JP2021500030A5 - - Google Patents
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- Publication number
- JP2021500030A5 JP2021500030A5 JP2020522014A JP2020522014A JP2021500030A5 JP 2021500030 A5 JP2021500030 A5 JP 2021500030A5 JP 2020522014 A JP2020522014 A JP 2020522014A JP 2020522014 A JP2020522014 A JP 2020522014A JP 2021500030 A5 JP2021500030 A5 JP 2021500030A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide
- seq
- composition
- interest
- capsid protein
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP17306430.4 | 2017-10-20 | ||
| EP17306430 | 2017-10-20 | ||
| PCT/EP2018/078856 WO2019077159A1 (en) | 2017-10-20 | 2018-10-22 | METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021500030A JP2021500030A (ja) | 2021-01-07 |
| JP2021500030A5 true JP2021500030A5 (enExample) | 2021-11-25 |
| JP7420710B2 JP7420710B2 (ja) | 2024-01-23 |
Family
ID=60201476
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522014A Active JP7420710B2 (ja) | 2017-10-20 | 2018-10-22 | 組換えａａｖ９由来ベクターの治療有効量の網膜下送達を含む、対象の錐体視細胞において目的のポリヌクレオチドを発現させる方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US11723988B2 (enExample) |
| EP (1) | EP3697448A1 (enExample) |
| JP (1) | JP7420710B2 (enExample) |
| KR (1) | KR102763312B1 (enExample) |
| CN (1) | CN111867635A (enExample) |
| WO (1) | WO2019077159A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2027889A1 (en) * | 2007-06-05 | 2009-02-25 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | New neuronal viability factor and use thereof |
| CN118360335A (zh) | 2017-04-05 | 2024-07-19 | 马萨诸塞大学 | 小基因治疗 |
| WO2020236815A1 (en) | 2019-05-20 | 2020-11-26 | University Of Massachusetts | Minigene therapy |
| US20230159609A1 (en) * | 2020-04-10 | 2023-05-25 | Sorbonne Université | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| CN113121655B (zh) * | 2021-04-19 | 2021-11-19 | 上海信致医药科技有限公司 | 眼部和肌肉特异靶向型腺相关病毒载体及其应用 |
| EP4326339B1 (en) | 2021-04-20 | 2025-04-02 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Compositions and methods for treating retinal degenerative disorders |
| JP2024520558A (ja) * | 2021-05-26 | 2024-05-24 | スパーリングヴィジョン | Ｇタンパク質ゲート－ｋ＋チャネルを介した桿体－錐体ジストロフィー（ｒｃｄ）における光感受性の増強 |
| EP4605537A1 (en) | 2022-10-20 | 2025-08-27 | Sparingvision | Compositions and methods for treating retinal degenerative disorders |
| WO2024110770A1 (en) * | 2022-11-22 | 2024-05-30 | INSERM (Institut National de la Santé et de la Recherche Médicale) | A new promoter for retinal pigment epithelium (rpe) targeted gene therapy |
| WO2024182558A1 (en) * | 2023-02-28 | 2024-09-06 | The Saban Research Institute; Children's Hospital Los Angeles | Fluorescent cone reporter ips cells, retinal organoids and uses thereof |
| CN117247434B (zh) * | 2023-11-10 | 2024-02-02 | 上海朗昇生物科技有限公司 | 衣壳修饰型病毒载体及其制备和用途 |
| CN119350454B (zh) * | 2024-12-27 | 2025-03-25 | 上海朗昇生物科技有限公司 | 衣壳重排型腺相关病毒载体及其用途 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| DE69939169D1 (de) | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| PT1127150E (pt) | 1998-11-05 | 2007-08-22 | Univ Pennsylvania | ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm'' |
| DE10216005A1 (de) | 2002-04-11 | 2003-10-30 | Max Planck Gesellschaft | Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| EP1919497B1 (en) | 2005-07-22 | 2020-02-12 | The Board of Trustees of the Leland Stanford Junior University | Light-activated cation channel and uses thereof |
| EP3437473A1 (en) | 2006-05-04 | 2019-02-06 | Wayne State University | Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids |
| EP2027889A1 (en) | 2007-06-05 | 2009-02-25 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | New neuronal viability factor and use thereof |
| DK4005603T3 (da) | 2011-04-22 | 2024-11-25 | Univ California | Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf |
| US10266845B2 (en) * | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| EP3653229A1 (en) | 2013-12-12 | 2020-05-20 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| US10308957B2 (en) * | 2014-03-04 | 2019-06-04 | University Of Florida Research Foundation, Inc. | rAAV vectors and methods for transduction of photoreceptors and RPE cells |
| ES3042252T3 (en) | 2014-03-17 | 2025-11-19 | Adverum Biotechnologies Inc | Compositions and methods for enhanced gene expression in cone cells |
| WO2015168666A2 (en) | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| PT3191139T (pt) * | 2014-07-24 | 2020-11-05 | Massachusetts Eye & Ear Infirmary | Terapia do gene rpgr para retinite pigmentosa |
| CN114480502A (zh) | 2015-03-02 | 2022-05-13 | 阿德夫拉姆生物技术股份有限公司 | 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法 |
| CN116333057A (zh) * | 2016-05-13 | 2023-06-27 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和其使用方法 |
| JP7237843B2 (ja) | 2017-02-21 | 2023-03-13 | ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． | 修飾されたａａｖキャプシドタンパク質およびその使用 |
| EP3697896A1 (en) | 2017-10-16 | 2020-08-26 | Vigeneron GmbH | Aav vectors |
-
2018
- 2018-10-22 US US16/756,988 patent/US11723988B2/en active Active
- 2018-10-22 KR KR1020207014057A patent/KR102763312B1/ko active Active
- 2018-10-22 WO PCT/EP2018/078856 patent/WO2019077159A1/en not_active Ceased
- 2018-10-22 JP JP2020522014A patent/JP7420710B2/ja active Active
- 2018-10-22 EP EP18789421.7A patent/EP3697448A1/en active Pending
- 2018-10-22 CN CN201880081245.8A patent/CN111867635A/zh active Pending
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