JP2019508481A5 - - Google Patents

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Publication number

JP2019508481A5

JP2019508481A5 JP2018550872A JP2018550872A JP2019508481A5 JP 2019508481 A5 JP2019508481 A5 JP 2019508481A5 JP 2018550872 A JP2018550872 A JP 2018550872A JP 2018550872 A JP2018550872 A JP 2018550872A JP 2019508481 A5 JP2019508481 A5 JP 2019508481A5

Authority

JP

Japan

Prior art keywords

dccpm

peptide

amino acids

cpa

bac

Prior art date

2016-12-21

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

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• 108090000765 processed proteins & peptides Proteins 0.000 claims description 29
• 150000001413 amino acids Chemical class 0.000 claims description 11
• 238000000034 method Methods 0.000 claims description 9
• 238000004132 cross linking Methods 0.000 claims description 8
• 150000001875 compounds Chemical class 0.000 claims description 6
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 6
• 230000001588 bifunctional effect Effects 0.000 claims description 4
• 239000003814 drug Substances 0.000 claims description 4
• 229940079593 drug Drugs 0.000 claims description 4
• 230000000087 stabilizing effect Effects 0.000 claims description 4
• 230000001737 promoting effect Effects 0.000 claims description 2
• 108091034117 Oligonucleotide Proteins 0.000 claims 9
• 239000000074 antisense oligonucleotide Substances 0.000 claims 4
• 238000012230 antisense oligonucleotides Methods 0.000 claims 4
• 230000007935 neutral effect Effects 0.000 claims 3
• 108090000623 proteins and genes Proteins 0.000 claims 3
• RTIIDBURLHHYRK-ZETCQYMHSA-N (2S)-2-(pent-1-enylamino)propanoic acid Chemical compound CCCC=CN[C@@H](C)C(O)=O RTIIDBURLHHYRK-ZETCQYMHSA-N 0.000 claims 2
• 239000004215 Carbon black (E152) Substances 0.000 claims 2
• 206010028980 Neoplasm Diseases 0.000 claims 2
• 108091093037 Peptide nucleic acid Proteins 0.000 claims 2
• 208000036142 Viral infection Diseases 0.000 claims 2
• 201000011510 cancer Diseases 0.000 claims 2
• 230000008878 coupling Effects 0.000 claims 2
• 238000010168 coupling process Methods 0.000 claims 2
• 238000005859 coupling reaction Methods 0.000 claims 2
• 208000016097 disease of metabolism Diseases 0.000 claims 2
• 229930195733 hydrocarbon Natural products 0.000 claims 2
• 150000002430 hydrocarbons Chemical class 0.000 claims 2
• 208000030159 metabolic disease Diseases 0.000 claims 2
• 208000015122 neurodegenerative disease Diseases 0.000 claims 2
• 208000018360 neuromuscular disease Diseases 0.000 claims 2
• 239000000126 substance Substances 0.000 claims 2
• JJAHTWIKCUJRDK-UHFFFAOYSA-N succinimidyl 4-(N-maleimidomethyl)cyclohexane-1-carboxylate Chemical compound C1CC(CN2C(C=CC2=O)=O)CCC1C(=O)ON1C(=O)CCC1=O JJAHTWIKCUJRDK-UHFFFAOYSA-N 0.000 claims 2
• 230000009385 viral infection Effects 0.000 claims 2
• RTIIDBURLHHYRK-SSDOTTSWSA-N C(=CCCC)N[C@H](C)C(=O)O Chemical compound C(=CCCC)N[C@H](C)C(=O)O RTIIDBURLHHYRK-SSDOTTSWSA-N 0.000 claims 1
• UOAXMMISMWRAMK-SNVBAGLBSA-N C(=CCCCCCC)N[C@H](C)C(=O)O Chemical compound C(=CCCCCCC)N[C@H](C)C(=O)O UOAXMMISMWRAMK-SNVBAGLBSA-N 0.000 claims 1
• 239000004971 Cross linker Substances 0.000 claims 1
• 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
• 108010069091 Dystrophin 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
• 150000001336 alkenes Chemical class 0.000 claims 1
• 230000005540 biological transmission Effects 0.000 claims 1
• 239000003795 chemical substances by application Substances 0.000 claims 1
• 201000010099 disease Diseases 0.000 claims 1
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
• 238000001990 intravenous administration Methods 0.000 claims 1
• 125000005439 maleimidyl group Chemical group C1(C=CC(N1*)=O)=O 0.000 claims 1
• 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
• 230000004048 modification Effects 0.000 claims 1
• 238000012986 modification Methods 0.000 claims 1
• JRZJOMJEPLMPRA-UHFFFAOYSA-N olefin Natural products CCCCCCCC=C JRZJOMJEPLMPRA-UHFFFAOYSA-N 0.000 claims 1
• 238000007911 parenteral administration Methods 0.000 claims 1
• 239000000546 pharmaceutical excipient Substances 0.000 claims 1
• NMHMNPHRMNGLLB-UHFFFAOYSA-N phloretic acid Chemical compound OC(=O)CCC1=CC=C(O)C=C1 NMHMNPHRMNGLLB-UHFFFAOYSA-N 0.000 claims 1
• 230000008685 targeting Effects 0.000 claims 1
• 238000011200 topical administration Methods 0.000 claims 1
• 230000000975 bioactive effect Effects 0.000 description 2
• 230000000149 penetrating effect Effects 0.000 description 1
• 230000035515 penetration Effects 0.000 description 1