JP2025016451A5 - - Google Patents
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- Publication number
- JP2025016451A5 JP2025016451A5 JP2024168632A JP2024168632A JP2025016451A5 JP 2025016451 A5 JP2025016451 A5 JP 2025016451A5 JP 2024168632 A JP2024168632 A JP 2024168632A JP 2024168632 A JP2024168632 A JP 2024168632A JP 2025016451 A5 JP2025016451 A5 JP 2025016451A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- sequence
- rna molecule
- seq
- synthetic rna
- 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
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662302122P | 2016-03-01 | 2016-03-01 | |
| US62/302,122 | 2016-03-01 | ||
| US201662398451P | 2016-09-22 | 2016-09-22 | |
| US62/398,451 | 2016-09-22 | ||
| PCT/US2017/020289 WO2017151823A1 (en) | 2016-03-01 | 2017-03-01 | Aav vectors for treatment of dominant retinitis pigmentosa |
| JP2018545818A JP7272795B2 (ja) | 2016-03-01 | 2017-03-01 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2022156496A JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022156496A Division JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025016451A JP2025016451A (ja) | 2025-02-04 |
| JP2025016451A5 true JP2025016451A5 (enExample) | 2025-05-30 |
Family
ID=59743213
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018545818A Active JP7272795B2 (ja) | 2016-03-01 | 2017-03-01 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2022156496A Active JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2024168632A Pending JP2025016451A (ja) | 2016-03-01 | 2024-09-27 | 優性網膜色素変性の治療のためのａａｖベクター |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018545818A Active JP7272795B2 (ja) | 2016-03-01 | 2017-03-01 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2022156496A Active JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11118185B2 (enExample) |
| EP (2) | EP3423582B1 (enExample) |
| JP (3) | JP7272795B2 (enExample) |
| CN (2) | CN115896106A (enExample) |
| AU (2) | AU2017227776C1 (enExample) |
| CA (1) | CA3014671A1 (enExample) |
| ES (1) | ES2923877T3 (enExample) |
| IL (2) | IL261014B2 (enExample) |
| WO (1) | WO2017151823A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11118185B2 (en) | 2016-03-01 | 2021-09-14 | University Of Florida Research Foundation, Incorporated | AAV vectors for treatment of dominant retinitis pigmentosa |
| WO2019094486A1 (en) * | 2017-11-07 | 2019-05-16 | The University Of North Carolina At Chapel Hill | Methods and compositions for circular rna molecules |
| WO2019222354A1 (en) * | 2018-05-15 | 2019-11-21 | University Of Washington | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
| WO2019232517A1 (en) * | 2018-06-01 | 2019-12-05 | University Of Florida Research Foundation, Incorporated | Compositions and methods for treatment of dominant retinitis pigmentosa |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| CN111518813B (zh) * | 2019-02-03 | 2023-04-28 | 武汉纽福斯生物科技有限公司 | 视紫红质的编码序列、其表达载体构建及其应用 |
| JP2022545378A (ja) * | 2019-08-15 | 2022-10-27 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法 |
| CN113038971B (zh) * | 2020-04-21 | 2022-06-24 | 北京中因科技有限公司 | RHO-adRP基于基因编辑的方法和组合物 |
| CN111926044B (zh) * | 2020-10-12 | 2021-01-22 | 北京大学第三医院（北京大学第三临床医学院） | 结合突变rho基因的核酸分子和试剂盒 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2025231487A1 (en) * | 2024-05-03 | 2025-11-06 | The Trustees Of The University Of Pennsylvania | Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration |
| WO2026087373A1 (en) * | 2024-10-21 | 2026-04-30 | MeiraGTx Ocular UK Limited | Promoters for driving gene expression in rod photoreceptors and uses thereof |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| GB9606961D0 (en) | 1996-04-02 | 1996-06-05 | Farrar Gwyneth J | Genetic strategy III |
| US20040234999A1 (en) | 1996-04-02 | 2004-11-25 | Farrar Gwenyth Jane | Genetic suppression and replacement |
| US8551970B2 (en) | 1996-04-02 | 2013-10-08 | Optigen Patents Limited | Genetic suppression and replacement |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| EP1615943A4 (en) | 2003-04-18 | 2006-08-16 | Univ Pennsylvania | COMPOSITION AND METHOD FOR SIRNA INHIBITION OF ANGIOPOIETIN 1 AND 2 AND THEIR RECIPIENTS TIE2 |
