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
Links
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 (enExample) | 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 (enExample) |
| JP (2) | JP7054675B2 (enExample) |
| CA (1) | CA3005254A1 (enExample) |
| ES (1) | ES2903394T3 (enExample) |
| WO (1) | WO2017106364A2 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| ES2882500T3 (es) | 2015-12-14 | 2021-12-02 | Cold Spring Harbor Laboratory | Oligómeros antisentido para el tratamiento del síndrome de Dravet |
| US11136577B2 (en) | 2016-03-09 | 2021-10-05 | Ionis Pharmaceuticals, Inc. | Methods and compositions for inhibiting PMP22 expression |
| SG11201906735RA (en) | 2017-01-23 | 2019-08-27 | Regeneron Pharma | Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof |
| JP2020516283A (ja) | 2017-04-11 | 2020-06-11 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | ヒドロキシステロイド（１７−ベータ）デヒドロゲナーゼ（ｈｓｄ１７ｂ）ファミリーのメンバーのモジュレーターの活性をスクリーニングするためのアッセイ |
| SI3673080T1 (sl) | 2017-08-25 | 2024-03-29 | Stoke Therapeutics, Inc. | Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni |
| KR20240125690A (ko) | 2017-10-11 | 2024-08-19 | 리제너론 파마슈티칼스 인코포레이티드 | Pnpla3 i148m 변이를 발현하는 환자의 간 질환의 치료에서의 hsd17b13의 저해 |
| US11661602B2 (en) * | 2018-03-02 | 2023-05-30 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Inhibition of polyomavirus replication |
| CN112020556A (zh) | 2018-03-21 | 2020-12-01 | 瑞泽恩制药公司 | 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法 |
| WO2019213525A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| WO2020132558A1 (en) * | 2018-12-21 | 2020-06-25 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing pmp22 expression |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
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 |
| JPH08501686A (ja) | 1992-09-25 | 1996-02-27 | ローン−プーラン・ロレ・ソシエテ・アノニム | 中枢神経系、特に脳における細胞への外来遺伝子の転移のためのアデノウィルスベクター |
| 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 |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| CN102006890A (zh) | 2007-12-04 | 2011-04-06 | 阿尔尼拉姆医药品有限公司 | 靶向脂质 |
| CN115227709A (zh) | 2009-06-17 | 2022-10-25 | 冷泉港实验室 | 用于在对象中调节smn2剪接的组合物和方法 |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| 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 |
| ES2779302T3 (es) | 2013-09-04 | 2020-08-14 | Cold Spring Harbor Laboratory | Reducción de la degradación de ARNm con mediación sin sentido |
-
2016
- 2016-12-14 CA CA3005254A patent/CA3005254A1/en active Pending
- 2016-12-14 JP JP2018529240A patent/JP7054675B2/ja not_active Expired - Fee Related
- 2016-12-14 WO PCT/US2016/066684 patent/WO2017106364A2/en not_active Ceased
- 2016-12-14 EP EP16876606.1A patent/EP3390642B1/en active Active
- 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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