CN115103710A - 用于治疗遗传性听力损失的腺相关病毒(aav)系统 - Google Patents

用于治疗遗传性听力损失的腺相关病毒(aav)系统 Download PDF

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Publication number
CN115103710A
CN115103710A CN202080083106.6A CN202080083106A CN115103710A CN 115103710 A CN115103710 A CN 115103710A CN 202080083106 A CN202080083106 A CN 202080083106A CN 115103710 A CN115103710 A CN 115103710A
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gjb2
aav
promoter
nucleic acid
expression
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克里斯·巴托洛梅
路易斯·大卫·贾拉米洛
阿德里安·蒂默尔斯
史蒂文·佩诺克
马克·希尔曼
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Eli Lilly and Co
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Applied Genetic Technologies Corp
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CN202080083106.6A 2019-09-30 2020-09-30 用于治疗遗传性听力损失的腺相关病毒(aav)系统 Pending CN115103710A (zh)

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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

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CN115103710A true CN115103710A (zh) 2022-09-23

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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=)
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CN115717166A (zh) * 2022-12-15 2023-02-28 东南大学 遗传性耳聋检测系统、标志物或探针、药物及应用
CN116925239A (zh) * 2023-07-17 2023-10-24 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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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
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

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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115717166A (zh) * 2022-12-15 2023-02-28 东南大学 遗传性耳聋检测系统、标志物或探针、药物及应用
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
CN116925239A (zh) * 2023-07-17 2023-10-24 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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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
JP2022549380A (ja) 2022-11-24
EP4037771A4 (en) 2023-10-25
JP2025110904A (ja) 2025-07-29
JP7713130B2 (ja) 2025-07-25
KR20220133854A (ko) 2022-10-05
CA3153133A1 (en) 2021-04-08

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