JP2019500349A5 - - Google Patents
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- JP2019500349A5 JP2019500349A5 JP2018529257A JP2018529257A JP2019500349A5 JP 2019500349 A5 JP2019500349 A5 JP 2019500349A5 JP 2018529257 A JP2018529257 A JP 2018529257A JP 2018529257 A JP2018529257 A JP 2018529257A JP 2019500349 A5 JP2019500349 A5 JP 2019500349A5
- Authority
- JP
- Japan
- Prior art keywords
- protein
- stxbp1
- mrna
- pharmaceutical composition
- composition according
- 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 |
|---|---|---|---|
| JP2022013867A JP2022062141A (ja) | 2015-12-14 | 2022-02-01 | 中枢神経系疾患の処置のための組成物および方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267256P | 2015-12-14 | 2015-12-14 | |
| US62/267,256 | 2015-12-14 | ||
| US201662319011P | 2016-04-06 | 2016-04-06 | |
| US62/319,011 | 2016-04-06 | ||
| PCT/US2016/066721 WO2017106382A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of central nervous system diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022013867A Division JP2022062141A (ja) | 2015-12-14 | 2022-02-01 | 中枢神経系疾患の処置のための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500349A JP2019500349A (ja) | 2019-01-10 |
| JP2019500349A5 true JP2019500349A5 (OSRAM) | 2020-01-30 |
| JP7049249B2 JP7049249B2 (ja) | 2022-04-06 |
Family
ID=59057524
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529257A Expired - Fee Related JP7049249B2 (ja) | 2015-12-14 | 2016-12-14 | 中枢神経系疾患の処置のための組成物および方法 |
| JP2022013867A Pending JP2022062141A (ja) | 2015-12-14 | 2022-02-01 | 中枢神経系疾患の処置のための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022013867A Pending JP2022062141A (ja) | 2015-12-14 | 2022-02-01 | 中枢神経系疾患の処置のための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| EP (2) | EP4104867A3 (OSRAM) |
| JP (2) | JP7049249B2 (OSRAM) |
| CA (1) | CA3005246A1 (OSRAM) |
| WO (1) | WO2017106382A1 (OSRAM) |
Families Citing this family (34)
| 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 |
| EP3390635A4 (en) * | 2015-12-14 | 2019-05-01 | Cold Spring Harbor Laboratory | Antisense oligomers for the treatment of a tuberous sclerosis complex |
| 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 |
| 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 |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| KR20240125690A (ko) | 2017-10-11 | 2024-08-19 | 리제너론 파마슈티칼스 인코포레이티드 | Pnpla3 i148m 변이를 발현하는 환자의 간 질환의 치료에서의 hsd17b13의 저해 |
| EP3700570B1 (en) * | 2017-10-23 | 2025-01-08 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases |
| 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 |
| CA3110211A1 (en) | 2018-08-20 | 2020-02-27 | Rogcon, Inc. | Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| WO2020191212A1 (en) | 2019-03-20 | 2020-09-24 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| WO2020227406A1 (en) * | 2019-05-06 | 2020-11-12 | The Children's Hospital Of Philadelphia | Micro-rna site blocking oligonucleotides for the treatment of epileptic encephalopathy and neurodevelopmental disorders |
| JP2022531809A (ja) * | 2019-05-14 | 2022-07-11 | デューク ユニバーシティ | ＡＴＰａｓｅ媒介性疾患の処置のための組成物および方法 |
| US20220333110A1 (en) * | 2019-09-30 | 2022-10-20 | The University Of Tokyo | Nucleic acid that inhibits expression of mex3b gene, mex3b gene expression inhibiting agent, method for inhibiting mex3b gene expression, and prophylactic or therapeutic agent for disease caused by mex3b gene expression |
| US20240294905A1 (en) * | 2020-04-27 | 2024-09-05 | Universität Ulm | Antisense oligonucleotides for increasing shank3 expression |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| JP7756166B2 (ja) * | 2020-12-23 | 2025-10-17 | イーライ リリー アンド カンパニー | Ｒｎａ治療薬及びその使用方法 |
| IL307167A (en) * | 2021-04-05 | 2023-11-01 | Itayandbiond Ltd | Small activating RNA that increases expression of SHANK and a method for treating intellectual disabilities and associated diseases associated with SHANK haploinsufficiency |
| CN118434858A (zh) * | 2021-12-07 | 2024-08-02 | 豪夫迈·罗氏有限公司 | 靶向actl6b的反义寡核苷酸 |
| CN118647405A (zh) * | 2022-01-25 | 2024-09-13 | 上海魁特迪生物科技有限公司 | 改善认知障碍的方法 |
| US20250170259A1 (en) | 2022-03-03 | 2025-05-29 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| CN114836532A (zh) * | 2022-04-28 | 2022-08-02 | 福建医科大学附属第一医院 | Eif2b5基因突变作为靶标在诊断消融性白质脑病中的应用 |
| WO2024126654A1 (en) * | 2022-12-14 | 2024-06-20 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides targeting actl6b |
| WO2024226471A2 (en) * | 2023-04-24 | 2024-10-31 | Biomarin Pharmaceutical Inc. | Compositions and methods for treating stxbp1 disorders |
| WO2025038842A1 (en) * | 2023-08-15 | 2025-02-20 | The Children's Medical Center Corporation | Systems and methods for modifying a polynucleotide |
| CN117122688B (zh) * | 2023-08-31 | 2024-06-04 | 中国科学院脑科学与智能技术卓越创新中心 | 作用于前脑兴奋性神经元的prrt2及其上调剂的应用 |
| WO2025217494A1 (en) * | 2024-04-11 | 2025-10-16 | The University Of Chicago | Splice-switching oligonucleotides for treating cacna1a-associated disorders |
| WO2025250031A1 (en) * | 2024-05-31 | 2025-12-04 | Bial - Portela & Ca., S.A. | Antisense oligonucleotides for treatment of stxbp1-related developmental epileptic encephalopathy |
Family Cites Families (15)
| 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 |
| WO2009084472A1 (ja) | 2007-12-28 | 2009-07-09 | Public University Corporation Yokohama City University | 新生児期～乳児期発症の難治性てんかんの検出方法 |
| 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 |
| US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| US20140298496A1 (en) * | 2011-06-23 | 2014-10-02 | Cold Spring Harbor Laboratory | Phenocopy model of disease |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| 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 EP EP22166699.3A patent/EP4104867A3/en not_active Withdrawn
- 2016-12-14 WO PCT/US2016/066721 patent/WO2017106382A1/en not_active Ceased
- 2016-12-14 EP EP16876621.0A patent/EP3389725B1/en active Active
- 2016-12-14 JP JP2018529257A patent/JP7049249B2/ja not_active Expired - Fee Related
- 2016-12-14 CA CA3005246A patent/CA3005246A1/en active Pending
-
2022
- 2022-02-01 JP JP2022013867A patent/JP2022062141A/ja active Pending
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