JP2020518268A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020518268A5 JP2020518268A5 JP2019560646A JP2019560646A JP2020518268A5 JP 2020518268 A5 JP2020518268 A5 JP 2020518268A5 JP 2019560646 A JP2019560646 A JP 2019560646A JP 2019560646 A JP2019560646 A JP 2019560646A JP 2020518268 A5 JP2020518268 A5 JP 2020518268A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- composition according
- seq
- optionally
- composition
- 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
Links
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023027073A JP7617600B2 (ja) | 2017-05-05 | 2023-02-24 | オトフェルリンを発現させるための組成物および方法 |
| JP2024226066A JP2025063057A (ja) | 2017-05-05 | 2024-12-23 | オトフェルリンを発現させるための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762502462P | 2017-05-05 | 2017-05-05 | |
| US62/502,462 | 2017-05-05 | ||
| PCT/US2018/031009 WO2018204734A1 (en) | 2017-05-05 | 2018-05-04 | Compositions and methods for expressing otoferlin |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023027073A Division JP7617600B2 (ja) | 2017-05-05 | 2023-02-24 | オトフェルリンを発現させるための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020518268A JP2020518268A (ja) | 2020-06-25 |
| JP2020518268A5 true JP2020518268A5 (enExample) | 2021-07-26 |
| JP7240675B2 JP7240675B2 (ja) | 2023-03-16 |
Family
ID=64016253
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019560646A Active JP7240675B2 (ja) | 2017-05-05 | 2018-05-04 | オトフェルリンを発現させるための組成物および方法 |
| JP2023027073A Active JP7617600B2 (ja) | 2017-05-05 | 2023-02-24 | オトフェルリンを発現させるための組成物および方法 |
| JP2024226066A Pending JP2025063057A (ja) | 2017-05-05 | 2024-12-23 | オトフェルリンを発現させるための組成物および方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023027073A Active JP7617600B2 (ja) | 2017-05-05 | 2023-02-24 | オトフェルリンを発現させるための組成物および方法 |
| JP2024226066A Pending JP2025063057A (ja) | 2017-05-05 | 2024-12-23 | オトフェルリンを発現させるための組成物および方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US12359221B2 (enExample) |
| EP (1) | EP3635100A4 (enExample) |
| JP (3) | JP7240675B2 (enExample) |
| KR (2) | KR20230167138A (enExample) |
| CN (1) | CN110892062A (enExample) |
| AU (2) | AU2018261769B2 (enExample) |
| CA (1) | CA3061955A1 (enExample) |
| MX (2) | MX2019013151A (enExample) |
| WO (1) | WO2018204734A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| CA3034527A1 (en) * | 2016-08-23 | 2018-03-01 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | 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 |
| JP7408228B2 (ja) * | 2018-02-22 | 2024-01-05 | 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及び方法 |
| WO2019210181A1 (en) | 2018-04-27 | 2019-10-31 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| WO2020093018A1 (en) * | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| SI3911354T1 (sl) * | 2019-01-18 | 2023-12-29 | Institut Pasteur | Aav posredovana genska terapija za obnovitev otoferlin gena |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN114072179B (zh) | 2019-02-08 | 2024-11-01 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| CN115066432A (zh) * | 2019-10-30 | 2022-09-16 | 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组合物和方法 |
| MX2022005236A (es) * | 2019-10-30 | 2022-06-08 | Decibel Therapeutics Inc | Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina. |
| BR112022016596A2 (pt) | 2020-02-21 | 2022-11-16 | Akouos Inc | Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano |
| CA3174500A1 (en) | 2020-04-01 | 2021-10-07 | University Of Florida Research Foundation, Incorporated | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| CN112125969A (zh) * | 2020-09-30 | 2020-12-25 | 东南大学 | 生物因子rimbp2在维持内耳毛细胞特性上的应用 |
| 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 |
| WO2025201333A1 (zh) * | 2024-03-28 | 2025-10-02 | 苏州星奥拓维生物技术有限公司 | 递送Otof基因的双载体系统和其用途 |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001070972A2 (en) * | 2000-03-24 | 2001-09-27 | Yasunaga, Shin'ichiro | Multiple human and mouse otoferlin isoforms |
| US8741650B2 (en) | 2004-01-22 | 2014-06-03 | Dnavec Research Inc. | Methods for producing minus-strand RNA viral vectors using hybrid promoter comprising cytomegalovirus enhancer and chicken β-actin promoter |
| 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 |
| CA2660286A1 (en) | 2006-08-09 | 2008-02-21 | Homestead Clinical Corporation | Organ-specific proteins and methods of their use |
| US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
| FR2919305B1 (fr) | 2007-07-26 | 2009-09-18 | Genethon Ass Loi De 1901 | Vecteurs viraux adeno-associes pour l'expression de la dysferline. |
| US8236557B2 (en) * | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| WO2010062905A2 (en) * | 2008-11-26 | 2010-06-03 | The Regents Of The University Of California | Lateral ventricle cell compositions and use for treating neural degenerative diseases |
