JPWO2020238758A5 - - Google Patents

Info

Publication number

JPWO2020238758A5

JPWO2020238758A5 JP2021569555A JP2021569555A JPWO2020238758A5 JP WO2020238758 A5 JPWO2020238758 A5 JP WO2020238758A5 JP 2021569555 A JP2021569555 A JP 2021569555A JP 2021569555 A JP2021569555 A JP 2021569555A JP WO2020238758 A5 JPWO2020238758 A5 JP WO2020238758A5

Authority

JP

Japan

Prior art keywords

nucleotide sequence

seq

nucleotide

sirna

group

Prior art date

2023-05-29

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

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• 125000003729 nucleotide group Chemical group 0.000 claims 275
• 239000002773 nucleotide Substances 0.000 claims 254
• 108020004459 Small interfering RNA Proteins 0.000 claims 71
• 230000000692 anti-sense effect Effects 0.000 claims 42
• 125000000008 (C1-C10) alkyl group Chemical group 0.000 claims 36
• 108091081021 Sense strand Proteins 0.000 claims 30
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 20
• 125000000217 alkyl group Chemical group 0.000 claims 16
• 230000000295 complement effect Effects 0.000 claims 12
• 125000005037 alkyl phenyl group Chemical group 0.000 claims 10
• 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 10
• 125000004432 carbon atom Chemical group C* 0.000 claims 4
• 108020004635 Complementary DNA Proteins 0.000 claims 3
• 239000000203 mixture Substances 0.000 claims 3
• 239000008194 pharmaceutical composition Substances 0.000 claims 3
• 229910052717 sulfur Inorganic materials 0.000 claims 3
• 230000008685 targeting Effects 0.000 claims 3
• 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 2
• 125000001313 C5-C10 heteroaryl group Chemical group 0.000 claims 2
• 125000000041 C6-C10 aryl group Chemical group 0.000 claims 2
• RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical group OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 2
• 125000004450 alkenylene group Chemical group 0.000 claims 2
• 125000002947 alkylene group Chemical group 0.000 claims 2
• 125000004419 alkynylene group Chemical group 0.000 claims 2
• 125000000732 arylene group Chemical group 0.000 claims 2
• 125000004429 atom Chemical group 0.000 claims 2
• 125000004093 cyano group Chemical group *C#N 0.000 claims 2
• 239000003937 drug carrier Substances 0.000 claims 2
• 125000001188 haloalkyl group Chemical group 0.000 claims 2
• 125000005843 halogen group Chemical group 0.000 claims 2
• 125000005549 heteroarylene group Chemical group 0.000 claims 2
• 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims 2
• 229910052757 nitrogen Inorganic materials 0.000 claims 2
• 229910052760 oxygen Inorganic materials 0.000 claims 2
• 125000001424 substituent group Chemical group 0.000 claims 2
• 102000005427 Asialoglycoprotein Receptor Human genes 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
• 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
• 208000001435 Thromboembolism Diseases 0.000 claims 1
• 125000003545 alkoxy group Chemical group 0.000 claims 1
• WQZGKKKJIJFFOK-PHYPRBDBSA-N alpha-D-galactose Chemical compound OC[C@H]1O[C@H](O)[C@H](O)[C@@H](O)[C@H]1O WQZGKKKJIJFFOK-PHYPRBDBSA-N 0.000 claims 1
• 108010006523 asialoglycoprotein receptor Proteins 0.000 claims 1
• 230000027455 binding Effects 0.000 claims 1
• 238000009739 binding Methods 0.000 claims 1
• 210000004027 cell Anatomy 0.000 claims 1
• 229930182830 galactose Natural products 0.000 claims 1
• 230000014509 gene expression Effects 0.000 claims 1
• 210000003494 hepatocyte Anatomy 0.000 claims 1
• 208000027866 inflammatory disease Diseases 0.000 claims 1
• 239000003446 ligand Substances 0.000 claims 1
• 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 1
• 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims 1
• 125000004437 phosphorous atom Chemical group 0.000 claims 1
• 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 1