JP2021513851A5 - - Google Patents
Info
- Publication number
- JP2021513851A5 JP2021513851A5 JP2020543576A JP2020543576A JP2021513851A5 JP 2021513851 A5 JP2021513851 A5 JP 2021513851A5 JP 2020543576 A JP2020543576 A JP 2020543576A JP 2020543576 A JP2020543576 A JP 2020543576A JP 2021513851 A5 JP2021513851 A5 JP 2021513851A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligonucleotide
- oligonucleotide according
- seq
- nucleic acid
- atp7b
- Prior art date
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862630565P | 2018-02-14 | 2018-02-14 | |
| US62/630,565 | 2018-02-14 | ||
| PCT/US2019/018076 WO2019161105A1 (en) | 2018-02-14 | 2019-02-14 | Oligonucleotide therapy for wilson disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021513851A JP2021513851A (ja) | 2021-06-03 |
| JP2021513851A5 true JP2021513851A5 (https=) | 2022-02-22 |
| JPWO2019161105A5 JPWO2019161105A5 (https=) | 2022-02-22 |
Family
ID=67619598
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020543576A Withdrawn JP2021513851A (ja) | 2018-02-14 | 2019-02-14 | ウィルソン病に対するオリゴヌクレオチド療法 |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US11578327B2 (https=) |
| EP (1) | EP3752614A4 (https=) |
| JP (1) | JP2021513851A (https=) |
| KR (1) | KR20200120675A (https=) |
| CN (1) | CN112041439A (https=) |
| AU (1) | AU2019222767A1 (https=) |
| BR (1) | BR112020016524A2 (https=) |
| CA (1) | CA3090517A1 (https=) |
| CL (1) | CL2020002100A1 (https=) |
| CO (1) | CO2020010226A2 (https=) |
| IL (1) | IL276701A (https=) |
| MX (1) | MX2020008533A (https=) |
| PH (1) | PH12020551219A1 (https=) |
| SG (1) | SG11202007644PA (https=) |
| WO (1) | WO2019161105A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US5578493A (en) * | 1993-09-01 | 1996-11-26 | The Trustees Of Columbia University In The City Of New York | Wilson's disease gene |
| CA2108927C (en) | 1993-09-21 | 2008-09-02 | Peter Bull | Wilson disease gene |
| AU1039397A (en) | 1995-11-22 | 1997-06-27 | Johns Hopkins University, The | Ligands to enhance cellular uptake of biomolecules |
| US20040101854A1 (en) * | 2002-11-21 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of BCL2-associated athanogene expression |
| US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| US20030180953A1 (en) | 2000-12-29 | 2003-09-25 | Elitra Pharmaceuticals, Inc. | Gene disruption methodologies for drug target discovery |
| US20040126761A1 (en) * | 2002-12-10 | 2004-07-01 | Isis Pharmaceuticals Inc. | Modulation of alpha-methylacyl-CoA racemase expression |
| WO2004035819A2 (en) | 2002-10-21 | 2004-04-29 | Exiqon A/S | Oligonucleotide analogues for detecting and analyzing nucleic acids |
| US20050244851A1 (en) | 2004-01-13 | 2005-11-03 | Affymetrix, Inc. | Methods of analysis of alternative splicing in human |
| CN1934251A (zh) * | 2004-01-29 | 2007-03-21 | 塞尔卓姆股份公司 | 使用atp7a-调控剂治疗神经变性疾病 |
| EP1752536A4 (en) | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
| CA2566519C (en) | 2004-05-14 | 2020-04-21 | Rosetta Genomics Ltd. | Micrornas and uses thereof |
| US20070048756A1 (en) | 2005-04-18 | 2007-03-01 | Affymetrix, Inc. | Methods for whole genome association studies |
| EP1783645A1 (en) | 2005-11-08 | 2007-05-09 | Actigenics | Methods for the identification of microRNA and their applications in research and human health |
| US8178503B2 (en) | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| JP4841279B2 (ja) | 2006-03-23 | 2011-12-21 | 一般財団法人 化学物質評価研究機構 | 被検物質の発がん性予測方法 |
| WO2009082607A2 (en) | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| CA2860676A1 (en) * | 2012-01-09 | 2013-07-18 | Novartis Ag | Organic compositions to treat beta-catenin-related diseases |
| WO2014036301A1 (en) * | 2012-08-30 | 2014-03-06 | Isis Pharmaceuticals, Inc. | Modulation of copper related diseases by ctr1 |
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| US20160258005A1 (en) * | 2013-07-02 | 2016-09-08 | The Trustees Of The University Of Pennsylvania | Methods for rapid ribonucleic acid fluorescence in situ hybridization |
