JP2020508648A5 - - Google Patents
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- Publication number
- JP2020508648A5 JP2020508648A5 JP2019542464A JP2019542464A JP2020508648A5 JP 2020508648 A5 JP2020508648 A5 JP 2020508648A5 JP 2019542464 A JP2019542464 A JP 2019542464A JP 2019542464 A JP2019542464 A JP 2019542464A JP 2020508648 A5 JP2020508648 A5 JP 2020508648A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- aav
- hair cell
- associated virus
- vector
- 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.)
- Withdrawn
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- 210000002768 hair cell Anatomy 0.000 claims description 16
- 241000702421 Dependoparvovirus Species 0.000 claims description 12
- 210000003027 ear inner Anatomy 0.000 claims description 12
- 230000008685 targeting Effects 0.000 claims description 12
- 102100024110 LHFPL tetraspan subfamily member 5 protein Human genes 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 7
- 206010011878 Deafness Diseases 0.000 claims description 6
- 102100037931 Harmonin Human genes 0.000 claims description 6
- 101710132730 Harmonin Proteins 0.000 claims description 6
- 102100036382 Protocadherin-15 Human genes 0.000 claims description 6
- 229920001184 polypeptide Polymers 0.000 claims description 6
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 6
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 6
- 208000014769 Usher Syndromes Diseases 0.000 claims description 5
- 102100031809 Espin Human genes 0.000 claims description 4
- 101710118108 Espin Proteins 0.000 claims description 4
- 101000981537 Homo sapiens LHFPL tetraspan subfamily member 5 protein Proteins 0.000 claims description 4
- 101000591234 Homo sapiens Phosphatidylinositol phosphatase PTPRQ Proteins 0.000 claims description 4
- 101001072259 Homo sapiens Protocadherin-15 Proteins 0.000 claims description 4
- 101000591236 Homo sapiens Receptor-type tyrosine-protein phosphatase R Proteins 0.000 claims description 4
- 101150050795 LHFPL5 gene Proteins 0.000 claims description 4
- 102100034101 Receptor-type tyrosine-protein phosphatase R Human genes 0.000 claims description 4
- 230000010370 hearing loss Effects 0.000 claims description 4
- 231100000888 hearing loss Toxicity 0.000 claims description 4
- 208000016354 hearing loss disease Diseases 0.000 claims description 4
- 102100032216 Calcium and integrin-binding protein 1 Human genes 0.000 claims description 2
- 101000943475 Homo sapiens Calcium and integrin-binding protein 1 Proteins 0.000 claims description 2
- 101000801040 Homo sapiens Transmembrane channel-like protein 1 Proteins 0.000 claims description 2
- 101000638069 Homo sapiens Transmembrane channel-like protein 2 Proteins 0.000 claims description 2
- 101100428000 Homo sapiens USH1G gene Proteins 0.000 claims description 2
- 206010048865 Hypoacusis Diseases 0.000 claims description 2
- 102000003505 Myosin Human genes 0.000 claims description 2
- 108060008487 Myosin Proteins 0.000 claims description 2
- 101710157841 Protocadherin-15 Proteins 0.000 claims description 2
- 102100033690 Transmembrane channel-like protein 1 Human genes 0.000 claims description 2
- 102100032054 Transmembrane channel-like protein 2 Human genes 0.000 claims description 2
- 201000008616 Usher syndrome type 1 Diseases 0.000 claims description 2
- 102100037929 Usher syndrome type-1G protein Human genes 0.000 claims description 2
- 210000000234 capsid Anatomy 0.000 claims description 2
- 230000002950 deficient Effects 0.000 claims description 2
- 230000006870 function Effects 0.000 claims description 2
- 210000000067 inner hair cell Anatomy 0.000 claims description 2
- 102000040430 polynucleotide Human genes 0.000 claims description 2
- 108091033319 polynucleotide Proteins 0.000 claims description 2
- 239000002157 polynucleotide Substances 0.000 claims description 2
- 238000004321 preservation Methods 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 8
- 238000000034 method Methods 0.000 description 11
- 239000000463 material Substances 0.000 description 5
- 239000000203 mixture Substances 0.000 description 2
- 102100022509 Cadherin-23 Human genes 0.000 description 1
- 101710196902 Cadherin-23 Proteins 0.000 description 1
- 208000011580 syndromic disease Diseases 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023000088A JP7601916B2 (ja) | 2017-02-06 | 2023-01-04 | 蝸牛および前庭細胞に核酸を送達するための物質および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762455197P | 2017-02-06 | 2017-02-06 | |
| US62/455,197 | 2017-02-06 | ||
| PCT/US2018/017104 WO2018145111A1 (en) | 2017-02-06 | 2018-02-06 | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023000088A Division JP7601916B2 (ja) | 2017-02-06 | 2023-01-04 | 蝸牛および前庭細胞に核酸を送達するための物質および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020508648A JP2020508648A (ja) | 2020-03-26 |
