KR20220133854A - 유전적 청력 상실 치료를 위한 아데노 관련 바이러스(aav) 시스템 - Google Patents

유전적 청력 상실 치료를 위한 아데노 관련 바이러스(aav) 시스템 Download PDF

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KR20220133854A
KR20220133854A KR1020227014445A KR20227014445A KR20220133854A KR 20220133854 A KR20220133854 A KR 20220133854A KR 1020227014445 A KR1020227014445 A KR 1020227014445A KR 20227014445 A KR20227014445 A KR 20227014445A KR 20220133854 A KR20220133854 A KR 20220133854A
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gjb2
aav
nucleic acid
promoter
expression
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크리스 바르톨로메
루이스 데이비드 자라밀로
아드리안 팀머스
스티븐 펜녹
마크 시어맨
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어플라이드 제네틱스 테크놀로지스 코퍼레이션
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Publication of KR20220133854A publication Critical patent/KR20220133854A/ko
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020227014445A 2019-09-30 2020-09-30 유전적 청력 상실 치료를 위한 아데노 관련 바이러스(aav) 시스템 Pending KR20220133854A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962907834P 2019-09-30 2019-09-30
US62/907,834 2019-09-30
PCT/US2020/053561 WO2021067448A1 (en) 2019-09-30 2020-09-30 Adeno-associated virus (aav) systems for treatment of genetic hearing loss

Publications (1)

Publication Number Publication Date
KR20220133854A true KR20220133854A (ko) 2022-10-05

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KR1020227014445A Pending KR20220133854A (ko) 2019-09-30 2020-09-30 유전적 청력 상실 치료를 위한 아데노 관련 바이러스(aav) 시스템

Country Status (9)

Country Link
US (2) US12241077B2 (https=)
EP (1) EP4037771A4 (https=)
JP (2) JP7713130B2 (https=)
KR (1) KR20220133854A (https=)
CN (1) CN115103710A (https=)
AU (1) AU2020357740A1 (https=)
CA (1) CA3153133A1 (https=)
IL (1) IL291789A (https=)
WO (1) WO2021067448A1 (https=)

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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
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
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
EP4164695A4 (en) * 2020-05-13 2024-06-05 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
US20230340038A1 (en) * 2020-09-14 2023-10-26 President And Fellows Of Harvard College Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
JP2023541443A (ja) * 2020-09-14 2023-10-02 プレジデント アンド フェローズ オブ ハーバード カレッジ ＧＪＢ２をコードする組換えアデノ随伴ウイルス（ｒＡＡＶ）およびその使用。
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
EP4278002A2 (en) * 2021-01-18 2023-11-22 Life Technologies Corporation Compositions, kits and methods for direct amplification from crude biological samples
US20240197920A1 (en) * 2021-04-27 2024-06-20 The Children's Hospital Of Philadelphia Adeno-associated viral vectors for transduction of cochlea
US20250339560A1 (en) * 2021-05-13 2025-11-06 Akouos, Inc. Gene therapy delivery compositions and methods for treating hearing loss
WO2023106256A1 (ja) * 2021-12-06 2023-06-15 学校法人順天堂 改変型アデノ随伴ウイルスベクター
US20240252685A1 (en) * 2022-05-09 2024-08-01 Akouos, Inc. Cell specific gene therapy delivery compositions and methods for treating hearing loss
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
US20260103728A1 (en) * 2022-10-12 2026-04-16 The Johns Hopkins University Method of treating lbsl by enhancing dars2 expression
CN115717166B (zh) * 2022-12-15 2026-04-14 东南大学 遗传性耳聋检测系统、标志物或探针、药物及应用
EP4658759A2 (en) * 2023-02-02 2025-12-10 Agathos Biologics Baby hamster kidney (bhk) cells transformed with the adenoviral e1 gene for production of recombinant adeno-associated virus
EP4713463A1 (en) * 2023-05-18 2026-03-25 Decibel Therapeutics, Inc. Compositions and methods for treating gjb2-related hearing loss
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
CN119510774A (zh) * 2024-11-26 2025-02-25 华中科技大学同济医学院附属协和医院 Sptbn1作为靶点在治疗gjb2相关感音神经性听力损失中的应用

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US20050281786A1 (en) * 2004-06-18 2005-12-22 David Poulsen AAV mediated gene delivery to cochlear cells
WO2007132169A1 (en) * 2006-05-11 2007-11-22 Crusade Laboratories Limited Mutant hsv, materials and methods for generation of mutant hsv
US8105575B2 (en) * 2006-10-10 2012-01-31 Viromed Co., Ltd. Expression vectors with improved safety
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GB201513176D0 (en) * 2015-07-27 2015-09-09 Glaxosmithkline Biolog Sa Novel methods for inducing an immune response
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WO2017136764A1 (en) * 2016-02-05 2017-08-10 The General Hospital Corporation Hybrid system for efficient gene delivery to cells of the inner ear
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
AU2018224044B2 (en) * 2017-02-21 2024-01-25 The Uab Research Foundation Modified AAV capsid proteins and uses thereof
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JP2023541443A (ja) * 2020-09-14 2023-10-02 プレジデント アンド フェローズ オブ ハーバード カレッジ ＧＪＢ２をコードする組換えアデノ随伴ウイルス（ｒＡＡＶ）およびその使用。

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Publication number Publication date
IL291789A (en) 2022-06-01
US20250236887A1 (en) 2025-07-24
US12241077B2 (en) 2025-03-04
US20210095313A1 (en) 2021-04-01
AU2020357740A1 (en) 2022-04-28
WO2021067448A1 (en) 2021-04-08
EP4037771A1 (en) 2022-08-10
CN115103710A (zh) 2022-09-23
JP2022549380A (ja) 2022-11-24
EP4037771A4 (en) 2023-10-25
JP2025110904A (ja) 2025-07-29
JP7713130B2 (ja) 2025-07-25
CA3153133A1 (en) 2021-04-08

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