JP2021513863A5 - - Google Patents

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Publication number
JP2021513863A5
JP2021513863A5 JP2020544478A JP2020544478A JP2021513863A5 JP 2021513863 A5 JP2021513863 A5 JP 2021513863A5 JP 2020544478 A JP2020544478 A JP 2020544478A JP 2020544478 A JP2020544478 A JP 2020544478A JP 2021513863 A5 JP2021513863 A5 JP 2021513863A5
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JP
Japan
Prior art keywords
aav
otoferrin
composition according
aav vectors
vectors
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.)
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Application number
JP2020544478A
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English (en)
Japanese (ja)
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JP2021513863A (ja
JP7408228B2 (ja
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Priority claimed from PCT/US2019/019268 external-priority patent/WO2019165292A1/en
Publication of JP2021513863A publication Critical patent/JP2021513863A/ja
Publication of JP2021513863A5 publication Critical patent/JP2021513863A5/ja
Priority to JP2023212464A priority Critical patent/JP2024023691A/ja
Application granted granted Critical
Publication of JP7408228B2 publication Critical patent/JP7408228B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2020544478A 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 Active JP7408228B2 (ja)

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 (en) 2018-02-22 2019-02-22 Compositions and methods for treating non-age-associated hearing impairment in a human subject

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
JP2021513863A5 true JP2021513863A5 (https=) 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 (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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