HRP20221547T1 - Inhibicija tmprss6 posredovana interferencijom rna - Google Patents
Inhibicija tmprss6 posredovana interferencijom rna Download PDFInfo
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- HRP20221547T1 HRP20221547T1 HRP20221547TT HRP20221547T HRP20221547T1 HR P20221547 T1 HRP20221547 T1 HR P20221547T1 HR P20221547T T HRP20221547T T HR P20221547TT HR P20221547 T HRP20221547 T HR P20221547T HR P20221547 T1 HRP20221547 T1 HR P20221547T1
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- Prior art keywords
- nucleic acid
- structural strand
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- acid according
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- 238000012228 RNA interference-mediated gene silencing Methods 0.000 title 1
- 230000009368 gene silencing by RNA Effects 0.000 title 1
- 230000005764 inhibitory process Effects 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 claims 32
- 150000007523 nucleic acids Chemical class 0.000 claims 32
- 102000039446 nucleic acids Human genes 0.000 claims 32
- 125000003729 nucleotide group Chemical group 0.000 claims 13
- 239000002773 nucleotide Substances 0.000 claims 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 3
- 208000035475 disorder Diseases 0.000 claims 3
- 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 2
- 208000019838 Blood disease Diseases 0.000 claims 2
- 208000007209 Erythropoietic Porphyria Diseases 0.000 claims 2
- 238000012413 Fluorescence activated cell sorting analysis Methods 0.000 claims 2
- 208000018565 Hemochromatosis Diseases 0.000 claims 2
- 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 2
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 2
- 206010036182 Porphyria acute Diseases 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 201000008230 cutaneous porphyria Diseases 0.000 claims 2
- 210000000267 erythroid cell Anatomy 0.000 claims 2
- 230000010437 erythropoiesis Effects 0.000 claims 2
- 208000014951 hematologic disease Diseases 0.000 claims 2
- 208000018706 hematopoietic system disease Diseases 0.000 claims 2
- 229910052742 iron Inorganic materials 0.000 claims 2
- 239000003446 ligand Substances 0.000 claims 2
- 238000012986 modification Methods 0.000 claims 2
- 230000004048 modification Effects 0.000 claims 2
- 210000003924 normoblast Anatomy 0.000 claims 2
- 210000000952 spleen Anatomy 0.000 claims 2
- 208000018240 Bone Marrow Failure disease Diseases 0.000 claims 1
- 208000035220 Dyserythropoietic Congenital Anemia Diseases 0.000 claims 1
- 102000001554 Hemoglobins Human genes 0.000 claims 1
- 108010054147 Hemoglobins Proteins 0.000 claims 1
- 101000798696 Homo sapiens Transmembrane protease serine 6 Proteins 0.000 claims 1
- 201000003793 Myelodysplastic syndrome Diseases 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
- 201000010273 Porphyria Cutanea Tarda Diseases 0.000 claims 1
- 206010036186 Porphyria non-acute Diseases 0.000 claims 1
- 206010041660 Splenomegaly Diseases 0.000 claims 1
- 101150114298 TMPRSS6 gene Proteins 0.000 claims 1
- 102100032452 Transmembrane protease serine 6 Human genes 0.000 claims 1
- 208000007502 anemia Diseases 0.000 claims 1
- 239000012752 auxiliary agent Substances 0.000 claims 1
- 208000005980 beta thalassemia Diseases 0.000 claims 1
- 210000001185 bone marrow Anatomy 0.000 claims 1
- 201000004440 congenital dyserythropoietic anemia Diseases 0.000 claims 1
- 210000003743 erythrocyte Anatomy 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 230000035800 maturation Effects 0.000 claims 1
- 230000001737 promoting effect Effects 0.000 claims 1
- 208000007056 sickle cell anemia Diseases 0.000 claims 1
- 229940126836 transmembrane protease serine 6 synthesis reducer Drugs 0.000 claims 1
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP17165007.0A EP3385380A1 (en) | 2017-04-05 | 2017-04-05 | Products and compositions |
| EP17201405 | 2017-11-13 | ||
| PCT/EP2018/058764 WO2018185240A1 (en) | 2017-04-05 | 2018-04-05 | Products and compositions |
| EP18715705.2A EP3607068B1 (en) | 2017-04-05 | 2018-04-05 | Rna interference mediated inhibition of tmprss6 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20221547T1 true HRP20221547T1 (hr) | 2023-03-03 |
Family
ID=61899310
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20221547TT HRP20221547T1 (hr) | 2017-04-05 | 2018-04-05 | Inhibicija tmprss6 posredovana interferencijom rna |
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 |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| 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 |
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