JPWO2019161105A5 - - Google Patents
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- Publication number
- JPWO2019161105A5 JPWO2019161105A5 JP2020543576A JP2020543576A JPWO2019161105A5 JP WO2019161105 A5 JPWO2019161105 A5 JP WO2019161105A5 JP 2020543576 A JP2020543576 A JP 2020543576A JP 2020543576 A JP2020543576 A JP 2020543576A JP WO2019161105 A5 JPWO2019161105 A5 JP WO2019161105A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligonucleotide
- seq
- nucleoside
- atp7b
- sequence
- 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.)
- Pending
Links
- 108091034117 Oligonucleotide Proteins 0.000 claims 44
- 239000000074 antisense oligonucleotide Substances 0.000 claims 40
- 238000012230 antisense oligonucleotides Methods 0.000 claims 40
- 239000002777 nucleoside Substances 0.000 claims 21
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 claims 13
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 claims 13
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 12
- 150000007523 nucleic acids Chemical group 0.000 claims 10
- 108020004707 nucleic acids Proteins 0.000 claims 9
- 102000039446 nucleic acids Human genes 0.000 claims 9
- 125000003835 nucleoside group Chemical group 0.000 claims 6
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 101150075644 ATP7B gene Proteins 0.000 claims 3
- 108091092195 Intron Proteins 0.000 claims 3
- 230000001594 aberrant effect Effects 0.000 claims 3
- 230000000692 anti-sense effect Effects 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 108700024394 Exon Proteins 0.000 claims 2
- 238000012986 modification Methods 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- VGGSQFUCUMXWEO-UHFFFAOYSA-N Ethene Chemical compound C=C VGGSQFUCUMXWEO-UHFFFAOYSA-N 0.000 claims 1
- 239000005977 Ethylene Substances 0.000 claims 1
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 208000018839 Wilson disease Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- -1 methoxyethoxy Chemical group 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
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 JP2021513851A5 (https=) | 2022-02-22 |
| JPWO2019161105A5 true 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
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