AU2019294603B2 - Methods of treating CLRN1-associated hearing loss and/or vision loss - Google Patents

Methods of treating CLRN1-associated hearing loss and/or vision loss

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Publication number
AU2019294603B2
AU2019294603B2 AU2019294603A AU2019294603A AU2019294603B2 AU 2019294603 B2 AU2019294603 B2 AU 2019294603B2 AU 2019294603 A AU2019294603 A AU 2019294603A AU 2019294603 A AU2019294603 A AU 2019294603A AU 2019294603 B2 AU2019294603 B2 AU 2019294603B2
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amino acids
nucleotides
amino acid
acid substitutions
seq
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AU2019294603A1 (en
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Robert NG
Emmanuel J. Simons
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Akouos Inc
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Akouos Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A61K9/0051—Ocular inserts or implants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
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    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
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  • Ophthalmology & Optometry (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
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  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Manufacturing & Machinery (AREA)
  • Acoustics & Sound (AREA)
AU2019294603A 2018-06-25 2019-06-25 Methods of treating CLRN1-associated hearing loss and/or vision loss Active AU2019294603B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2026201371A AU2026201371A1 (en) 2018-06-25 2026-02-24 Methods of treating CLRN1-associated hearing loss and/or vision loss

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862689660P 2018-06-25 2018-06-25
US62/689,660 2018-06-25
PCT/US2019/039030 WO2020005974A1 (en) 2018-06-25 2019-06-25 Methods of treating clrn1-associated hearing loss and/or vision loss

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AU2019294603B2 true AU2019294603B2 (en) 2025-11-27

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EP (1) EP3824090A4 (enExample)
JP (3) JP2021529519A (enExample)
KR (1) KR20210057720A (enExample)
CN (1) CN112639107B (enExample)
AU (2) AU2019294603B2 (enExample)
BR (1) BR112020026606A2 (enExample)
CA (1) CA3104330A1 (enExample)
CL (1) CL2020003346A1 (enExample)
EA (1) EA202190114A1 (enExample)
IL (1) IL279551A (enExample)
MX (1) MX2020013628A (enExample)
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Families Citing this family (3)

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Publication number Priority date Publication date Assignee Title
US20230340038A1 (en) * 2020-09-14 2023-10-26 President And Fellows Of Harvard College Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof
IL304006A (en) * 2020-12-29 2023-08-01 Akouos Inc Compositions and methods for treating hearing loss and/or vision loss associated with CLRN1
EP4409009A4 (en) * 2021-09-30 2025-12-17 Akouos Inc Compositions for administering gene therapy and methods for treating hearing loss

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073900A1 (en) * 2014-11-06 2016-05-12 Case Western Reserve University Compositions and methods of treating usher syndrome iii

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US8980952B2 (en) * 2002-03-20 2015-03-17 University Of Maryland, Baltimore Methods for treating brain swelling with a compound that blocks a non-selective cation channel
WO2006084243A2 (en) * 2005-02-03 2006-08-10 Biotech Institute For International Innovation, Inc. Compositions and methods for the therapeutic treatment of diabetes
CA2624615A1 (en) * 2005-10-05 2007-04-19 Bayhill Therapeutics, Inc. Compositions and methods for treatment of autoimmune disease
US20090215178A1 (en) * 2008-02-22 2009-08-27 Zequn Tang Methods to enhance the stability and homogeneity of transgene expression in clonal cell lines
JP2013511258A (ja) * 2009-11-19 2013-04-04 株式会社免疫工学研究所 所望のポリペプチドを産生する抗体産生細胞の製造方法
MX362981B (es) * 2012-03-27 2019-02-28 Curevac Ag Moleculas artificiales de acido nucleico para la expresion mejorada de proteina o peptido.
CN102978284A (zh) * 2012-11-19 2013-03-20 金子兵 一种筛查各类眼科遗传性疾病的基因芯片及其制备和使用方法
EP2970972B1 (en) * 2013-03-11 2018-08-15 Fondazione Telethon Mir-204 and mir-211 and uses thereof
AU2014255665B2 (en) * 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
BR112017026523A2 (pt) * 2015-06-12 2018-08-14 Glaxosmithkline Biologicals Sa vetor recombinante, adenovírus recombinante, composição, uso de um vetor recombinante, de um adenovírus recombinante ou de uma composição, método para induzir uma resposta imune em um indivíduo, e, polinucleotídeo isolado.
SG10201913266UA (en) * 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
EP3510161A4 (en) * 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON

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Publication number Priority date Publication date Assignee Title
WO2016073900A1 (en) * 2014-11-06 2016-05-12 Case Western Reserve University Compositions and methods of treating usher syndrome iii

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JP2021529519A (ja) 2021-11-04
JP2025156349A (ja) 2025-10-14
IL279551A (en) 2021-01-31
EP3824090A4 (en) 2022-06-01
AU2019294603A1 (en) 2021-01-14
CL2020003346A1 (es) 2021-05-14
CN112639107A (zh) 2021-04-09
CN112639107B (zh) 2025-03-04
AU2026201371A1 (en) 2026-03-19
SG11202012710WA (en) 2021-01-28
KR20210057720A (ko) 2021-05-21
JP2023153320A (ja) 2023-10-17
EP3824090A1 (en) 2021-05-26
CA3104330A1 (en) 2020-01-02
EA202190114A1 (ru) 2021-05-12
MX2020013628A (es) 2021-05-27
BR112020026606A2 (pt) 2021-04-06
US20210277417A1 (en) 2021-09-09
WO2020005974A1 (en) 2020-01-02

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