A1 CA 3129422A1 CA 3129422 A CA3129422 A CA 3129422A CA 3129422 A CA3129422 A CA 3129422A CA 3129422 A1 CA3129422 A1 CA 3129422A1
Authority
CA
Canada
Prior art keywords
seq
sequence
polynucleotide
functional portion
nucleic acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3129422A
Other languages
English (en)
French (fr)
Inventor
Joseph Burns
Kathryn ELLIS
Adam Palermo
Martin Schwander
Jonathon WHITTON
Leah SABIN
Christos Kyratsous
Meghan DRUMMOND SAMUELSON
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regeneron Pharmaceuticals Inc
Decibel Therapeutics Inc
Original Assignee
Regeneron Pharmaceuticals Inc
Decibel Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regeneron Pharmaceuticals Inc, Decibel Therapeutics Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of CA3129422A1 publication Critical patent/CA3129422A1/en
Pending legal-status Critical Current

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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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CA3129422A 2019-02-08 2020-02-07 Myosin 15 promoters and uses thereof Pending CA3129422A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962802874P 2019-02-08 2019-02-08
US62/802,874 2019-02-08
US201962928311P 2019-10-30 2019-10-30
US62/928,311 2019-10-30
US202062965773P 2020-01-24 2020-01-24
US62/965,773 2020-01-24
PCT/US2020/017292 WO2020163761A1 (en) 2019-02-08 2020-02-07 Myosin 15 promoters and uses thereof

Publications (1)

Publication Number Publication Date
CA3129422A1 true CA3129422A1 (en) 2020-08-13

Family

ID=71947522

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3129422A Pending CA3129422A1 (en) 2019-02-08 2020-02-07 Myosin 15 promoters and uses thereof

Country Status (7)

Country Link
US (2) US12252520B2 (https=)
EP (1) EP3920974A4 (https=)
JP (3) JP7562544B2 (https=)
CN (3) CN119320772A (https=)
AU (1) AU2020218547A1 (https=)
CA (1) CA3129422A1 (https=)
WO (1) WO2020163761A1 (https=)

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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
CA3159549A1 (en) * 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
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
CN112194717B (zh) * 2020-09-30 2021-12-14 东南大学 一种促进耳蜗毛细胞再生的肽段Tr及其应用
JP2025504263A (ja) 2021-12-23 2025-02-10 ユニバーシティ オブ ロチェスター 内耳への薬剤の送達のためのｃｓｆ輸送経路
JP2025503508A (ja) 2021-12-23 2025-02-04 ユニバーシティ オブ ロチェスター 内耳への薬剤の送達のための組成物及び方法
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
WO2024250774A1 (zh) * 2023-06-07 2024-12-12 苏州星奥拓维生物技术有限公司 耳蜗外毛细胞特异性启动子及其应用
CN117486995A (zh) * 2023-11-03 2024-02-02 首都医科大学附属北京胸科医院 Kncn蛋白在结核诊断试剂中的应用

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Also Published As

Publication number Publication date
JP2025000750A (ja) 2025-01-07
CN114072179B (zh) 2024-11-01
JP2026020285A (ja) 2026-02-06
JP2022519750A (ja) 2022-03-24
AU2020218547A1 (en) 2021-08-26
JP7778879B2 (ja) 2025-12-02
CN119307494A (zh) 2025-01-14
EP3920974A1 (en) 2021-12-15
US20250304637A1 (en) 2025-10-02
JP7562544B2 (ja) 2024-10-07
US20210388045A1 (en) 2021-12-16
CN114072179A (zh) 2022-02-18
US12252520B2 (en) 2025-03-18
WO2020163761A1 (en) 2020-08-13
EP3920974A4 (en) 2022-11-30
CN119320772A (zh) 2025-01-17

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