JP2021500030A5 - - Google Patents
Download PDFInfo
- 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 (https=) | 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 (https=) |
| EP (1) | EP3697448A1 (https=) |
| JP (1) | JP7420710B2 (https=) |
| KR (1) | KR102763312B1 (https=) |
| CN (1) | CN111867635A (https=) |
| WO (1) | WO2019077159A1 (https=) |
Families Citing this family (13)
| 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 |
| CN110709095B (zh) | 2017-04-05 | 2024-05-03 | 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) |
| CA3195696A1 (en) * | 2020-09-17 | 2022-03-24 | Restore Vision Inc. | Composition for treating or preventing diseases, disorders, or conditions associated with endoplasmic reticulum stress or all-trans-retinal, protecting retinal thickness, or suppressing reduction in retinal thickness or progress of reduction in retinal thickness |
| 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 |
| WO2022223644A2 (en) | 2021-04-20 | 2022-10-27 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Compositions and methods for treating retinal degenerative disorders |
| CA3221439A1 (en) * | 2021-05-26 | 2022-12-01 | Sparingvision | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| IL320351A (en) | 2022-10-20 | 2025-06-01 | 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 |
|---|---|---|---|---|
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, 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 |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| 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 |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| 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 |
| CN101484005A (zh) | 2006-05-04 | 2009-07-15 | 韦恩州立大学 | 通过向体内递送视紫红质核酸恢复视觉响应 |
| 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 |
| CN105755044A (zh) * | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| US10266845B2 (en) * | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| JP7103750B2 (ja) * | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用 |
| EP3113787B1 (en) * | 2014-03-04 | 2019-12-04 | University of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
| EP3800191B1 (en) * | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| HUE068183T2 (hu) * | 2014-07-24 | 2024-12-28 | Massachusetts Eye & Ear Infirmary | RPGR génterápia a retinitis pigmentosa terápiájára |
| US11021519B2 (en) | 2015-03-02 | 2021-06-01 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| US11364308B2 (en) * | 2016-05-13 | 2022-06-21 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| EP3585883A4 (en) | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| US12043848B2 (en) * | 2017-10-16 | 2024-07-23 | Vigeneron Gmbh | AAV vectors |
-
2018
- 2018-10-22 CN CN201880081245.8A patent/CN111867635A/zh active Pending
- 2018-10-22 EP EP18789421.7A patent/EP3697448A1/en active Pending
- 2018-10-22 JP JP2020522014A patent/JP7420710B2/ja active Active
- 2018-10-22 KR KR1020207014057A patent/KR102763312B1/ko active Active
- 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
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021500030A5 (https=) | ||
| Liu et al. | Gene therapy for ocular diseases | |
| US9597415B2 (en) | Methods for expressing a polynucleotide of interest in the retina of a subject | |
| CN107530399A (zh) | 治疗分子在体外和体内的有效递送 | |
| AU2017222948B2 (en) | Gene therapy for the treatment of a disease of retinal cone cells | |
| JP2016535034A5 (https=) | ||
| JP2022110004A (ja) | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 | |
| HUE034510T2 (en) | Adeno-associated virus virions with capsid variant and methods for their use | |
| HRP20201225T1 (hr) | Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31 | |
| EP3533877A3 (de) | Viraler vektor für den zielgerichteten gentransfer in gehirn und rückenmark | |
| Torrecilla et al. | Targeting corneal inflammation by gene therapy: Emerging strategies for keratitis | |
| JP2017523239A5 (https=) | ||
| JP2014524302A (ja) | 眼疾患の処置のための装置 | |
| JP2020073536A5 (https=) | ||
| JP2011511090A5 (https=) | ||
| FI3821912T3 (fi) | Verkkokalvorappeuman RPGR-geeniterapia | |
| US20250171738A1 (en) | Process for producing cone photoreceptor cells | |
| JP2017523983A5 (https=) | ||
| CN116406304A (zh) | 使用AIMP2-DX2和任选地miR-142的靶序列及其组合物治疗年龄相关的黄斑疾病的方法 | |
| US20060031946A1 (en) | Gene therapy with chimeric oligonucleotides delivered by a method comprising a step of iontophoresis | |
| HK1253299A1 (zh) | 一种通过基因治疗治疗眼疾病的改进方法 | |
| EP2909232B1 (en) | Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (amd) | |
| WO2009058970A3 (en) | A novel gene therapy approach for treating the metabolic disorder obesity | |
| Tuo et al. | AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2−/−/Cx3cr1−/− mice | |
| Wei et al. | A novel AAV capsid-mediated RS1 gene therapy restored retinal function to wild-type levels in Rs1R213W mouse model |