JPWO2022099159A5 - - Google Patents

Info

Publication number

JPWO2022099159A5

JPWO2022099159A5 JP2023527250A JP2023527250A JPWO2022099159A5 JP WO2022099159 A5 JPWO2022099159 A5 JP WO2022099159A5 JP 2023527250 A JP2023527250 A JP 2023527250A JP 2023527250 A JP2023527250 A JP 2023527250A JP WO2022099159 A5 JPWO2022099159 A5 JP WO2022099159A5

Authority

JP

Japan

Prior art keywords

oligonucleotide

nucleic acid

target nucleic

composition

oligonucleotides

Prior art date

2024-11-20

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.)

Pending

Links

• Espacenet
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• 108091034117 Oligonucleotide Proteins 0.000 claims 107
• 108020004707 nucleic acids Proteins 0.000 claims 48
• 102000039446 nucleic acids Human genes 0.000 claims 48
• 150000007523 nucleic acids Chemical class 0.000 claims 48
• OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 42
• 239000000203 mixture Substances 0.000 claims 41
• 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 31
• 239000002126 C01EB10 - Adenosine Substances 0.000 claims 21
• 229960005305 adenosine Drugs 0.000 claims 21
• 230000004048 modification Effects 0.000 claims 20
• 238000012986 modification Methods 0.000 claims 20
• 238000000034 method Methods 0.000 claims 14
• 230000000295 complement effect Effects 0.000 claims 13
• 235000000346 sugar Nutrition 0.000 claims 12
• 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims 11
• 102000055025 Adenosine deaminases Human genes 0.000 claims 11
• 150000008163 sugars Chemical class 0.000 claims 5
• 239000008186 active pharmaceutical agent Substances 0.000 claims 4
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
• 239000003446 ligand Substances 0.000 claims 4
• 239000002777 nucleoside Substances 0.000 claims 4
• 150000008300 phosphoramidites Chemical class 0.000 claims 4
• 150000003839 salts Chemical class 0.000 claims 3
• 125000001931 aliphatic group Chemical group 0.000 claims 2
• 201000010099 disease Diseases 0.000 claims 2
• 208000035475 disorder Diseases 0.000 claims 2
• 230000001788 irregular Effects 0.000 claims 2
• 150000003833 nucleoside derivatives Chemical class 0.000 claims 2
• 125000003835 nucleoside group Chemical group 0.000 claims 2
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
• 101150075175 Asgr1 gene Proteins 0.000 claims 1
• OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 1
• 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 1
• 229910019142 PO4 Inorganic materials 0.000 claims 1
• OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 1
• 239000002253 acid Substances 0.000 claims 1
• 125000002619 bicyclic group Chemical group 0.000 claims 1
• 150000001875 compounds Chemical class 0.000 claims 1
• 230000008878 coupling Effects 0.000 claims 1
• 238000010168 coupling process Methods 0.000 claims 1
• 238000005859 coupling reaction Methods 0.000 claims 1
• 239000003937 drug carrier Substances 0.000 claims 1
• 125000005842 heteroatom Chemical group 0.000 claims 1
• 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 1
• 125000002950 monocyclic group Chemical group 0.000 claims 1
• 230000035772 mutation Effects 0.000 claims 1
• 239000002773 nucleotide Substances 0.000 claims 1
• 239000008194 pharmaceutical composition Substances 0.000 claims 1
• 239000010452 phosphate Substances 0.000 claims 1
• 229910052698 phosphorus Inorganic materials 0.000 claims 1
• 239000011574 phosphorus Substances 0.000 claims 1
• 125000003367 polycyclic group Chemical group 0.000 claims 1