JP2023068035A5 - - Google Patents
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- JP2023068035A5 JP2023068035A5 JP2023039774A JP2023039774A JP2023068035A5 JP 2023068035 A5 JP2023068035 A5 JP 2023068035A5 JP 2023039774 A JP2023039774 A JP 2023039774A JP 2023039774 A JP2023039774 A JP 2023039774A JP 2023068035 A5 JP2023068035 A5 JP 2023068035A5
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- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- strand
- nucleic acid
- composition according
- ome
- 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.)
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- 239000008194 pharmaceutical composition Substances 0.000 claims 15
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 150000007523 nucleic acids Chemical class 0.000 claims 12
- 102000039446 nucleic acids Human genes 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 239000002773 nucleotide Substances 0.000 claims 7
- 239000003446 ligand Substances 0.000 claims 3
- 208000018565 Hemochromatosis Diseases 0.000 claims 2
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical group CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- ZGYYPTJWJBEXBC-QYYRPYCUSA-N (2r,3r,4r,5r)-5-(6-aminopurin-9-yl)-4-fluoro-2-(hydroxymethyl)oxolan-3-ol Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1F ZGYYPTJWJBEXBC-QYYRPYCUSA-N 0.000 claims 1
- 208000033981 Hereditary haemochromatosis Diseases 0.000 claims 1
- 101000798696 Homo sapiens Transmembrane protease serine 6 Proteins 0.000 claims 1
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims 1
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims 1
- 101150114298 TMPRSS6 gene Proteins 0.000 claims 1
- 102100032452 Transmembrane protease serine 6 Human genes 0.000 claims 1
- 208000005980 beta thalassemia Diseases 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 229940126836 transmembrane protease serine 6 synthesis reducer Drugs 0.000 claims 1
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP17165007.0A EP3385380A1 (en) | 2017-04-05 | 2017-04-05 | Products and compositions |
| EP17165007.0 | 2017-04-05 | ||
| EP17201405 | 2017-11-13 | ||
| EP17201405.2 | 2017-11-13 | ||
| PCT/EP2018/058764 WO2018185240A1 (en) | 2017-04-05 | 2018-04-05 | Products and compositions |
| JP2020504435A JP7247167B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020504435A Division JP7247167B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023068035A JP2023068035A (ja) | 2023-05-16 |
| JP2023068035A5 true JP2023068035A5 (https=) | 2023-07-06 |
| JP7495544B2 JP7495544B2 (ja) | 2024-06-04 |
Family
ID=61899310
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020504435A Active JP7247167B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
| JP2020554254A Active JP7419252B2 (ja) | 2017-04-05 | 2019-04-05 | アンチセンス鎖の５’末端でビニルホスホネートを有するｓｉＲＮＡ |
| JP2023039774A Active JP7495544B2 (ja) | 2017-04-05 | 2023-03-14 | 産物及び組成物 |
| JP2024001366A Pending JP2024036347A (ja) | 2017-04-05 | 2024-01-09 | アンチセンス鎖の５’末端でビニルホスホネートを有するｓｉＲＮＡ |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020504435A Active JP7247167B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
| JP2020554254A Active JP7419252B2 (ja) | 2017-04-05 | 2019-04-05 | アンチセンス鎖の５’末端でビニルホスホネートを有するｓｉＲＮＡ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024001366A Pending JP2024036347A (ja) | 2017-04-05 | 2024-01-09 | アンチセンス鎖の５’末端でビニルホスホネートを有するｓｉＲＮＡ |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US11174483B2 (https=) |
| EP (2) | EP3607068B1 (https=) |
| JP (4) | JP7247167B2 (https=) |
| KR (1) | KR102589803B1 (https=) |
| CN (2) | CN110573619B (https=) |
| AU (2) | AU2018247924B2 (https=) |
| CA (2) | CA3057561C (https=) |
| DK (1) | DK3607068T3 (https=) |
| ES (1) | ES2936109T3 (https=) |
| FI (1) | FI3607068T3 (https=) |
| HR (1) | HRP20221547T1 (https=) |
| HU (1) | HUE061894T2 (https=) |
| IL (2) | IL269576B2 (https=) |
| LT (1) | LT3607068T (https=) |
| PL (1) | PL3607068T3 (https=) |
| RS (1) | RS63887B1 (https=) |
| SI (1) | SI3607068T1 (https=) |
| WO (1) | WO2018185240A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102365961B1 (ko) | 2011-03-29 | 2022-02-23 | 알닐람 파마슈티칼스 인코포레이티드 | Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법 |
| EP4029942A1 (en) | 2017-04-05 | 2022-07-20 | Silence Therapeutics GmbH | Products and compositions |
| JP7155239B2 (ja) * | 2017-04-05 | 2022-10-18 | サイレンス・セラピューティクス・ゲーエムベーハー | 産物及び組成物 |
| EP3598995A1 (en) * | 2018-07-26 | 2020-01-29 | Silence Therapeutics GmbH | Products and compositions |
