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.Info
- 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
Links
- 102000016774 Otoferlin Human genes 0.000 title abstract 5
- 108050006335 Otoferlin Proteins 0.000 title abstract 5
- 239000013598 vector Substances 0.000 title abstract 4
- 206010011891 Deafness neurosensory Diseases 0.000 title abstract 3
- 208000009966 Sensorineural Hearing Loss Diseases 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 3
- 231100000879 sensorineural hearing loss Toxicity 0.000 title abstract 3
- 208000023573 sensorineural hearing loss disease Diseases 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 230000009977 dual effect Effects 0.000 title 1
- 102000001708 Protein Isoforms Human genes 0.000 abstract 2
- 108010029485 Protein Isoforms Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 102000040430 polynucleotide Human genes 0.000 abstract 2
- 108091033319 polynucleotide Proteins 0.000 abstract 2
- 239000002157 polynucleotide Substances 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 101150006256 Otof gene Proteins 0.000 abstract 1
- 208000002982 auditory neuropathy Diseases 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
Classifications
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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; 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Cell Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Luminescent Compositions (AREA)
- Peptides Or Proteins (AREA)
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) | US12589168B2 (enExample) |
| EP (1) | EP4051698A4 (enExample) |
| JP (2) | JP7665609B2 (enExample) |
| KR (1) | KR20220130093A (enExample) |
| AU (1) | AU2020375947B2 (enExample) |
| BR (1) | BR112022008214A2 (enExample) |
| CA (1) | CA3159549A1 (enExample) |
| IL (1) | IL292620A (enExample) |
| MX (1) | MX2022005236A (enExample) |
| TW (1) | TWI896570B (enExample) |
| WO (1) | WO2021087296A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| IL325122A (en) | 2018-02-22 | 2026-02-01 | Akouos Inc | Compositions and methods for treating non-age-related hearing impairment in humans |
| 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 |
| CN113924109A (zh) | 2019-01-18 | 2022-01-11 | 巴斯德研究所 | Aav介导的基因疗法恢复耳畸蛋白基因 |
| 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 |
| WO2020163761A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| TWI896570B (zh) | 2019-10-30 | 2025-09-11 | 美商迪賽博治療公司 | 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法 |
| CA3168055A1 (en) | 2020-02-21 | 2021-08-26 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| IL295741A (en) | 2020-04-01 | 2022-10-01 | Univ Florida | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| JP2024534991A (ja) * | 2021-09-10 | 2024-09-26 | インスティチュート パスツール | オトフェリンのアイソフォーム５をコードする二重組換えａａｖ８ベクター系およびその使用 |
| CA3250345A1 (en) * | 2022-02-04 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | GJB2 REGULATORY ELEMENTS AND THEIR USES |
| CN117106824B (zh) * | 2022-05-17 | 2025-09-02 | 上海佑音医药生物科技有限公司 | 一种治疗听力损伤的双载体系统及其应用 |
| AR129387A1 (es) * | 2022-05-19 | 2024-08-21 | Akouos Inc | Composiciones y métodos para el tratamiento de deficiencias auditivas no asociadas a la edad en un sujeto humano |
| CN121002049A (zh) * | 2023-02-17 | 2025-11-21 | 分贝治疗公司 | 用于施用耳畸蛋白双载体系统的方法和组合物 |
| WO2025221253A1 (en) * | 2024-04-17 | 2025-10-23 | Decibel Therapeutics, Inc. | Methods for treating sensorineural hearing loss using otoferlin dual vector systems |
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| WO2020163761A1 (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 |
| TWI896570B (zh) | 2019-10-30 | 2025-09-11 | 美商迪賽博治療公司 | 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法 |
| CA3168055A1 (en) | 2020-02-21 | 2021-08-26 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| IL295741A (en) | 2020-04-01 | 2022-10-01 | Univ Florida | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| KR20240004253A (ko) | 2021-02-19 | 2024-01-11 | 데시벨 테라퓨틱스, 인크. | 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 방법 |
| CN121002049A (zh) | 2023-02-17 | 2025-11-21 | 分贝治疗公司 | 用于施用耳畸蛋白双载体系统的方法和组合物 |
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| US12589168B2 (en) | 2026-03-31 |
| BR112022008214A2 (pt) | 2022-07-12 |
| EP4051698A1 (en) | 2022-09-07 |
| IL292620A (en) | 2022-07-01 |
| TWI896570B (zh) | 2025-09-11 |
| JP2022554300A (ja) | 2022-12-28 |
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| WO2021087296A8 (en) | 2021-08-05 |
| EP4051698A4 (en) | 2024-03-27 |
| KR20220130093A (ko) | 2022-09-26 |
| AU2020375947A1 (en) | 2022-05-26 |
| WO2021087296A1 (en) | 2021-05-06 |
| JP7665609B2 (ja) | 2025-04-21 |
| JP7892830B2 (ja) | 2026-07-21 |
| TW202130654A (zh) | 2021-08-16 |
| AU2020375947B2 (en) | 2026-05-21 |
| CA3159549A1 (en) | 2021-05-06 |
| US20220265865A1 (en) | 2022-08-25 |
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