JPWO2021087296A5 - - Google Patents

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JPWO2021087296A5
JPWO2021087296A5 JP2022525387A JP2022525387A JPWO2021087296A5 JP WO2021087296 A5 JPWO2021087296 A5 JP WO2021087296A5 JP 2022525387 A JP2022525387 A JP 2022525387A JP 2022525387 A JP2022525387 A JP 2022525387A JP WO2021087296 A5 JPWO2021087296 A5 JP WO2021087296A5
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sequence
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double
vector system
otof
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JP2022554300A (ja
JP7665609B2 (ja
JP2022554300A5 (https=
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JP2022525387A 2019-10-30 2020-10-30 オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物 Active JP7665609B2 (ja)

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JP2025064211A JP2025106464A (ja) 2019-10-30 2025-04-09 オトフェリン二重ベクター系を使用して、感音難聴を治療するための組成物及び方法

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US201962928290P 2019-10-30 2019-10-30
US62/928,290 2019-10-30
US202062965776P 2020-01-24 2020-01-24
US62/965,776 2020-01-24
US202062971504P 2020-02-07 2020-02-07
US62/971,504 2020-02-07
US202063023058P 2020-05-11 2020-05-11
US63/023,058 2020-05-11
PCT/US2020/058265 WO2021087296A1 (en) 2019-10-30 2020-10-30 Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems

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JP2025064211A Division JP2025106464A (ja) 2019-10-30 2025-04-09 オトフェリン二重ベクター系を使用して、感音難聴を治療するための組成物及び方法

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JP2022554300A JP2022554300A (ja) 2022-12-28
JPWO2021087296A5 true JPWO2021087296A5 (https=) 2023-11-02
JP2022554300A5 JP2022554300A5 (https=) 2023-11-02
JP7665609B2 JP7665609B2 (ja) 2025-04-21

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JP2022525387A Active JP7665609B2 (ja) 2019-10-30 2020-10-30 オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
JP2025064211A Pending JP2025106464A (ja) 2019-10-30 2025-04-09 オトフェリン二重ベクター系を使用して、感音難聴を治療するための組成物及び方法

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US (1) US12589168B2 (https=)
EP (1) EP4051698A4 (https=)
JP (2) JP7665609B2 (https=)
KR (1) KR20220130093A (https=)
AU (1) AU2020375947A1 (https=)
BR (1) BR112022008214A2 (https=)
CA (1) CA3159549A1 (https=)
IL (1) IL292620A (https=)
MX (1) MX2022005236A (https=)
TW (1) TWI896570B (https=)
WO (1) WO2021087296A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
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
WO2020148458A1 (en) 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
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
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
CA3250345A1 (en) * 2022-02-04 2023-08-10 Decibel Therapeutics, Inc. GJB2 REGULATORY ELEMENTS AND THEIR USES
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
EP4665751A2 (en) * 2023-02-17 2025-12-24 Decibel Therapeutics, Inc. Methods and compositions for administering otoferlin dual vector systems
WO2025221253A1 (en) * 2024-04-17 2025-10-23 Decibel Therapeutics, Inc. Methods for treating sensorineural hearing loss using otoferlin dual vector systems

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1224313A1 (en) 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-associated viruses and uses thereof
WO2001029243A1 (en) 1999-10-15 2001-04-26 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
WO2001070972A2 (en) 2000-03-24 2001-09-27 Yasunaga, Shin'ichiro Multiple human and mouse otoferlin isoforms
US20040072154A1 (en) 2000-12-22 2004-04-15 Morris David W. Novel compositions and methods for cancer
CA2442670A1 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
JP2003088388A (ja) 2001-09-14 2003-03-25 Herikkusu Kenkyusho:Kk 新規な全長cDNA
JP4999330B2 (ja) 2004-01-22 2012-08-15 株式会社ディナベック研究所 サイトメガロウイルスエンハンサーおよびニワトリβ−アクチンプロモーターを含むハイブリッドプロモーターを利用したマイナス鎖RNAウイルスベクターの製造方法
US20070161031A1 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
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
EP2520935A3 (en) 2006-08-09 2013-02-13 Homestead Clinical Corporation Organ-specific proteins and methods of their use
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にnNOSを回復させる合成ミニ/ミクロジストロフィン遺伝子
FR2919305B1 (fr) 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
EP2732819B1 (en) 2008-02-07 2019-10-16 Massachusetts Eye & Ear Infirmary Compounds that enhance Atoh-1 expression
US20090215879A1 (en) * 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
US8962314B2 (en) 2008-11-26 2015-02-24 The Regents Of The University Of California Lateral ventricle cell compositions and use for treating neural degenerative diseases
HRP20190144T1 (hr) 2010-04-23 2019-03-22 University Of Florida Research Foundation, Inc. Pripravci raav-gvanilat ciklaze i postupci za liječenje leberove urođene amauroze-1 (lca1)
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
AU2013221212B2 (en) * 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
AU2013249202B2 (en) * 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
CA2884309A1 (en) 2012-09-07 2014-03-13 Massachusetts Eye And Ear Infirmary Methods and compositions for regenerating hair cells and/or supporting cells from differentiated cochlear cells or differentiated utricular cells by modulation of notch and cmyc activity
ES2697249T3 (es) 2013-03-11 2019-01-22 Fond Telethon miR-204 y miR-211 y usos de los mismos
CA2909733C (en) * 2013-04-18 2023-10-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors
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
US11000597B2 (en) 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
US10751385B2 (en) 2015-02-20 2020-08-25 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
RS63416B1 (sr) 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
AU2016324317A1 (en) 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
SG11201804814YA (en) * 2015-12-11 2018-07-30 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2017108931A1 (en) 2015-12-22 2017-06-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Improved hybrid dual recombinant aav vector systems for gene therapy
BR112018075855A2 (pt) 2016-06-15 2019-04-02 Oxford University Innovation Limited sistema de vetor viral adeno-associado com dupla sobreposição para a expressão abc4a
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
WO2018145111A1 (en) 2017-02-06 2018-08-09 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells
US11541131B2 (en) 2017-03-10 2023-01-03 Genethon Treatment of glycogen storage disease III
AU2018261769B2 (en) 2017-05-05 2024-03-14 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
IL325122A (en) * 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
US20190345516A1 (en) * 2018-03-12 2019-11-14 Musc Foundation For Research Development Methods for treating eye disease
CA3094828A1 (en) 2018-03-23 2019-09-26 Massachusetts Eye And Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome
US11660353B2 (en) * 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
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
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
CN118497276A (zh) 2018-11-07 2024-08-16 阿库斯股份有限公司 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途
WO2020148458A1 (en) 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
JP2022522995A (ja) 2019-01-18 2022-04-21 ボイジャー セラピューティクス インコーポレイテッド Aav粒子を生成するための方法及びシステム
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
AU2020218547A1 (en) * 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
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
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
EP4665751A2 (en) 2023-02-17 2025-12-24 Decibel Therapeutics, Inc. Methods and compositions for administering otoferlin dual vector systems

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