JP2018538288A5 - - Google Patents
Download PDFInfo
- 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 (enExample) | 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 (enExample) |
| JP (2) | JP2018538288A (enExample) |
| CA (1) | CA3005245A1 (enExample) |
| WO (1) | WO2017106210A1 (enExample) |
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 |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| 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 |
| CN116059236A (zh) | 2015-12-14 | 2023-05-05 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| CA3049980A1 (en) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof |
| JP7048574B2 (ja) | 2017-03-10 | 2022-04-05 | 国立研究開発法人国立成育医療研究センター | アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物 |
| CA3059348A1 (en) | 2017-04-11 | 2018-10-18 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family |
| CA3071033A1 (en) | 2017-08-18 | 2019-02-21 | Ionis Pharmaceuticals, Inc. | Modulation of the notch signaling pathway for treatment of respiratory disorders |
| KR20260009385A (ko) | 2017-08-25 | 2026-01-19 | 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 |
| WO2019172286A1 (ja) * | 2018-03-09 | 2019-09-12 | 第一三共株式会社 | 糖原病Ｉａ型治療薬 |
| MY207352A (en) | 2018-03-21 | 2025-02-21 | Regeneron Pharma | 17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof |
| WO2019210279A1 (en) * | 2018-04-27 | 2019-10-31 | The Regents Of The University Of California | De novo formation of the biliary system by hepatocyte transdifferentiation |
| 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 |
| CN119193702A (zh) * | 2018-09-06 | 2024-12-27 | 加利福尼亚大学董事会 | 经由改造的adar募集的rna和dna碱基编辑 |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| US12338437B2 (en) | 2020-05-11 | 2025-06-24 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
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 |
| DE69334238D1 (de) | 1992-09-25 | 2008-09-25 | 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 |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | 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 |
| EP2229186A2 (en) | 2007-12-04 | 2010-09-22 | Alnylam Pharmaceuticals Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| KR20220084437A (ko) | 2009-06-17 | 2022-06-21 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| WO2015035091A1 (en) | 2013-09-04 | 2015-03-12 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
-
2016
- 2016-12-13 EP EP16876499.1A patent/EP3389671A4/en not_active Withdrawn
- 2016-12-13 CA CA3005245A patent/CA3005245A1/en not_active Abandoned
- 2016-12-13 WO PCT/US2016/066414 patent/WO2017106210A1/en not_active Ceased
- 2016-12-13 JP JP2018529221A patent/JP2018538288A/ja not_active Ceased
-
2022
- 2022-04-20 JP JP2022069164A patent/JP2022106803A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019501892A5 (enExample) | ||
| JP2019500350A5 (enExample) | ||
| JP2018538287A5 (enExample) | ||
| JP7420679B2 (ja) | 状態および疾患の処置のためのアンチセンスオリゴマー | |
| JP2019500349A5 (enExample) | ||
| JP2019500347A5 (enExample) | ||
| JP2019500346A5 (enExample) | ||
| KR102728481B1 (ko) | Lpa의 유전자 발현을 저해하기 위한 조성물 및 방법 | |
| JP7051683B2 (ja) | 結節性硬化症の処置のためのアンチセンスオリゴマー | |
| JP2024056778A5 (enExample) | ||
| JP2019500345A5 (enExample) | ||
| JP2017519766A5 (enExample) | ||
| TW201831686A (zh) | α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法 | |
| JP7660515B2 (ja) | 病態及び疾患の治療用アンチセンスオリゴマー | |
| CA3140045A1 (en) | Splice modulating oligonucleotides targeting receptor for advanced glycation end products and methods of use | |
| JPWO2018164275A1 (ja) | アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物 | |
| US20200407721A1 (en) | Compounds of chemically modified oligonucleotides and methods of use thereof | |
| JP2021523227A5 (enExample) | ||
| JP2024524974A (ja) | ナンセンス変異依存ｒｎａ分解機構に基づく病態及び疾患の処置のためのアンチセンスオリゴマー | |
| JP2021523227A (ja) | 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 | |
| CN118043461A (zh) | 方法 | |
| JP2024507717A5 (enExample) | ||
| TW202242113A (zh) | 用於治療與多囊蛋白表現相關之病況及疾病之組合物 | |
| US20200263204A1 (en) | Composition for treating hemophilia a by crispr/cas system of reverting fviii gene inversion | |
| WO2026065401A1 (zh) | 一种用于调控SCN1A基因mRNA和蛋白表达的方法和化合物 |