CA3104330A1 - Methods of treating clrn1-associated hearing loss and/or vision loss - Google Patents

Methods of treating clrn1-associated hearing loss and/or vision loss Download PDF

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Publication number
CA3104330A1
CA3104330A1 CA3104330A CA3104330A CA3104330A1 CA 3104330 A1 CA3104330 A1 CA 3104330A1 CA 3104330 A CA3104330 A CA 3104330A CA 3104330 A CA3104330 A CA 3104330A CA 3104330 A1 CA3104330 A1 CA 3104330A1
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composition
nucleotides
amino acids
seq
sequence
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CA3104330A
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English (en)
French (fr)
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Emmanuel J. Simons
Robert NG
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Akouos Inc
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Akouos Inc
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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
    • C—CHEMISTRY; METALLURGY
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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
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/0012—Galenical forms characterised by the site of application
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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
    • A—HUMAN NECESSITIES
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • 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
    • C12N5/0618—Cells of the nervous system
    • C12N5/062—Sensory transducers, e.g. photoreceptors; Sensory neurons, e.g. for hearing, taste, smell, pH, touch, temperature, pain
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    • 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
    • C12N5/0618—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
    • 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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  • Health & Medical Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
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  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Manufacturing & Machinery (AREA)
  • Analytical Chemistry (AREA)
CA3104330A 2018-06-25 2019-06-25 Methods of treating clrn1-associated hearing loss and/or vision loss Pending CA3104330A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3104330A1 true CA3104330A1 (en) 2020-01-02

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Family Applications (1)

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CA3104330A Pending CA3104330A1 (en) 2018-06-25 2019-06-25 Methods of treating clrn1-associated hearing loss and/or vision loss

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US (1) US20210277417A1 (enExample)
EP (1) EP3824090A4 (enExample)
JP (3) JP2021529519A (enExample)
KR (2) 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)
SG (1) SG11202012710WA (enExample)
WO (1) WO2020005974A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
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
CN117157108A (zh) * 2020-12-29 2023-12-01 阿库斯股份有限公司 用于治疗clrn1相关听力损失和/或视力损失的组合物和方法
JP2024537796A (ja) * 2021-09-30 2024-10-16 アコーオス インコーポレイテッド 難聴を治療するための遺伝子療法送達組成物及び方法

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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
JP2008529498A (ja) * 2005-02-03 2008-08-07 バイオテック インスティテュート フォー インターナショナル イノベーション，インク． 糖尿病治療用組成物類および方法
US20100160415A1 (en) * 2005-10-05 2010-06-24 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
US20130071881A1 (en) * 2009-11-19 2013-03-21 Immuno Tec Laboratory Co. Ltd Methods for producing antibody-producing cells that produce desired polypeptides
RU2658490C2 (ru) * 2012-03-27 2018-06-21 Кьюрвак Аг Искусственные молекулы нуклеиновых кислот для улучшенной экспрессии белков или пептидов
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
CN105209619B (zh) * 2013-03-11 2018-10-12 泰莱托恩基金会 miR-204和miR-211及其用途
DK2986635T3 (en) * 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
WO2016073900A1 (en) * 2014-11-06 2016-05-12 Case Western Reserve University Compositions and methods of treating usher syndrome iii
WO2016198621A1 (en) * 2015-06-12 2016-12-15 Glaxosmithkline Biologicals Sa Adenovirus polynucleotides and polypeptides
CA3007476A1 (en) * 2015-12-11 2017-06-15 Massachusetts Eye And Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2018039375A1 (en) * 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject

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Publication number Publication date
JP2023153320A (ja) 2023-10-17
KR20260073466A (ko) 2026-05-21
CN112639107B (zh) 2025-03-04
CL2020003346A1 (es) 2021-05-14
AU2019294603A1 (en) 2021-01-14
BR112020026606A2 (pt) 2021-04-06
CN112639107A (zh) 2021-04-09
WO2020005974A1 (en) 2020-01-02
KR20210057720A (ko) 2021-05-21
US20210277417A1 (en) 2021-09-09
AU2019294603B2 (en) 2025-11-27
EP3824090A1 (en) 2021-05-26
MX2020013628A (es) 2021-05-27
EP3824090A4 (en) 2022-06-01
JP2025156349A (ja) 2025-10-14
JP2021529519A (ja) 2021-11-04
AU2026201371A1 (en) 2026-03-19
IL279551A (en) 2021-01-31
SG11202012710WA (en) 2021-01-28
EA202190114A1 (ru) 2021-05-12

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