JPWO2023036966A5 - - Google Patents

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Publication number
JPWO2023036966A5
JPWO2023036966A5 JP2024515645A JP2024515645A JPWO2023036966A5 JP WO2023036966 A5 JPWO2023036966 A5 JP WO2023036966A5 JP 2024515645 A JP2024515645 A JP 2024515645A JP 2024515645 A JP2024515645 A JP 2024515645A JP WO2023036966 A5 JPWO2023036966 A5 JP WO2023036966A5
Authority
JP
Japan
Prior art keywords
polynucleotide
sequence
nucleotides
seq
inverted terminal
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
JP2024534991A5 (https=
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 JPWO2023036966A5 publication Critical patent/JPWO2023036966A5/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 (3)

Publication Number Publication Date
JP2024534991A JP2024534991A (ja) 2024-09-26
JPWO2023036966A5 true JPWO2023036966A5 (https=) 2025-08-05
JP2024534991A5 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
WO2025128559A1 (en) * 2023-12-11 2025-06-19 Akouos, Inc. Compositions and methods for treating hearing loss in a human subject having a defective otoferlin gene

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
US10751385B2 (en) * 2015-02-20 2020-08-25 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
US12188041B2 (en) * 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
WO2020148458A1 (en) * 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CA3159549A1 (en) * 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
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

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