JP2023520488A5 - - Google Patents
Info
- Publication number
- JP2023520488A5 JP2023520488A5 JP2022560108A JP2022560108A JP2023520488A5 JP 2023520488 A5 JP2023520488 A5 JP 2023520488A5 JP 2022560108 A JP2022560108 A JP 2022560108A JP 2022560108 A JP2022560108 A JP 2022560108A JP 2023520488 A5 JP2023520488 A5 JP 2023520488A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide
- aav vector
- promoter
- sequence
- reverse
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063003774P | 2020-04-01 | 2020-04-01 | |
| US63/003,774 | 2020-04-01 | ||
| PCT/US2021/025281 WO2021202817A1 (en) | 2020-04-01 | 2021-03-31 | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023520488A JP2023520488A (ja) | 2023-05-17 |
| JP2023520488A5 true JP2023520488A5 (https=) | 2024-04-10 |
| JP7785359B2 JP7785359B2 (ja) | 2025-12-15 |
Family
ID=77929961
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022560108A Active JP7785359B2 (ja) | 2020-04-01 | 2021-03-31 | Ｕｓｈ１ｂの処置のための安全性が改善されたデュアルａａｖ－ｍｙｏ７ａベクター |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US12377169B2 (https=) |
| EP (1) | EP4126071A4 (https=) |
| JP (1) | JP7785359B2 (https=) |
| CN (1) | CN115666658A (https=) |
| AU (1) | AU2021247172A1 (https=) |
| CA (1) | CA3174500A1 (https=) |
| IL (1) | IL295741A (https=) |
| WO (1) | WO2021202817A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102606810B1 (ko) | 2017-05-05 | 2023-11-27 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 오토펄린을 발현시키기 위한 조성물 및 방법 |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| 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 |
| KR20220130093A (ko) | 2019-10-30 | 2022-09-26 | 데시벨 테라퓨틱스, 인크. | 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법 |
| US20240167056A1 (en) * | 2022-05-09 | 2024-05-23 | Akouos, Inc. | Cell specific gene therapy delivery compositions and methods for treating 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 |
| WO2025153705A1 (en) * | 2024-01-18 | 2025-07-24 | Splicebio, Sl | Use of split intein for the treatment of myo7a-associated disease |
| CN120400250A (zh) * | 2024-02-01 | 2025-08-01 | 苏州星奥拓维生物技术有限公司 | 表达myo7a蛋白的双载体系统及其用途 |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU4445701A (en) | 2000-03-24 | 2001-10-03 | Centre National De La Recherche Scientifique-Cnrs | Multiple human and mouse otoferlin isoforms |
| CN1934260B (zh) | 2004-01-22 | 2013-03-13 | 株式会社载体研究所 | 利用含有巨细胞病毒增强子和鸡β-肌动蛋白启动子的杂合启动子制造负链RNA病毒载体的方法 |
| WO2007127428A2 (en) | 2006-04-28 | 2007-11-08 | University Of Florida Research Foundation, Inc. | Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery |
| AU2007284651B2 (en) | 2006-08-09 | 2014-03-20 | Institute For Systems Biology | Organ-specific proteins and methods of their use |
| CN101711164B (zh) | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 |
| 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 |
| SG10201800541SA (en) | 2010-04-23 | 2018-03-28 | Univ Florida | 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 |
| WO2013151665A2 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| EP2970972B1 (en) * | 2013-03-11 | 2018-08-15 | Fondazione Telethon | Mir-204 and mir-211 and uses thereof |
| AU2014255665B2 (en) | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| EP3492597A3 (en) | 2013-05-21 | 2019-08-28 | University of Florida Research Foundation, Inc. | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer |
| US11000597B2 (en) | 2015-01-21 | 2021-05-11 | University Of Florida Research Foundation, Incorporated | Engineered receptor/ligand system for delivery of therapeutic agents |
| EP3258955A1 (en) | 2015-02-20 | 2017-12-27 | Institut Pasteur | Prevention and/or treatment of hearing loss or impairment |
| SI3265571T1 (sl) | 2015-03-03 | 2022-10-28 | Fondazione Telethon | Več vektorski sistem in njegove uporabe |
| CN107112767B (zh) | 2015-06-30 | 2019-06-07 | 深圳市大疆创新科技有限公司 | 充电控制电路、充电装置、充电系统及充电控制方法 |
| AU2016324317A1 (en) | 2015-09-17 | 2018-03-08 | Coda Biotherapeutics, Inc. | Compositions and methods for treating neurological disorders |
| SG10201913266UA (en) | 2015-12-11 | 2020-02-27 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| JP7007273B2 (ja) | 2015-12-22 | 2022-01-24 | アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） | 遺伝子治療用の改良された複合型二重組換えａａｖベクターシステム |
| EP3472328A1 (en) * | 2016-06-15 | 2019-04-24 | Oxford University Innovation Limited | Dual overlapping adeno-associated viral vector system for expressing abc4a |
| EP3510161A4 (en) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON |
| WO2018162748A1 (en) | 2017-03-10 | 2018-09-13 | Genethon | Treatment of glycogen storage disease iii |
| KR102606810B1 (ko) | 2017-05-05 | 2023-11-27 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 오토펄린을 발현시키기 위한 조성물 및 방법 |
| IL325122A (en) | 2018-02-22 | 2026-02-01 | Akouos Inc | Compositions and methods for treating non-age-related hearing impairment in humans |
| BR112020019079A2 (pt) * | 2018-03-23 | 2020-12-29 | Massachusetts Eye And Ear Infirmary | Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| EP4295913A3 (en) | 2019-01-18 | 2024-03-06 | Institut Pasteur | Aav-mediated gene therapy restoring the otoferlin gene |
| KR20220130093A (ko) | 2019-10-30 | 2022-09-26 | 데시벨 테라퓨틱스, 인크. | 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법 |
| KR20220157944A (ko) | 2020-02-21 | 2022-11-29 | 아카우오스, 인크. | 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법 |
-
2021
- 2021-03-31 CN CN202180032038.5A patent/CN115666658A/zh active Pending
- 2021-03-31 CA CA3174500A patent/CA3174500A1/en active Pending
- 2021-03-31 AU AU2021247172A patent/AU2021247172A1/en active Pending
- 2021-03-31 US US17/916,308 patent/US12377169B2/en active Active
- 2021-03-31 JP JP2022560108A patent/JP7785359B2/ja active Active
- 2021-03-31 EP EP21781660.2A patent/EP4126071A4/en active Pending
- 2021-03-31 IL IL295741A patent/IL295741A/en unknown
- 2021-03-31 WO PCT/US2021/025281 patent/WO2021202817A1/en not_active Ceased
-
2025
- 2025-06-23 US US19/245,523 patent/US20250381298A1/en active Pending
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