CA3106261A1 - Methods of treating non-syndromic sensorineural hearing loss - Google Patents

Methods of treating non-syndromic sensorineural hearing loss Download PDF

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Publication number
CA3106261A1
CA3106261A1 CA3106261A CA3106261A CA3106261A1 CA 3106261 A1 CA3106261 A1 CA 3106261A1 CA 3106261 A CA3106261 A CA 3106261A CA 3106261 A CA3106261 A CA 3106261A CA 3106261 A1 CA3106261 A1 CA 3106261A1
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Canada
Prior art keywords
amino acids
amino acid
acid substitutions
amino
acids
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Pending
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CA3106261A
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English (en)
French (fr)
Inventor
Robert NG
Emmanuel J. Simons
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Akouos Inc
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Akouos Inc
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Publication of CA3106261A1 publication Critical patent/CA3106261A1/en
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    • 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
    • A61K48/005—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
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • 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
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • 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
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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
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
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    • A01K2217/07—Animals genetically altered by homologous recombination
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    • A—HUMAN NECESSITIES
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    • A01K2227/103—Ovine
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
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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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    • C12N2750/14011—Parvoviridae
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CA3106261A 2018-07-13 2019-07-12 Methods of treating non-syndromic sensorineural hearing loss Pending CA3106261A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862697652P 2018-07-13 2018-07-13
US62/697,652 2018-07-13
PCT/US2019/041625 WO2020014625A1 (en) 2018-07-13 2019-07-12 Methods of treating non-syndromic sensorineural hearing loss

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Publication Number Publication Date
CA3106261A1 true CA3106261A1 (en) 2020-01-16

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CA3106261A Pending CA3106261A1 (en) 2018-07-13 2019-07-12 Methods of treating non-syndromic sensorineural hearing loss

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US (2) US12226493B2 (enExample)
EP (1) EP3821019A4 (enExample)
JP (2) JP2021530227A (enExample)
CN (1) CN112639108A (enExample)
AU (1) AU2019300045A1 (enExample)
CA (1) CA3106261A1 (enExample)
WO (1) WO2020014625A1 (enExample)

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JP2021530227A (ja) 2018-07-13 2021-11-11 アコーオス インコーポレイテッド 非症候性感音性聴力喪失の治療方法
US12667628B2 (en) 2019-07-03 2026-06-30 Children's Medical Center Corporation Compositions and methods for gene replacement
US20230090778A1 (en) * 2020-02-07 2023-03-23 The Children's Medical Center Corporation Large gene vectors and delivery and uses thereof
WO2022026516A1 (en) * 2020-07-29 2022-02-03 The Board Of Regents Of The University Of Texas System Transgene cassettes, aav vectors, and aav viral vectors for expression of human codon-optimized cstb
CA3219054A1 (en) * 2021-05-05 2022-11-10 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using stereocilin dual vector systems
JP2024518552A (ja) * 2021-05-14 2024-05-01 レスキュー ヒヤリング インコーポレイテッド 難聴治療のための遺伝子治療用構築物および方法
WO2023064388A2 (en) * 2021-10-12 2023-04-20 Decibel Therapeutics, Inc. Stereocilin promoters and uses thereof

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JP2024518552A (ja) 2021-05-14 2024-05-01 レスキュー ヒヤリング インコーポレイテッド 難聴治療のための遺伝子治療用構築物および方法
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JP2021530227A (ja) 2021-11-11
US20210330814A1 (en) 2021-10-28
EP3821019A1 (en) 2021-05-19
CN112639108A (zh) 2021-04-09
US20260000787A1 (en) 2026-01-01
EP3821019A4 (en) 2022-10-05
AU2019300045A1 (en) 2021-01-07
JP2024109708A (ja) 2024-08-14
WO2020014625A1 (en) 2020-01-16
US12226493B2 (en) 2025-02-18

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