JPWO2020148458A5 - - Google Patents
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- Publication number
- JPWO2020148458A5 JPWO2020148458A5 JP2021541524A JP2021541524A JPWO2020148458A5 JP WO2020148458 A5 JPWO2020148458 A5 JP WO2020148458A5 JP 2021541524 A JP2021541524 A JP 2021541524A JP 2021541524 A JP2021541524 A JP 2021541524A JP WO2020148458 A5 JPWO2020148458 A5 JP WO2020148458A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- sequence
- otoferlin
- seq
- polynucleotide
- 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
- 108091033319 polynucleotide Proteins 0.000 claims description 18
- 239000002157 polynucleotide Substances 0.000 claims description 18
- 102000040430 polynucleotide Human genes 0.000 claims description 18
- 108050006335 Otoferlin Proteins 0.000 claims description 17
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 8
- 239000002245 particle Substances 0.000 claims description 8
- 229920001184 polypeptide Polymers 0.000 claims description 7
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 7
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 7
- 108091026890 Coding region Proteins 0.000 claims description 6
- 210000004899 c-terminal region Anatomy 0.000 claims description 5
- 230000008488 polyadenylation Effects 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 18
- 102100034198 Otoferlin Human genes 0.000 claims 8
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 206010011878 Deafness Diseases 0.000 claims 4
- 201000006093 autosomal recessive nonsyndromic deafness 9 Diseases 0.000 claims 4
- 208000032337 autosomal recessive nonsyndromic hearing loss 9 Diseases 0.000 claims 4
- 239000012634 fragment Substances 0.000 claims 4
- 230000010370 hearing loss Effects 0.000 claims 4
- 231100000888 hearing loss Toxicity 0.000 claims 4
- 208000016354 hearing loss disease Diseases 0.000 claims 4
- 230000035772 mutation Effects 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 101001134169 Homo sapiens Otoferlin Proteins 0.000 claims 2
- 230000006698 induction Effects 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 230000006798 recombination Effects 0.000 claims 2
- 238000005215 recombination Methods 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 210000000067 inner hair cell Anatomy 0.000 claims 1
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims 1
- 102220024085 rs397515607 Human genes 0.000 claims 1
- 241000894007 species Species 0.000 claims 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims 1
- 102000016774 Otoferlin Human genes 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024204975A JP2025032155A (ja) | 2019-01-18 | 2024-11-25 | オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP19305071.3 | 2019-01-18 | ||
| EP19305071 | 2019-01-18 | ||
| PCT/EP2020/051283 WO2020148458A1 (en) | 2019-01-18 | 2020-01-20 | Aav-mediated gene therapy restoring the otoferlin gene |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024204975A Division JP2025032155A (ja) | 2019-01-18 | 2024-11-25 | オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022522996A JP2022522996A (ja) | 2022-04-21 |
| JP2022522996A5 JP2022522996A5 (https=) | 2024-05-10 |
| JPWO2020148458A5 true JPWO2020148458A5 (https=) | 2024-05-10 |
| JP7595013B2 JP7595013B2 (ja) | 2024-12-05 |
Family
ID=65351986
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021541524A Active JP7595013B2 (ja) | 2019-01-18 | 2020-01-20 | オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 |
| JP2024204975A Pending JP2025032155A (ja) | 2019-01-18 | 2024-11-25 | オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024204975A Pending JP2025032155A (ja) | 2019-01-18 | 2024-11-25 | オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US20220125875A1 (https=) |
| EP (2) | EP4295913A3 (https=) |
| JP (2) | JP7595013B2 (https=) |
| KR (2) | KR20260046255A (https=) |
| CN (1) | CN113924109A (https=) |
| AU (2) | AU2020208933B2 (https=) |
| BR (1) | BR112021013996A2 (https=) |
| CA (1) | CA3126884A1 (https=) |
| DK (1) | DK3911354T5 (https=) |
| ES (1) | ES2960880T3 (https=) |
| FI (1) | FI3911354T3 (https=) |
| HR (1) | HRP20231296T1 (https=) |
| HU (1) | HUE063458T2 (https=) |
| IL (1) | IL284742B2 (https=) |
