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 | 2017-10-20 | ||
| EP17306430.4 | 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 |
| CN114206353B (zh) | 2019-05-20 | 2024-11-08 | 马萨诸塞大学 | 小基因疗法 |
| EP4132961A1 (en) * | 2020-04-10 | 2023-02-15 | 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 |
| US20240197913A1 (en) | 2021-04-20 | 2024-06-20 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Compositions and methods for treating retinal degenerative disorders |
| WO2022248634A1 (en) * | 2021-05-26 | 2022-12-01 | Sparingvision | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| 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 |
|---|---|---|---|---|
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, 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 |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| DE10216005A1 (de) | 2002-04-11 | 2003-10-30 | Max Planck Gesellschaft | Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle |
| EP2292780B1 (en) | 2003-09-30 | 2017-08-23 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US8906360B2 (en) | 2005-07-22 | 2014-12-09 | The Board Of Trustees Of The Leland Stanford Junior University | Light-activated cation channel and uses thereof |
| EP2019588A4 (en) | 2006-05-04 | 2010-11-24 | Univ Wayne State | RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN |
| 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 |
| DK2699270T3 (en) * | 2011-04-22 | 2017-10-02 | Univ California | ADENO-ASSOCIATED VIRUS VIRUSES WITH VARIANT CAPSID AND METHODS FOR USING IT |
| US10266845B2 (en) * | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| WO2015089462A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| ES2768763T3 (es) | 2014-03-04 | 2020-06-23 | Univ Florida | Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR |
| JP6689249B2 (ja) * | 2014-03-17 | 2020-05-20 | アドヴェラム バイオテクノロジーズ， インコーポレイテッド | 錐体細胞における増強された遺伝子発現のための組成物および方法 |
| PE20170260A1 (es) * | 2014-05-02 | 2017-04-12 | Genzyme Corp | Vectores de aav para la terapia genica de la retina y el snc |
| DK3821912T3 (da) * | 2014-07-24 | 2024-08-05 | Massachusetts Eye & Ear Infirmary | RPGR-genterapi mod retinitis pigmentosa |
| AU2016226289B2 (en) | 2015-03-02 | 2021-04-29 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| DK4206216T3 (da) * | 2016-05-13 | 2025-12-01 | 4D Molecular Therapeutics Inc | Adeno-associerede virusvarianter og fremgangsmåder til brug deraf |
| EP3585883A4 (en) * | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| 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 WO PCT/EP2018/078856 patent/WO2019077159A1/en not_active Ceased
- 2018-10-22 CN CN201880081245.8A patent/CN111867635A/zh active Pending
- 2018-10-22 JP JP2020522014A patent/JP7420710B2/ja active Active
- 2018-10-22 EP EP18789421.7A patent/EP3697448A1/en active Pending
- 2018-10-22 KR KR1020207014057A patent/KR102763312B1/ko active Active
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