JPWO2019165292A5 - - Google Patents
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- Publication number
- JPWO2019165292A5 JPWO2019165292A5 JP2020544478A JP2020544478A JPWO2019165292A5 JP WO2019165292 A5 JPWO2019165292 A5 JP WO2019165292A5 JP 2020544478 A JP2020544478 A JP 2020544478A JP 2020544478 A JP2020544478 A JP 2020544478A JP WO2019165292 A5 JPWO2019165292 A5 JP WO2019165292A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- otoferin
- aav vectors
- composition
- item
- Prior art date
- Legal status (The legal status 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 status listed.)
- Granted
Links
- 239000000203 mixture Substances 0.000 claims description 77
- 239000013607 AAV vector Substances 0.000 claims description 63
- 239000013598 vector Substances 0.000 claims description 55
- 108090000623 proteins and genes Proteins 0.000 claims description 54
- 150000007523 nucleic acids Chemical group 0.000 claims description 16
- 210000004962 mammalian cell Anatomy 0.000 claims description 15
- 108700024394 Exon Proteins 0.000 claims description 12
- 210000003477 cochlea Anatomy 0.000 claims description 9
- 102000001708 Protein Isoforms Human genes 0.000 claims description 7
- 108010029485 Protein Isoforms Proteins 0.000 claims description 7
- 241000702421 Dependoparvovirus Species 0.000 claims description 5
- 230000006801 homologous recombination Effects 0.000 claims description 5
- 238000002744 homologous recombination Methods 0.000 claims description 5
- 230000008488 polyadenylation Effects 0.000 claims description 5
- 230000000368 destabilizing effect Effects 0.000 claims description 4
- 230000001939 inductive effect Effects 0.000 claims description 3
- 229920001184 polypeptide Polymers 0.000 claims 13
- 108090000765 processed proteins & peptides Proteins 0.000 claims 13
- 102000004196 processed proteins & peptides Human genes 0.000 claims 13
- 206010011878 Deafness Diseases 0.000 claims 5
- 210000000234 capsid Anatomy 0.000 claims 5
- 231100000895 deafness Toxicity 0.000 claims 5
- 208000016354 hearing loss disease Diseases 0.000 claims 5
- 210000004027 cell Anatomy 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 28
- 102000004169 proteins and genes Human genes 0.000 description 28
- 108091026890 Coding region Proteins 0.000 description 24
- 108010076504 Protein Sorting Signals Proteins 0.000 description 14
- 108020004707 nucleic acids Proteins 0.000 description 14
- 102000039446 nucleic acids Human genes 0.000 description 14
- 239000002773 nucleotide Substances 0.000 description 13
- 125000003729 nucleotide group Chemical group 0.000 description 13
- 125000003275 alpha amino acid group Chemical group 0.000 description 10
- 230000002950 deficient Effects 0.000 description 10
- 241000124008 Mammalia Species 0.000 description 8
- 150000001413 amino acids Chemical class 0.000 description 8
- 210000004899 c-terminal region Anatomy 0.000 description 7
- 125000000539 amino acid group Chemical group 0.000 description 6
- 108020004414 DNA Proteins 0.000 description 5
- 239000003550 marker Substances 0.000 description 5
- 230000001965 increasing effect Effects 0.000 description 4
- 210000000067 inner hair cell Anatomy 0.000 description 3
- 206010011891 Deafness neurosensory Diseases 0.000 description 2
- 101000634196 Homo sapiens Neurotrophin-3 Proteins 0.000 description 2
- 102100029268 Neurotrophin-3 Human genes 0.000 description 2
- 208000009966 Sensorineural Hearing Loss Diseases 0.000 description 2
- 210000004436 artificial bacterial chromosome Anatomy 0.000 description 2
- 210000004507 artificial chromosome Anatomy 0.000 description 2
- 210000001106 artificial yeast chromosome Anatomy 0.000 description 2
- 210000000688 human artificial chromosome Anatomy 0.000 description 2
- 210000005260 human cell Anatomy 0.000 description 2
- 208000023573 sensorineural hearing loss disease Diseases 0.000 description 2
- 239000013603 viral vector Substances 0.000 description 2
- 102000002260 Alkaline Phosphatase Human genes 0.000 description 1
- 108020004774 Alkaline Phosphatase Proteins 0.000 description 1
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 1
- 210000003030 auditory receptor cell Anatomy 0.000 description 1
- VJBCNMFKFZIXHC-UHFFFAOYSA-N azanium;2-(4-methyl-5-oxo-4-propan-2-yl-1h-imidazol-2-yl)quinoline-3-carboxylate Chemical group N.N1C(=O)C(C(C)C)(C)N=C1C1=NC2=CC=CC=C2C=C1C(O)=O VJBCNMFKFZIXHC-UHFFFAOYSA-N 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 239000000463 material Substances 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
- 239000013612 plasmid Substances 0.000 description 1
- 229940071643 prefilled syringe Drugs 0.000 description 1
