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.)
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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 (https=) | 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) | EP3389725B1 (https=) |
| JP (2) | JP7049249B2 (https=) |
| CA (1) | CA3005246A1 (https=) |
| WO (1) | WO2017106382A1 (https=) |
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 |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| EP3390635A4 (en) * | 2015-12-14 | 2019-05-01 | Cold Spring Harbor Laboratory | Antisense oligomers for the treatment of a tuberous sclerosis complex |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| HUE070436T2 (hu) * | 2017-10-23 | 2025-06-28 | Stoke Therapeutics Inc | Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| BR112021003224A2 (pt) | 2018-08-20 | 2021-07-20 | Rogcon, Inc. | oligonucleotídeos antissentido direcionados a scn2a para o tratamento de encefalopatias por scn1a |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| US12595479B2 (en) | 2019-03-20 | 2026-04-07 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| EP3965777A4 (en) * | 2019-05-06 | 2024-08-07 | The Children's Hospital of Philadelphia | MICRORNA SITE-BLOCKING OLIGONUCLEOTIDES FOR THE TREATMENT OF EPILEPTIC ENCEPHALOPATHY AND NEURODEVELOPMENTAL DISORDERS |
| CA3140049A1 (en) * | 2019-05-14 | 2020-11-19 | Duke University | Compositions and methods for the treatment of atpase-mediated diseases |
| 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 |
| EP4143317A1 (en) | 2020-04-27 | 2023-03-08 | Universität Ulm | Antisense oligonucleotides for increasing shank3 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 |
| WO2022140264A1 (en) * | 2020-12-23 | 2022-06-30 | Eli Lilly And Company | Rna therapeutics and methods of use thereof |
| WO2022215066A1 (en) * | 2021-04-05 | 2022-10-13 | Itayandbiond Ltd | Small activating rna increasing shank expression and method of treating intellectual disabilities and associated comorbidities associated with shank haploinsufficiency |
| WO2023104693A1 (en) * | 2021-12-07 | 2023-06-15 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides targeting actl6b |
| 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 |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | 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 |
| EP4702143A2 (en) * | 2023-04-24 | 2026-03-04 | 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 |
| ATE404683T1 (de) | 1992-09-25 | 2008-08-15 | Aventis Pharma Sa | Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn |
| 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 |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| JP5608863B2 (ja) | 2007-12-28 | 2014-10-15 | 公立大学法人横浜市立大学 | 新生児期〜乳児期発症の難治性てんかんの検出方法 |
| ES2699827T3 (es) | 2009-06-17 | 2019-02-13 | Biogen Ma Inc | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto |
| WO2012138487A2 (en) * | 2011-04-07 | 2012-10-11 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| WO2012178122A2 (en) * | 2011-06-23 | 2012-12-27 | Cold Spring Harbor Laboratory | Phenocopy model of disease |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| EP3041958B1 (en) | 2013-09-04 | 2019-12-04 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
-
2016
- 2016-12-14 JP JP2018529257A patent/JP7049249B2/ja not_active Expired - Fee Related
- 2016-12-14 EP EP16876621.0A patent/EP3389725B1/en active Active
- 2016-12-14 EP EP22166699.3A patent/EP4104867A3/en not_active Withdrawn
- 2016-12-14 CA CA3005246A patent/CA3005246A1/en active Pending
- 2016-12-14 WO PCT/US2016/066721 patent/WO2017106382A1/en not_active Ceased
-
2022
- 2022-02-01 JP JP2022013867A patent/JP2022062141A/ja active Pending
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