JP2016521753A5 - - Google Patents
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- JP2016521753A5 JP2016521753A5 JP2016519662A JP2016519662A JP2016521753A5 JP 2016521753 A5 JP2016521753 A5 JP 2016521753A5 JP 2016519662 A JP2016519662 A JP 2016519662A JP 2016519662 A JP2016519662 A JP 2016519662A JP 2016521753 A5 JP2016521753 A5 JP 2016521753A5
- Authority
- JP
- Japan
- Prior art keywords
- disease
- composition
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- treatment
- therapeutic
- 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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- 239000000203 mixture Substances 0.000 claims 38
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 32
- 108091034117 Oligonucleotide Proteins 0.000 claims 28
- 230000001225 therapeutic effect Effects 0.000 claims 25
- 201000010099 disease Diseases 0.000 claims 15
- 208000035475 disorder Diseases 0.000 claims 15
- 150000007523 nucleic acids Chemical class 0.000 claims 13
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 102000039446 nucleic acids Human genes 0.000 claims 12
- 102000004169 proteins and genes Human genes 0.000 claims 10
- 108090000623 proteins and genes Proteins 0.000 claims 9
- 108020004999 messenger RNA Proteins 0.000 claims 8
- 230000000692 anti-sense effect Effects 0.000 claims 7
- 230000002018 overexpression Effects 0.000 claims 6
- 230000009452 underexpressoin Effects 0.000 claims 6
- 108091005461 Nucleic proteins Proteins 0.000 claims 5
- 239000002679 microRNA Substances 0.000 claims 5
- 239000002773 nucleotide Substances 0.000 claims 5
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- 208000024172 Cardiovascular disease Diseases 0.000 claims 4
- 208000012902 Nervous system disease Diseases 0.000 claims 4
- 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 4
- 239000000074 antisense oligonucleotide Substances 0.000 claims 4
- 238000012230 antisense oligonucleotides Methods 0.000 claims 4
- 208000027866 inflammatory disease Diseases 0.000 claims 4
- 208000030159 metabolic disease Diseases 0.000 claims 4
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 3
- 206010048643 Hypereosinophilic syndrome Diseases 0.000 claims 3
- 230000001594 aberrant effect Effects 0.000 claims 3
- 230000002159 abnormal effect Effects 0.000 claims 3
- 230000005284 excitation Effects 0.000 claims 3
- 230000014509 gene expression Effects 0.000 claims 3
- 238000001727 in vivo Methods 0.000 claims 3
- 239000002777 nucleoside Substances 0.000 claims 3
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 3
- VEXZGXHMUGYJMC-UHFFFAOYSA-M Chloride anion Chemical compound [Cl-] VEXZGXHMUGYJMC-UHFFFAOYSA-M 0.000 claims 2
- 208000035473 Communicable disease Diseases 0.000 claims 2
- 108020004414 DNA Proteins 0.000 claims 2
- 206010028980 Neoplasm Diseases 0.000 claims 2
- 208000025966 Neurological disease Diseases 0.000 claims 2
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 2
- 201000011510 cancer Diseases 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 208000016097 disease of metabolism Diseases 0.000 claims 2
- NAGJZTKCGNOGPW-UHFFFAOYSA-N dithiophosphoric acid Chemical class OP(O)(S)=S NAGJZTKCGNOGPW-UHFFFAOYSA-N 0.000 claims 2
- 208000030533 eye disease Diseases 0.000 claims 2
- 230000009368 gene silencing by RNA Effects 0.000 claims 2
- 208000026278 immune system disease Diseases 0.000 claims 2
- 208000015181 infectious disease Diseases 0.000 claims 2
