CA3233097A1 - Compositions and methods for treating kcnq4-associated hearing loss - Google Patents

Compositions and methods for treating kcnq4-associated hearing loss Download PDF

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Publication number
CA3233097A1
CA3233097A1 CA3233097A CA3233097A CA3233097A1 CA 3233097 A1 CA3233097 A1 CA 3233097A1 CA 3233097 A CA3233097 A CA 3233097A CA 3233097 A CA3233097 A CA 3233097A CA 3233097 A1 CA3233097 A1 CA 3233097A1
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CA
Canada
Prior art keywords
seq
construct
exemplified
promoter
kcnq4
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
CA3233097A
Other languages
English (en)
French (fr)
Inventor
Katherine Diane GRIBBLE
Robert NG
Emmanuel John Simons
Gregory Scott ROBINSON
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.)
Akouos Inc
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Akouos 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
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Application filed by Akouos Inc filed Critical Akouos Inc
Publication of CA3233097A1 publication Critical patent/CA3233097A1/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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3233097A 2021-09-30 2022-09-29 Compositions and methods for treating kcnq4-associated hearing loss Pending CA3233097A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163250857P 2021-09-30 2021-09-30
US63/250,857 2021-09-30
US202263305740P 2022-02-02 2022-02-02
US63/305,740 2022-02-02
US202263309061P 2022-02-11 2022-02-11
US63/309,061 2022-02-11
PCT/US2022/077222 WO2023056329A1 (en) 2021-09-30 2022-09-29 Compositions and methods for treating kcnq4-associated hearing loss

Publications (1)

Publication Number Publication Date
CA3233097A1 true CA3233097A1 (en) 2023-04-06

Family

ID=84358215

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3233097A Pending CA3233097A1 (en) 2021-09-30 2022-09-29 Compositions and methods for treating kcnq4-associated hearing loss

Country Status (15)

Country Link
US (1) US20250288692A1 (https=)
EP (1) EP4408872A1 (https=)
JP (1) JP2024537775A (https=)
KR (1) KR20240067112A (https=)
AU (1) AU2022356427A1 (https=)
CA (1) CA3233097A1 (https=)
CO (1) CO2024003631A2 (https=)
CR (1) CR20240169A (https=)
DO (1) DOP2024000054A (https=)
EC (1) ECSP24032712A (https=)
IL (1) IL311572A (https=)
JO (1) JOP20240061A1 (https=)
MX (1) MX2024003778A (https=)
PE (1) PE20241065A1 (https=)
WO (1) WO2023056329A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4695406A1 (en) * 2023-04-13 2026-02-18 F. Hoffmann-La Roche AG Improved recombinant polyadenylation signal sequences and use thereof

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Publication number Publication date
IL311572A (en) 2024-05-01
US20250288692A1 (en) 2025-09-18
WO2023056329A1 (en) 2023-04-06
JOP20240061A1 (ar) 2024-03-26
KR20240067112A (ko) 2024-05-16
PE20241065A1 (es) 2024-05-13
AU2022356427A1 (en) 2024-05-09
MX2024003778A (es) 2024-04-10
WO2023056329A8 (en) 2023-05-25
DOP2024000054A (es) 2024-06-30
CO2024003631A2 (es) 2024-04-18
CR20240169A (es) 2024-06-04
ECSP24032712A (es) 2024-05-31
JP2024537775A (ja) 2024-10-16
EP4408872A1 (en) 2024-08-07

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