JP2013514321A5 - - Google Patents
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- JP2013514321A5 JP2013514321A5 JP2012543756A JP2012543756A JP2013514321A5 JP 2013514321 A5 JP2013514321 A5 JP 2013514321A5 JP 2012543756 A JP2012543756 A JP 2012543756A JP 2012543756 A JP2012543756 A JP 2012543756A JP 2013514321 A5 JP2013514321 A5 JP 2013514321A5
- Authority
- JP
- Japan
- Prior art keywords
- strand
- composition
- rnai agent
- sequence
- hsf1
- 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.)
- Granted
Links
- 108091030071 RNAI Proteins 0.000 claims 17
- 239000003795 chemical substances by application Substances 0.000 claims 17
- 230000009368 gene silencing by RNA Effects 0.000 claims 17
- 102100032606 Heat shock factor protein 1 Human genes 0.000 claims 10
- 101000867525 Homo sapiens Heat shock factor protein 1 Proteins 0.000 claims 10
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 239000002773 nucleotide Substances 0.000 claims 6
- 150000001720 carbohydrates Chemical class 0.000 claims 4
- 235000014633 carbohydrates Nutrition 0.000 claims 4
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 206010028980 Neoplasm Diseases 0.000 claims 3
- 201000011510 cancer Diseases 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- KIUKXJAPPMFGSW-DNGZLQJQSA-N (2S,3S,4S,5R,6R)-6-[(2S,3R,4R,5S,6R)-3-Acetamido-2-[(2S,3S,4R,5R,6R)-6-[(2R,3R,4R,5S,6R)-3-acetamido-2,5-dihydroxy-6-(hydroxymethyl)oxan-4-yl]oxy-2-carboxy-4,5-dihydroxyoxan-3-yl]oxy-5-hydroxy-6-(hydroxymethyl)oxan-4-yl]oxy-3,4,5-trihydroxyoxane-2-carboxylic acid Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O[C@H]1[C@H](O)[C@@H](O)[C@H](O[C@H]2[C@@H]([C@@H](O[C@H]3[C@@H]([C@@H](O)[C@H](O)[C@H](O3)C(O)=O)O)[C@H](O)[C@@H](CO)O2)NC(C)=O)[C@@H](C(O)=O)O1 KIUKXJAPPMFGSW-DNGZLQJQSA-N 0.000 claims 2
- 229960000549 4-dimethylaminophenol Drugs 0.000 claims 2
- VHYFNPMBLIVWCW-UHFFFAOYSA-N 4-dimethylaminopyridine Substances CN(C)C1=CC=NC=C1 VHYFNPMBLIVWCW-UHFFFAOYSA-N 0.000 claims 2
- 229920002101 Chitin Polymers 0.000 claims 2
- 229920001661 Chitosan Polymers 0.000 claims 2
- 229920000858 Cyclodextrin Polymers 0.000 claims 2
- 229920002307 Dextran Polymers 0.000 claims 2
- 229920001202 Inulin Polymers 0.000 claims 2
- 102000004856 Lectins Human genes 0.000 claims 2
- 108090001090 Lectins Proteins 0.000 claims 2
- SMEROWZSTRWXGI-UHFFFAOYSA-N Lithocholsaeure Natural products C1CC2CC(O)CCC2(C)C2C1C1CCC(C(CCC(O)=O)C)C1(C)CC2 SMEROWZSTRWXGI-UHFFFAOYSA-N 0.000 claims 2
- 229920001218 Pullulan Polymers 0.000 claims 2
- 239000004373 Pullulan Substances 0.000 claims 2
- 102000004338 Transferrin Human genes 0.000 claims 2
- 108090000901 Transferrin Proteins 0.000 claims 2
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- 239000002253 acid Substances 0.000 claims 2