- 108091070501 miRNA Proteins 0.000 claims 2
- 125000001147 pentyl group Chemical group C(CCCC)* 0.000 claims 2
- 150000008039 phosphoramides Chemical class 0.000 claims 2
- 230000002062 proliferating effect Effects 0.000 claims 2
- MWWATHDPGQKSAR-UHFFFAOYSA-N propyne Chemical compound CC#C MWWATHDPGQKSAR-UHFFFAOYSA-N 0.000 claims 2
- 208000017520 skin disease Diseases 0.000 claims 2
- 238000012385 systemic delivery Methods 0.000 claims 2
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 208000030852 Parasitic disease Diseases 0.000 claims 1
- 108091081024 Start codon Proteins 0.000 claims 1
- 108091036066 Three prime untranslated region Proteins 0.000 claims 1
- 208000036142 Viral infection Diseases 0.000 claims 1
- 230000033228 biological regulation Effects 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 208000032839 leukemia Diseases 0.000 claims 1
- 208000037819 metastatic cancer Diseases 0.000 claims 1
- 208000011575 metastatic malignant neoplasm Diseases 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 125000003835 nucleoside group Chemical group 0.000 claims 1
- 230000009385 viral infection Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361834383P | 2013-06-12 | 2013-06-12 | |
| US61/834,383 | 2013-06-12 | ||
| PCT/US2014/042202 WO2014201306A1 (en) | 2013-06-12 | 2014-06-12 | Systemic in vivo delivery of oligonucleotides |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019088413A Division JP2019135259A (ja) | 2013-06-12 | 2019-05-08 | オリゴヌクレオチドの全身性イン−ビボ送達 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016521753A JP2016521753A (ja) | 2016-07-25 |
| JP2016521753A5 true JP2016521753A5 (cg-RX-API-DMAC7.html) | 2021-07-29 |
| JP7011389B2 JP7011389B2 (ja) | 2022-01-26 |
Family
ID=52022784
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016519662A Active JP7011389B2 (ja) | 2013-06-12 | 2014-06-12 | オリゴヌクレオチドの全身性イン－ビボ送達 |
| JP2019088413A Pending JP2019135259A (ja) | 2013-06-12 | 2019-05-08 | オリゴヌクレオチドの全身性イン−ビボ送達 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019088413A Pending JP2019135259A (ja) | 2013-06-12 | 2019-05-08 | オリゴヌクレオチドの全身性イン−ビボ送達 |
Country Status (5)
| Country | Link |
|---|---|
| US (3) | US20160367587A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3007705A4 (cg-RX-API-DMAC7.html) |
| JP (2) | JP7011389B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2951816A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2014201306A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 十分に安定化された非対称ｓｉｒｎａ |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| CA3008289C (en) | 2015-12-16 | 2023-10-31 | The Walter And Eliza Hall Institute Of Medical Research | Inhibition of cytokine-induced sh2 protein in nk cells |
| JOP20200228A1 (ar) | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| US20210371855A1 (en) * | 2018-06-15 | 2021-12-02 | Ideaya Biosciences, Inc. | Methods of inhibiting proliferative cells |
| BR112021002440A2 (pt) | 2018-08-10 | 2021-05-04 | University Of Massachusetts | oligonucleotídeos modificados visando snps |
| WO2020041769A1 (en) | 2018-08-23 | 2020-02-27 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| US11820985B2 (en) | 2019-03-26 | 2023-11-21 | University Of Massachusetts | Modified oligonucleotides with increased stability |
| AU2020252374A1 (en) * | 2019-04-03 | 2021-11-11 | Bristol-Myers Squibb Company | ANGPTL2 antisense oligonucleotides and uses thereof |
