CA3098198A1 - Myosin 15 promoters and uses thereof - Google Patents

Myosin 15 promoters and uses thereof Download PDF

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Publication number
CA3098198A1
CA3098198A1 CA3098198A CA3098198A CA3098198A1 CA 3098198 A1 CA3098198 A1 CA 3098198A1 CA 3098198 A CA3098198 A CA 3098198A CA 3098198 A CA3098198 A CA 3098198A CA 3098198 A1 CA3098198 A1 CA 3098198A1
Authority
CA
Canada
Prior art keywords
seq
sequence
polynucleotide
nucleic acid
functional portion
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
CA3098198A
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English (en)
French (fr)
Inventor
Joseph Burns
Kathryn ELLIS
Adam Palermo
Martin Schwander
Jonathon WHITTON
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.)
Decibel Therapeutics Inc
Original Assignee
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 Decibel Therapeutics Inc filed Critical Decibel Therapeutics Inc
Publication of CA3098198A1 publication Critical patent/CA3098198A1/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
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0625—Epidermal cells, skin cells; Cells of the oral mucosa
    • C12N5/0627—Hair 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Cell Biology (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3098198A 2018-04-27 2019-04-26 Myosin 15 promoters and uses thereof Pending CA3098198A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862663679P 2018-04-27 2018-04-27
US62/663,679 2018-04-27
PCT/US2019/029366 WO2019210181A1 (en) 2018-04-27 2019-04-26 Myosin 15 promoters and uses thereof

Publications (1)

Publication Number Publication Date
CA3098198A1 true CA3098198A1 (en) 2019-10-31

Family

ID=68294246

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3098198A Pending CA3098198A1 (en) 2018-04-27 2019-04-26 Myosin 15 promoters and uses thereof

Country Status (6)

Country Link
US (2) US12233136B2 (https=)
JP (3) JP7331011B2 (https=)
CN (3) CN112703016B (https=)
AU (1) AU2019257782B2 (https=)
CA (1) CA3098198A1 (https=)
WO (1) WO2019210181A1 (https=)

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EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
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
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
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
KR20220130093A (ko) * 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
WO2021091938A1 (en) * 2019-11-04 2021-05-14 Decibel Therapeutics, Inc. Cochlear outer hair cell promoters and uses thereof
EP4055175A4 (en) 2019-11-04 2023-11-29 Decibel Therapeutics, Inc. Cochlear inner hair cell promoters and uses thereof
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
WO2021222682A1 (en) * 2020-04-30 2021-11-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Application of aav44.9 vector in gene therapy for the inner ear
WO2023147383A1 (en) * 2022-01-25 2023-08-03 Bridgebio Services Inc. Gene therapy for genetic hearing loss

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Also Published As

Publication number Publication date
CN112703016B (zh) 2024-11-22
JP2025122095A (ja) 2025-08-20
WO2019210181A1 (en) 2019-10-31
US20250319205A1 (en) 2025-10-16
EP3784289A1 (en) 2021-03-03
AU2019257782A1 (en) 2020-11-19
CN119410637A (zh) 2025-02-11
US12233136B2 (en) 2025-02-25
CN119410636A (zh) 2025-02-11
JP2021521842A (ja) 2021-08-30
US20210236654A1 (en) 2021-08-05
AU2019257782B2 (en) 2025-11-20
JP2023138741A (ja) 2023-10-02
JP7686709B2 (ja) 2025-06-02
JP7331011B2 (ja) 2023-08-22
CN112703016A (zh) 2021-04-23

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