MX2020008763A - Composiciones y metodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano. - Google Patents

Composiciones y metodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano.

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Publication number
MX2020008763A
MX2020008763A MX2020008763A MX2020008763A MX2020008763A MX 2020008763 A MX2020008763 A MX 2020008763A MX 2020008763 A MX2020008763 A MX 2020008763A MX 2020008763 A MX2020008763 A MX 2020008763A MX 2020008763 A MX2020008763 A MX 2020008763A
Authority
MX
Mexico
Prior art keywords
compositions
age
methods
human subject
hearing impairment
Prior art date
Application number
MX2020008763A
Other languages
English (en)
Spanish (es)
Inventor
Robert Ng
Emmanuel John Simons
Ellen Reisinger
Hanan Al-Moyed
Sebastian Kügler
Original Assignee
Akouos 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 Akouos Inc filed Critical Akouos Inc
Publication of MX2020008763A publication Critical patent/MX2020008763A/es

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    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276Knock-out vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0046Ear
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/513Organic macromolecular compounds; Dendrimers
    • A61K9/5146Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A61K9/5153Polyesters, e.g. poly(lactide-co-glycolide)
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2840/44Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

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MX2020008763A 2018-02-22 2019-02-22 Composiciones y metodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano. MX2020008763A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862634088P 2018-02-22 2018-02-22
PCT/US2019/019268 WO2019165292A1 (en) 2018-02-22 2019-02-22 Compositions and methods for treating non-age-associated hearing impairment in a human subject

Publications (1)

Publication Number Publication Date
MX2020008763A true MX2020008763A (es) 2021-02-15

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MX2020008763A MX2020008763A (es) 2018-02-22 2019-02-22 Composiciones y metodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano.
MX2025012429A MX2025012429A (es) 2018-02-22 2020-08-21 Composiciones y metodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano

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MX2025012429A MX2025012429A (es) 2018-02-22 2020-08-21 Composiciones y metodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano

Country Status (15)

Country Link
US (2) US20200390905A1 (https=)
EP (1) EP3755805A4 (https=)
JP (2) JP7408228B2 (https=)
KR (2) KR20260015297A (https=)
CN (1) CN112020561A (https=)
AU (2) AU2019224121B2 (https=)
BR (1) BR112020016919A2 (https=)
CA (1) CA3091676A1 (https=)
CL (1) CL2020002161A1 (https=)
EA (1) EA202091995A1 (https=)
IL (2) IL325122A (https=)
MA (1) MA51918A (https=)
MX (2) MX2020008763A (https=)
SG (1) SG11202007491UA (https=)
WO (1) WO2019165292A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
EP4295913A3 (en) * 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
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
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
IL298128A (en) 2020-05-13 2023-01-01 Akouos Inc Compositions and methods for treating slc26a4-associated hearing loss
IL304006A (en) 2020-12-29 2023-08-01 Akouos Inc Compositions and methods for treating hearing loss and/or vision loss associated with CLRN1
EP4294460A4 (en) * 2021-02-19 2025-06-25 Decibel Therapeutics, Inc. Methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN113063944B (zh) * 2021-03-05 2023-03-24 李朴 血清gsdme在b淋巴细胞白血病诊断、疗效监测及预后评估上的用途
CN113337507B (zh) * 2021-08-05 2021-11-12 首都医科大学附属北京友谊医院 Otof 1273 (C>T)基因定点突变的耳聋小鼠模型的构建方法及其应用
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
CN114807224B (zh) * 2022-04-25 2023-09-29 中国人民解放军军事科学院军事医学研究院 一种可视化斑马鱼胆汁酸代谢模型的构建方法及其应用
CN114854762A (zh) * 2022-06-09 2022-08-05 上海鼎新基因科技有限公司 编码人otof的核酸序列及其应用
CN115141896A (zh) * 2022-09-04 2022-10-04 上海鼎新基因科技有限公司 一种AAV组织分布的qPCR检测方法
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
CN117305367A (zh) * 2023-08-21 2023-12-29 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4445701A (en) * 2000-03-24 2001-10-03 Centre National De La Recherche Scientifique-Cnrs Multiple human and mouse otoferlin isoforms
US8173792B2 (en) * 2007-02-09 2012-05-08 The Board Of Trustees Of The Leland Stanford Junior University Method for regulating protein function in cells using synthetic small molecules
HK1220488A1 (zh) * 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
AU2014255665B2 (en) * 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
SI3265571T1 (sl) * 2015-03-03 2022-10-28 Fondazione Telethon Več vektorski sistem in njegove uporabe
EP3510161A4 (en) * 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
KR20220157944A (ko) * 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법

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SG11202007491UA (en) 2020-09-29
JP2021513863A (ja) 2021-06-03
IL276764B1 (en) 2026-01-01
MX2025012429A (es) 2025-11-03
AU2025263792A1 (en) 2025-11-27
CL2020002161A1 (es) 2021-01-08
EP3755805A4 (en) 2022-01-19
KR20260015297A (ko) 2026-02-02
IL325122A (en) 2026-02-01
JP7408228B2 (ja) 2024-01-05
EA202091995A1 (ru) 2021-03-30
US20200390905A1 (en) 2020-12-17
CA3091676A1 (en) 2019-08-29
AU2019224121A1 (en) 2020-08-27
JP2024023691A (ja) 2024-02-21
AU2019224121B2 (en) 2025-08-07
KR20200126997A (ko) 2020-11-09
WO2019165292A1 (en) 2019-08-29
IL276764A (en) 2020-10-29
CN112020561A (zh) 2020-12-01
BR112020016919A2 (pt) 2020-12-15
MA51918A (fr) 2020-12-30
KR102907773B1 (ko) 2026-01-06
EP3755805A1 (en) 2020-12-30
US20220040327A1 (en) 2022-02-10

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