- 230000001177 retroviral effect Effects 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 108700026220 vif Genes Proteins 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023212464A JP2024023691A (ja) | 2018-02-22 | 2023-12-15 | 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及び方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862634088P | 2018-02-22 | 2018-02-22 | |
| US62/634,088 | 2018-02-22 | ||
| PCT/US2019/019268 WO2019165292A1 (fr) | 2018-02-22 | 2019-02-22 | Compositions et méthodes de traitement d'une hypoacousie non associée à l'âge chez un sujet humain |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023212464A Division JP2024023691A (ja) | 2018-02-22 | 2023-12-15 | 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及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021513863A JP2021513863A (ja) | 2021-06-03 |
| JPWO2019165292A5 true JPWO2019165292A5 (fr) | 2022-03-03 |
| JP7408228B2 JP7408228B2 (ja) | 2024-01-05 |
Family
ID=67687298
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020544478A Active JP7408228B2 (ja) | 2018-02-22 | 2019-02-22 | 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及び方法 |
| JP2023212464A Pending JP2024023691A (ja) | 2018-02-22 | 2023-12-15 | 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及び方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023212464A Pending JP2024023691A (ja) | 2018-02-22 | 2023-12-15 | 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及び方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20200390905A1 (fr) |
| EP (1) | EP3755805A4 (fr) |
| JP (2) | JP7408228B2 (fr) |
| KR (1) | KR20200126997A (fr) |
| CN (1) | CN112020561A (fr) |
| AU (1) | AU2019224121A1 (fr) |
| BR (1) | BR112020016919A2 (fr) |
| CA (1) | CA3091676A1 (fr) |
| CL (1) | CL2020002161A1 (fr) |
| EA (1) | EA202091995A1 (fr) |
| IL (1) | IL276764A (fr) |
| MA (1) | MA51918A (fr) |
| MX (1) | MX2020008763A (fr) |
| SG (1) | SG11202007491UA (fr) |
| WO (1) | WO2019165292A1 (fr) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110225975A (zh) | 2016-08-23 | 2019-09-10 | 阿库斯股份有限公司 | 用于治疗人受试者中非年龄相关的听力损害的组合物和方法 |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| WO2021021674A1 (fr) | 2019-07-26 | 2021-02-04 | Akouos, Inc. | Méthodes de traitement de la perte auditive à l'aide d'une protéine cible sécrétée |
| BR112022008214A2 (pt) * | 2019-10-30 | 2022-07-12 | Decibel Therapeutics Inc | Composições e métodos para tratar perda auditiva neurossensorial usando sistemas de vetor duplo de otoferlina |
| WO2021168362A1 (fr) | 2020-02-21 | 2021-08-26 | Akouos, Inc. | Compositions et méthodes de traitement d'une hypoacousie non associée à l'âge chez un sujet humain |
| CA3183171A1 (fr) | 2020-05-13 | 2021-11-18 | Akouos, Inc. | Compositions et methodes pour traiter une perte auditive associee a slc26a4 |
| IL304006A (en) | 2020-12-29 | 2023-08-01 | Akouos Inc | Compositions and methods for treating hearing loss and/or vision loss associated with CLRN1 |
| 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 |
| IL311345A (en) * | 2021-09-10 | 2024-05-01 | Pasteur Institut | Dual recombinant AAV8 vector system for encoding isoform 5 of autoperlin 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 |
| CN116925239A (zh) * | 2023-07-17 | 2023-10-24 | 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 |
Family Cites Families (4)
| 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 |
| WO2014170480A1 (fr) * | 2013-04-18 | 2014-10-23 | Fondazione Telethon | Administration efficace de grands gènes par des vecteurs aav doubles |
| CN110225975A (zh) * | 2016-08-23 | 2019-09-10 | 阿库斯股份有限公司 | 用于治疗人受试者中非年龄相关的听力损害的组合物和方法 |
| CA3061955A1 (fr) | 2017-05-05 | 2018-11-08 | University Of Florida Research Foundation, Incorporated | Compositions et procedes pour exprimer l'otoferline |
-
2019
- 2019-02-22 CA CA3091676A patent/CA3091676A1/fr active Pending
- 2019-02-22 MA MA051918A patent/MA51918A/fr unknown
- 2019-02-22 SG SG11202007491UA patent/SG11202007491UA/en unknown
- 2019-02-22 JP JP2020544478A patent/JP7408228B2/ja active Active
- 2019-02-22 CN CN201980026498.XA patent/CN112020561A/zh active Pending
- 2019-02-22 EA EA202091995A patent/EA202091995A1/ru unknown
- 2019-02-22 WO PCT/US2019/019268 patent/WO2019165292A1/fr unknown
- 2019-02-22 AU AU2019224121A patent/AU2019224121A1/en active Pending
- 2019-02-22 EP EP19757886.7A patent/EP3755805A4/fr active Pending
- 2019-02-22 US US16/971,922 patent/US20200390905A1/en not_active Abandoned
- 2019-02-22 MX MX2020008763A patent/MX2020008763A/es unknown
- 2019-02-22 KR KR1020207027137A patent/KR20200126997A/ko unknown
- 2019-02-22 BR BR112020016919-6A patent/BR112020016919A2/pt unknown
-
2020
- 2020-08-17 IL IL276764A patent/IL276764A/en unknown
- 2020-08-21 CL CL2020002161A patent/CL2020002161A1/es unknown
-
2021
- 2021-04-08 US US17/225,621 patent/US20220040327A1/en active Pending
-
2023
- 2023-12-15 JP JP2023212464A patent/JP2024023691A/ja active Pending
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