JP2011517676A5 - - Google Patents
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- JP2011517676A5 JP2011517676A5 JP2011502065A JP2011502065A JP2011517676A5 JP 2011517676 A5 JP2011517676 A5 JP 2011517676A5 JP 2011502065 A JP2011502065 A JP 2011502065A JP 2011502065 A JP2011502065 A JP 2011502065A JP 2011517676 A5 JP2011517676 A5 JP 2011517676A5
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- 125000003729 nucleotide group Chemical group 0.000 claims 20
- 210000001519 tissue Anatomy 0.000 claims 16
- 239000002773 nucleotide Substances 0.000 claims 14
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 claims 10
- 108091034117 Oligonucleotide Proteins 0.000 claims 10
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol group Chemical group [C@@H]1(CC[C@H]2[C@@H]3CC=C4C[C@@H](O)CC[C@]4(C)[C@H]3CC[C@]12C)[C@H](C)CCCC(C)C HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 9
- 238000000034 method Methods 0.000 claims 9
- 239000008194 pharmaceutical composition Substances 0.000 claims 8
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 6
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 6
- 230000000295 complement effect Effects 0.000 claims 5
- 239000000468 intravenous fat emulsion Substances 0.000 claims 5
- 235000012000 cholesterol Nutrition 0.000 claims 4
- 235000011187 glycerol Nutrition 0.000 claims 4
- 210000005003 heart tissue Anatomy 0.000 claims 4
- 210000004072 lung Anatomy 0.000 claims 4
- 210000003205 muscle Anatomy 0.000 claims 4
- 239000002245 particle Substances 0.000 claims 4
- 239000000126 substance Substances 0.000 claims 4
- 102000018616 Apolipoproteins B Human genes 0.000 claims 3
- 108010027006 Apolipoproteins B Proteins 0.000 claims 3
- 101100356682 Caenorhabditis elegans rho-1 gene Proteins 0.000 claims 3
- 101150111584 RHOA gene Proteins 0.000 claims 3
- 230000014509 gene expression Effects 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 241000124008 Mammalia Species 0.000 claims 2
- 230000000692 anti-sense effect Effects 0.000 claims 2
- 229940068998 egg yolk phospholipid Drugs 0.000 claims 2
- 239000008344 egg yolk phospholipid Substances 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 102000019758 lipid binding proteins Human genes 0.000 claims 2
- 108091016323 lipid binding proteins Proteins 0.000 claims 2
- 150000003904 phospholipids Chemical class 0.000 claims 2
- 235000012424 soybean oil Nutrition 0.000 claims 2
- 239000003549 soybean oil Substances 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 2
- AGNTUZCMJBTHOG-UHFFFAOYSA-N 3-[3-(2,3-dihydroxypropoxy)-2-hydroxypropoxy]propane-1,2-diol Chemical compound OCC(O)COCC(O)COCC(O)CO AGNTUZCMJBTHOG-UHFFFAOYSA-N 0.000 claims 1
- 208000035657 Abasia Diseases 0.000 claims 1
- 108091081021 Sense strand Proteins 0.000 claims 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 1
- 238000001727 in vivo Methods 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 125000001921 locked nucleotide group Chemical group 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- ONKSSDKXDIVIHK-UHFFFAOYSA-N n,n-didecyldodecanamide Chemical group CCCCCCCCCCCC(=O)N(CCCCCCCCCC)CCCCCCCCCC ONKSSDKXDIVIHK-UHFFFAOYSA-N 0.000 claims 1
- 238000012385 systemic delivery Methods 0.000 claims 1
- 238000012384 transportation and delivery Methods 0.000 claims 1
- DCXXMTOCNZCJGO-UHFFFAOYSA-N tristearoylglycerol Chemical compound CCCCCCCCCCCCCCCCCC(=O)OCC(OC(=O)CCCCCCCCCCCCCCCCC)COC(=O)CCCCCCCCCCCCCCCCC DCXXMTOCNZCJGO-UHFFFAOYSA-N 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP08005800.1 | 2008-03-27 | ||
| EP08005800A EP2105145A1 (en) | 2008-03-27 | 2008-03-27 | Method for muscle-specific delivery lipid-conjugated oligonucleotides |
| US4038608P | 2008-03-28 | 2008-03-28 | |
| US61/040,386 | 2008-03-28 | ||
| PCT/US2009/038437 WO2009120887A2 (en) | 2008-03-27 | 2009-03-26 | Compositions and methods for mediating rnai in vivo |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2011517676A JP2011517676A (ja) | 2011-06-16 |
