AR122078A1 - Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4 - Google Patents

Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4

Info

Publication number
AR122078A1
AR122078A1 ARP210101313A ARP210101313A AR122078A1 AR 122078 A1 AR122078 A1 AR 122078A1 AR P210101313 A ARP210101313 A AR P210101313A AR P210101313 A ARP210101313 A AR P210101313A AR 122078 A1 AR122078 A1 AR 122078A1
Authority
AR
Argentina
Prior art keywords
methods
hearing loss
slc26a4
compositions
treating
Prior art date
Application number
ARP210101313A
Other languages
English (en)
Inventor
Emmanuel John Simons
Robert Ng
Yen-Fu Cheng
Original Assignee
Akouos Inc
Taipei Veterans General Hospital
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, Taipei Veterans General Hospital filed Critical Akouos Inc
Publication of AR122078A1 publication Critical patent/AR122078A1/es

Links

Classifications

    • 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
    • A61K48/005—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
    • A61K48/0075—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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic 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.
ARP210101313A 2020-05-13 2021-05-12 Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4 AR122078A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063024466P 2020-05-13 2020-05-13

Publications (1)

Publication Number Publication Date
AR122078A1 true AR122078A1 (es) 2022-08-10

Family

ID=76584538

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210101313A AR122078A1 (es) 2020-05-13 2021-05-12 Composiciones y métodos para tratar la pérdida auditiva asociada a 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
WO2023122720A1 (en) * 2021-12-23 2023-06-29 University Of Rochester Compositions and methods for delivery of agents to inner ear

Family Cites Families (34)

* Cited by examiner, † Cited by third party
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EP0173552B1 (en) 1984-08-24 1991-10-09 The Upjohn Company Recombinant dna compounds and the expression of polypeptides such as tpa
US5168062A (en) 1985-01-30 1992-12-01 University Of Iowa Research Foundation Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
WO1998046728A1 (en) 1997-04-14 1998-10-22 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
AU2001268149B2 (en) 2000-06-01 2005-08-18 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
EP1399183B1 (en) 2001-05-31 2010-06-30 Novartis Vaccines and Diagnostics, Inc. Chimeric alphavirus replicon particles
AU2002327614B2 (en) 2001-09-06 2007-12-06 Alphavax, Inc. Alphavirus replicon vector systems
NZ600121A (en) 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
SI2292780T1 (sl) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba
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EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
AU2013240248B2 (en) 2012-03-26 2018-12-20 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Delivery of packaged RNA to mammalian cells
GB201518979D0 (en) 2015-10-27 2015-12-09 Univ Leuven Kath Treatment of hepatic steatosis related oligo-ovulation
US20190038778A1 (en) * 2016-02-05 2019-02-07 The General Hospital Corporation Hybrid System for Efficient Gene Delivery to Cells of the Inner Ear
CN110225975A (zh) 2016-08-23 2019-09-10 阿库斯股份有限公司 用于治疗人受试者中非年龄相关的听力损害的组合物和方法
EA202091995A1 (ru) 2018-02-22 2021-03-30 Акуос, Инк. Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека
KR20210113160A (ko) * 2018-10-11 2021-09-15 데시벨 테라퓨틱스, 인크. Aav1 벡터 및 귀 적응증의 치료를 위한 이의 용도

Also Published As

Publication number Publication date
WO2021231567A2 (en) 2021-11-18
WO2021231567A3 (en) 2021-12-23
BR112022022906A2 (pt) 2023-01-17
IL298128A (en) 2023-01-01
CN116801911A (zh) 2023-09-22
MX2022014204A (es) 2023-04-14
US20230201372A1 (en) 2023-06-29
CA3183171A1 (en) 2021-11-18
CL2022003153A1 (es) 2023-07-28
EP4165195A2 (en) 2023-04-19
TW202208405A (zh) 2022-03-01
JP2023526047A (ja) 2023-06-20
KR20230041965A (ko) 2023-03-27
AU2021272971A1 (en) 2023-02-02

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