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 |
|---|---|---|---|
| EP08005800A EP2105145A1 (en) | 2008-03-27 | 2008-03-27 | Method for muscle-specific delivery lipid-conjugated oligonucleotides |
| EP08005800.1 | 2008-03-27 | ||
| 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 (enExample) | 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 (enExample) |
| EP (2) | EP2105145A1 (enExample) |
| JP (1) | JP2011517676A (enExample) |
| WO (1) | WO2009120887A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| CA2705325C (en) | 2007-11-09 | 2016-11-01 | The Board Of Regents Of The University Of Texas System | Micro-rnas of the mir-15 family modulate cardiomyocyte survival and cardiac repair |
| 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 |
| US20130034599A1 (en) | 2010-01-19 | 2013-02-07 | Northwestern University | Synthetic nanostructures including nucleic acids and/or other entities |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| 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 |
| WO2015013673A1 (en) | 2013-07-25 | 2015-01-29 | Aurasense Therapeutics, Llc | Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use |
| US10568898B2 (en) | 2013-08-13 | 2020-02-25 | Northwestern University | Lipophilic nanoparticles for drug delivery |
| KR102507624B1 (ko) | 2013-11-22 | 2023-03-09 | 미나 테라퓨틱스 리미티드 | Ｃ/ｅｂｐ 알파 짧은 활성화 ｒｎａ 조성물 및 사용 방법 |
| MX389323B (es) * | 2013-12-03 | 2025-03-20 | Univ Northwestern | Particulas liposomales, metodos para elaborarlas y sus usos. |
| US10513710B2 (en) | 2014-04-18 | 2019-12-24 | University Of Massachusetts | Exosomal loading using hydrophobically modified oligonucleotides |
| US10434064B2 (en) | 2014-06-04 | 2019-10-08 | Exicure, Inc. | Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications |
| EP3183007B1 (en) | 2014-08-19 | 2020-06-17 | Northwestern University | Protein/oligonucleotide core-shell nanoparticle therapeutics |
| JP6741673B2 (ja) | 2014-10-06 | 2020-08-19 | イグジキュア, インコーポレーテッドExicure, Inc. | 抗ｔｎｆ化合物 |
| CA2968531A1 (en) | 2014-11-21 | 2016-05-26 | Northwestern University | The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates |
| EP3247796A4 (en) | 2015-01-14 | 2018-07-11 | Exicure, Inc. | Nucleic acid nanostructures with core motifs |
| AU2016334086B2 (en) * | 2015-10-07 | 2022-10-20 | Cormedix Inc. | Skin-penetrating formulation of taurolidine |
| US10967072B2 (en) | 2016-04-27 | 2021-04-06 | Northwestern University | Short interfering RNA templated lipoprotein particles (siRNA-TLP) |
| KR102617833B1 (ko) | 2016-05-06 | 2023-12-27 | 엑시큐어 오퍼레이팅 컴퍼니 | 인터류킨 17 수용체 mRNA의 특이적 녹다운을 위한 안티센스 올리고뉴클레오티드 (ASO)를 제시하는 리포좀성 구형 핵산 (SNA) 구축물 |
| WO2018039629A2 (en) | 2016-08-25 | 2018-03-01 | Northwestern University | Micellar spherical nucleic acids from thermoresponsive, traceless templates |
| 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 |
| KR20200028997A (ko) | 2017-07-13 | 2020-03-17 | 노오쓰웨스턴 유니버시티 | 올리고뉴클레오타이드-작용화된 금속-유기 프레임워크 나노입자를 제조하는 일반적이고 직접적인 방법 |
| AU2018330495A1 (en) | 2017-09-08 | 2020-03-26 | Mina Therapeutics Limited | Stabilized hnf4a sarna compositions and methods of use |
| EP3679140B1 (en) | 2017-09-08 | 2022-11-16 | MiNA Therapeutics Limited | Stabilized cebpa sarna compositions and methods of use |
| US11566246B2 (en) | 2018-04-12 | 2023-01-31 | Mina Therapeutics Limited | SIRT1-saRNA compositions and methods of use |
| WO2020208361A1 (en) | 2019-04-12 | 2020-10-15 | Mina Therapeutics Limited | Sirt1-sarna compositions and methods of use |
| JP7463410B2 (ja) * | 2019-07-02 | 2024-04-08 | アルゴノート アールエヌエー リミテッド | アポリポタンパク質ｂアンタゴニスト |
| 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 |
| EP4634388A1 (en) | 2022-12-14 | 2025-10-22 | 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 |
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| CA2329130A1 (en) * | 1998-05-21 | 1999-11-25 | Isis Pharmaceuticals Inc. | Compositions and methods for non-parenteral delivery of oligonucleotides |
| EP1156812A4 (en) | 1999-02-23 | 2004-09-29 | Isis Pharmaceuticals Inc | MULTIPARTICULAR FORMULATION |
| GB9930533D0 (en) * | 1999-12-23 | 2000-02-16 | Mitsubishi Tokyo Pharm Inc | Nucleic acid delivery |
| US20070026394A1 (en) * | 2000-02-11 | 2007-02-01 | Lawrence Blatt | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies |
| RU2322500C2 (ru) * | 2000-12-01 | 2008-04-20 | Макс-Планк-Гезелльшафт Цур Фердерунг Дер Виссеншафтен Е.Ф. | Малые молекулы рнк, опосредующие интерференцию рнк |
| 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 |
| US7923547B2 (en) * | 2002-09-05 | 2011-04-12 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| DK2284266T3 (da) * | 2002-11-14 | 2014-01-13 | Thermo Fisher Scient Biosciences Inc | sIRNA-MOLEKYLE MOD TP53 |
| 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 |
| KR101407707B1 (ko) * | 2006-04-03 | 2014-06-19 | 산타리스 팔마 에이/에스 | Ａｎｔｉ-ｍｉｒｎａ 안티센스 올리고뉴클레오타이드를 함유하는 약학적 조성물 |
| 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 EP EP09724197A patent/EP2265289A2/en not_active Withdrawn
- 2009-03-26 WO PCT/US2009/038437 patent/WO2009120887A2/en not_active Ceased
- 2009-03-26 US US12/412,206 patent/US8148344B2/en active Active
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