| JP2011517676A5 true JP2011517676A5 (https=) | 2012-05-17 |
Family
ID=39683892
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011502065A Pending JP2011517676A (ja) | 2008-03-27 | 2009-03-26 | インビボでｒｎａ干渉を媒介するための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US8148344B2 (https=) |
| EP (2) | EP2105145A1 (https=) |
| JP (1) | JP2011517676A (https=) |
| WO (1) | WO2009120887A2 (https=) |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| JP5654352B2 (ja) | 2007-11-09 | 2015-01-14 | ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム | ｍｉＲ−１５ファミリーのマイクロＲＮＡによる心筋細胞生存及び心臓修復の調節 |
| CA2718520C (en) | 2008-03-17 | 2020-01-07 | The Board Of Regents Of The University Of Texas System | Identification of micro-rnas involved in neuromuscular synapse maintenance and regeneration |
| EP2105145A1 (en) | 2008-03-27 | 2009-09-30 | ETH Zürich | Method for muscle-specific delivery lipid-conjugated oligonucleotides |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US20100233270A1 (en) | 2009-01-08 | 2010-09-16 | Northwestern University | Delivery of Oligonucleotide-Functionalized Nanoparticles |
| WO2011091065A2 (en) | 2010-01-19 | 2011-07-28 | Northwestern University | Synthetic nanostructures including nucleic acids and/or other entities |
| CN112251436A (zh) | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| JP2013256452A (ja) * | 2012-06-11 | 2013-12-26 | Kawaken Fine Chem Co Ltd | メラニン産生抑制剤とその組成物 |
| US9163235B2 (en) | 2012-06-21 | 2015-10-20 | MiRagen Therapeutics, Inc. | Inhibitors of the miR-15 family of micro-RNAs |
| CN105579582A (zh) | 2013-07-25 | 2016-05-11 | 埃克西奎雷股份有限公司 | 用于预防和治疗用途的作为免疫刺激剂的基于球形核酸的构建体 |
| WO2015023797A1 (en) | 2013-08-13 | 2015-02-19 | Northwestern University | Lipophilic nanoparticles for drug delivery |
| CA3236835A1 (en) | 2013-11-22 | 2015-05-28 | Mina Therapeutics Limited | C/ebp alpha short activating rna compositions and methods of use |
| JP6527516B2 (ja) * | 2013-12-03 | 2019-06-05 | ノースウェスタン ユニバーシティ | リポソーム粒子、前述のものを作製する方法及びその使用 |
| EP3733856A1 (en) | 2014-04-18 | 2020-11-04 | University of Massachusetts | Exosomal loading using hydrophobically modified oligonucleotides |
| AU2015269412B2 (en) | 2014-06-04 | 2020-03-12 | Exicure Operating Company | Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications |
| WO2016028940A1 (en) | 2014-08-19 | 2016-02-25 | Northwestern University | Protein/oligonucleotide core-shell nanoparticle therapeutics |
| CA2963931A1 (en) | 2014-10-06 | 2016-04-14 | Exicure, Inc. | Anti-tnf compounds |
| KR20170078843A (ko) | 2014-11-21 | 2017-07-07 | 노오쓰웨스턴 유니버시티 | 구형 핵산 나노입자 접합체의 서열 특이적 세포 흡수 |
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| KR20180105115A (ko) * | 2015-10-07 | 2018-09-27 | 코르메딕스, 인코포레이티드 | 타우로리딘의 피부-침투성 제제 |
| US10967072B2 (en) | 2016-04-27 | 2021-04-06 | Northwestern University | Short interfering RNA templated lipoprotein particles (siRNA-TLP) |
| EP3452598A4 (en) | 2016-05-06 | 2020-04-29 | Exicure, Inc. | LIPOSOMAL SPHERICAL NUCLEIC ACID (ANS) CONSTRUCTS HAVING ANTISENSE OLIGONUCLEOTIDES (ASO) FOR THE SPECIFIC INACTIVATION OF INTERLEUKIN 17 RECEPTOR RNA |
| US11364304B2 (en) | 2016-08-25 | 2022-06-21 | Northwestern University | Crosslinked micellar spherical nucleic acids |
| WO2018152327A1 (en) * | 2017-02-15 | 2018-08-23 | Northwestern University | Enhancing stability and immunomodulatory activity of liposomal spherical nucleic acids |
| US11696954B2 (en) | 2017-04-28 | 2023-07-11 | Exicure Operating Company | Synthesis of spherical nucleic acids using lipophilic moieties |
| JP2020526558A (ja) | 2017-07-13 | 2020-08-31 | ノースウェスタン ユニバーシティ | オリゴヌクレオチド官能化金属有機構造体ナノ粒子を調製するための一般的かつ直接的な方法 |
| CA3075205A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | Stabilized hnf4a sarna compositions and methods of use |
| WO2019048645A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE |
| EP3775211B1 (en) | 2018-04-12 | 2023-04-05 | MiNA Therapeutics Limited | Sirt1-sarna compositions and methods of use |
