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 (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 |
| 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 |
| EP3390636B1 (en) | 2015-12-14 | 2021-05-19 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of dravet syndrome |
| WO2018136758A1 (en) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Hsd17b13 variants and uses thereof |
| CN110536964A (zh) | 2017-03-10 | 2019-12-03 | 国立研究开发法人国立成育医疗研究中心 | 反义寡核苷酸和糖原贮积病Ia型预防或治疗用组合物 |
| 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). |
| EP3668984A4 (en) | 2017-08-18 | 2021-09-08 | Ionis Pharmaceuticals, Inc. | MODULATION OF THE NOTCH SIGNALING PATH FOR THE TREATMENT OF RESPIRATORY DISORDERS |
| EP3673080B1 (en) | 2017-08-25 | 2023-10-18 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| 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 |
| CN111511915A (zh) | 2018-03-09 | 2020-08-07 | 第一三共株式会社 | 糖原病Ia型治疗药 |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| US20210238257A1 (en) * | 2018-04-27 | 2021-08-05 | 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 |
| JP7720623B6 (ja) * | 2018-09-06 | 2025-09-18 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 遺伝子操作されたａｄａｒのリクルートを介したｒｎａ及びｄｎａ塩基の編集 |
| 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 |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | 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 |
| 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 |
| 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 |
| 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 |
| WO2007048628A2 (en) * | 2005-10-28 | 2007-05-03 | Max Planck Gesellschaft | Structures of active guide rna molecules and method of selection |
| CA2910760C (en) | 2007-12-04 | 2019-07-09 | Muthiah Manoharan | Targeting lipids |
| PT3449926T (pt) | 2009-06-17 | 2019-11-12 | Cold Spring Harbor Laboratory | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| 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
- 2016-12-13 CA CA3005245A patent/CA3005245A1/en not_active Abandoned
- 2016-12-13 WO PCT/US2016/066414 patent/WO2017106210A1/en not_active Ceased
- 2016-12-13 EP EP16876499.1A patent/EP3389671A4/en not_active Withdrawn
- 2016-12-13 JP JP2018529221A patent/JP2018538288A/ja not_active Ceased
-
2022
- 2022-04-20 JP JP2022069164A patent/JP2022106803A/ja active Pending