| JP2020508648A5 true JP2020508648A5 (https=) | 2021-03-18 |
Family
ID=63040112
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019542464A Withdrawn JP2020508648A (ja) | 2017-02-06 | 2018-02-06 | 蝸牛および前庭細胞に核酸を送達するための物質および方法 |
| JP2023000088A Active JP7601916B2 (ja) | 2017-02-06 | 2023-01-04 | 蝸牛および前庭細胞に核酸を送達するための物質および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023000088A Active JP7601916B2 (ja) | 2017-02-06 | 2023-01-04 | 蝸牛および前庭細胞に核酸を送達するための物質および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US11730827B2 (https=) |
| EP (1) | EP3576696A4 (https=) |
| JP (2) | JP2020508648A (https=) |
| CN (1) | CN111698975A (https=) |
| AU (2) | AU2018215785B2 (https=) |
| CA (1) | CA3052704A1 (https=) |
| WO (1) | WO2018145111A1 (https=) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| DE102018103924A1 (de) * | 2018-02-21 | 2019-08-22 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin | Gentherapeutische Behandlung von Schwerhörigkeit |
| JP7430652B2 (ja) | 2018-03-05 | 2024-02-13 | ザ チルドレンズ メディカル センター コーポレーション | 蝸牛および前庭細胞に核酸を送達するための組成物および方法 |
| 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 |
| WO2020069320A1 (en) | 2018-09-27 | 2020-04-02 | Akouos, Inc. | A method for treating an auditory neuropathy spectrum disorder |
| CA3115810A1 (en) | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
| CN113825521A (zh) * | 2018-12-21 | 2021-12-21 | 美利坚合众国由健康及人类服务部部长代表 | 腺相关病毒及其用于内耳疗法的用途 |
| 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 |
| WO2020219990A1 (en) * | 2019-04-26 | 2020-10-29 | President And Fellows Of Harvard College | Aav vectors encoding mini-pcdh15 and uses thereof |
| EP3959226A2 (en) | 2019-04-26 | 2022-03-02 | Sangamo Therapeutics, Inc. | Engineering aav |
| US20220396806A1 (en) * | 2019-07-26 | 2022-12-15 | Akouos, Inc. | Methods of treating hearing loss using a secreted target protein |
| CA3159549A1 (en) | 2019-10-30 | 2021-05-06 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| WO2021091950A1 (en) * | 2019-11-04 | 2021-05-14 | Decibel Therapeutics, Inc. | Vestibular supporting cell promoters and uses thereof |
| EP4055175A4 (en) * | 2019-11-04 | 2023-11-29 | Decibel Therapeutics, Inc. | Cochlear inner hair cell promoters and uses thereof |
| WO2021150850A1 (en) * | 2020-01-24 | 2021-07-29 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Svcs. | Adeno-associated viruses and their uses for inner ear therapy |
| 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 |
| WO2022129543A1 (en) * | 2020-12-18 | 2022-06-23 | Institut Pasteur | Gene therapy for treating usher syndrome |
| CN114762733B (zh) * | 2021-01-15 | 2025-03-18 | 中国科学院脑科学与智能技术卓越创新中心 | 耳蜗外毛细胞特异性顺式调节元件及其应用 |
| KR102751019B1 (ko) * | 2021-03-03 | 2025-01-09 | 연세대학교 산학협력단 | Kcnq4 유전자 교정용 가이드 rna 및 이를 포함하는 난청 치료용 조성물 |
| US20250114480A1 (en) | 2022-01-25 | 2025-04-10 | Bridgebio Gene Therapy Research, Inc. | Gene therapy for genetic hearing loss |
| EP4717770A1 (en) * | 2023-05-23 | 2026-04-01 | Otovia Therapeutics | Cochlear hair cell specific promoter and use thereof |
| CN119061007A (zh) * | 2023-06-02 | 2024-12-03 | 苏州星奥拓维生物技术有限公司 | 耳蜗支持细胞基因治疗和再生的方法 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8673634B2 (en) * | 2003-11-13 | 2014-03-18 | Massachusetts Eye & Ear Infirmary | Method for the treatment of hearing loss |
| WO2011075838A1 (en) | 2009-12-21 | 2011-06-30 | Audigen Inc. | Method of treating or preventing hearing loss |
| KR100990160B1 (ko) * | 2010-03-15 | 2010-10-29 | 도태환 | 기전력 안정화 영구자석 발전기 |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| AU2014361781B2 (en) | 2013-12-12 | 2021-04-01 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing |
| SG11201804814YA (en) | 2015-12-11 | 2018-07-30 | 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 |
| US20190254997A1 (en) * | 2016-07-21 | 2019-08-22 | The Regents Of The University Of California | Methods for inducing stereocilia on hair cells |
-
2018
- 2018-02-06 WO PCT/US2018/017104 patent/WO2018145111A1/en not_active Ceased
- 2018-02-06 JP JP2019542464A patent/JP2020508648A/ja not_active Withdrawn
- 2018-02-06 CN CN201880022337.9A patent/CN111698975A/zh active Pending
- 2018-02-06 US US16/483,668 patent/US11730827B2/en active Active
- 2018-02-06 EP EP18747407.7A patent/EP3576696A4/en active Pending
- 2018-02-06 CA CA3052704A patent/CA3052704A1/en active Pending
- 2018-02-06 AU AU2018215785A patent/AU2018215785B2/en active Active
-
2023
- 2023-01-04 JP JP2023000088A patent/JP7601916B2/ja active Active
- 2023-07-03 US US18/346,720 patent/US20230330268A1/en active Pending
- 2023-08-09 AU AU2023214288A patent/AU2023214288A1/en active Pending
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