JP2018538288A5 - - Google Patents
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- Publication number
- JP2018538288A5 JP2018538288A5 JP2018529221A JP2018529221A JP2018538288A5 JP 2018538288 A5 JP2018538288 A5 JP 2018538288A5 JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018538288 A5 JP2018538288 A5 JP 2018538288A5
- Authority
- JP
- Japan
- Prior art keywords
- mrna
- pharmaceutical composition
- item
- composition according
- 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.)
- Ceased
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022069164A JP2022106803A (ja) | 2015-12-14 | 2022-04-20 | アラジール症候群の処置のためのアンチセンスオリゴマー |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267210P | 2015-12-14 | 2015-12-14 | |
| US62/267,210 | 2015-12-14 | ||
| PCT/US2016/066414 WO2017106210A1 (en) | 2015-12-14 | 2016-12-13 | Antisense oligomers for treatment of alagille syndrome |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022069164A Division JP2022106803A (ja) | 2015-12-14 | 2022-04-20 | アラジール症候群の処置のためのアンチセンスオリゴマー |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018538288A JP2018538288A (ja) | 2018-12-27 |
| JP2018538288A5 true JP2018538288A5 (https=) | 2020-01-30 |
Family
ID=59057483
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529221A Ceased JP2018538288A (ja) | 2015-12-14 | 2016-12-13 | アラジール症候群の処置のためのアンチセンスオリゴマー |
| JP2022069164A Pending JP2022106803A (ja) | 2015-12-14 | 2022-04-20 | アラジール症候群の処置のためのアンチセンスオリゴマー |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022069164A Pending JP2022106803A (ja) | 2015-12-14 | 2022-04-20 | アラジール症候群の処置のためのアンチセンスオリゴマー |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP3389671A4 (https=) |
| JP (2) | JP2018538288A (https=) |
| CA (1) | CA3005245A1 (https=) |
| WO (1) | WO2017106210A1 (https=) |
Families Citing this family (18)
| 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 |
| 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 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| EP3594346A4 (en) | 2017-03-10 | 2020-12-16 | National Center For Child Health And Development | ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| WO2019036613A1 (en) | 2017-08-18 | 2019-02-21 | Ionis Pharmaceuticals, Inc. | MODULATION OF THE NOTCH SIGNALING PATHWAY FOR THE TREATMENT OF RESPIRATORY DISORDERS |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| 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 |
| KR102398295B1 (ko) * | 2018-03-09 | 2022-05-17 | 다이이찌 산쿄 가부시키가이샤 | 당원병 Ia형 치료약 |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| EP3784224A4 (en) * | 2018-04-27 | 2022-08-03 | The Regents of the University of California | DE NOVO FORMATION OF THE BILIARY SYSTEM BY HEPATOCYTE TRANSDIFFERENTIATION |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| US20220010333A1 (en) * | 2018-09-06 | 2022-01-13 | The Regents Of The University Of California | Rna and dna base editing via engineered adar recruitment |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| 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 |
Family Cites Families (16)
| 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 |
| US20040102401A1 (en) * | 2002-11-22 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of jagged 1 expression |
| GB0326578D0 (en) * | 2003-11-14 | 2003-12-17 | Univ Belfast | Cancer diagnosis and therapy |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| US20080280848A1 (en) | 2005-10-28 | 2008-11-13 | Volker Patzel | Structures of Active Guide Rna Molecules and Method of Selection |
| WO2007048629A2 (en) | 2005-10-28 | 2007-05-03 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Modulation of rna silencing efficiency by argonaute proteins |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| 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 |
| 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
- 2016-12-13 CA CA3005245A patent/CA3005245A1/en not_active Abandoned
- 2016-12-13 EP EP16876499.1A patent/EP3389671A4/en not_active Withdrawn
- 2016-12-13 JP JP2018529221A patent/JP2018538288A/ja not_active Ceased
- 2016-12-13 WO PCT/US2016/066414 patent/WO2017106210A1/en not_active Ceased
-
2022
- 2022-04-20 JP JP2022069164A patent/JP2022106803A/ja active Pending
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