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MX2022014204A - Composiciones y metodos para tratar la perdida auditiva asociada al gen slc26a4. - Google Patents

Composiciones y metodos para tratar la perdida auditiva asociada al gen slc26a4.

Info

Publication number
MX2022014204A
MX2022014204A MX2022014204A MX2022014204A MX2022014204A MX 2022014204 A MX2022014204 A MX 2022014204A MX 2022014204 A MX2022014204 A MX 2022014204A MX 2022014204 A MX2022014204 A MX 2022014204A MX 2022014204 A MX2022014204 A MX 2022014204A
Authority
MX
Mexico
Prior art keywords
methods
hearing loss
slc26a4
compositions
treating
Prior art date
Application number
MX2022014204A
Other languages
English (en)
Inventor
Robert Ng
Emmanuel John Simons
Yen-Fu Cheng
Original Assignee
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
Publication date
Application filed by Akouos Inc filed Critical Akouos Inc
Publication of MX2022014204A publication Critical patent/MX2022014204A/es

Links

Classifications

    • 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
    • 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
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)

Abstract

La presente invención se refiere a constructos que comprenden una secuencia codificante enlazada operativamente a un promotor, en donde la secuencia codificante codifica una proteína pendrina. Los constructos ejemplares incluyen constructos de AAV. También se proporcionan métodos de uso de constructos descritos para el tratamiento de la pérdida auditiva y/o sordera.
MX2022014204A 2020-05-13 2021-05-12 Composiciones y metodos para tratar la perdida auditiva asociada al gen slc26a4. MX2022014204A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063024466P 2020-05-13 2020-05-13
PCT/US2021/031983 WO2021231567A2 (en) 2020-05-13 2021-05-12 Compositions and methods for treating slc26a4-associated hearing loss

Publications (1)

Publication Number Publication Date
MX2022014204A true MX2022014204A (es) 2023-04-14

Family

ID=76584538

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022014204A MX2022014204A (es) 2020-05-13 2021-05-12 Composiciones y metodos para tratar la perdida auditiva asociada al gen slc26a4.

Country Status (14)

Country Link
US (1) US20230201372A1 (es)
EP (1) EP4165195A2 (es)
JP (1) JP2023526047A (es)
KR (1) KR20230041965A (es)
CN (1) CN116801911A (es)
AR (1) AR122078A1 (es)
AU (1) AU2021272971A1 (es)
BR (1) BR112022022906A2 (es)
CA (1) CA3183171A1 (es)
CL (1) CL2022003153A1 (es)
IL (1) IL298128A (es)
MX (1) MX2022014204A (es)
TW (1) TW202208405A (es)
WO (1) WO2021231567A2 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4453220A1 (en) * 2021-12-23 2024-10-30 University of Rochester Compositions and methods for delivery of agents to inner ear

Family Cites Families (34)

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US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
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AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
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WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
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ES2624854T3 (es) 2001-05-31 2017-07-17 Glaxosmithkline Biologicals Sa Partículas de replicón de alfavirus quimérico
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US20190038778A1 (en) * 2016-02-05 2019-02-07 The General Hospital Corporation Hybrid System for Efficient Gene Delivery to Cells of the Inner Ear
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CN113365603A (zh) * 2018-10-11 2021-09-07 分贝治疗公司 Aav1载体及其用于治疗耳部适应症的用途

Also Published As

Publication number Publication date
IL298128A (en) 2023-01-01
US20230201372A1 (en) 2023-06-29
CL2022003153A1 (es) 2023-07-28
TW202208405A (zh) 2022-03-01
JP2023526047A (ja) 2023-06-20
WO2021231567A3 (en) 2021-12-23
KR20230041965A (ko) 2023-03-27
BR112022022906A2 (pt) 2023-01-17
AR122078A1 (es) 2022-08-10
AU2021272971A1 (en) 2023-02-02
WO2021231567A2 (en) 2021-11-18
CA3183171A1 (en) 2021-11-18
EP4165195A2 (en) 2023-04-19
CN116801911A (zh) 2023-09-22

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