CL2023003260A1 - Composiciones y métodos para el tratamiento de la hipoacusia neurosensorial mediante sistemas de vector dual de estereocilina. - Google Patents

Composiciones y métodos para el tratamiento de la hipoacusia neurosensorial mediante sistemas de vector dual de estereocilina.

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Publication number
CL2023003260A1
CL2023003260A1 CL2023003260A CL2023003260A CL2023003260A1 CL 2023003260 A1 CL2023003260 A1 CL 2023003260A1 CL 2023003260 A CL2023003260 A CL 2023003260A CL 2023003260 A CL2023003260 A CL 2023003260A CL 2023003260 A1 CL2023003260 A1 CL 2023003260A1
Authority
CL
Chile
Prior art keywords
stereocilin
hearing loss
treatment
vector systems
compositions
Prior art date
Application number
CL2023003260A
Other languages
English (en)
Inventor
Burns Joseph
Wu Xudong
Becker Lars
Pan Ning
Martin Schwander
Tyler Gibson
Original Assignee
Decibel Therapeutics Inc
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 Decibel Therapeutics Inc filed Critical Decibel Therapeutics Inc
Publication of CL2023003260A1 publication Critical patent/CL2023003260A1/es

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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
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4728—Calcium binding proteins, e.g. calmodulin
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • A01K2227/105—Murine
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • 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
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

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  • Health & Medical Sciences (AREA)
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  • Public Health (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Peptides Or Proteins (AREA)
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  • Electroluminescent Light Sources (AREA)
  • Luminescent Compositions (AREA)
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Abstract

The disclosure provides compositions containing polynucleotides that encode a stereocilin protein under regulatory control of an outer hair cell-specific promoter, as well as two-vector systems containing the same, that can be used to promote expression of stereocilin specifically in outer hair cells. Additionally, the compositions described herein may be used for the treatment of subjects having or at risk of developing hearing loss, such as hearing loss associated with a mutation in stereocilin.
CL2023003260A 2021-05-05 2023-11-02 Composiciones y métodos para el tratamiento de la hipoacusia neurosensorial mediante sistemas de vector dual de estereocilina. CL2023003260A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163184737P 2021-05-05 2021-05-05

Publications (1)

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CL2023003260A1 true CL2023003260A1 (es) 2024-05-31

Family

ID=83932494

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CL2023003260A CL2023003260A1 (es) 2021-05-05 2023-11-02 Composiciones y métodos para el tratamiento de la hipoacusia neurosensorial mediante sistemas de vector dual de estereocilina.

Country Status (13)

Country Link
US (1) US20240216540A1 (es)
EP (1) EP4333905A4 (es)
JP (1) JP2024517843A (es)
KR (1) KR20240021166A (es)
CN (1) CN117750983A (es)
AU (1) AU2022271265A1 (es)
BR (1) BR112023022833A2 (es)
CA (1) CA3219054A1 (es)
CL (1) CL2023003260A1 (es)
CO (1) CO2023016804A2 (es)
IL (1) IL308142A (es)
MX (1) MX2023013053A (es)
WO (1) WO2022235933A2 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3821019A4 (en) 2018-07-13 2022-10-05 Akouos, Inc. METHODS OF TREATMENT OF NONSYNDROMIC INNER EAR DEAF
US20220054640A1 (en) * 2018-12-11 2022-02-24 Decibel Therapeutics, Inc. Compositions and methods for the delivery of therapeutic agents across the round window membrane
AU2022364798A1 (en) * 2021-10-12 2024-05-02 Decibel Therapeutics, Inc. Stereocilin promoters and uses thereof
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
US20260027327A1 (en) 2024-07-26 2026-01-29 Regeneron Pharmaceuticals, Inc. Otic drug delivery system and methods

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090018031A1 (en) * 2006-12-07 2009-01-15 Switchgear Genomics Transcriptional regulatory elements of biological pathways tools, and methods
EP3821019A4 (en) * 2018-07-13 2022-10-05 Akouos, Inc. METHODS OF TREATMENT OF NONSYNDROMIC INNER EAR DEAF
WO2020077295A1 (en) * 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
EP3880827A4 (en) * 2018-11-07 2023-04-26 Akouos, Inc. COMPOSITIONS AND METHODS FOR INDUCING DIFFERENTIATION OF A HAIR CELL
EP3921032A4 (en) * 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS

Also Published As

Publication number Publication date
AU2022271265A1 (en) 2023-12-14
CN117750983A (zh) 2024-03-22
CA3219054A1 (en) 2022-11-10
JP2024517843A (ja) 2024-04-23
CO2023016804A2 (es) 2024-02-26
WO2022235933A8 (en) 2023-02-02
IL308142A (en) 2023-12-01
EP4333905A4 (en) 2025-03-26
WO2022235933A2 (en) 2022-11-10
EP4333905A2 (en) 2024-03-13
KR20240021166A (ko) 2024-02-16
AU2022271265A9 (en) 2024-01-25
WO2022235933A3 (en) 2022-12-29
MX2023013053A (es) 2024-01-12
BR112023022833A2 (pt) 2024-02-15
US20240216540A1 (en) 2024-07-04

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