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- 235000012000 cholesterol Nutrition 0.000 claims 2
- 150000001875 compounds Chemical class 0.000 claims 2
- 150000002148 esters Chemical class 0.000 claims 2
- 230000002496 gastric effect Effects 0.000 claims 2
- 229920002674 hyaluronan Polymers 0.000 claims 2
- 229960003160 hyaluronic acid Drugs 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 102000006495 integrins Human genes 0.000 claims 2
- 108010044426 integrins Proteins 0.000 claims 2
- JYJIGFIDKWBXDU-MNNPPOADSA-N inulin Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)OC[C@]1(OC[C@]2(OC[C@]3(OC[C@]4(OC[C@]5(OC[C@]6(OC[C@]7(OC[C@]8(OC[C@]9(OC[C@]%10(OC[C@]%11(OC[C@]%12(OC[C@]%13(OC[C@]%14(OC[C@]%15(OC[C@]%16(OC[C@]%17(OC[C@]%18(OC[C@]%19(OC[C@]%20(OC[C@]%21(OC[C@]%22(OC[C@]%23(OC[C@]%24(OC[C@]%25(OC[C@]%26(OC[C@]%27(OC[C@]%28(OC[C@]%29(OC[C@]%30(OC[C@]%31(OC[C@]%32(OC[C@]%33(OC[C@]%34(OC[C@]%35(OC[C@]%36(O[C@@H]%37[C@@H]([C@@H](O)[C@H](O)[C@@H](CO)O%37)O)[C@H]([C@H](O)[C@@H](CO)O%36)O)[C@H]([C@H](O)[C@@H](CO)O%35)O)[C@H]([C@H](O)[C@@H](CO)O%34)O)[C@H]([C@H](O)[C@@H](CO)O%33)O)[C@H]([C@H](O)[C@@H](CO)O%32)O)[C@H]([C@H](O)[C@@H](CO)O%31)O)[C@H]([C@H](O)[C@@H](CO)O%30)O)[C@H]([C@H](O)[C@@H](CO)O%29)O)[C@H]([C@H](O)[C@@H](CO)O%28)O)[C@H]([C@H](O)[C@@H](CO)O%27)O)[C@H]([C@H](O)[C@@H](CO)O%26)O)[C@H]([C@H](O)[C@@H](CO)O%25)O)[C@H]([C@H](O)[C@@H](CO)O%24)O)[C@H]([C@H](O)[C@@H](CO)O%23)O)[C@H]([C@H](O)[C@@H](CO)O%22)O)[C@H]([C@H](O)[C@@H](CO)O%21)O)[C@H]([C@H](O)[C@@H](CO)O%20)O)[C@H]([C@H](O)[C@@H](CO)O%19)O)[C@H]([C@H](O)[C@@H](CO)O%18)O)[C@H]([C@H](O)[C@@H](CO)O%17)O)[C@H]([C@H](O)[C@@H](CO)O%16)O)[C@H]([C@H](O)[C@@H](CO)O%15)O)[C@H]([C@H](O)[C@@H](CO)O%14)O)[C@H]([C@H](O)[C@@H](CO)O%13)O)[C@H]([C@H](O)[C@@H](CO)O%12)O)[C@H]([C@H](O)[C@@H](CO)O%11)O)[C@H]([C@H](O)[C@@H](CO)O%10)O)[C@H]([C@H](O)[C@@H](CO)O9)O)[C@H]([C@H](O)[C@@H](CO)O8)O)[C@H]([C@H](O)[C@@H](CO)O7)O)[C@H]([C@H](O)[C@@H](CO)O6)O)[C@H]([C@H](O)[C@@H](CO)O5)O)[C@H]([C@H](O)[C@@H](CO)O4)O)[C@H]([C@H](O)[C@@H](CO)O3)O)[C@H]([C@H](O)[C@@H](CO)O2)O)[C@@H](O)[C@H](O)[C@@H](CO)O1 JYJIGFIDKWBXDU-MNNPPOADSA-N 0.000 claims 2
- 229940029339 inulin Drugs 0.000 claims 2
- 239000002523 lectin Substances 0.000 claims 2
- 150000002632 lipids Chemical class 0.000 claims 2
- 150000002634 lipophilic molecules Chemical class 0.000 claims 2
- SMEROWZSTRWXGI-HVATVPOCSA-N lithocholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)CC1 SMEROWZSTRWXGI-HVATVPOCSA-N 0.000 claims 2
- 238000012986 modification Methods 0.000 claims 2
- 229920005615 natural polymer Polymers 0.000 claims 2
- 229920000768 polyamine Polymers 0.000 claims 2
- 229920000642 polymer Polymers 0.000 claims 2
- 235000019423 pullulan Nutrition 0.000 claims 2
