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
    • 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
    • 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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    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/11—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/103—Ovine
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
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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
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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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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ID=69142651

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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 (https=)
EP (1) EP3821019A4 (https=)
JP (2) JP2021530227A (https=)
CN (1) CN112639108A (https=)
AU (1) AU2019300045A1 (https=)
CA (1) CA3106261A1 (https=)
WO (1) WO2020014625A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020014625A1 (en) 2018-07-13 2020-01-16 Akouos, Inc. Methods of treating non-syndromic sensorineural hearing loss
JP7846626B2 (ja) * 2020-02-07 2026-04-15 ザ チルドレンズ メディカル センター コーポレーション 大型遺伝子ベクターならびにその送達および使用の方法
US11946065B2 (en) * 2020-07-29 2024-04-02 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
JP2024517843A (ja) * 2021-05-05 2024-04-23 デシベル セラピューティクス インコーポレイテッド ステレオシリン二重ベクター系を使用して感音性難聴を治療するための組成物及び方法
IL308328A (en) * 2021-05-14 2024-01-01 Rescue Hearing Inc Structures and methods of gene therapy to treat hearing loss
EP4416294A2 (en) * 2021-10-12 2024-08-21 Decibel Therapeutics, Inc. Stereocilin promoters and uses thereof

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WO2020014625A1 (en) 2018-07-13 2020-01-16 Akouos, Inc. Methods of treating non-syndromic sensorineural hearing loss
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JP7846626B2 (ja) 2020-02-07 2026-04-15 ザ チルドレンズ メディカル センター コーポレーション 大型遺伝子ベクターならびにその送達および使用の方法
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JP2024517843A (ja) 2021-05-05 2024-04-23 デシベル セラピューティクス インコーポレイテッド ステレオシリン二重ベクター系を使用して感音性難聴を治療するための組成物及び方法
IL308328A (en) 2021-05-14 2024-01-01 Rescue Hearing Inc Structures and methods of gene therapy to treat hearing loss
EP4416294A2 (en) 2021-10-12 2024-08-21 Decibel Therapeutics, Inc. Stereocilin promoters and uses thereof
JP2025504263A (ja) 2021-12-23 2025-02-10 ユニバーシティ オブ ロチェスター 内耳への薬剤の送達のためのｃｓｆ輸送経路
WO2024151703A2 (en) 2023-01-12 2024-07-18 Ohio State Innovation Foundation Gene therapy for treating hearing loss disorders

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CN112639108A (zh) 2021-04-09
JP2021530227A (ja) 2021-11-11
US12226493B2 (en) 2025-02-18
AU2019300045A1 (en) 2021-01-07
US20260000787A1 (en) 2026-01-01
US20210330814A1 (en) 2021-10-28
EP3821019A4 (en) 2022-10-05
EP3821019A1 (en) 2021-05-19
JP2024109708A (ja) 2024-08-14
WO2020014625A1 (en) 2020-01-16

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