JP2022549380A5 - - Google Patents

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Publication number
JP2022549380A5
JP2022549380A5 JP2022520059A JP2022520059A JP2022549380A5 JP 2022549380 A5 JP2022549380 A5 JP 2022549380A5 JP 2022520059 A JP2022520059 A JP 2022520059A JP 2022520059 A JP2022520059 A JP 2022520059A JP 2022549380 A5 JP2022549380 A5 JP 2022549380A5
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JP
Japan
Prior art keywords
cell
gjb2
promoter
sequence
nucleic acid
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.)
Granted
Application number
JP2022520059A
Other languages
English (en)
Japanese (ja)
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JP7713130B2 (ja
JP2022549380A (ja
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Priority claimed from PCT/US2020/053561 external-priority patent/WO2021067448A1/en
Publication of JP2022549380A publication Critical patent/JP2022549380A/ja
Publication of JP2022549380A5 publication Critical patent/JP2022549380A5/ja
Priority to JP2025035714A priority Critical patent/JP2025110904A/ja
Application granted granted Critical
Publication of JP7713130B2 publication Critical patent/JP7713130B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2022520059A 2019-09-30 2020-09-30 遺伝性難聴の処置のためのアデノ随伴ウイルス(aav)システム Active JP7713130B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025035714A JP2025110904A (ja) 2019-09-30 2025-03-06 遺伝性難聴の処置のためのアデノ随伴ウイルス(aav)システム

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025035714A Division JP2025110904A (ja) 2019-09-30 2025-03-06 遺伝性難聴の処置のためのアデノ随伴ウイルス(aav)システム

Publications (3)

Publication Number Publication Date
JP2022549380A JP2022549380A (ja) 2022-11-24
JP2022549380A5 true JP2022549380A5 (https=) 2023-10-10
JP7713130B2 JP7713130B2 (ja) 2025-07-25

Family

ID=75161339

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022520059A Active JP7713130B2 (ja) 2019-09-30 2020-09-30 遺伝性難聴の処置のためのアデノ随伴ウイルス(aav)システム
JP2025035714A Pending JP2025110904A (ja) 2019-09-30 2025-03-06 遺伝性難聴の処置のためのアデノ随伴ウイルス(aav)システム

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025035714A Pending JP2025110904A (ja) 2019-09-30 2025-03-06 遺伝性難聴の処置のためのアデノ随伴ウイルス(aav)システム

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=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
WO2021231808A2 (en) * 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating gjb2-associated hearing loss
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Dna分子の組成物、その作製方法、及びその使用方法
TW202227476A (zh) * 2020-09-14 2022-07-16 哈佛大學校長及研究員協會 編碼GJB2之重組腺相關病毒(rAAV)及其用途
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
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
WO2022155588A2 (en) * 2021-01-18 2022-07-21 Life Technologies Corporation Compositions, kits and methods for direct amplification from crude biological samples
EP4330413A4 (en) * 2021-04-27 2026-01-28 Childrens Hospital Philadelphia Adeno-associated viral vectors for cochlear transduction
MX2023013394A (es) * 2021-05-13 2023-12-14 Akouos Inc Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva.
CN118043468A (zh) * 2021-06-11 2024-05-14 分贝治疗公司 用于内耳中的细胞类型特异性基因表达的组合物和方法
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
WO2024081687A2 (en) * 2022-10-12 2024-04-18 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
US20240263143A1 (en) * 2023-02-02 2024-08-08 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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1755400A2 (en) * 2004-06-18 2007-02-28 The University Of Montana Aav mediated gene delivery to cochlear cells
US9085777B2 (en) * 2006-05-11 2015-07-21 Virttu Biologics Limited Mutant HSV, materials and methods for generation of mutant HSV
JP2010505442A (ja) * 2006-10-10 2010-02-25 ビロメッド カンパニー, リミテッド 安全性の改善された発現ベクター
WO2015130840A2 (en) * 2014-02-25 2015-09-03 Coda Therapeutics, Inc. Treatment of resistant lesions
GB201513176D0 (en) * 2015-07-27 2015-09-09 Glaxosmithkline Biolog Sa Novel methods for inducing an immune response
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
SG10201913266UA (en) * 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2017136764A1 (en) * 2016-02-05 2017-08-10 The General Hospital Corporation Hybrid system for efficient gene delivery to cells of the inner ear
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
CN109055499B (zh) * 2018-08-30 2021-01-19 杭州杰毅生物技术有限公司 基于CRISPR-Cas的等温核酸检测方法及试剂盒
TW202227476A (zh) * 2020-09-14 2022-07-16 哈佛大學校長及研究員協會 編碼GJB2之重組腺相關病毒(rAAV)及其用途

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