JPWO2021195307A5 - - Google Patents

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Publication number

JPWO2021195307A5

JPWO2021195307A5 JP2022557819A JP2022557819A JPWO2021195307A5 JP WO2021195307 A5 JPWO2021195307 A5 JP WO2021195307A5 JP 2022557819 A JP2022557819 A JP 2022557819A JP 2022557819 A JP2022557819 A JP 2022557819A JP WO2021195307 A5 JPWO2021195307 A5 JP WO2021195307A5

Authority

JP

Japan

Prior art keywords

nucleotides

nucleotide sequence

nucleotide

seq

phosphate

Prior art date

2024-04-03

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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• 125000003729 nucleotide group Chemical group 0.000 claims 129
• 239000002773 nucleotide Substances 0.000 claims 115
• 239000003795 chemical substances by application Substances 0.000 claims 53
• 230000000692 anti-sense effect Effects 0.000 claims 41
• 108091081021 Sense strand Proteins 0.000 claims 23
• 239000000203 mixture Substances 0.000 claims 19
• 241000711573 Coronaviridae Species 0.000 claims 17
• 229910019142 PO4 Inorganic materials 0.000 claims 14
• 230000002401 inhibitory effect Effects 0.000 claims 12
• 239000010452 phosphate Substances 0.000 claims 12
• 239000008194 pharmaceutical composition Substances 0.000 claims 10
• 235000021317 phosphate Nutrition 0.000 claims 10
• 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 9
• 108091030071 RNAI Proteins 0.000 claims 9
• 230000009368 gene silencing by RNA Effects 0.000 claims 9
• NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 8
• 230000000295 complement effect Effects 0.000 claims 6
• LETAFIVATRZLTE-HUBRGWSESA-N P(=O)(O)(O)O[C@H]1[C@H]([C@@H](O[C@@H]1CO)N1C=NC=2C(N)=NC=NC1=2)OCCCCCCCCCCCCCCCC Chemical class P(=O)(O)(O)O[C@H]1[C@H]([C@@H](O[C@@H]1CO)N1C=NC=2C(N)=NC=NC1=2)OCCCCCCCCCCCCCCCC LETAFIVATRZLTE-HUBRGWSESA-N 0.000 claims 5
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 5
• QQMSZHORHNORLP-KVQBGUIXSA-N 2'-deoxyguanosine 3'-monophosphate Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@H]1C[C@H](OP(O)(O)=O)[C@@H](CO)O1 QQMSZHORHNORLP-KVQBGUIXSA-N 0.000 claims 4
• IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical group O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims 4
• 108020004999 messenger RNA Proteins 0.000 claims 4
• 238000000034 method Methods 0.000 claims 4
• 108020004707 nucleic acids Proteins 0.000 claims 4
• 102000039446 nucleic acids Human genes 0.000 claims 4
• 229920002477 rna polymer Polymers 0.000 claims 4
• ZTWTYVWXUKTLCP-UHFFFAOYSA-L ethenyl-dioxido-oxo-$l^{5}-phosphane Chemical compound [O-]P([O-])(=O)C=C ZTWTYVWXUKTLCP-UHFFFAOYSA-L 0.000 claims 3
• 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 3
• MPCAJMNYNOGXPB-UHFFFAOYSA-N 1,5-Anhydro-mannit Natural products OCC1OCC(O)C(O)C1O MPCAJMNYNOGXPB-UHFFFAOYSA-N 0.000 claims 2
• UEUPTUCWIHOIMK-RRKCRQDMSA-N 2'-deoxyadenosine 3'-monophosphate Chemical class C1=NC=2C(N)=NC=NC=2N1[C@H]1C[C@H](OP(O)(O)=O)[C@@H](CO)O1 UEUPTUCWIHOIMK-RRKCRQDMSA-N 0.000 claims 2
• QQMSZHORHNORLP-UHFFFAOYSA-N 2'-deoxyguanosine 3'-monophosphate Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1CC(OP(O)(O)=O)C(CO)O1 QQMSZHORHNORLP-UHFFFAOYSA-N 0.000 claims 2
• MXHRCPNRJAMMIM-SHYZEUOFSA-N 2'-deoxyuridine Chemical class C1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 MXHRCPNRJAMMIM-SHYZEUOFSA-N 0.000 claims 2
• 208000035657 Abasia Diseases 0.000 claims 2
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
• BQMQLJQPTQPEOV-UHFFFAOYSA-N OP(=O)OC=C Chemical class OP(=O)OC=C BQMQLJQPTQPEOV-UHFFFAOYSA-N 0.000 claims 2