- 125000006853 reporter group Chemical group 0.000 claims 2
- HFHDHCJBZVLPGP-UHFFFAOYSA-N schardinger α-dextrin Chemical compound O1C(C(C2O)O)C(CO)OC2OC(C(C2O)O)C(CO)OC2OC(C(C2O)O)C(CO)OC2OC(C(O)C2O)C(CO)OC2OC(C(C2O)O)C(CO)OC2OC2C(O)C(O)C1OC2CO HFHDHCJBZVLPGP-UHFFFAOYSA-N 0.000 claims 2
- 150000003431 steroids Chemical class 0.000 claims 2
- 239000000126 substance Substances 0.000 claims 2
- 150000003505 terpenes Chemical class 0.000 claims 2
- 235000007586 terpenes Nutrition 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 239000012581 transferrin Substances 0.000 claims 2
- 150000003648 triterpenes Chemical class 0.000 claims 2
- XUARCIYIVXVTAE-ZAPOICBTSA-N uvaol Chemical compound C1C[C@H](O)C(C)(C)[C@@H]2CC[C@@]3(C)[C@]4(C)CC[C@@]5(CO)CC[C@@H](C)[C@H](C)[C@H]5C4=CC[C@@H]3[C@]21C XUARCIYIVXVTAE-ZAPOICBTSA-N 0.000 claims 2
- 235000013343 vitamin Nutrition 0.000 claims 2
- 239000011782 vitamin Substances 0.000 claims 2
- 229940088594 vitamin Drugs 0.000 claims 2
- 229930003231 vitamin Natural products 0.000 claims 2
- 208000036762 Acute promyelocytic leukaemia Diseases 0.000 claims 1
- 239000004971 Cross linker Substances 0.000 claims 1
- KVSNMTUIMXZPLU-UHFFFAOYSA-N D:A-friedo-oleanane Natural products CC12CCC3(C)C4CC(C)(C)CCC4(C)CCC3(C)C2CCC2(C)C1CCCC2C KVSNMTUIMXZPLU-UHFFFAOYSA-N 0.000 claims 1
- 208000006168 Ewing Sarcoma Diseases 0.000 claims 1
- 201000008808 Fibrosarcoma Diseases 0.000 claims 1
- JUUHNUPNMCGYDT-UHFFFAOYSA-N Friedelin Natural products CC1CC2C(C)(CCC3(C)C4CC(C)(C)CCC4(C)CCC23C)C5CCC(=O)C(C)C15 JUUHNUPNMCGYDT-UHFFFAOYSA-N 0.000 claims 1
- QOLRLLFJMZLYQJ-LOBDNJQFSA-N Hecogenin Chemical compound O([C@@H]1[C@@H]([C@]2(C(=O)C[C@@H]3[C@@]4(C)CC[C@H](O)C[C@@H]4CC[C@H]3[C@@H]2C1)C)[C@@H]1C)[C@]11CC[C@@H](C)CO1 QOLRLLFJMZLYQJ-LOBDNJQFSA-N 0.000 claims 1
- OXLGJTRVVNGJRK-UHFFFAOYSA-N Hecogenin Natural products CC1CCC2(CC3CC4C5CCC6CC(O)CCC6(C)C5CC(=O)C4(C)C3C2C)OC1 OXLGJTRVVNGJRK-UHFFFAOYSA-N 0.000 claims 1
- 208000017604 Hodgkin disease Diseases 0.000 claims 1
- 208000010747 Hodgkins lymphoma Diseases 0.000 claims 1
- 206010025323 Lymphomas Diseases 0.000 claims 1
- UVLDESQWQRMYKD-UHFFFAOYSA-N Neobotogenin Natural products CC1C(C2(C(=O)CC3C4(C)CCC(O)CC4=CCC3C2C2)C)C2OC11CCC(C)CO1 UVLDESQWQRMYKD-UHFFFAOYSA-N 0.000 claims 1
- 206010029260 Neuroblastoma Diseases 0.000 claims 1
- GMBQZIIUCVWOCD-WWASVFFGSA-N Sarsasapogenin Natural products O([C@@H]1[C@@H]([C@]2(CC[C@@H]3[C@@]4(C)CC[C@H](O)C[C@H]4CC[C@H]3[C@@H]2C1)C)[C@@H]1C)[C@]11CC[C@H](C)CO1 GMBQZIIUCVWOCD-WWASVFFGSA-N 0.000 claims 1
- 208000005718 Stomach Neoplasms Diseases 0.000 claims 1
- 208000024313 Testicular Neoplasms Diseases 0.000 claims 1
- 206010057644 Testis cancer Diseases 0.000 claims 1
