MX2022005236A - Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina. - Google Patents

Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina.

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Publication number
MX2022005236A
MX2022005236A MX2022005236A MX2022005236A MX2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A
Authority
MX
Mexico
Prior art keywords
compositions
hearing loss
sensorineural hearing
otoferlin
methods
Prior art date
Application number
MX2022005236A
Other languages
English (en)
Inventor
Christos Kyratsous
Adam Palermo
Leah Sabin
Samuelson Meghan Drummond
Joseph Burns
Kathryn Ellis
Tyler Gibson
Martin Schwander
Jonathon Whitton
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 MX2022005236A publication Critical patent/MX2022005236A/es

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    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Abstract

La divulgación presenta composiciones y métodos para el tratamiento de la pérdida auditiva neurosensorial y la neuropatía auditiva, en particular las formas de la enfermedad que están asociadas con una mutación en la otoferlina (OTOF), por medio de la terapia génica OTOF. La divulgación proporciona una variedad de composiciones que incluyen un primer vector de ácido nucleico que contiene un polinucleótido que codifica una parte de extremo N de una proteína de la isoforma 5 de OTOF y un segundo vector de ácido nucleico que contiene un polinucleótido que codifica una parte de extremo C de una proteína de la isoforma 5 de OTOF. Estos vectores pueden usarse para aumentar la expresión de OTOF en un sujeto, tal como un sujeto humano que padece pérdida auditiva neurosensorial.
MX2022005236A 2019-10-30 2020-10-30 Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina. MX2022005236A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962928290P 2019-10-30 2019-10-30
US202062965776P 2020-01-24 2020-01-24
US202062971504P 2020-02-07 2020-02-07
US202063023058P 2020-05-11 2020-05-11
PCT/US2020/058265 WO2021087296A1 (en) 2019-10-30 2020-10-30 Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems

Publications (1)

Publication Number Publication Date
MX2022005236A true MX2022005236A (es) 2022-06-08

Family

ID=75716489

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022005236A MX2022005236A (es) 2019-10-30 2020-10-30 Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina.

Country Status (11)

Country Link
US (1) US20220265865A1 (es)
EP (1) EP4051698A4 (es)
JP (1) JP2022554300A (es)
KR (1) KR20220130093A (es)
AU (1) AU2020375947A1 (es)
BR (1) BR112022008214A2 (es)
CA (1) CA3159549A1 (es)
IL (1) IL292620A (es)
MX (1) MX2022005236A (es)
TW (1) TW202130654A (es)
WO (1) WO2021087296A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
AU2021225035A1 (en) 2020-02-21 2022-10-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
WO2023150689A2 (en) * 2022-02-04 2023-08-10 Decible Therapeutics, Inc. Gjb2 regulatory elements and uses thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11167042B2 (en) * 2015-12-11 2021-11-09 Massachusetts Eye And Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP2019530737A (ja) * 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
CN110892062A (zh) * 2017-05-05 2020-03-17 佛罗里达大学研究基金会 表达耳畸蛋白的组合物和方法
AU2019224121A1 (en) * 2018-02-22 2020-08-27 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
AU2019257782A1 (en) * 2018-04-27 2020-11-19 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
US11660353B2 (en) * 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2020163743A1 (en) * 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems

Also Published As

Publication number Publication date
KR20220130093A (ko) 2022-09-26
BR112022008214A2 (pt) 2022-07-12
CA3159549A1 (en) 2021-05-06
IL292620A (en) 2022-07-01
TW202130654A (zh) 2021-08-16
WO2021087296A1 (en) 2021-05-06
AU2020375947A1 (en) 2022-05-26
WO2021087296A8 (en) 2021-08-05
US20220265865A1 (en) 2022-08-25
EP4051698A1 (en) 2022-09-07
EP4051698A4 (en) 2024-03-27
JP2022554300A (ja) 2022-12-28

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