| AU2011242527B2 (en) * | 2010-04-23 | 2016-05-19 | University Of Florida Research Foundation, Inc. | rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1) |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| HK1206601A1 (en) | 2012-04-02 | 2016-01-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| CN105209619B (zh) | 2013-03-11 | 2018-10-12 | 泰莱托恩基金会 | miR-204和miR-211及其用途 |
| DK2986635T3 (en) | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
| CN105408486B (zh) | 2013-05-21 | 2020-07-14 | 佛罗里达大学研究基金会有限公司 | 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途 |
| US11000597B2 (en) | 2015-01-21 | 2021-05-11 | University Of Florida Research Foundation, Incorporated | Engineered receptor/ligand system for delivery of therapeutic agents |
| WO2016131981A1 (en) * | 2015-02-20 | 2016-08-25 | Institut Pasteur | Prevention and/or treatment of hearing loss or impairment |
| KR102240180B1 (ko) | 2015-03-03 | 2021-04-14 | 폰다지오네 텔레톤 | 다중 벡터 시스템 및 이의 용도 |
| CN108348528A (zh) | 2015-09-17 | 2018-07-31 | 科达生物治疗公司 | 治疗神经障碍的组合物和方法 |
| KR102794870B1 (ko) | 2015-12-11 | 2025-04-10 | 매사추세츠 아이 앤드 이어 인퍼머리 | 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법 |
| CA3008956C (en) | 2015-12-22 | 2023-01-03 | Inserm (Institut National De La Sante Et De La Recherche Medicale) | Improved hybrid dual recombinant aav vector systems for gene therapy |
| EP3472328A1 (en) | 2016-06-15 | 2019-04-24 | Oxford University Innovation Limited | Dual overlapping adeno-associated viral vector system for expressing abc4a |
| CA3034527A1 (en) | 2016-08-23 | 2018-03-01 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| WO2018162748A1 (en) | 2017-03-10 | 2018-09-13 | Genethon | Treatment of glycogen storage disease iii |
| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| JP7408228B2 (ja) | 2018-02-22 | 2024-01-05 | 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及び方法 |
| US12331328B2 (en) | 2018-03-23 | 2025-06-17 | Massachusetts Eye And Ear Infirmary | CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated usher syndrome |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| SI3911354T1 (sl) | 2019-01-18 | 2023-12-29 | Institut Pasteur | Aav posredovana genska terapija za obnovitev otoferlin gena |
| MX2022005236A (es) | 2019-10-30 | 2022-06-08 | Decibel Therapeutics Inc | Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina. |
| BR112022016596A2 (pt) | 2020-02-21 | 2022-11-16 | Akouos Inc | Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano |
| CA3174500A1 (en) | 2020-04-01 | 2021-10-07 | University Of Florida Research Foundation, Incorporated | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
-
2018
- 2018-05-04 US US16/611,049 patent/US12359221B2/en active Active
- 2018-05-04 CN CN201880029806.XA patent/CN110892062A/zh active Pending
- 2018-05-04 MX MX2019013151A patent/MX2019013151A/es unknown
- 2018-05-04 AU AU2018261769A patent/AU2018261769B2/en active Active
- 2018-05-04 JP JP2019560646A patent/JP7240675B2/ja active Active
- 2018-05-04 WO PCT/US2018/031009 patent/WO2018204734A1/en not_active Ceased
- 2018-05-04 EP EP18793935.0A patent/EP3635100A4/en active Pending
- 2018-05-04 KR KR1020237040309A patent/KR20230167138A/ko not_active Ceased
- 2018-05-04 CA CA3061955A patent/CA3061955A1/en active Pending
- 2018-05-04 KR KR1020197035845A patent/KR102606810B1/ko active Active
-
2019
- 2019-11-04 MX MX2023013435A patent/MX2023013435A/es unknown
-
2023
- 2023-02-24 JP JP2023027073A patent/JP7617600B2/ja active Active
-
2024
- 2024-05-09 AU AU2024203084A patent/AU2024203084A1/en active Pending
- 2024-12-23 JP JP2024226066A patent/JP2025063057A/ja active Pending
-
2025
- 2025-06-10 US US19/233,471 patent/US20250367325A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020518268A5 (enExample) | ||
| JP2023070682A5 (enExample) | ||
| JP6832280B2 (ja) | 新規のマイクロジストロフィンおよび使用の関連する方法 | |
| JP2024020346A (ja) | 筋疾患の治療のための改変aavカプシドポリペプチド | |
| JP2018138049A5 (enExample) | ||
| JP2015521465A5 (enExample) | ||
| JP2025063057A5 (enExample) | ||
| JP2020520662A5 (enExample) | ||
| NZ603348A (en) | Simian adenovirus nucleic acid- and amino acid-sequences, vectors containing same, and uses thereof | |
| JP7427208B2 (ja) | 視覚機能再生剤又は視覚機能低下予防剤 | |
| CN116440292A (zh) | Danon病和其它自噬障碍的治疗方法 | |
| JP2018537089A5 (enExample) | ||
| JPWO2019156137A5 (enExample) | ||
| CN114929735A (zh) | 因子viii构建体 | |
| US20240002462A1 (en) | Treatment of neuropathy with igf-1-encoding dna constructs and hgf-encoding dna constructs | |
| AU2017301819B2 (en) | Gene therapy for the treatment of aldehyde dehydrogenase deficiency | |
| JPWO2019165292A5 (enExample) | ||
| WO2017151717A1 (en) | Compositions and methods for adeno-associated virus mediated gene expression in myofibroblast-like cells | |
| JP2020535804A5 (enExample) | ||
| JP2020127419A5 (enExample) | ||
| ES2729872T3 (es) | Terapias basadas en la transcriptasa inversa de la telomerasa | |
| JPWO2020047467A5 (enExample) | ||
| US20180140720A1 (en) | Compositions and methods for tissue regeneration | |
| EP4153745A1 (en) | Viral vectors and nucleic acids for regulated gene therapy | |
| JP2008502323A5 (enExample) |