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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JP2019560646A
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Japanese (ja)
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JP2020518268A (ja
JP7240675B2 (ja
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Priority claimed from PCT/US2018/031009 external-priority patent/WO2018204734A1/en
Publication of JP2020518268A publication Critical patent/JP2020518268A/ja
Publication of JP2020518268A5 publication Critical patent/JP2020518268A5/ja
Priority to JP2023027073A priority Critical patent/JP7617600B2/ja
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Publication of JP7240675B2 publication Critical patent/JP7240675B2/ja
Priority to JP2024226066A priority patent/JP2025063057A/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 JP2025063057A (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)

Publication Number Publication Date
JP2020518268A JP2020518268A (ja) 2020-06-25
JP2020518268A5 true JP2020518268A5 (enExample) 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 Pending JP2025063057A (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 Pending JP2025063057A (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)

Country Link
US (2) US12359221B2 (enExample)
EP (1) EP3635100A4 (enExample)
JP (3) JP7240675B2 (enExample)
KR (2) KR20230167138A (enExample)
CN (1) CN110892062A (enExample)
AU (2) AU2018261769B2 (enExample)
CA (1) CA3061955A1 (enExample)
MX (2) MX2019013151A (enExample)
WO (1) WO2018204734A1 (enExample)

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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
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 (en) * 2017-05-05 2018-11-08 University Of Florida Research Foundation, Incorporated Compositions and methods for expressing otoferlin
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
AU2019224121B2 (en) * 2018-02-22 2025-08-07 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
CN119410637A (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
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
EP3911354B8 (en) * 2019-01-18 2024-04-03 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CN119320772A (zh) 2019-02-08 2025-01-17 分贝治疗公司 肌球蛋白15启动子及其用途
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
AU2020375947A1 (en) * 2019-10-30 2022-05-26 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN115066432A (zh) * 2019-10-30 2022-09-16 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
EP4114958A1 (en) 2020-02-21 2023-01-11 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
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
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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US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
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WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
FR2919305B1 (fr) 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
US8962314B2 (en) * 2008-11-26 2015-02-24 The Regents Of The University Of California Lateral ventricle cell compositions and use for treating neural degenerative diseases
KR20190060881A (ko) * 2010-04-23 2019-06-03 유니버시티 오브 플로리다 리서치 파운데이션, 인크. 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
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JP7007273B2 (ja) 2015-12-22 2022-01-24 アンセルム(アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル) 遺伝子治療用の改良された複合型二重組換えaavベクターシステム
BR112018075855A2 (pt) 2016-06-15 2019-04-02 Oxford University Innovation Limited sistema de vetor viral adeno-associado com dupla sobreposição para a expressão abc4a
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
US11541131B2 (en) 2017-03-10 2023-01-03 Genethon Treatment of glycogen storage disease III
CA3061955A1 (en) 2017-05-05 2018-11-08 University Of Florida Research Foundation, Incorporated Compositions and methods for expressing otoferlin
AU2019224121B2 (en) 2018-02-22 2025-08-07 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
EP4524247A3 (en) 2018-03-23 2025-06-11 Massachusetts Eye and Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
EP3911354B8 (en) 2019-01-18 2024-04-03 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
AU2020375947A1 (en) 2019-10-30 2022-05-26 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP4114958A1 (en) 2020-02-21 2023-01-11 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
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

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