JP2020518268A5 - - Google Patents

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JP2020518268A5
JP2020518268A5 JP2019560646A JP2019560646A JP2020518268A5 JP 2020518268 A5 JP2020518268 A5 JP 2020518268A5 JP 2019560646 A JP2019560646 A JP 2019560646A JP 2019560646 A JP2019560646 A JP 2019560646A JP 2020518268 A5 JP2020518268 A5 JP 2020518268A5
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JP7240675B2 (ja
JP2020518268A (ja
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Priority claimed from PCT/US2018/031009 external-priority patent/WO2018204734A1/en
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Priority to JP2023027073A priority Critical patent/JP7617600B2/ja
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Priority to JP2024226066A priority patent/JP7851563B2/ja
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JP2019560646A 2017-05-05 2018-05-04 オトフェルリンを発現させるための組成物および方法 Active JP7240675B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023027073A JP7617600B2 (ja) 2017-05-05 2023-02-24 オトフェルリンを発現させるための組成物および方法
JP2024226066A JP7851563B2 (ja) 2017-05-05 2024-12-23 オトフェルリンを発現させるための組成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762502462P 2017-05-05 2017-05-05
US62/502,462 2017-05-05
PCT/US2018/031009 WO2018204734A1 (en) 2017-05-05 2018-05-04 Compositions and methods for expressing otoferlin

Related Child Applications (1)

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JP2023027073A Division JP7617600B2 (ja) 2017-05-05 2023-02-24 オトフェルリンを発現させるための組成物および方法

Publications (3)

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JP2020518268A JP2020518268A (ja) 2020-06-25
JP2020518268A5 true JP2020518268A5 (https=) 2021-07-26
JP7240675B2 JP7240675B2 (ja) 2023-03-16

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JP2019560646A Active JP7240675B2 (ja) 2017-05-05 2018-05-04 オトフェルリンを発現させるための組成物および方法
JP2023027073A Active JP7617600B2 (ja) 2017-05-05 2023-02-24 オトフェルリンを発現させるための組成物および方法
JP2024226066A Active JP7851563B2 (ja) 2017-05-05 2024-12-23 オトフェルリンを発現させるための組成物および方法

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JP2023027073A Active JP7617600B2 (ja) 2017-05-05 2023-02-24 オトフェルリンを発現させるための組成物および方法
JP2024226066A Active JP7851563B2 (ja) 2017-05-05 2024-12-23 オトフェルリンを発現させるための組成物および方法

Country Status (9)

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US (2) US12359221B2 (https=)
EP (1) EP3635100A4 (https=)
JP (3) JP7240675B2 (https=)
KR (2) KR102606810B1 (https=)
CN (1) CN110892062A (https=)
AU (2) AU2018261769B2 (https=)
CA (1) CA3061955A1 (https=)
MX (2) MX2019013151A (https=)
WO (1) WO2018204734A1 (https=)

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WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
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
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
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
WO2020093018A1 (en) * 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
EP4295913A3 (en) * 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
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
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
KR20220130093A (ko) * 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
CN115066432B (zh) * 2019-10-30 2026-04-17 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组合物和方法
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
CN112125969A (zh) * 2020-09-30 2020-12-25 东南大学 生物因子rimbp2在维持内耳毛细胞特性上的应用
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
WO2025201333A1 (zh) * 2024-03-28 2025-10-02 苏州星奥拓维生物技术有限公司 递送Otof基因的双载体系统和其用途

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CN1934260B (zh) 2004-01-22 2013-03-13 株式会社载体研究所 利用含有巨细胞病毒增强子和鸡β-肌动蛋白启动子的杂合启动子制造负链RNA病毒载体的方法
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
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EP3510161A4 (en) * 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
IL325122A (en) 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
BR112020019079A2 (pt) 2018-03-23 2020-12-29 Massachusetts Eye And Ear Infirmary Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
EP4295913A3 (en) 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
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

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