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 JP7851563B2 (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 | ||
| PCT/US2018/031009 WO2018204734A1 (en) | 2017-05-05 | 2018-05-04 | Compositions and methods for expressing otoferlin |
| 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 |
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 JP7851563B2 (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 (https=) | 2023-07-18 |
| JP7617600B2 JP7617600B2 (ja) | 2025-01-20 |
Family
ID=64016253
Family Applications (2)
| 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 |
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 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US12359221B2 (https=) |
| EP (1) | EP3635100A4 (https=) |
| JP (2) | JP7240675B2 (https=) |
| KR (2) | KR20230167138A (https=) |
| CN (1) | CN110892062A (https=) |
| AU (2) | AU2018261769B2 (https=) |
| CA (1) | CA3061955A1 (https=) |
| MX (2) | MX2019013151A (https=) |
| WO (1) | WO2018204734A1 (https=) |
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 |
| 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 |
| AU2018261769B2 (en) * | 2017-05-05 | 2024-03-14 | 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 |
| IL325122A (en) * | 2018-02-22 | 2026-02-01 | Akouos Inc | Compositions and methods for treating non-age-related hearing impairment in humans |
| CN119410636A (zh) | 2018-04-27 | 2025-02-11 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| US12188041B2 (en) | 2018-11-01 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Codon optimized otoferlin AAV dual vector gene therapy |
| WO2020148458A1 (en) * | 2019-01-18 | 2020-07-23 | Institut Pasteur | Aav-mediated gene therapy restoring the otoferlin gene |
| AU2020218547A1 (en) | 2019-02-08 | 2021-08-26 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| 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 |
| CA3159549A1 (en) | 2019-10-30 | 2021-05-06 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN115066432B (zh) * | 2019-10-30 | 2026-04-17 | 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 |
| 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 |
| AU2021247172A1 (en) | 2020-04-01 | 2022-09-15 | University Of Florida Research Foundation, Incorporated | Dual AAV-MY07A 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 |
| JP4999330B2 (ja) | 2004-01-22 | 2012-08-15 | 株式会社ディナベック研究所 | サイトメガロウイルスエンハンサーおよびニワトリβ−アクチンプロモーターを含むハイブリッドプロモーターを利用したマイナス鎖ＲＮＡウイルスベクターの製造方法 |
| 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 |
| EP2520935A3 (en) | 2006-08-09 | 2013-02-13 | Homestead Clinical Corporation | Organ-specific proteins and methods of their use |
| 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 |
| HRP20190144T1 (hr) * | 2010-04-23 | 2019-03-22 | University Of Florida Research Foundation, Inc. | Pripravci raav-gvanilat ciklaze i postupci za liječenje leberove urođene amauroze-1 (lca1) |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| AU2013243948A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| ES2697249T3 (es) | 2013-03-11 | 2019-01-22 | Fond Telethon | miR-204 y miR-211 y usos de los mismos |
| CA2909733C (en) * | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| KR102234672B1 (ko) | 2013-05-21 | 2021-04-06 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도 |
| US11000597B2 (en) | 2015-01-21 | 2021-05-11 | University Of Florida Research Foundation, Incorporated | Engineered receptor/ligand system for delivery of therapeutic agents |
| US10751385B2 (en) * | 2015-02-20 | 2020-08-25 | Institut Pasteur | Prevention and/or treatment of hearing loss or impairment |
| RS63416B1 (sr) * | 2015-03-03 | 2022-08-31 | Fond Telethon | Sistem višestrukih vektora i njegove primene |
| AU2016324317A1 (en) | 2015-09-17 | 2018-03-08 | Coda Biotherapeutics, Inc. | Compositions and methods for treating neurological disorders |
| SG11201804814YA (en) | 2015-12-11 | 2018-07-30 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| WO2017108931A1 (en) | 2015-12-22 | 2017-06-29 | 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 |
| 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 |
| US11541131B2 (en) | 2017-03-10 | 2023-01-03 | Genethon | Treatment of glycogen storage disease III |
| AU2018261769B2 (en) | 2017-05-05 | 2024-03-14 | The Regents Of The University Of California | Compositions and methods for expressing Otoferlin |
| IL325122A (en) | 2018-02-22 | 2026-02-01 | Akouos Inc | Compositions and methods for treating non-age-related hearing impairment in humans |
| CA3094828A1 (en) | 2018-03-23 | 2019-09-26 | Massachusetts Eye And Ear Infirmary | Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome |
| US12188041B2 (en) | 2018-11-01 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Codon optimized otoferlin AAV dual vector gene therapy |
| WO2020148458A1 (en) | 2019-01-18 | 2020-07-23 | Institut Pasteur | Aav-mediated gene therapy restoring the otoferlin gene |
| CA3159549A1 (en) | 2019-10-30 | 2021-05-06 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| 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 |
| AU2021247172A1 (en) | 2020-04-01 | 2022-09-15 | University Of Florida Research Foundation, Incorporated | Dual AAV-MY07A vectors with improved safety for the treatment of USH1B |
-
2018
- 2018-05-04 AU AU2018261769A patent/AU2018261769B2/en active Active
- 2018-05-04 KR KR1020237040309A patent/KR20230167138A/ko not_active Ceased
- 2018-05-04 EP EP18793935.0A patent/EP3635100A4/en active Pending
- 2018-05-04 JP JP2019560646A patent/JP7240675B2/ja active Active
- 2018-05-04 CN CN201880029806.XA patent/CN110892062A/zh active Pending
- 2018-05-04 KR KR1020197035845A patent/KR102606810B1/ko active Active
- 2018-05-04 MX MX2019013151A patent/MX2019013151A/es unknown
- 2018-05-04 WO PCT/US2018/031009 patent/WO2018204734A1/en not_active Ceased
- 2018-05-04 CA CA3061955A patent/CA3061955A1/en active Pending
- 2018-05-04 US US16/611,049 patent/US12359221B2/en 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/AU2024203084B2/en active Active
-
2025
- 2025-06-10 US US19/233,471 patent/US20250367325A1/en active Pending
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