JP2021502978A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2021502978A5 JP2021502978A5 JP2020526473A JP2020526473A JP2021502978A5 JP 2021502978 A5 JP2021502978 A5 JP 2021502978A5 JP 2020526473 A JP2020526473 A JP 2020526473A JP 2020526473 A JP2020526473 A JP 2020526473A JP 2021502978 A5 JP2021502978 A5 JP 2021502978A5
- Authority
- JP
- Japan
- Prior art keywords
- adeno
- associated virus
- virus vector
- human
- seq
- 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.)
- Pending
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023121924A JP7615238B2 (ja) | 2017-11-15 | 2023-07-26 | 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762586624P | 2017-11-15 | 2017-11-15 | |
| US62/586,624 | 2017-11-15 | ||
| PCT/US2018/061319 WO2019099696A1 (en) | 2017-11-15 | 2018-11-15 | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023121924A Division JP7615238B2 (ja) | 2017-11-15 | 2023-07-26 | 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021502978A JP2021502978A (ja) | 2021-02-04 |
| JP2021502978A5 true JP2021502978A5 (https=) | 2021-12-23 |
Family
ID=66431192
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020526473A Pending JP2021502978A (ja) | 2017-11-15 | 2018-11-15 | 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 |
| JP2023121924A Active JP7615238B2 (ja) | 2017-11-15 | 2023-07-26 | 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023121924A Active JP7615238B2 (ja) | 2017-11-15 | 2023-07-26 | 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 |
Country Status (27)
| Country | Link |
|---|---|
| US (2) | US20190142909A1 (https=) |
| EP (2) | EP4364803A3 (https=) |
| JP (2) | JP2021502978A (https=) |
| KR (1) | KR102704296B1 (https=) |
| CN (2) | CN111886340A (https=) |
| AU (1) | AU2018367606B2 (https=) |
| BR (1) | BR112020009828A2 (https=) |
| DK (1) | DK3710590T3 (https=) |
| EA (1) | EA202091194A1 (https=) |
| ES (1) | ES2983990T3 (https=) |
| FI (1) | FI3710590T3 (https=) |
| HR (1) | HRP20241073T1 (https=) |
| HU (1) | HUE068393T2 (https=) |
| IL (2) | IL308645B2 (https=) |
| LT (1) | LT3710590T (https=) |
| MA (1) | MA50914A (https=) |
| MX (2) | MX2020005008A (https=) |
| MY (1) | MY204271A (https=) |
| PH (1) | PH12020551319A1 (https=) |
| PL (1) | PL3710590T3 (https=) |
| PT (1) | PT3710590T (https=) |
| RS (1) | RS65881B1 (https=) |
| SG (1) | SG11202004527YA (https=) |
| SI (1) | SI3710590T1 (https=) |
| SM (1) | SMT202400321T1 (https=) |
| WO (1) | WO2019099696A1 (https=) |
| ZA (1) | ZA202003416B (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018009814A1 (en) | 2016-07-08 | 2018-01-11 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving rdh12 |
| WO2022232249A1 (en) * | 2021-04-27 | 2022-11-03 | The Trustees Of Columbia University In The City Of New York | Methods and compositions to regulate cholesterol efflux to prevent, treat, or cure macular degeneration |
| CN115725659A (zh) * | 2022-04-01 | 2023-03-03 | 首都医科大学附属北京同仁医院 | 一种应用视网膜原位基因编辑制作非人灵长类动物先天性黑矇模型的方法 |
| CN118453915A (zh) * | 2024-04-29 | 2024-08-09 | 中国医学科学院北京协和医院 | 一种针对rdh12突变的基因治疗药物 |
| CN120290554B (zh) * | 2024-09-10 | 2026-04-14 | 广州市未来基因递送研究院 | 感光细胞特异性双向启动子及其用途 |
| CN119432856B (zh) * | 2024-12-30 | 2025-08-12 | 云舟生物科技(广州)股份有限公司 | 核酸分子、病毒颗粒及其制备方法和应用 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20120172419A1 (en) * | 2009-09-15 | 2012-07-05 | Medical College Of Wisconsin Research Foundation Inc. | Reagents and methods for modulating cone photoreceptor activity |
| SG10201800541SA (en) * | 2010-04-23 | 2018-03-28 | Univ Florida | Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1) |
| WO2012167109A2 (en) | 2011-06-03 | 2012-12-06 | Massachusetts Eye & Ear Infirmary | Rpgrip1 gene therapy for leber congenital amaurosis |
| EP2872183B1 (en) * | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| WO2015075154A2 (en) | 2013-11-20 | 2015-05-28 | Fondazione Telethon | Artificial dna-binding proteins and uses thereof |
| 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 |
| GB201412011D0 (en) * | 2014-07-04 | 2014-08-20 | Ucl Business Plc | Treatments |
| HUE068183T2 (hu) * | 2014-07-24 | 2024-12-28 | Massachusetts Eye & Ear Infirmary | RPGR génterápia a retinitis pigmentosa terápiájára |
