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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| JP (2) | JP7049249B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3005246A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017106382A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (33)
| 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 |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| 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 |
| 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 |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| 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 |
| RS66633B1 (sr) * | 2017-10-23 | 2025-08-29 | Stoke Therapeutics Inc | Antisens oligomeri za lečenje bolesti i stanja na bazi besmisleno posredovanog raspadanja rnk |
| 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 |
| US11939582B2 (en) | 2018-08-20 | 2024-03-26 | Rogcon, Inc. | Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies |
| US20220154182A1 (en) | 2019-03-20 | 2022-05-19 | 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 |
| CN114072514A (zh) * | 2019-05-14 | 2022-02-18 | 杜克大学 | 用于治疗atp酶介导的疾病的组合物和方法 |
| ES3035200T3 (en) * | 2019-09-30 | 2025-08-29 | Univ 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 |
| EP4267738A1 (en) * | 2020-12-23 | 2023-11-01 | 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 |
| EP4486386A1 (en) | 2022-03-03 | 2025-01-08 | 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 |
| 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 |
| 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 |
| 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 |
| US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| AU2012272656A1 (en) * | 2011-06-23 | 2014-02-06 | Cold Spring Harbor Laboratory | Phenocopy model of disease |
| ES2626488T3 (es) | 2011-07-19 | 2017-07-25 | Wave Life Sciences Pte. Ltd. | Procedimientos para la síntesis de ácidos nucleicos funcionalizados |
| PL3041958T3 (pl) | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
-
2016
- 2016-12-14 EP EP22166699.3A patent/EP4104867A3/en not_active Withdrawn
- 2016-12-14 EP EP16876621.0A patent/EP3389725B1/en active Active
- 2016-12-14 CA CA3005246A patent/CA3005246A1/en active Pending
- 2016-12-14 JP JP2018529257A patent/JP7049249B2/ja not_active Expired - Fee Related
- 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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