JP2018510621A5 - - Google Patents

Info

Publication number

JP2018510621A5

JP2018510621A5 JP2017542133A JP2017542133A JP2018510621A5 JP 2018510621 A5 JP2018510621 A5 JP 2018510621A5 JP 2017542133 A JP2017542133 A JP 2017542133A JP 2017542133 A JP2017542133 A JP 2017542133A JP 2018510621 A5 JP2018510621 A5 JP 2018510621A5

Authority

JP

Japan

Prior art keywords

double

rnai agent

stranded rnai

pnpla3

stranded

Prior art date

2016-02-11

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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• 229920002477 rna polymer Polymers 0.000 claims 43
• 239000003795 chemical substances by application Substances 0.000 claims 41
• 125000003729 nucleotide group Chemical group 0.000 claims 14
• 239000002773 nucleotide Substances 0.000 claims 12
• 102100031251 1-acylglycerol-3-phosphate O-acyltransferase PNPLA3 Human genes 0.000 claims 9
• 101001129184 Homo sapiens 1-acylglycerol-3-phosphate O-acyltransferase PNPLA3 Proteins 0.000 claims 9
• 230000004048 modification Effects 0.000 claims 8
• 238000012986 modification Methods 0.000 claims 8
• 230000000692 anti-sense effect Effects 0.000 claims 7
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 7
• 239000008194 pharmaceutical composition Substances 0.000 claims 7
• 108091081021 Sense strand Proteins 0.000 claims 5
• 239000003446 ligand Substances 0.000 claims 5
• 201000010099 disease Diseases 0.000 claims 4
• 230000002401 inhibitory effect Effects 0.000 claims 4
• 208000008338 non-alcoholic fatty liver disease Diseases 0.000 claims 4
• 101100244213 Homo sapiens PNPLA3 gene Proteins 0.000 claims 3
• 239000000872 buffer Substances 0.000 claims 3
• 238000000034 method Methods 0.000 claims 3
• 108091034117 Oligonucleotide Proteins 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
• 208000035475 disorder Diseases 0.000 claims 2
• 206010053219 non-alcoholic steatohepatitis Diseases 0.000 claims 2
• 239000002953 phosphate buffered saline Substances 0.000 claims 2
• 230000008410 smoothened signaling pathway Effects 0.000 claims 2
• FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
• QTBSBXVTEAMEQO-UHFFFAOYSA-M Acetate Chemical compound CC([O-])=O QTBSBXVTEAMEQO-UHFFFAOYSA-M 0.000 claims 1
• BVKZGUZCCUSVTD-UHFFFAOYSA-L Carbonate Chemical compound [O-]C([O-])=O BVKZGUZCCUSVTD-UHFFFAOYSA-L 0.000 claims 1
• KRKNYBCHXYNGOX-UHFFFAOYSA-K Citrate Chemical compound [O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O KRKNYBCHXYNGOX-UHFFFAOYSA-K 0.000 claims 1
• 208000004930 Fatty Liver Diseases 0.000 claims 1
• 206010019708 Hepatic steatosis Diseases 0.000 claims 1
• 229910019142 PO4 Inorganic materials 0.000 claims 1
• FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 1
• 239000000427 antigen Substances 0.000 claims 1
• 108091007433 antigens Proteins 0.000 claims 1
• 102000036639 antigens Human genes 0.000 claims 1
• 239000007853 buffer solution Substances 0.000 claims 1
• 239000008366 buffered solution Substances 0.000 claims 1
• 230000015556 catabolic process Effects 0.000 claims 1
• 230000000295 complement effect Effects 0.000 claims 1
• 238000006731 degradation reaction Methods 0.000 claims 1
• 208000010706 fatty liver disease Diseases 0.000 claims 1
• 239000012634 fragment Substances 0.000 claims 1
• 150000002632 lipids Chemical class 0.000 claims 1
• 108020004999 messenger RNA Proteins 0.000 claims 1
• YACKEPLHDIMKIO-UHFFFAOYSA-N methylphosphonic acid Chemical compound CP(O)(O)=O YACKEPLHDIMKIO-UHFFFAOYSA-N 0.000 claims 1
• NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
• 239000010452 phosphate Substances 0.000 claims 1
• 108060006613 prolamin Proteins 0.000 claims 1
• 239000011780 sodium chloride Substances 0.000 claims 1
• 231100000240 steatosis hepatitis Toxicity 0.000 claims 1
• 208000024891 symptom Diseases 0.000 claims 1
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
• XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 1
• 230000003442 weekly effect Effects 0.000 claims 1