JP2022522996A5 - - Google Patents

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Publication number
JP2022522996A5
JP2022522996A5 JP2021541524A JP2021541524A JP2022522996A5 JP 2022522996 A5 JP2022522996 A5 JP 2022522996A5 JP 2021541524 A JP2021541524 A JP 2021541524A JP 2021541524 A JP2021541524 A JP 2021541524A JP 2022522996 A5 JP2022522996 A5 JP 2022522996A5
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JP
Japan
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JP2021541524A
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Japanese (ja)
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JPWO2020148458A5 (https=
JP2022522996A (ja
JP7595013B2 (ja
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Priority claimed from PCT/EP2020/051283 external-priority patent/WO2020148458A1/en
Publication of JP2022522996A publication Critical patent/JP2022522996A/ja
Publication of JP2022522996A5 publication Critical patent/JP2022522996A5/ja
Publication of JPWO2020148458A5 publication Critical patent/JPWO2020148458A5/ja
Priority to JP2024204975A priority Critical patent/JP2025032155A/ja
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JP2021541524A 2019-01-18 2020-01-20 オトフェリン遺伝子を回復させるaav媒介遺伝子治療 Active JP7595013B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024204975A JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるaav媒介遺伝子治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19305071.3 2019-01-18
EP19305071 2019-01-18
PCT/EP2020/051283 WO2020148458A1 (en) 2019-01-18 2020-01-20 Aav-mediated gene therapy restoring the otoferlin gene

Related Child Applications (1)

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JP2024204975A Division JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるaav媒介遺伝子治療

Publications (4)

Publication Number Publication Date
JP2022522996A JP2022522996A (ja) 2022-04-21
JP2022522996A5 true JP2022522996A5 (https=) 2024-05-10
JPWO2020148458A5 JPWO2020148458A5 (https=) 2024-05-10
JP7595013B2 JP7595013B2 (ja) 2024-12-05

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ID=65351986

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JP2021541524A Active JP7595013B2 (ja) 2019-01-18 2020-01-20 オトフェリン遺伝子を回復させるaav媒介遺伝子治療
JP2024204975A Pending JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるaav媒介遺伝子治療

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JP2024204975A Pending JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるaav媒介遺伝子治療

Country Status (22)

Country Link
US (1) US20220125875A1 (https=)
EP (2) EP4295913A3 (https=)
JP (2) JP7595013B2 (https=)
KR (2) KR20260046255A (https=)
CN (1) CN113924109A (https=)
AU (2) AU2020208933B2 (https=)
BR (1) BR112021013996A2 (https=)
CA (1) CA3126884A1 (https=)
DK (1) DK3911354T5 (https=)
ES (1) ES2960880T3 (https=)
FI (1) FI3911354T3 (https=)
HR (1) HRP20231296T1 (https=)
HU (1) HUE063458T2 (https=)
IL (1) IL284742B2 (https=)
LT (1) LT3911354T (https=)
MX (1) MX2021008622A (https=)
PL (1) PL3911354T3 (https=)
PT (1) PT3911354T (https=)
RS (1) RS64794B1 (https=)
SI (1) SI3911354T1 (https=)
SM (1) SMT202300359T1 (https=)
WO (1) WO2020148458A1 (https=)

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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
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
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
AU2021376225A1 (en) * 2020-11-06 2023-06-22 Eli Lilly And Company Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss
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
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
CN117106824B (zh) * 2022-05-17 2025-09-02 上海佑音医药生物科技有限公司 一种治疗听力损伤的双载体系统及其应用
EP4527933A4 (en) * 2022-05-17 2025-12-24 Eye & Ent Hospital Of Fudan Univ DUAL-SUPPORT SYSTEM FOR THE TREATMENT OF HEARING LOSS, AND ITS USE
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

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DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
WO2003059396A1 (en) 2002-01-11 2003-07-24 Sergei Zolotukhin Adiponectin gene therapy
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
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
WO2011075838A1 (en) 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
KR102234672B1 (ko) 2013-05-21 2021-04-06 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US10751385B2 (en) * 2015-02-20 2020-08-25 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
SG11201804814YA (en) * 2015-12-11 2018-07-30 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
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
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
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems

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