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
Application number
JP2020544478A
Other languages
English (en)
Japanese (ja)
Other versions
JP7408228B2 (ja
JPWO2019165292A5 (https=
JP2021513863A (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 JPWO2019165292A5 publication Critical patent/JPWO2019165292A5/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)

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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 (4)

Publication Number Publication Date
JP2021513863A JP2021513863A (ja) 2021-06-03
JPWO2019165292A5 JPWO2019165292A5 (https=) 2022-03-03
JP2021513863A5 true JP2021513863A5 (https=) 2022-03-03
JP7408228B2 JP7408228B2 (ja) 2024-01-05

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ID=67687298

Family Applications (2)

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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)

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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=)

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Publication number Priority date Publication date Assignee Title
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
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2020148458A1 (en) * 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
AU2020218547A1 (en) 2019-02-08 2021-08-26 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
US20220396806A1 (en) 2019-07-26 2022-12-15 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7807380B2 (ja) * 2020-02-21 2026-01-27 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関連聴力障害を処置するための組成物および方法
AU2021247172A1 (en) 2020-04-01 2022-09-15 University Of Florida Research Foundation, Incorporated Dual AAV-MY07A vectors with improved safety for the treatment of USH1B
JP2023526047A (ja) 2020-05-13 2023-06-20 アコーオス インコーポレイテッド Ｓｌｃ２６ａ４関連難聴を治療するための組成物及び方法
MX2023007800A (es) 2020-12-29 2023-07-11 Akouos Inc Composiciones y metodos para tratar la perdida de audicion y/o la perdida de vision asociada a clrn1.
CN117295529A (zh) * 2021-02-19 2023-12-26 分贝治疗公司 使用耳畸蛋白双载体系统治疗感觉神经性听力损失的方法
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
WO2001070972A2 (en) * 2000-03-24 2001-09-27 Yasunaga, Shin'ichiro 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
MX384795B (es) * 2013-03-15 2025-03-14 Childrens Hospital Philadelphia Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso.
CA2909733C (en) * 2013-04-18 2023-10-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors
RS63416B1 (sr) * 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
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
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
JP7807380B2 (ja) * 2020-02-21 2026-01-27 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関連聴力障害を処置するための組成物および方法

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