- DWCSNWXARWMZTG-UHFFFAOYSA-N Trigonegenin A Natural products CC1C(C2(CCC3C4(C)CCC(O)C=C4CCC3C2C2)C)C2OC11CCC(C)CO1 DWCSNWXARWMZTG-UHFFFAOYSA-N 0.000 claims 1
- 210000000988 bone and bone Anatomy 0.000 claims 1
- 210000000481 breast Anatomy 0.000 claims 1
- 210000003679 cervix uteri Anatomy 0.000 claims 1
- 210000001072 colon Anatomy 0.000 claims 1
- 239000003431 cross linking reagent Substances 0.000 claims 1
- WQLVFSAGQJTQCK-VKROHFNGSA-N diosgenin Chemical compound O([C@@H]1[C@@H]([C@]2(CC[C@@H]3[C@@]4(C)CC[C@H](O)CC4=CC[C@H]3[C@@H]2C1)C)[C@@H]1C)[C@]11CC[C@@H](C)CO1 WQLVFSAGQJTQCK-VKROHFNGSA-N 0.000 claims 1
- WQLVFSAGQJTQCK-UHFFFAOYSA-N diosgenin Natural products CC1C(C2(CCC3C4(C)CCC(O)CC4=CCC3C2C2)C)C2OC11CCC(C)CO1 WQLVFSAGQJTQCK-UHFFFAOYSA-N 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000002357 endometrial effect Effects 0.000 claims 1
- OFMXGFHWLZPCFL-SVRPQWSVSA-N friedelin Chemical compound C([C@H]1[C@]2(C)CC[C@@]34C)C(C)(C)CC[C@]1(C)CC[C@]2(C)[C@H]4CC[C@@]1(C)[C@H]3CCC(=O)[C@@H]1C OFMXGFHWLZPCFL-SVRPQWSVSA-N 0.000 claims 1
- MFVJCHSUSSRHRH-UHFFFAOYSA-N friedeline Natural products CC1(C)CCC2(C)CCC3C4(C)CCC5C(C)(C)C(=O)CCC5(C)C4CCC3(C)C2C1 MFVJCHSUSSRHRH-UHFFFAOYSA-N 0.000 claims 1
- 206010017758 gastric cancer Diseases 0.000 claims 1
- 230000002489 hematologic effect Effects 0.000 claims 1
- 230000005764 inhibitory process Effects 0.000 claims 1
- 210000000936 intestine Anatomy 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 208000032839 leukemia Diseases 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004072 lung Anatomy 0.000 claims 1
- 201000008968 osteosarcoma Diseases 0.000 claims 1
- 230000002611 ovarian Effects 0.000 claims 1
- 239000000816 peptidomimetic Substances 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 210000002307 prostate Anatomy 0.000 claims 1
- 201000011549 stomach cancer Diseases 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 201000003120 testicular cancer Diseases 0.000 claims 1
- SYFNOXYZEIYOSE-UHFFFAOYSA-N uvaol Natural products CC1CCC2(O)CCC3(C)C(=CCC4(C)C5(C)CCC(O)C(C)(C)C5CCC34C)C2C1C SYFNOXYZEIYOSE-UHFFFAOYSA-N 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US28813709P | 2009-12-18 | 2009-12-18 | |
| US61/288,137 | 2009-12-18 | ||
| PCT/EP2010/069917 WO2011073326A2 (en) | 2009-12-18 | 2010-12-16 | Organic compositions to treat hsf1-related diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015195916A Division JP5981625B2 (ja) | 2009-12-18 | 2015-10-01 | Ｈｓｆ１関連疾患を処置するための有機組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013514321A JP2013514321A (ja) | 2013-04-25 |
| JP2013514321A5 true JP2013514321A5 (enExample) | 2014-01-16 |
| JP5819848B2 JP5819848B2 (ja) | 2015-11-24 |
Family
ID=43646461