| CN111973619B (zh) * | 2019-05-23 | 2024-01-30 | 苏州瑞博生物技术股份有限公司 | 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途 |
| US20240240187A1 (en) | 2021-04-27 | 2024-07-18 | Silence Therapeutics Gmbh | Sirna targeting tmprss6 for the treatment of myeloproliferative disorders |
| TWI868755B (zh) | 2022-06-24 | 2025-01-01 | 丹麥商諾佛 儂迪克股份有限公司 | 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 |
| WO2024060649A1 (zh) * | 2022-09-29 | 2024-03-28 | 广州必贝特医药股份有限公司 | 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用 |
| EP4687983A1 (en) | 2023-03-27 | 2026-02-11 | Silence Therapeutics GmbH | Compounds and compositions for use in stem cell transplantation |
| WO2025132845A1 (en) | 2023-12-21 | 2025-06-26 | Silence Therapeutics Gmbh | Galnac phosphoramidites and their uses |
| WO2025163186A1 (en) * | 2024-02-01 | 2025-08-07 | Silence Therapeutics Gmbh | Conjugated nucleic acids and nucleic acids comprising locked nucleosides and inverted nucleotides for inhibiting gene expression in a cell |
| WO2026003163A1 (en) | 2024-06-26 | 2026-01-02 | Silence Therapeutics Gmbh | Sirna against tmprss6 for treating polycythemia vera |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NL9201440A (nl) | 1992-08-11 | 1994-03-01 | Univ Leiden | Triantennaire clusterglycosiden, hun bereiding en toepassing. |
| EP2305812A3 (en) * | 2002-11-14 | 2012-06-06 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| KR101869570B1 (ko) * | 2010-04-28 | 2018-06-20 | 아이오니스 파마수티컬즈, 인코포레이티드 | 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물 |
| KR102365961B1 (ko) * | 2011-03-29 | 2022-02-23 | 알닐람 파마슈티칼스 인코포레이티드 | Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법 |
| EP3650544A1 (en) * | 2011-11-07 | 2020-05-13 | Ionis Pharmaceuticals, Inc. | Modulation of tmprss6 expression |
| KR102486617B1 (ko) | 2013-05-22 | 2023-01-12 | 알닐람 파마슈티칼스 인코포레이티드 | Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법 |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| WO2016055601A1 (en) * | 2014-10-10 | 2016-04-14 | F. Hoffmann-La Roche Ag | Galnac phosphoramidites, nucleic acid conjugates thereof and their use |
| WO2016085852A1 (en) * | 2014-11-24 | 2016-06-02 | Alnylam Pharmaceuticals, Inc. | Tmprss6 irna compositions and methods of use thereof |
| US20160272970A1 (en) * | 2015-03-17 | 2016-09-22 | Arrowhead Madison Inc. | RNA Interference Agents |
| EP3228326A1 (en) | 2016-04-05 | 2017-10-11 | Silence Therapeutics GmbH | Nucleic acid linked to a trivalent glycoconjugate |
| JP7155239B2 (ja) * | 2017-04-05 | 2022-10-18 | サイレンス・セラピューティクス・ゲーエムベーハー | 産物及び組成物 |
| EP4029942A1 (en) * | 2017-04-05 | 2022-07-20 | Silence Therapeutics GmbH | Products and compositions |
-
2018
- 2018-04-05 WO PCT/EP2018/058764 patent/WO2018185240A1/en not_active Ceased
- 2018-04-05 KR KR1020197032297A patent/KR102589803B1/ko active Active
- 2018-04-05 EP EP18715705.2A patent/EP3607068B1/en active Active
- 2018-04-05 US US16/500,703 patent/US11174483B2/en active Active
- 2018-04-05 FI FIEP18715705.2T patent/FI3607068T3/fi active
- 2018-04-05 IL IL269576A patent/IL269576B2/en unknown
- 2018-04-05 JP JP2020504435A patent/JP7247167B2/ja active Active
- 2018-04-05 EP EP22202522.3A patent/EP4219714A3/en active Pending
- 2018-04-05 SI SI201830837T patent/SI3607068T1/sl unknown
- 2018-04-05 RS RS20230024A patent/RS63887B1/sr unknown
- 2018-04-05 ES ES18715705T patent/ES2936109T3/es active Active
- 2018-04-05 HU HUE18715705A patent/HUE061894T2/hu unknown
- 2018-04-05 CN CN201880021535.3A patent/CN110573619B/zh active Active
- 2018-04-05 HR HRP20221547TT patent/HRP20221547T1/hr unknown
- 2018-04-05 DK DK18715705.2T patent/DK3607068T3/da active
- 2018-04-05 AU AU2018247924A patent/AU2018247924B2/en active Active
- 2018-04-05 CA CA3057561A patent/CA3057561C/en active Active
- 2018-04-05 PL PL18715705.2T patent/PL3607068T3/pl unknown
- 2018-04-05 LT LTEPPCT/EP2018/058764T patent/LT3607068T/lt unknown
-
2019
- 2019-04-05 CN CN201980037644.9A patent/CN112424354A/zh active Pending
- 2019-04-05 CA CA3095523A patent/CA3095523A1/en active Pending
- 2019-04-05 AU AU2019247637A patent/AU2019247637B2/en active Active
- 2019-04-05 JP JP2020554254A patent/JP7419252B2/ja active Active
- 2019-04-05 IL IL277636A patent/IL277636B2/en unknown
-
2021
- 2021-10-05 US US17/450,064 patent/US20220025377A1/en active Pending
-
2023
- 2023-03-14 JP JP2023039774A patent/JP7495544B2/ja active Active
-
2024
- 2024-01-09 JP JP2024001366A patent/JP2024036347A/ja active Pending
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