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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2022522996A JP2022522996A (ja) | 2022-04-21 |
| JPWO2020148458A5 true JPWO2020148458A5 (enExample) | 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 (enExample) |
| EP (2) | EP3911354B8 (enExample) |
| JP (2) | JP7595013B2 (enExample) |
| KR (1) | KR20210118112A (enExample) |
| CN (1) | CN113924109A (enExample) |
| AU (1) | AU2020208933B2 (enExample) |
| BR (1) | BR112021013996A2 (enExample) |
| CA (1) | CA3126884A1 (enExample) |
| DK (1) | DK3911354T5 (enExample) |
| ES (1) | ES2960880T3 (enExample) |
| FI (1) | FI3911354T3 (enExample) |
| HR (1) | HRP20231296T1 (enExample) |
| HU (1) | HUE063458T2 (enExample) |
| IL (1) | IL284742B2 (enExample) |
| LT (1) | LT3911354T (enExample) |
| MX (1) | MX2021008622A (enExample) |
| PL (1) | PL3911354T3 (enExample) |
| PT (1) | PT3911354T (enExample) |
| RS (1) | RS64794B1 (enExample) |
| SI (1) | SI3911354T1 (enExample) |
| SM (1) | SMT202300359T1 (enExample) |
| WO (1) | WO2020148458A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| WO2019210181A1 (en) | 2018-04-27 | 2019-10-31 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN114072179B (zh) | 2019-02-08 | 2024-11-01 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| BR112022016596A2 (pt) | 2020-02-21 | 2022-11-16 | Akouos Inc | Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano |
| CA3174500A1 (en) | 2020-04-01 | 2021-10-07 | University Of Florida Research Foundation, Incorporated | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| CA3197592A1 (en) * | 2020-11-06 | 2022-05-12 | Steven Pennock | Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss |
| WO2022178298A1 (en) * | 2021-02-19 | 2022-08-25 | Decibel Therapeutics, Inc. | Methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| IL311345A (en) * | 2021-09-10 | 2024-05-01 | Pasteur Institut | 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 |
| 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 (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2359869T (pt) | 2001-12-17 | 2019-04-16 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| AU2003209226A1 (en) | 2002-01-11 | 2003-07-30 | Michael Tennant | 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 |
| CN105408486B (zh) | 2013-05-21 | 2020-07-14 | 佛罗里达大学研究基金会有限公司 | 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途 |
| KR102794870B1 (ko) | 2015-12-11 | 2025-04-10 | 매사추세츠 아이 앤드 이어 인퍼머리 | 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법 |
| CA3034527A1 (en) * | 2016-08-23 | 2018-03-01 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| JP7408228B2 (ja) | 2018-02-22 | 2024-01-05 | 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及び方法 |
| US11660353B2 (en) * | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| 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. |
-
2020
- 2020-01-20 SI SI202030290T patent/SI3911354T1/sl unknown
- 2020-01-20 PT PT207019514T patent/PT3911354T/pt unknown
- 2020-01-20 JP JP2021541524A patent/JP7595013B2/ja active Active
- 2020-01-20 SM SM20230359T patent/SMT202300359T1/it unknown
- 2020-01-20 IL IL284742A patent/IL284742B2/en unknown
- 2020-01-20 BR BR112021013996-6A patent/BR112021013996A2/pt unknown
- 2020-01-20 KR KR1020217026056A patent/KR20210118112A/ko active Pending
- 2020-01-20 LT LTEPPCT/EP2020/051283T patent/LT3911354T/lt unknown
- 2020-01-20 ES ES20701951T patent/ES2960880T3/es active Active
- 2020-01-20 HR HRP20231296TT patent/HRP20231296T1/hr unknown
- 2020-01-20 AU AU2020208933A patent/AU2020208933B2/en active Active
- 2020-01-20 DK DK20701951.4T patent/DK3911354T5/da active
- 2020-01-20 EP EP20701951.4A patent/EP3911354B8/en active Active
- 2020-01-20 FI FIEP20701951.4T patent/FI3911354T3/fi active
- 2020-01-20 CA CA3126884A patent/CA3126884A1/en active Pending
- 2020-01-20 PL PL20701951.4T patent/PL3911354T3/pl unknown
- 2020-01-20 EP EP23184106.5A patent/EP4295913A3/en active Pending
- 2020-01-20 WO PCT/EP2020/051283 patent/WO2020148458A1/en not_active Ceased
- 2020-01-20 CN CN202080022300.3A patent/CN113924109A/zh active Pending
- 2020-01-20 HU HUE20701951A patent/HUE063458T2/hu unknown
- 2020-01-20 RS RS20230958A patent/RS64794B1/sr unknown
- 2020-01-20 MX MX2021008622A patent/MX2021008622A/es unknown
-
2021
- 2021-01-20 US US17/422,737 patent/US20220125875A1/en active Pending
-
2024
- 2024-11-25 JP JP2024204975A patent/JP2025032155A/ja active Pending
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