JP2023070682A5 - - Google Patents
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- JP2023070682A5 JP2023070682A5 JP2023027073A JP2023027073A JP2023070682A5 JP 2023070682 A5 JP2023070682 A5 JP 2023070682A5 JP 2023027073 A JP2023027073 A JP 2023027073A JP 2023027073 A JP2023027073 A JP 2023027073A JP 2023070682 A5 JP2023070682 A5 JP 2023070682A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- sequence
- polynucleotide
- seq
- promoter
- 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.)
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- 108091033319 polynucleotide Proteins 0.000 claims 8
- 239000002157 polynucleotide Substances 0.000 claims 8
- 102000040430 polynucleotide Human genes 0.000 claims 8
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 108020005067 RNA Splice Sites Proteins 0.000 claims 4
- 239000002245 particle Substances 0.000 claims 4
- 229920001184 polypeptide Polymers 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims 4
- 102100034198 Otoferlin Human genes 0.000 claims 3
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 230000010370 hearing loss Effects 0.000 claims 3
- 231100000888 hearing loss Toxicity 0.000 claims 3
- 208000016354 hearing loss disease Diseases 0.000 claims 3
- 108091026890 Coding region Proteins 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 101001134169 Homo sapiens Otoferlin Proteins 0.000 claims 2
- 208000027601 Inner ear disease Diseases 0.000 claims 2
- 108050006335 Otoferlin Proteins 0.000 claims 2
- 108091081062 Repeated sequence (DNA) Proteins 0.000 claims 2
- 201000006093 autosomal recessive nonsyndromic deafness 9 Diseases 0.000 claims 2
- 208000032337 autosomal recessive nonsyndromic hearing loss 9 Diseases 0.000 claims 2
- 108010006025 bovine growth hormone Proteins 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 208000027491 vestibular disease Diseases 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 206010011878 Deafness Diseases 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 101150006256 Otof gene Proteins 0.000 claims 1
- 108010029485 Protein Isoforms Proteins 0.000 claims 1
- 102000001708 Protein Isoforms Human genes 0.000 claims 1
- 210000004899 c-terminal region Anatomy 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 102000052168 human OTOF Human genes 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762502462P | 2017-05-05 | 2017-05-05 | |
| US62/502,462 | 2017-05-05 | ||
| JP2019560646A 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 |
| PCT/US2018/031009 WO2018204734A1 (en) | 2017-05-05 | 2018-05-04 | Compositions and methods for expressing otoferlin |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019560646A Division 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024226066A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023070682A JP2023070682A (ja) | 2023-05-19 |
| JP2023070682A5 true JP2023070682A5 (enExample) | 2023-07-18 |
| JP7617600B2 JP7617600B2 (ja) | 2025-01-20 |
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 Before (1)
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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) | KR102606810B1 (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 |
| EP3510161A4 (en) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT |
| CN110892062A (zh) * | 2017-05-05 | 2020-03-17 | 佛罗里达大学研究基金会 | 表达耳畸蛋白的组合物和方法 |
| DE102018103924A1 (de) * | 2018-02-21 | 2019-08-22 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin | Gentherapeutische Behandlung von Schwerhörigkeit |
| AU2019224121B2 (en) * | 2018-02-22 | 2025-08-07 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| JP7331011B2 (ja) | 2018-04-27 | 2023-08-22 | デシベル セラピューティクス インコーポレイテッド | ミオシン１５プロモーター及びその使用 |
| WO2020093018A1 (en) * | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| AU2020208933B2 (en) * | 2019-01-18 | 2025-08-28 | Centre National De La Recherche Scientifique (Cnrs) | AAV-mediated gene therapy restoring the otoferlin gene |