| US20070009899A1 (en) | 2003-10-02 | 2007-01-11 | Mounts William M | Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases |
| GB0403600D0 (en) * | 2004-02-18 | 2004-03-24 | Trinity College Dublin | Methods and reagents for treating disease |
| WO2005090572A2 (en) | 2004-03-24 | 2005-09-29 | Oncotherapy Science, Inc. | Compositions and methods for treating pancreatic cancer |
| US20080221057A1 (en) | 2007-02-16 | 2008-09-11 | Wyeth | Secreted protein ccdc80 regulates adipocyte differentiation |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| CA2683469C (en) * | 2007-04-12 | 2019-11-26 | The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| WO2009035792A1 (en) * | 2007-08-03 | 2009-03-19 | Melba Ketchum | Compositions, methods and systems for the simultaneous determination of parentage, identity, sex, genotype and/or phenotype and breed determination in animals |
| US8450473B2 (en) * | 2009-04-30 | 2013-05-28 | The Research Foundation Of State University Of New York | Compositions and methods for therapy of macular degeneration |
| KR20200047642A (ko) * | 2010-04-23 | 2020-05-07 | 유니버시티 오브 플로리다 리서치 파운데이션, 인크. | 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법 |
| US8968746B2 (en) | 2010-07-30 | 2015-03-03 | Curevac Gmbh | Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation |
| CN102061308B (zh) | 2010-10-29 | 2013-05-08 | 北京未名凯拓作物设计中心有限公司 | 一种用百草枯筛选转基因植物的方法 |
| CN102061305B (zh) | 2010-12-15 | 2012-07-18 | 深圳市百恩维生物科技有限公司 | 一种多ShRNA高效协同沉默基因方法及载体 |
| US20130064815A1 (en) | 2011-09-12 | 2013-03-14 | The Trustees Of Princeton University | Inducing apoptosis in quiescent cells |
| WO2013134766A2 (en) * | 2012-03-09 | 2013-09-12 | The Johns Hopkins University | Identification of molecular pathways and methods of use thereof for treating retinal neurodegeneration and other neurodegenerative disorders |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| US9695443B2 (en) * | 2012-05-25 | 2017-07-04 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| US20140289881A1 (en) | 2013-03-14 | 2014-09-25 | Commonwealth Scientific And Industrial Research Organisation | Double-stranded rna |
| DK3628334T5 (da) * | 2014-03-21 | 2024-09-02 | Genzyme Corp | Genterapi til behandling af retinitis pigmentosa |
| US10426789B2 (en) | 2015-02-26 | 2019-10-01 | Ionis Pharmaceuticals, Inc. | Allele specific modulators of P23H rhodopsin |
| US20190275168A1 (en) | 2015-04-30 | 2019-09-12 | The Trustees Of Columbia University In The City Of New York | Gene therapy for autosomal dominant diseases |
| US11096955B2 (en) | 2016-02-09 | 2021-08-24 | Fondazione Telethon | Synthetic promoters and uses thereof |
| US20170348387A1 (en) | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| US11118185B2 (en) | 2016-03-01 | 2021-09-14 | University Of Florida Research Foundation, Incorporated | AAV vectors for treatment of dominant retinitis pigmentosa |
-
2017
- 2017-03-01 US US16/081,307 patent/US11118185B2/en active Active
- 2017-03-01 CN CN202210934990.0A patent/CN115896106A/zh active Pending
- 2017-03-01 IL IL261014A patent/IL261014B2/en unknown
- 2017-03-01 ES ES17760752T patent/ES2923877T3/es active Active
- 2017-03-01 IL IL302455A patent/IL302455A/en unknown
- 2017-03-01 AU AU2017227776A patent/AU2017227776C1/en active Active
- 2017-03-01 EP EP17760752.0A patent/EP3423582B1/en active Active
- 2017-03-01 WO PCT/US2017/020289 patent/WO2017151823A1/en not_active Ceased
- 2017-03-01 CA CA3014671A patent/CA3014671A1/en active Pending
- 2017-03-01 JP JP2018545818A patent/JP7272795B2/ja active Active
- 2017-03-01 CN CN201780014309.8A patent/CN109154002B/zh active Active
- 2017-03-01 EP EP22170988.4A patent/EP4119668A1/en active Pending
-
2021
- 2021-05-21 US US17/327,609 patent/US20210324387A1/en active Pending
-
2022
- 2022-09-29 JP JP2022156496A patent/JP7642211B2/ja active Active
-
2023
- 2023-09-27 AU AU2023237106A patent/AU2023237106B2/en active Active
-
2024
- 2024-09-27 JP JP2024168632A patent/JP2025016451A/ja active Pending
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