JP2019500347A5 - - Google Patents
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- Publication number
- JP2019500347A5 JP2019500347A5 JP2018529240A JP2018529240A JP2019500347A5 JP 2019500347 A5 JP2019500347 A5 JP 2019500347A5 JP 2018529240 A JP2018529240 A JP 2018529240A JP 2018529240 A JP2018529240 A JP 2018529240A JP 2019500347 A5 JP2019500347 A5 JP 2019500347A5
- Authority
- JP
- Japan
- Prior art keywords
- protein
- mrna
- prpf8
- pharmaceutical composition
- nucleobases
- 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
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022062557A JP2022088621A (ja) | 2015-12-14 | 2022-04-04 | 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267261P | 2015-12-14 | 2015-12-14 | |
| US62/267,261 | 2015-12-14 | ||
| PCT/US2016/066684 WO2017106364A2 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022062557A Division JP2022088621A (ja) | 2015-12-14 | 2022-04-04 | 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500347A JP2019500347A (ja) | 2019-01-10 |
| JP2019500347A5 true JP2019500347A5 (cg-RX-API-DMAC7.html) | 2020-01-30 |
| JP7054675B2 JP7054675B2 (ja) | 2022-04-14 |
Family
ID=59057547
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529240A Expired - Fee Related JP7054675B2 (ja) | 2015-12-14 | 2016-12-14 | 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 |
| JP2022062557A Pending JP2022088621A (ja) | 2015-12-14 | 2022-04-04 | 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022062557A Pending JP2022088621A (ja) | 2015-12-14 | 2022-04-04 | 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 |
Country Status (5)
| Country | Link |
|---|---|
| EP (1) | EP3390642B1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP7054675B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3005254A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2903394T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017106364A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| KR102422625B1 (ko) | 2015-10-09 | 2022-07-20 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| AU2017229778A1 (en) | 2016-03-09 | 2018-08-16 | Ionis Pharmaceuticals, Inc. | Methods and compositions for inhibiting PMP22 expression |
| CN118325899A (zh) | 2017-01-23 | 2024-07-12 | 瑞泽恩制药公司 | Hsd17b13变体及其应用 |
| RU2019135845A (ru) | 2017-04-11 | 2021-05-11 | Ридженерон Фармасьютикалз, Инк. | Анализы для скрининга активности модуляторов членов семейства (17-бета)гидроксистероиддегидрогеназ (hsd17b) |
| CN111278991B (zh) | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| EP4234719B1 (en) | 2017-10-11 | 2025-11-26 | Regeneron Pharmaceuticals, Inc. | Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation |
| CN112218949B (zh) * | 2018-03-02 | 2025-02-11 | 莱顿大学医学中心附属莱顿教学医院 | 多瘤病毒复制的抑制 |
| BR112020018758A2 (pt) | 2018-03-21 | 2021-01-26 | Regeneron Pharmaceuticals, Inc. | agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| US20220112503A1 (en) * | 2018-12-21 | 2022-04-14 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing pmp22 expression |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4866042A (en) | 1987-11-18 | 1989-09-12 | Neuwelt Edward A | Method for the delivery of genetic material across the blood brain barrier |
| US6294520B1 (en) | 1989-03-27 | 2001-09-25 | Albert T. Naito | Material for passage through the blood-brain barrier |
| US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
| NZ256018A (en) | 1992-09-25 | 1997-07-27 | Rhone Poulenc Rorer Sa | Recombinant adenoviral vectors and their use in directing expression and transcription of selected nucleotide sequences in cells of the central nervous system |
| US5656612A (en) | 1994-05-31 | 1997-08-12 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
| FR2727867B1 (fr) | 1994-12-13 | 1997-01-31 | Rhone Poulenc Rorer Sa | Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux |
| US6936589B2 (en) | 2001-09-28 | 2005-08-30 | Albert T. Naito | Parenteral delivery systems |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of LMNA expression |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| JP5707396B2 (ja) | 2009-06-17 | 2015-04-30 | アイシス ファーマシューティカルズ， インコーポレーテッド | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| ES2626488T3 (es) | 2011-07-19 | 2017-07-25 | Wave Life Sciences Pte. Ltd. | Procedimientos para la síntesis de ácidos nucleicos funcionalizados |
| CN104004826B (zh) * | 2013-01-07 | 2016-03-02 | 赵晨 | 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用 |
| PL3041958T3 (pl) | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
-
2016
- 2016-12-14 EP EP16876606.1A patent/EP3390642B1/en active Active
- 2016-12-14 CA CA3005254A patent/CA3005254A1/en active Pending
- 2016-12-14 WO PCT/US2016/066684 patent/WO2017106364A2/en not_active Ceased
- 2016-12-14 JP JP2018529240A patent/JP7054675B2/ja not_active Expired - Fee Related
- 2016-12-14 ES ES16876606T patent/ES2903394T3/es active Active
-
2022
- 2022-04-04 JP JP2022062557A patent/JP2022088621A/ja active Pending
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