| CA3129422A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| WO2020163743A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| 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. |
| 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 |
| WO2021168362A1 (en) | 2020-02-21 | 2021-08-26 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| CN115666658A (zh) | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | 具有提高的治疗ush1b的安全性的双aav-myo7a载体 |
| 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 |
| EP2434020B1 (en) | 2004-01-22 | 2014-11-05 | Dnavec Research Inc. | Method of producing Paramyxoviridae viral vectors using a hybrid promoter comprising a cytomegalovirus enhancer and a chicken beta-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 |
| JP5244103B2 (ja) | 2006-08-09 | 2013-07-24 | ホームステッド クリニカル コーポレイション | 器官特異的蛋白質およびその使用方法 |
| JP5575486B2 (ja) | 2007-01-18 | 2014-08-20 | ユニヴァーシティ オブ ミズーリー−コロンビア | 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子 |
| 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 |
| US8962314B2 (en) * | 2008-11-26 | 2015-02-24 | The Regents Of The University Of California | Lateral ventricle cell compositions and use for treating neural degenerative diseases |
| EA202091105A1 (ru) * | 2010-04-23 | 2020-12-30 | Юниверсити Оф Флорида Рисерч Фаундейшн, Инк. | Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 1 (lca1) |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
| WO2014140051A1 (en) | 2013-03-11 | 2014-09-18 | Fondazione Telethon | Mir-204 and mir-211 and uses thereof |
| US10494645B2 (en) * | 2013-04-18 | 2019-12-03 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| AU2014274457B2 (en) | 2013-05-21 | 2019-10-24 | University Of Florida Research Foundation, Inc. | Capsid-modified, rAAV3 vector compositions and uses 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 |
| DK3265571T3 (da) * | 2015-03-03 | 2022-06-27 | Fond Telethon | Fler-vektorsystem og anvendelse heraf |
| KR20200108514A (ko) | 2015-09-17 | 2020-09-21 | 코다 바이오테라퓨틱스 인코포레이티드 | 신경 장애를 치료하기 위한 조성물 및 방법 |
| JP6990182B2 (ja) | 2015-12-11 | 2022-02-15 | マサチューセッツ アイ アンド イヤー インファーマリー | 蝸牛および前庭細胞に核酸を送達するための材料および方法 |
| EP3393522B1 (en) | 2015-12-22 | 2021-07-07 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Improved hybrid dual recombinant aav vector systems for gene therapy |
| BR112018075855A2 (pt) | 2016-06-15 | 2019-04-02 | Oxford University Innovation Limited | sistema de vetor viral adeno-associado com dupla sobreposição para a expressão abc4a |
| EP3510161A4 (en) * | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT |
| EP3592848A1 (en) | 2017-03-10 | 2020-01-15 | Genethon | Treatment of glycogen storage disease iii |
| CN110892062A (zh) | 2017-05-05 | 2020-03-17 | 佛罗里达大学研究基金会 | 表达耳畸蛋白的组合物和方法 |
| AU2019224121B2 (en) | 2018-02-22 | 2025-08-07 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| 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 |
| AU2020208933B2 (en) | 2019-01-18 | 2025-08-28 | Centre National De La Recherche Scientifique (Cnrs) | AAV-mediated gene therapy restoring the otoferlin gene |
| 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. |
| WO2021168362A1 (en) | 2020-02-21 | 2021-08-26 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| CN115666658A (zh) | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | 具有提高的治疗ush1b的安全性的双aav-myo7a载体 |
-
2018
- 2018-05-04 CN CN201880029806.XA patent/CN110892062A/zh active Pending
- 2018-05-04 AU AU2018261769A patent/AU2018261769B2/en active Active
- 2018-05-04 KR KR1020197035845A patent/KR102606810B1/ko active Active
- 2018-05-04 CA CA3061955A patent/CA3061955A1/en active Pending
- 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 MX MX2019013151A patent/MX2019013151A/es unknown
- 2018-05-04 US US16/611,049 patent/US12359221B2/en active Active
- 2018-05-04 JP JP2019560646A patent/JP7240675B2/ja 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
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