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
Prior art keywords
sequence
pharmaceutical composition
composition according
polynucleotide
gene
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JP2021541524A
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English (en)
Japanese (ja)
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JP7595013B2 (ja
JP2022522996A (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
Priority to JP2024204975A priority Critical patent/JP2025032155A/ja
Application granted granted Critical
Publication of JP7595013B2 publication Critical patent/JP7595013B2/ja
Active legal-status Critical Current
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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)

Application Number Title Priority Date Filing Date
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 (3)

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

Family

ID=65351986

Family Applications (2)

Application Number Title Priority Date Filing Date
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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
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
CA3129422A1 (en) 2019-02-08 2020-08-13 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
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
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
EP4294460A4 (en) * 2021-02-19 2025-06-25 Decibel Therapeutics, Inc. Methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
JP2025530726A (ja) * 2022-05-17 2025-09-17 復旦大学附属眼耳鼻喉科医院 聴力障害を治療するためのデュアルベクターシステム及びその使用
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
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 (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
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
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
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
EP3492597A3 (en) 2013-05-21 2019-08-28 University of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
EP3258955A1 (en) * 2015-02-20 2017-12-27 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
SG10201913266UA (en) 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
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
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
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법

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