| AU2020329155A1 (en) | 2019-08-09 | 2022-03-10 | University Of Massachusetts | Chemically modified oligonucleotides targeting SNPs |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| WO2021132648A1 (ja) * | 2019-12-27 | 2021-07-01 | ルクサナバイオテク株式会社 | Ｒｅｃｑｌの発現を阻害するアンチセンスオリゴヌクレオチド及びその用途 |
| JP2023509979A (ja) * | 2020-01-11 | 2023-03-10 | オンコイミューニン，インコーポレイティド | オリゴヌクレオチドの標的化及び局所インビボ送達 |
| CA3172168A1 (en) | 2020-03-26 | 2021-09-30 | Anastasia Khvorova | Synthesis of modified oligonucleotides with increased stability |
| WO2025171411A1 (en) | 2024-02-09 | 2025-08-14 | Herophilus, Inc. | Compositions and methods related to modulating macrophage migration inhibitory factor (mif)-cd74 signaling and related treatments for neuroinflammatory conditions |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6893868B2 (en) * | 1997-02-20 | 2005-05-17 | Onco Immunin, Inc. | Homo-doubly labeled compositions for the detection of enzyme activity in biological samples |
| US6133032A (en) * | 1999-09-09 | 2000-10-17 | Isis Pharmaceutical Inc. | Antisense modulation of PI3 kinase p110 beta expression |
| WO2002059137A1 (en) * | 2000-11-03 | 2002-08-01 | Isis Pharmaceuticals, Inc. | Nuclease-based method for detecting and quantitating oligonucleotides |
| US7060809B2 (en) * | 2001-09-04 | 2006-06-13 | Exiqon A/S | LNA compositions and uses thereof |
| EP1501930A2 (en) * | 2002-04-05 | 2005-02-02 | Santaris Pharma A/S | Oligomeric compounds for the modulation hif-1alpha expression |
| EP1532271A4 (en) * | 2002-06-12 | 2006-10-18 | Ambion Inc | METHODS AND COMPOSITIONS RELATED TO POLYPEPTIDES WITH RNASE III DOMAINS THAT TRANSFER RNA INTERFERENCE |
| US20050142581A1 (en) * | 2003-09-04 | 2005-06-30 | Griffey Richard H. | Microrna as ligands and target molecules |
| US20080199960A1 (en) * | 2004-05-13 | 2008-08-21 | Juliano Rudolph L | Methods for the Delivery of Oligomeric Compounds |
| WO2008134593A1 (en) * | 2007-04-25 | 2008-11-06 | President And Fellows Of Harvard College | Molecular circuits |
| RU2010101882A (ru) * | 2007-06-22 | 2011-07-27 | Киджин Н.В. (Nl) | Направленная нуклеотидная замена при применении улучшенных модифицированных олигонуклеотидов |
| US20100280098A1 (en) * | 2007-10-05 | 2010-11-04 | Juliano Rudolph L | Receptor targeted oligonucleotides |
| US8911948B2 (en) * | 2008-04-30 | 2014-12-16 | Integrated Dna Technologies, Inc. | RNase H-based assays utilizing modified RNA monomers |
| US20120087949A1 (en) * | 2008-12-19 | 2012-04-12 | University Of Florida Research Foundation, Inc. | Oligonucleotide micelles |
| AU2010328104B2 (en) * | 2009-12-09 | 2014-10-30 | Nitto Denko Corporation | Modulation of hsp47 expression |
| JP2015502365A (ja) * | 2011-12-12 | 2015-01-22 | オンコイミューニン，インコーポレイティド | オリゴヌクレオチドのイン−ビボ送達 |
-
2014
- 2014-06-12 CA CA2951816A patent/CA2951816A1/en not_active Abandoned
- 2014-06-12 JP JP2016519662A patent/JP7011389B2/ja active Active
- 2014-06-12 WO PCT/US2014/042202 patent/WO2014201306A1/en not_active Ceased
- 2014-06-12 US US14/897,872 patent/US20160367587A1/en not_active Abandoned
- 2014-06-12 EP EP14811033.1A patent/EP3007705A4/en not_active Withdrawn
-
2018
- 2018-10-29 US US16/174,091 patent/US20190183918A1/en not_active Abandoned
-
2019
- 2019-05-08 JP JP2019088413A patent/JP2019135259A/ja active Pending
-
2021
- 2021-05-04 US US17/307,350 patent/US20220033815A1/en not_active Abandoned
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