JP2020521491A5 - - Google Patents

Info

Publication number

JP2020521491A5

JP2020521491A5 JP2019566236A JP2019566236A JP2020521491A5 JP 2020521491 A5 JP2020521491 A5 JP 2020521491A5 JP 2019566236 A JP2019566236 A JP 2019566236A JP 2019566236 A JP2019566236 A JP 2019566236A JP 2020521491 A5 JP2020521491 A5 JP 2020521491A5

Authority

JP

Japan

Prior art keywords

pharmaceutically acceptable

pharmaceutical composition

oligonucleotide

nucleoside

acceptable salt

Prior art date

2018-05-30

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
• Global Dossier
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• 108091034117 Oligonucleotide Proteins 0.000 claims 19
• 239000002777 nucleoside Substances 0.000 claims 19
• 239000008194 pharmaceutical composition Substances 0.000 claims 12
• 150000003839 salts Chemical class 0.000 claims 11
• 150000003833 nucleoside derivatives Chemical class 0.000 claims 10
• 239000000074 antisense oligonucleotide Substances 0.000 claims 9
• 238000012230 antisense oligonucleotides Methods 0.000 claims 9
• LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 7
• 125000003835 nucleoside group Chemical group 0.000 claims 7
• 208000002780 macular degeneration Diseases 0.000 claims 4
• 239000002773 nucleotide Substances 0.000 claims 4
• 125000003729 nucleotide group Chemical group 0.000 claims 4
• 208000008069 Geographic Atrophy Diseases 0.000 claims 3
• 101001041393 Homo sapiens Serine protease HTRA1 Proteins 0.000 claims 3
• 102100021119 Serine protease HTRA1 Human genes 0.000 claims 3
• 206010064930 age-related macular degeneration Diseases 0.000 claims 3
• 239000003085 diluting agent Substances 0.000 claims 3
• 239000003814 drug Substances 0.000 claims 3
• 208000011325 dry age related macular degeneration Diseases 0.000 claims 3
• 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 1
• -1 LNA nucleoside Chemical class 0.000 claims 1
• RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 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
• 239000002671 adjuvant Substances 0.000 claims 1
• OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical group NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 1
• 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 1
• 238000000338 in vitro Methods 0.000 claims 1
• 238000001727 in vivo Methods 0.000 claims 1
• 238000004519 manufacturing process Methods 0.000 claims 1
• 238000000034 method Methods 0.000 claims 1
• 150000007523 nucleic acids Chemical group 0.000 claims 1
• 239000002953 phosphate buffered saline Substances 0.000 claims 1
• 230000002265 prevention Effects 0.000 claims 1
• 230000001105 regulatory effect Effects 0.000 claims 1
• 159000000000 sodium salts Chemical group 0.000 claims 1
• 239000002904 solvent Substances 0.000 claims 1
• 230000008685 targeting Effects 0.000 claims 1