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- Withdrawn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 (enExample) | 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 (enExample) |
| EP (1) | EP3576696A4 (enExample) |
| JP (2) | JP2020508648A (enExample) |
| CN (1) | CN111698975A (enExample) |
| AU (2) | AU2018215785B2 (enExample) |
| CA (1) | CA3052704A1 (enExample) |
| WO (1) | WO2018145111A1 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018039375A1 (en) | 2016-08-23 | 2018-03-01 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| 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 |
| 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 |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN112703016B (zh) | 2018-04-27 | 2024-11-22 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| US12491223B2 (en) | 2018-09-27 | 2025-12-09 | Akouos, Inc. | Method for treating an auditory neuropathy spectrum disorder |
| US12534739B2 (en) | 2018-10-11 | 2026-01-27 | 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 |
| 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 |
| WO2020163761A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| EP3959324B1 (en) * | 2019-04-26 | 2025-09-17 | President and Fellows of Harvard College | Aav vectors encoding mini-pcdh15 and uses thereof |
| CN114269926A (zh) | 2019-04-26 | 2022-04-01 | 美商圣加莫治疗股份有限公司 | Aav工程化 |
| US12667628B2 (en) | 2019-07-03 | 2026-06-30 | Children's Medical Center Corporation | Compositions and methods for gene replacement |
| EP4007814A1 (en) | 2019-07-26 | 2022-06-08 | Akouos, Inc. | Methods of treating hearing loss using a secreted target protein |
| TWI896570B (zh) | 2019-10-30 | 2025-09-11 | 美商迪賽博治療公司 | 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法 |
| EP4055175A4 (en) * | 2019-11-04 | 2023-11-29 | Decibel Therapeutics, Inc. | Cochlear inner hair cell promoters and uses thereof |
| CN119391775A (zh) * | 2019-11-04 | 2025-02-07 | 分贝治疗公司 | 前庭支持细胞启动子和其用途 |
| 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 |
| CA3168055A1 (en) | 2020-02-21 | 2021-08-26 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| 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 |
| WO2023147383A1 (en) | 2022-01-25 | 2023-08-03 | Bridgebio Services Inc. | Gene therapy for genetic hearing loss |
| CN116606885A (zh) * | 2023-02-21 | 2023-08-18 | 东南大学 | 一种Lhfpl5基因敲除鼠构建实验方法 |
| WO2024239226A1 (zh) * | 2023-05-23 | 2024-11-28 | 苏州星奥拓维生物技术有限公司 | 耳蜗毛细胞特异性启动子及其用途 |
| 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 |
| CN111206032B (zh) | 2013-12-12 | 2024-07-19 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
| CA3007476A1 (en) * | 2015-12-11 | 2017-06-15 | Massachusetts Eye And 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 CN CN201880022337.9A patent/CN111698975A/zh active Pending
- 2018-02-06 WO PCT/US2018/017104 patent/WO2018145111A1/en not_active Ceased
- 2018-02-06 EP EP18747407.7A patent/EP3576696A4/en active Pending
- 2018-02-06 JP JP2019542464A patent/JP2020508648A/ja not_active Withdrawn
- 2018-02-06 CA CA3052704A patent/CA3052704A1/en active Pending
- 2018-02-06 US US16/483,668 patent/US11730827B2/en active Active
- 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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