| EP3953473A1 (en) | 2019-04-12 | 2022-02-16 | MiNA Therapeutics Limited | Sirt1-sarna compositions and methods of use |
| US20230027604A1 (en) * | 2019-07-02 | 2023-01-26 | Argonaute RNA Limited | Apolipoprotein b antagonist |
| US12319711B2 (en) | 2019-09-20 | 2025-06-03 | Northwestern University | Spherical nucleic acids with tailored and active protein coronae |
| US12378560B2 (en) | 2019-10-29 | 2025-08-05 | Northwestern University | Sequence multiplicity within spherical nucleic acids |
| GB2603454A (en) | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
| EP4314292A1 (en) | 2021-03-26 | 2024-02-07 | MiNA Therapeutics Limited | Tmem173 sarna compositions and methods of use |
| WO2023099884A1 (en) | 2021-12-01 | 2023-06-08 | Mina Therapeutics Limited | Pax6 sarna compositions and methods of use |
| GB202117758D0 (en) | 2021-12-09 | 2022-01-26 | Ucl Business Ltd | Therapeutics for the treatment of neurodegenerative disorders |
| WO2023170435A1 (en) | 2022-03-07 | 2023-09-14 | Mina Therapeutics Limited | Il10 sarna compositions and methods of use |
| WO2024125597A1 (en) | 2022-12-14 | 2024-06-20 | Providence Therapeutics Holdings Inc. | Compositions and methods for infectious diseases |
| WO2024134199A1 (en) | 2022-12-22 | 2024-06-27 | Mina Therapeutics Limited | Chemically modified sarna compositions and methods of use |
| WO2026027887A2 (en) | 2024-08-02 | 2026-02-05 | Mina Therapeutics Limited | Hbg1/2-sarna compositions and methods of use |
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| EP1080103A4 (en) | 1998-05-21 | 2003-07-02 | Isis Pharmaceuticals Inc | PREPARATIONS AND METHOD FOR THE NON-PARENTERAL ADMINISTRATION OF OLIGONUCLEOTIDES |
| JP2002537343A (ja) | 1999-02-23 | 2002-11-05 | アイシス・ファーマシューティカルス・インコーポレーテッド | 多重粒子製剤 |
| GB9930533D0 (en) * | 1999-12-23 | 2000-02-16 | Mitsubishi Tokyo Pharm Inc | Nucleic acid delivery |
| WO2002081628A2 (en) | 2001-04-05 | 2002-10-17 | Ribozyme Pharmaceuticals, Incorporated | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies |
| US20040259247A1 (en) | 2000-12-01 | 2004-12-23 | Thomas Tuschl | Rna interference mediating small rna molecules |
| WO2002087594A1 (en) * | 2001-04-30 | 2002-11-07 | The Regents Of The University Of California | Non-viral vesicle vector for cardiac specific gene delivery |
| US20050277133A1 (en) * | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| GB0118517D0 (en) * | 2001-07-30 | 2001-09-19 | Mitsubishi Tokyo Pharm Inc | Compound |
| US7956176B2 (en) | 2002-09-05 | 2011-06-07 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| EP2305812A3 (en) | 2002-11-14 | 2012-06-06 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| DE10302421A1 (de) | 2003-01-21 | 2004-07-29 | Ribopharma Ag | Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit |
| US20060003976A1 (en) | 2004-06-04 | 2006-01-05 | Yuehua Zhang | Cholesterol/bile acid/bile acid derivative-modified therapeutic drug compounds |
| US7740861B2 (en) | 2004-06-16 | 2010-06-22 | University Of Massachusetts | Drug delivery product and methods |
| FR2894582B1 (fr) * | 2005-12-12 | 2008-02-22 | Fabre Pierre Dermo Cosmetique | Sirna anti myosine va et depigmentation de la peau |
| CA2648132C (en) * | 2006-04-03 | 2019-05-28 | Santaris Pharma A/S | Pharmaceutical composition comprising anti-mirna antisense oligonucleotides |
| EP1844772A1 (en) * | 2006-04-06 | 2007-10-17 | Polyplus-Transfection SA | Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications |
| EP2105145A1 (en) | 2008-03-27 | 2009-09-30 | ETH Zürich | Method for muscle-specific delivery lipid-conjugated oligonucleotides |
-
2008
- 2008-03-27 EP EP08005800A patent/EP2105145A1/en not_active Withdrawn
-
2009
- 2009-03-26 JP JP2011502065A patent/JP2011517676A/ja active Pending
- 2009-03-26 US US12/412,206 patent/US8148344B2/en active Active
- 2009-03-26 EP EP09724197A patent/EP2265289A2/en not_active Withdrawn
- 2009-03-26 WO PCT/US2009/038437 patent/WO2009120887A2/en not_active Ceased
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