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012543756A Active JP5819848B2 (ja) | 2009-12-18 | 2010-12-16 | Ｈｓｆ１関連疾患を処置するための有機組成物 |
| JP2015195916A Active JP5981625B2 (ja) | 2009-12-18 | 2015-10-01 | Ｈｓｆ１関連疾患を処置するための有機組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015195916A Active JP5981625B2 (ja) | 2009-12-18 | 2015-10-01 | Ｈｓｆ１関連疾患を処置するための有機組成物 |
Country Status (14)
| Country | Link |
|---|---|
| US (8) | US8293718B2 (enExample) |
| EP (4) | EP3406720A1 (enExample) |
| JP (2) | JP5819848B2 (enExample) |
| KR (2) | KR101605932B1 (enExample) |
| CN (2) | CN105125572A (enExample) |
| AR (1) | AR079494A1 (enExample) |
| AU (1) | AU2010332881B2 (enExample) |
| BR (1) | BR112012014760A2 (enExample) |
| CA (2) | CA2784783C (enExample) |
| HK (1) | HK1214949A1 (enExample) |
| MX (3) | MX339050B (enExample) |
| TW (1) | TWI576108B (enExample) |
| UY (1) | UY33114A (enExample) |
| WO (1) | WO2011073326A2 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2322145T3 (es) | 2002-07-26 | 2009-06-17 | Novartis Vaccines And Diagnostics, Inc. | Moleculas pequeñas modificadas de adn inerferente y procedimiento de uso. |
| EP1796732B1 (en) * | 2004-10-01 | 2013-10-30 | Novartis Vaccines and Diagnostics, Inc. | Modified small interfering rna molecules and methods of use |
| US8765704B1 (en) | 2008-02-28 | 2014-07-01 | Novartis Ag | Modified small interfering RNA molecules and methods of use |
| EP3406720A1 (en) | 2009-12-18 | 2018-11-28 | Arrowhead Pharmaceuticals, Inc. | Organic compositions to treat hsf1-related diseases |
| JP2014525435A (ja) * | 2011-09-02 | 2014-09-29 | ノバルティス アーゲー | Ｈｓｆ１関連疾患を処置するための有機組成物 |
| BR112014016870A2 (pt) * | 2012-01-09 | 2017-06-27 | Huesken Dieter | composições orgânicas para tratar doenças relacionadas com beta-catenina |
| BR112015013440B1 (pt) * | 2012-12-13 | 2020-12-08 | Aduro Biotech, Inc | composições compreendendo dinucleotídeos de purina cíclicos com estereoquímicas |
| GB201317609D0 (en) | 2013-10-04 | 2013-11-20 | Cancer Rec Tech Ltd | Inhibitor compounds |
| LT3119888T (lt) | 2014-03-19 | 2021-10-11 | Ionis Pharmaceuticals, Inc. | Kompozicijos, skirtos ataksino 2 raiškos moduliavimui |
| GB201505658D0 (en) | 2015-04-01 | 2015-05-13 | Cancer Rec Tech Ltd | Inhibitor compounds |
| GB201617103D0 (en) | 2016-10-07 | 2016-11-23 | Cancer Research Technology Limited | Compound |
| FR3082274B1 (fr) | 2018-06-06 | 2021-11-19 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante |
| TWI843738B (zh) | 2018-07-25 | 2024-06-01 | 美商Ionis製藥公司 | 用於減少atxn2表現之化合物及方法 |
| CN113862361B (zh) * | 2021-10-25 | 2023-08-15 | 中山大学孙逸仙纪念医院 | 一种诊断和治疗膀胱癌的分子标志物hsf1及其用途 |
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| AUPP249298A0 (en) * | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
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