JP2024534991A5 - - Google Patents

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Publication number
JP2024534991A5
JP2024534991A5 JP2024515645A JP2024515645A JP2024534991A5 JP 2024534991 A5 JP2024534991 A5 JP 2024534991A5 JP 2024515645 A JP2024515645 A JP 2024515645A JP 2024515645 A JP2024515645 A JP 2024515645A JP 2024534991 A5 JP2024534991 A5 JP 2024534991A5
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JP
Japan
Prior art keywords
polynucleotide
sequence
nucleotides
seq
gene
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024515645A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024534991A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2022/075185 external-priority patent/WO2023036966A1/en
Publication of JP2024534991A publication Critical patent/JP2024534991A/ja
Publication of JP2024534991A5 publication Critical patent/JP2024534991A5/ja
Pending legal-status Critical Current

Links

JP2024515645A 2021-09-10 2022-09-09 オトフェリンのアイソフォーム5をコードする二重組換えaav8ベクター系およびその使用 Pending JP2024534991A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP21306245.8 2021-09-10
EP21306245 2021-09-10
US202263303743P 2022-01-27 2022-01-27
US63/303,743 2022-01-27
PCT/EP2022/075185 WO2023036966A1 (en) 2021-09-10 2022-09-09 Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof

Publications (2)

Publication Number Publication Date
JP2024534991A JP2024534991A (ja) 2024-09-26
JP2024534991A5 true JP2024534991A5 (https=) 2025-08-05

Family

ID=83598432

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024515645A Pending JP2024534991A (ja) 2021-09-10 2022-09-09 オトフェリンのアイソフォーム5をコードする二重組換えaav8ベクター系およびその使用

Country Status (9)

Country Link
US (1) US20250295716A1 (https=)
EP (1) EP4398943A1 (https=)
JP (1) JP2024534991A (https=)
KR (1) KR20240053630A (https=)
AU (1) AU2022344528A1 (https=)
CA (1) CA3231374A1 (https=)
IL (1) IL311345A (https=)
MX (1) MX2024003052A (https=)
WO (1) WO2023036966A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
TW202532649A (zh) * 2023-12-11 2025-08-16 美商阿科奧斯公司 用於治療具有缺陷耳畸蛋白基因之人類個體之聽力損失的組合物及方法

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
EP3258955A1 (en) * 2015-02-20 2017-12-27 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
IL325122A (en) * 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
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
EP4294460A4 (en) * 2021-02-19 2025-06-25 Decibel Therapeutics, Inc. Methods for treating sensorineural hearing loss using otoferlin dual vector systems

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