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 (enExample) | 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 (enExample) |
| JP (2) | JP7049249B2 (enExample) |
| CA (1) | CA3005246A1 (enExample) |
| WO (1) | WO2017106382A1 (enExample) |
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 |
| EP3390636B1 (en) | 2015-12-14 | 2021-05-19 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of dravet syndrome |
| JP7051683B2 (ja) * | 2015-12-14 | 2022-04-11 | コールド スプリング ハーバー ラボラトリー | 結節性硬化症の処置のためのアンチセンスオリゴマー |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2018136758A1 (en) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Hsd17b13 variants and uses thereof |
| MX2019012169A (es) | 2017-04-11 | 2019-12-11 | Regeneron Pharma | Ensayos para evaluar la actividad de moduladores de miembros de la familia de hidroxiesteroide (17-beta) deshidrogenasa (hsd17b). |
| EP3673080B1 (en) | 2017-08-25 | 2023-10-18 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| JP7434151B2 (ja) | 2017-10-11 | 2024-02-20 | 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 | Ｐｎｐｌａ３ ｉ１４８ｍの変異を発現している患者の肝疾患の治療におけるｈｓｄ１７ｂ１３の阻害 |
| RS66633B1 (sr) * | 2017-10-23 | 2025-08-29 | Stoke Therapeutics Inc | Antisens oligomeri za lečenje bolesti i stanja na bazi besmisleno posredovanog raspadanja rnk |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス，インク． | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| EA202190581A1 (ru) | 2018-08-20 | 2021-07-13 | Рогкон, Инк. | Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a |
| CN113748209A (zh) | 2019-02-27 | 2021-12-03 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| EP3942049A4 (en) | 2019-03-20 | 2023-10-18 | President And Fellows Of Harvard College | ANTISENSE OLIGONUCLEOTIDE BASED PROGRANULIN AUGMENTATION THERAPY FOR 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 |
| MX2021013913A (es) * | 2019-05-14 | 2022-03-17 | Univ Duke | Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa. |
| EP4039282B1 (en) * | 2019-09-30 | 2025-06-04 | 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 |
| JP2023524671A (ja) * | 2020-04-27 | 2023-06-13 | ウニベルジテート ウルム | Ｓｈａｎｋ３の発現を増加させるアンチセンスオリゴヌクレオチド |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| TWI899403B (zh) * | 2020-12-23 | 2025-10-01 | 美商美國禮來大藥廠 | Rna治療劑及其使用方法 |
| US20240218360A1 (en) * | 2021-04-05 | 2024-07-04 | Itayandbiond Ltd | Small activating rna increasing shank expression and method of treating intellectual disabilities and associated comorbidities associated with shank haploinsufficiency |
| JP2025501457A (ja) * | 2021-12-07 | 2025-01-22 | エフ． ホフマン－ラ ロシュ アーゲー | Ａｃｔｌ６ｂを標的とするアンチセンスオリゴヌクレオチド |
| WO2023143425A1 (zh) * | 2022-01-25 | 2023-08-03 | 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 |
| CA2145535C (en) | 1992-09-25 | 2007-07-17 | Axel Kahn | Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain |
| 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 |
| CA2910760C (en) | 2007-12-04 | 2019-07-09 | Muthiah Manoharan | Targeting lipids |
| 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 |
| PT3449926T (pt) | 2009-06-17 | 2019-11-12 | Cold Spring Harbor Laboratory | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| WO2012138487A2 (en) * | 2011-04-07 | 2012-10-11 | 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 |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| DK3041958T3 (da) | 2013-09-04 | 2020-03-09 | Cold Spring Harbor Laboratory | Reducering af nonsense-medieret mrna-degradering |
-
2016
- 2016-12-14 CA CA3005246A patent/CA3005246A1/en active Pending
- 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 EP EP22166699.3A patent/EP4104867A3/en not_active Withdrawn
-
2022
- 2022-02-01 JP JP2022013867A patent/JP2022062141A/ja active Pending
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