| AU2014346658A1 (en) | 2013-11-06 | 2016-06-02 | Solstice Biologics, Ltd. | Polynucleotide constructs having disulfide groups |
| JP2017522046A (ja) | 2014-06-06 | 2017-08-10 | ソルスティス バイオロジクス,リミティッド | 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物 |
| WO2016004043A1 (en) | 2014-06-30 | 2016-01-07 | Blend Therapeutics, Inc. | Targeted conjugates and particles and formulations thereof |
| JP2017533721A (ja) | 2014-11-14 | 2017-11-16 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | タンパク質の調節のための化合物及び方法 |
| EP3247366A4 (en) | 2015-01-21 | 2018-10-31 | Sangamo Therapeutics, Inc. | Methods and compositions for identification of highly specific nucleases |
| CN105986015B (zh) * | 2015-02-05 | 2020-10-23 | 大连晶泰生物技术有限公司 | 一种基于高通量测序的多样本的一个或多个靶序列的检测方法和试剂盒 |
| EP3286318A2 (en) | 2015-04-22 | 2018-02-28 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| AU2016365828A1 (en) | 2015-12-08 | 2018-07-05 | Solstice Biologics, Ltd. | Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate |
| CA3005090A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR101743211B1 (ko) | 2016-06-24 | 2017-06-15 | 주식회사 녹십자지놈 | 선천성 기능장애 진단용 조성물 및 이의 용도 |
| EP3840758A1 (en) * | 2018-08-21 | 2021-06-30 | Deep Genomics Incorporated | Therapeutic splice-switching oligonucleotides |
| US20240358845A1 (en) * | 2021-05-10 | 2024-10-31 | Entrada Therapeutics, Inc. | Compositions and methods for intracellular therapeutics |
| WO2022256283A2 (en) * | 2021-06-01 | 2022-12-08 | Korro Bio, Inc. | Methods for restoring protein function using adar |
-
2019
- 2019-02-14 CN CN201980018495.1A patent/CN112041439A/zh active Pending
- 2019-02-14 BR BR112020016524-7A patent/BR112020016524A2/pt not_active IP Right Cessation
- 2019-02-14 SG SG11202007644PA patent/SG11202007644PA/en unknown
- 2019-02-14 AU AU2019222767A patent/AU2019222767A1/en not_active Abandoned
- 2019-02-14 EP EP19754072.7A patent/EP3752614A4/en active Pending
- 2019-02-14 CA CA3090517A patent/CA3090517A1/en active Pending
- 2019-02-14 KR KR1020207025862A patent/KR20200120675A/ko not_active Withdrawn
- 2019-02-14 JP JP2020543576A patent/JP2021513851A/ja not_active Withdrawn
- 2019-02-14 WO PCT/US2019/018076 patent/WO2019161105A1/en not_active Ceased
- 2019-02-14 MX MX2020008533A patent/MX2020008533A/es unknown
- 2019-02-14 PH PH1/2020/551219A patent/PH12020551219A1/en unknown
- 2019-02-14 US US16/970,088 patent/US11578327B2/en active Active
-
2020
- 2020-08-13 CL CL2020002100A patent/CL2020002100A1/es unknown
- 2020-08-13 IL IL276701A patent/IL276701A/en unknown
- 2020-08-19 CO CONC2020/0010226A patent/CO2020010226A2/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CN108271351B (zh) | 用于调节血管紧张素原表达的化合物和方法 | |
| US7307069B2 (en) | Antisense oligonucleotide modulation of STAT3 expression | |
| JP7379387B2 (ja) | Atxn2発現を制御するためのオリゴヌクレオチド | |
| JP2024056820A (ja) | Scn9a発現を調節するためのオリゴヌクレオチド | |
| JP2016513976A5 (https=) | ||
| JP2024105284A5 (https=) | ||
| JP2018519811A5 (https=) | ||
| JP2012147790A (ja) | エキソンスキッピングを誘発する為の手段及び方法 | |
| CA2453301A1 (en) | Synthetic double-stranded oligonucleotides for specific inhibition of gene expression | |
| WO2000061810A1 (en) | Antisense oligonucleotides comprising universal and/or degenerate bases | |
| CN107406852A (zh) | 用于莱伯氏先天性黑蒙的寡核苷酸疗法 | |
| CA3072314A1 (en) | Nucleic acid molecule for reduction of papd5 and papd7 mrna for treating hepatitis b infection | |
| JP2023503635A (ja) | Rna分子を合成する方法 | |
| CN114901821A (zh) | Sept9抑制剂用于治疗乙型肝炎病毒感染的用途 | |
| JP2021513851A5 (https=) | ||
| JP7499267B2 (ja) | Atxn2発現を調節するためのオリゴヌクレオチド | |
| TW202329983A (zh) | Rna編輯 | |
| CN115698290A (zh) | 用于治疗神经系统疾病的补体组分4抑制剂以及使用它们的相关组合物、系统和方法 | |
| EP2175023A1 (en) | Anti-tumor activity of small double-stranded oligodeoxynucleotides targeting telomerase RNA | |
| CN115551519A (zh) | 用于治疗神经系统疾病的补体组分c1s抑制剂以及相关的组合物、系统和使用它们的方法 | |
| JPWO2019161105A5 (https=) | ||
| CN114829601A (zh) | Sbds抑制剂用于治疗乙型肝炎病毒感染的用途 | |
| TW202136510A (zh) | 用於抑制scn9a表現之增強型寡核苷酸 | |
| CN115605592A (zh) | 用于治疗神经系统疾病的补体组分c1r抑制剂以及相关的组合物、系统和使用它们的方法 | |
| JP2026074094A (ja) | Fubp1発現を調節するための増強されたオリゴヌクレオチド |