JP2020096629A5 - - Google Patents

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Publication number

JP2020096629A5

JP2020096629A5 JP2020025372A JP2020025372A JP2020096629A5 JP 2020096629 A5 JP2020096629 A5 JP 2020096629A5 JP 2020025372 A JP2020025372 A JP 2020025372A JP 2020025372 A JP2020025372 A JP 2020025372A JP 2020096629 A5 JP2020096629 A5 JP 2020096629A5

Authority

JP

Japan

Prior art keywords

modified oligonucleotide

seq

compound

modified

compound according

Prior art date

2020-02-18

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

• Espacenet
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• 150000001875 compounds Chemical class 0.000 claims 36
• 108091034117 Oligonucleotide Proteins 0.000 claims 35
• 239000002777 nucleoside Substances 0.000 claims 21
• 125000003835 nucleoside group Chemical group 0.000 claims 9
• 239000000203 mixture Substances 0.000 claims 5
• 230000000295 complement effect Effects 0.000 claims 4
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
• 108020004707 nucleic acids Proteins 0.000 claims 4
• 102000039446 nucleic acids Human genes 0.000 claims 4
• 150000007523 nucleic acids Chemical class 0.000 claims 4
• 150000003833 nucleoside derivatives Chemical class 0.000 claims 4
• 239000008194 pharmaceutical composition Substances 0.000 claims 3
• LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 2
• 208000005189 Embolism Diseases 0.000 claims 2
• 206010019860 Hereditary angioedema 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
• 125000002619 bicyclic group Chemical group 0.000 claims 2
• OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 2
• 239000003085 diluting agent Substances 0.000 claims 2
• LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical group P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims 2
• 201000010099 disease Diseases 0.000 claims 2
• 208000035475 disorder Diseases 0.000 claims 2
• 208000027866 inflammatory disease Diseases 0.000 claims 2
• 239000002953 phosphate buffered saline Substances 0.000 claims 2
• 208000028185 Angioedema Diseases 0.000 claims 1
• 206010048962 Brain oedema Diseases 0.000 claims 1
• 206010051055 Deep vein thrombosis Diseases 0.000 claims 1
• 206010052139 Eye oedema Diseases 0.000 claims 1
• 101001081555 Homo sapiens Plasma protease C1 inhibitor Proteins 0.000 claims 1
• 206010061216 Infarction Diseases 0.000 claims 1
• 208000001344 Macular Edema Diseases 0.000 claims 1
• 206010025415 Macular oedema 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
• 206010030113 Oedema Diseases 0.000 claims 1
• 102100027637 Plasma protease C1 inhibitor Human genes 0.000 claims 1
• 208000010378 Pulmonary Embolism Diseases 0.000 claims 1
• 208000006011 Stroke Diseases 0.000 claims 1
• 208000001435 Thromboembolism Diseases 0.000 claims 1
• 208000007536 Thrombosis Diseases 0.000 claims 1
• 206010047249 Venous thrombosis Diseases 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 Chemical class 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
• 208000006752 brain edema Diseases 0.000 claims 1
• 229940104302 cytosine Drugs 0.000 claims 1
• 239000005549 deoxyribonucleoside Substances 0.000 claims 1
• 239000003937 drug carrier Substances 0.000 claims 1
• 210000000744 eyelid Anatomy 0.000 claims 1
• 230000007574 infarction Effects 0.000 claims 1
• 239000003446 ligand Substances 0.000 claims 1
• 201000010230 macular retinal edema Diseases 0.000 claims 1
• 208000010125 myocardial infarction Diseases 0.000 claims 1
• 230000001575 pathological effect Effects 0.000 claims 1
• -1 phosphorothioate nucleoside Chemical class 0.000 claims 1
• 150000003839 salts Chemical class 0.000 claims 1
• 239000000126 substance Substances 0.000 claims 1
• 238000006467 substitution reaction Methods 0.000 claims 1
• 230000008961 swelling Effects 0.000 claims 1