JP2018525015A5 - - Google Patents

Info

Publication number

JP2018525015A5

JP2018525015A5 JP2018510718A JP2018510718A JP2018525015A5 JP 2018525015 A5 JP2018525015 A5 JP 2018525015A5 JP 2018510718 A JP2018510718 A JP 2018510718A JP 2018510718 A JP2018510718 A JP 2018510718A JP 2018525015 A5 JP2018525015 A5 JP 2018525015A5

Authority

JP

Japan

Prior art keywords

alkyl

formula

seq

cpp

integer

Prior art date

2016-08-26

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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• 150000001875 compounds Chemical class 0.000 claims 23
• 230000008685 targeting Effects 0.000 claims 22
• -1 4-aminopiperidin-1-yl moiety Chemical group 0.000 claims 10
• ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 8
• 125000002252 acyl group Chemical group 0.000 claims 8
• 230000000692 anti-sense effect Effects 0.000 claims 8
• OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 8
• RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 8
• 125000005647 linker group Chemical group 0.000 claims 6
• 125000002221 trityl group Chemical group [H]C1=C([H])C([H])=C([H])C([H])=C1C([*])(C1=C(C(=C(C(=C1[H])[H])[H])[H])[H])C1=C([H])C([H])=C([H])C([H])=C1[H] 0.000 claims 6
• 125000003729 nucleotide group Chemical group 0.000 claims 5
• LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 4
• 229940104302 cytosine Drugs 0.000 claims 4
• 239000002773 nucleotide Substances 0.000 claims 4
• 150000003839 salts Chemical class 0.000 claims 4
• 229940113082 thymine Drugs 0.000 claims 4
• 229940035893 uracil Drugs 0.000 claims 4
• 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 3
• 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 3
• 125000000217 alkyl group Chemical group 0.000 claims 3
• 125000003710 aryl alkyl group Chemical group 0.000 claims 3
• 210000004899 c-terminal region Anatomy 0.000 claims 3
• 239000012634 fragment Substances 0.000 claims 3
• 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 3
• 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims 2
• 108091093037 Peptide nucleic acid Proteins 0.000 claims 2
• 102100021947 Survival motor neuron protein Human genes 0.000 claims 2
• ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims 2
• UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 2
• FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims 2
• 108020004999 messenger RNA Proteins 0.000 claims 2
• 239000000203 mixture Substances 0.000 claims 2
• 108020004707 nucleic acids Proteins 0.000 claims 2
• 150000007523 nucleic acids Chemical class 0.000 claims 2
• 102000039446 nucleic acids Human genes 0.000 claims 2
• 208000002320 spinal muscular atrophy Diseases 0.000 claims 2
• MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims 1
• 229930024421 Adenine Natural products 0.000 claims 1
• 239000004475 Arginine Substances 0.000 claims 1
• 239000005977 Ethylene Substances 0.000 claims 1
• UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims 1
• 229930010555 Inosine Natural products 0.000 claims 1
• UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims 1
• 125000002777 acetyl group Chemical group [H]C([H])([H])C(*)=O 0.000 claims 1
• 229960000643 adenine Drugs 0.000 claims 1
• GFFGJBXGBJISGV-UHFFFAOYSA-N adenyl group Chemical group N1=CN=C2N=CNC2=C1N GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 1
• ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
• 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims 1
• 210000004027 cell Anatomy 0.000 claims 1
• 230000000295 complement effect Effects 0.000 claims 1
• DMSZORWOGDLWGN-UHFFFAOYSA-N ctk1a3526 Chemical compound NP(N)(N)=O DMSZORWOGDLWGN-UHFFFAOYSA-N 0.000 claims 1
• 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
• 229960003786 inosine Drugs 0.000 claims 1
• 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
• 239000008194 pharmaceutical composition Substances 0.000 claims 1
• PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims 1
• 108090000765 processed proteins & peptides Proteins 0.000 claims 1
• 125000003696 stearoyl group Chemical group O=C([*])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])C([H])([H])C([H])([H])[H] 0.000 claims 1
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1