• RPYCUZNHGSRXHN-PTGPVQHPSA-N P(=O)(O)(O)O[C@H]1[C@H]([C@@H](O[C@@H]1CO)N1C=NC=2C(=O)NC(N)=NC1=2)OCCCCCCCCCCCCCCCC Chemical class P(=O)(O)(O)O[C@H]1[C@H]([C@@H](O[C@@H]1CO)N1C=NC=2C(=O)NC(N)=NC1=2)OCCCCCCCCCCCCCCCC RPYCUZNHGSRXHN-PTGPVQHPSA-N 0.000 claims 2
• UAYXCKWHISTQAV-MSNJVRRCSA-N [(2R,3R,4R,5R)-5-(4-amino-2-oxopyrimidin-1-yl)-4-hexadecoxy-2-(hydroxymethyl)oxolan-3-yl] dihydrogen phosphate Chemical class CCCCCCCCCCCCCCCCO[C@@H]1[C@H](OP(O)(O)=O)[C@@H](CO)O[C@H]1N1C(=O)N=C(N)C=C1 UAYXCKWHISTQAV-MSNJVRRCSA-N 0.000 claims 2
• FVSAHFFHPOLBLL-SHYZEUOFSA-N [(2r,3s,5r)-5-(4-amino-2-oxopyrimidin-1-yl)-2-(hydroxymethyl)oxolan-3-yl] dihydrogen phosphate Chemical class O=C1N=C(N)C=CN1[C@@H]1O[C@H](CO)[C@@H](OP(O)(O)=O)C1 FVSAHFFHPOLBLL-SHYZEUOFSA-N 0.000 claims 2
• 150000001345 alkine derivatives Chemical class 0.000 claims 2
• 150000001412 amines Chemical class 0.000 claims 2
• 150000001540 azides Chemical class 0.000 claims 2
• 150000001732 carboxylic acid derivatives Chemical class 0.000 claims 2
• HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol group Chemical group [C@@H]1(CC[C@H]2[C@@H]3CC=C4C[C@@H](O)CC[C@]4(C)[C@H]3CC[C@]12C)[C@H](C)CCCC(C)C HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
• YQUAKORMLHPSLZ-XVFCMESISA-N cytidine 2'-phosphate Chemical class O=C1N=C(N)C=CN1[C@H]1[C@H](OP(O)(O)=O)[C@H](O)[C@@H](CO)O1 YQUAKORMLHPSLZ-XVFCMESISA-N 0.000 claims 2
• 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 2
• 125000000524 functional group Chemical group 0.000 claims 2
• WTIFIAZWCCBCGE-UUOKFMHZSA-N guanosine 2'-monophosphate Chemical class C1=2NC(N)=NC(=O)C=2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1OP(O)(O)=O WTIFIAZWCCBCGE-UUOKFMHZSA-N 0.000 claims 2
• 125000001183 hydrocarbyl group Chemical group 0.000 claims 2
• 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 2
• 125000001921 locked nucleotide group Chemical group 0.000 claims 2
• 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 2
• ONKSSDKXDIVIHK-UHFFFAOYSA-N n,n-didecyldodecanamide Chemical group CCCCCCCCCCCC(=O)N(CCCCCCCCCC)CCCCCCCCCC ONKSSDKXDIVIHK-UHFFFAOYSA-N 0.000 claims 2
• 150000008298 phosphoramidates Chemical class 0.000 claims 2
• 150000003013 phosphoric acid derivatives Chemical class 0.000 claims 2
• 230000002685 pulmonary effect Effects 0.000 claims 2
• 229920006395 saturated elastomer Polymers 0.000 claims 2
• BDHFUVZGWQCTTF-UHFFFAOYSA-M sulfonate Chemical compound [O-]S(=O)=O BDHFUVZGWQCTTF-UHFFFAOYSA-M 0.000 claims 2
• 150000003573 thiols Chemical class 0.000 claims 2
• -1 thymidine-glycol nucleic acid Chemical class 0.000 claims 2
• 208000001528 Coronaviridae Infections Diseases 0.000 claims 1
• SGEUBSPLWALIEU-MSNJVRRCSA-N [(2r,3r,4r,5r)-5-(2,4-dioxopyrimidin-1-yl)-4-hexadecoxy-2-(hydroxymethyl)oxolan-3-yl] dihydrogen phosphate Chemical class CCCCCCCCCCCCCCCCO[C@@H]1[C@H](OP(O)(O)=O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 SGEUBSPLWALIEU-MSNJVRRCSA-N 0.000 claims 1
• 150000001334 alicyclic compounds Chemical class 0.000 claims 1
• 125000002723 alicyclic group Chemical group 0.000 claims 1
• 150000007824 aliphatic compounds Chemical class 0.000 claims 1
• 125000001931 aliphatic group Chemical group 0.000 claims 1
• 230000015556 catabolic process Effects 0.000 claims 1
• 150000001875 compounds Chemical class 0.000 claims 1
• 238000006731 degradation reaction Methods 0.000 claims 1
• 238000000338 in vitro Methods 0.000 claims 1
• 125000000913 palmityl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims 1
• 238000007920 subcutaneous administration Methods 0.000 claims 1
• BSYVTEYKTMYBMK-UHFFFAOYSA-N tetrahydrofurfuryl alcohol Chemical compound OCC1CCCO1 BSYVTEYKTMYBMK-UHFFFAOYSA-N 0.000 claims 1