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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JP7240675B2 (ja
JP2020518268A (ja
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Priority claimed from PCT/US2018/031009 external-priority patent/WO2018204734A1/en
Publication of JP2020518268A publication Critical patent/JP2020518268A/ja
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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)

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

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Application Number Title Priority Date Filing Date
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
CA3034527A1 (en) * 2016-08-23 2018-03-01 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12359221B2 (en) 2017-05-05 2025-07-15 The Regents Of The University Of California 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
JP7408228B2 (ja) * 2018-02-22 2024-01-05 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及び方法
WO2019210181A1 (en) 2018-04-27 2019-10-31 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2020093018A1 (en) * 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
SI3911354T1 (sl) * 2019-01-18 2023-12-29 Institut Pasteur Aav posredovana genska terapija za obnovitev otoferlin gena
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN114072179B (zh) 2019-02-08 2024-11-01 分贝治疗公司 肌球蛋白15启动子及其用途
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
MX2022005236A (es) * 2019-10-30 2022-06-08 Decibel Therapeutics Inc Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina.
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a 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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US8741650B2 (en) 2004-01-22 2014-06-03 Dnavec Research Inc. Methods for producing minus-strand RNA viral vectors using hybrid promoter comprising cytomegalovirus enhancer and chicken β-actin promoter
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
CA2660286A1 (en) 2006-08-09 2008-02-21 Homestead Clinical Corporation Organ-specific proteins and methods of their use
US7892824B2 (en) 2007-01-18 2011-02-22 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
WO2010062905A2 (en) * 2008-11-26 2010-06-03 The Regents Of The University Of California Lateral ventricle cell compositions and use for treating neural degenerative diseases
AU2011242527B2 (en) * 2010-04-23 2016-05-19 University Of Florida Research Foundation, Inc. rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1)
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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KR102794870B1 (ko) 2015-12-11 2025-04-10 매사추세츠 아이 앤드 이어 인퍼머리 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법
CA3008956C (en) 2015-12-22 2023-01-03 Inserm (Institut National De La Sante Et De La Recherche Medicale) Improved hybrid dual recombinant aav vector systems for gene therapy
EP3472328A1 (en) 2016-06-15 2019-04-24 Oxford University Innovation Limited Dual overlapping adeno-associated viral vector system for expressing abc4a
CA3034527A1 (en) 2016-08-23 2018-03-01 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
US12359221B2 (en) 2017-05-05 2025-07-15 The Regents Of The University Of California Compositions and methods for expressing otoferlin
JP7408228B2 (ja) 2018-02-22 2024-01-05 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及び方法
US12331328B2 (en) 2018-03-23 2025-06-17 Massachusetts Eye And Ear Infirmary CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated usher syndrome
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
SI3911354T1 (sl) 2019-01-18 2023-12-29 Institut Pasteur Aav posredovana genska terapija za obnovitev otoferlin gena
MX2022005236A (es) 2019-10-30 2022-06-08 Decibel Therapeutics Inc Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina.
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a vectors with improved safety for the treatment of ush1b

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