| US10392622B2 (en) | 2014-08-01 | 2019-08-27 | The Trustees Of The University Of Pennsylvania | Compositions and methods for self-regulated inducible gene expression |
| EP3268481B1 (en) * | 2015-03-11 | 2020-05-27 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Rpgr vectors for treating x-linked retinitis pigmentosa |
| EP3289080B1 (en) * | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| WO2018009814A1 (en) | 2016-07-08 | 2018-01-11 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving rdh12 |
| TR2021022284A2 (tr) | 2021-12-31 | 2022-01-21 | T C Ueskuedar Ueniversitesi | Retinitis pigmentosa (rp) hastalığının tedavisinde kullanılmak üzere retinol dehidrojenaz 12 (rdh12) gen mutasyonlarına yönelik crıspr-pe sistemi. |
-
2018
- 2018-11-15 KR KR1020207016694A patent/KR102704296B1/ko active Active
- 2018-11-15 MY MYPI2020002341A patent/MY204271A/en unknown
- 2018-11-15 SM SM20240321T patent/SMT202400321T1/it unknown
- 2018-11-15 CN CN201880086649.6A patent/CN111886340A/zh active Pending
- 2018-11-15 FI FIEP18878835.0T patent/FI3710590T3/fi active
- 2018-11-15 EP EP24163209.0A patent/EP4364803A3/en active Pending
- 2018-11-15 EA EA202091194A patent/EA202091194A1/ru unknown
- 2018-11-15 CN CN202311261068.0A patent/CN117752822A/zh active Pending
- 2018-11-15 SI SI201831135T patent/SI3710590T1/sl unknown
- 2018-11-15 BR BR112020009828-0A patent/BR112020009828A2/pt unknown
- 2018-11-15 SG SG11202004527YA patent/SG11202004527YA/en unknown
- 2018-11-15 IL IL308645A patent/IL308645B2/en unknown
- 2018-11-15 EP EP18878835.0A patent/EP3710590B1/en active Active
- 2018-11-15 IL IL274609A patent/IL274609B2/en unknown
- 2018-11-15 RS RS20240857A patent/RS65881B1/sr unknown
- 2018-11-15 AU AU2018367606A patent/AU2018367606B2/en active Active
- 2018-11-15 MA MA050914A patent/MA50914A/fr unknown
- 2018-11-15 JP JP2020526473A patent/JP2021502978A/ja active Pending
- 2018-11-15 PT PT188788350T patent/PT3710590T/pt unknown
- 2018-11-15 ES ES18878835T patent/ES2983990T3/es active Active
- 2018-11-15 US US16/192,382 patent/US20190142909A1/en not_active Abandoned
- 2018-11-15 MX MX2020005008A patent/MX2020005008A/es unknown
- 2018-11-15 LT LTEPPCT/US2018/061319T patent/LT3710590T/lt unknown
- 2018-11-15 DK DK18878835.0T patent/DK3710590T3/da active
- 2018-11-15 PL PL18878835.0T patent/PL3710590T3/pl unknown
- 2018-11-15 HU HUE18878835A patent/HUE068393T2/hu unknown
- 2018-11-15 WO PCT/US2018/061319 patent/WO2019099696A1/en not_active Ceased
- 2018-11-15 HR HRP20241073TT patent/HRP20241073T1/hr unknown
-
2020
- 2020-05-15 PH PH12020551319A patent/PH12020551319A1/en unknown
- 2020-06-08 ZA ZA2020/03416A patent/ZA202003416B/en unknown
- 2020-07-13 MX MX2026001243A patent/MX2026001243A/es unknown
-
2022
- 2022-08-09 US US17/818,623 patent/US12589136B2/en active Active
-
2023
- 2023-07-26 JP JP2023121924A patent/JP7615238B2/ja active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021502978A5 (https=) | ||
| JP7182873B2 (ja) | 多重ベクターシステム及びその使用 | |
| JP2023093461A (ja) | ハンチントン病治療組成物および方法 | |
| Trapani et al. | Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease | |
| EP3151866B1 (en) | Chimeric capsids | |
| JP6805174B2 (ja) | 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質 | |
| McClements et al. | Gene therapy for retinal disease | |
| JP2020073536A5 (https=) | ||
| JP2015523379A5 (https=) | ||
| IL274609B2 (en) | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies | |
| JP2017523239A5 (https=) | ||
| RU2019100525A (ru) | Векторная система на основе аденоассоциированного вируса | |
| EP2262899A1 (en) | Method of treating genetic disorders | |
| CA3133555A1 (en) | Triple helix terminator for efficient rna trans-splicing | |
| JP7671790B2 (ja) | 眼疾患の遺伝子治療 | |
| JP2022535412A5 (https=) | ||
| JP2019524090A5 (https=) | ||
| JP2018526994A5 (https=) | ||
| JP2019520405A5 (https=) | ||
| JP2021533801A5 (https=) | ||
| JP2018509154A5 (https=) | ||
| JP2021534796A5 (https=) | ||
| US20170137480A1 (en) | Rpgr-ofr15 variant gene in the treatment of retinitis pigmentosa | |
| WO2016073900A1 (en) | Compositions and methods of treating usher syndrome iii | |
| CN108103096B (zh) | 一种先天性黒曚症的基因治疗药物 |