| LT (1) | LT3911354T (https=) |
| MX (1) | MX2021008622A (https=) |
| PL (1) | PL3911354T3 (https=) |
| PT (1) | PT3911354T (https=) |
| RS (1) | RS64794B1 (https=) |
| SI (1) | SI3911354T1 (https=) |
| SM (1) | SMT202300359T1 (https=) |
| WO (1) | WO2020148458A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018261769B2 (en) | 2017-05-05 | 2024-03-14 | The Regents Of The University Of California | Compositions and methods for expressing Otoferlin |
| 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 |
| 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 |
| 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 |
| AU2021376225A1 (en) * | 2020-11-06 | 2023-06-22 | Eli Lilly And Company | Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss |
| CN117295529A (zh) * | 2021-02-19 | 2023-12-26 | 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方法 |
| WO2023036966A1 (en) * | 2021-09-10 | 2023-03-16 | Institut Pasteur | Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof |
| CN117106824B (zh) * | 2022-05-17 | 2025-09-02 | 上海佑音医药生物科技有限公司 | 一种治疗听力损伤的双载体系统及其应用 |
| EP4527933A4 (en) * | 2022-05-17 | 2025-12-24 | Eye & Ent Hospital Of Fudan Univ | DUAL-SUPPORT SYSTEM FOR THE TREATMENT OF HEARING LOSS, AND ITS USE |
| 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 |
| CN117305367A (zh) * | 2023-08-21 | 2023-12-29 | 复旦大学附属眼耳鼻喉科医院 | 一种表达全长耳畸蛋白的双aav载体系统及其应用 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| WO2003059396A1 (en) | 2002-01-11 | 2003-07-24 | Sergei Zolotukhin | Adiponectin gene therapy |
| WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
| 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 |
| WO2011075838A1 (en) | 2009-12-21 | 2011-06-30 | Audigen Inc. | Method of treating or preventing hearing loss |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| 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 |
| US10751385B2 (en) * | 2015-02-20 | 2020-08-25 | Institut Pasteur | Prevention and/or treatment of hearing loss or impairment |
| SG11201804814YA (en) * | 2015-12-11 | 2018-07-30 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| 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 |
| IL325122A (en) * | 2018-02-22 | 2026-02-01 | Akouos Inc | Compositions and methods for treating non-age-related hearing impairment in humans |
| US11660353B2 (en) * | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual 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 |
-
2020
- 2020-01-20 WO PCT/EP2020/051283 patent/WO2020148458A1/en not_active Ceased
- 2020-01-20 EP EP23184106.5A patent/EP4295913A3/en active Pending
- 2020-01-20 SM SM20230359T patent/SMT202300359T1/it unknown
- 2020-01-20 CA CA3126884A patent/CA3126884A1/en active Pending
- 2020-01-20 CN CN202080022300.3A patent/CN113924109A/zh active Pending
- 2020-01-20 SI SI202030290T patent/SI3911354T1/sl unknown
- 2020-01-20 KR KR1020267009428A patent/KR20260046255A/ko active Pending
- 2020-01-20 LT LTEPPCT/EP2020/051283T patent/LT3911354T/lt unknown
- 2020-01-20 RS RS20230958A patent/RS64794B1/sr unknown
- 2020-01-20 MX MX2021008622A patent/MX2021008622A/es unknown
- 2020-01-20 BR BR112021013996-6A patent/BR112021013996A2/pt unknown
- 2020-01-20 PT PT207019514T patent/PT3911354T/pt unknown
- 2020-01-20 PL PL20701951.4T patent/PL3911354T3/pl unknown
- 2020-01-20 ES ES20701951T patent/ES2960880T3/es active Active
- 2020-01-20 HR HRP20231296TT patent/HRP20231296T1/hr unknown
- 2020-01-20 KR KR1020217026056A patent/KR102946735B1/ko active Active
- 2020-01-20 JP JP2021541524A patent/JP7595013B2/ja active Active
- 2020-01-20 EP EP20701951.4A patent/EP3911354B8/en active Active
- 2020-01-20 HU HUE20701951A patent/HUE063458T2/hu unknown
- 2020-01-20 AU AU2020208933A patent/AU2020208933B2/en active Active
- 2020-01-20 IL IL284742A patent/IL284742B2/en unknown
- 2020-01-20 DK DK20701951.4T patent/DK3911354T5/da active
- 2020-01-20 FI FIEP20701951.4T patent/FI3911354T3/fi active
-
2021
- 2021-01-20 US US17/422,737 patent/US20220125875A1/en active Pending
-
2024
- 2024-11-25 JP JP2024204975A patent/JP2025032155A/ja active Pending
-
2025
- 2025-11-28 AU AU2025271467A patent/AU2025271467A1/en not_active Abandoned
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