JPWO2020132227A5 - - Google Patents
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- JPWO2020132227A5 JPWO2020132227A5 JP2021536024A JP2021536024A JPWO2020132227A5 JP WO2020132227 A5 JPWO2020132227 A5 JP WO2020132227A5 JP 2021536024 A JP2021536024 A JP 2021536024A JP 2021536024 A JP2021536024 A JP 2021536024A JP WO2020132227 A5 JPWO2020132227 A5 JP WO2020132227A5
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- JP
- Japan
- Prior art keywords
- nucleotides
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- double
- strand
- app
- Prior art date
- 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.)
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- 125000003729 nucleotide group Chemical group 0.000 claims 166
- 239000002773 nucleotide Substances 0.000 claims 139
- 229920002477 rna polymer Polymers 0.000 claims 115
- 230000000692 anti-sense Effects 0.000 claims 97
- 239000003795 chemical substances by application Substances 0.000 claims 95
- 229920000972 Sense strand Polymers 0.000 claims 79
- 230000004048 modification Effects 0.000 claims 75
- 238000006011 modification reaction Methods 0.000 claims 75
- 102100017796 APP Human genes 0.000 claims 72
- 108060000460 APP Proteins 0.000 claims 72
- 229920002277 Glycol nucleic acid Polymers 0.000 claims 30
- 206010001897 Alzheimer's disease Diseases 0.000 claims 25
- 230000027455 binding Effects 0.000 claims 22
- 239000003446 ligand Substances 0.000 claims 22
- 230000000295 complement Effects 0.000 claims 19
- 230000002401 inhibitory effect Effects 0.000 claims 18
- 239000000203 mixture Substances 0.000 claims 17
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 17
- 229920000272 Oligonucleotide Polymers 0.000 claims 15
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 12
- 210000004027 cells Anatomy 0.000 claims 11
- 201000010099 disease Diseases 0.000 claims 11
- 108020004999 Messenger RNA Proteins 0.000 claims 9
- 229920002106 messenger RNA Polymers 0.000 claims 9
- 239000008194 pharmaceutical composition Substances 0.000 claims 7
- -1 polycyclic aliphatic compound Chemical class 0.000 claims 7
- NBIIXXVUZAFLBC-UHFFFAOYSA-K [O-]P([O-])([O-])=O Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 5
- 239000010452 phosphate Substances 0.000 claims 5
- 208000005145 Cerebral Amyloid Angiopathy Diseases 0.000 claims 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N Thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 4
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 4
- BQMQLJQPTQPEOV-UHFFFAOYSA-M [O-]P(=O)OC=C Chemical class [O-]P(=O)OC=C BQMQLJQPTQPEOV-UHFFFAOYSA-M 0.000 claims 4
- 239000000969 carrier Substances 0.000 claims 4
- 238000003776 cleavage reaction Methods 0.000 claims 4
- YACKEPLHDIMKIO-UHFFFAOYSA-L methylphosphonate(2-) Chemical compound CP([O-])([O-])=O YACKEPLHDIMKIO-UHFFFAOYSA-L 0.000 claims 4
- 210000001519 tissues Anatomy 0.000 claims 4
- 229920000160 (ribonucleotides)n+m Polymers 0.000 claims 3
- 210000004556 Brain Anatomy 0.000 claims 3
- 238000009825 accumulation Methods 0.000 claims 3
- 150000001412 amines Chemical group 0.000 claims 3
- 239000007853 buffer solution Substances 0.000 claims 3
- 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 3
- 125000004122 cyclic group Chemical group 0.000 claims 3
- BNKAXGCRDYRABM-UHFFFAOYSA-L ethenyl phosphate Chemical group [O-]P([O-])(=O)OC=C BNKAXGCRDYRABM-UHFFFAOYSA-L 0.000 claims 3
- 125000001153 fluoro group Chemical group F* 0.000 claims 3
- 125000001183 hydrocarbyl group Chemical group 0.000 claims 3
- 150000002632 lipids Chemical group 0.000 claims 3
- YPFDHNVEDLHUCE-UHFFFAOYSA-N 1,3-Propanediol Chemical group OCCCO YPFDHNVEDLHUCE-UHFFFAOYSA-N 0.000 claims 2
- 206010049460 Abasia Diseases 0.000 claims 2
- BVKZGUZCCUSVTD-UHFFFAOYSA-L Carbonate dianion Chemical compound [O-]C([O-])=O BVKZGUZCCUSVTD-UHFFFAOYSA-L 0.000 claims 2
- TUNFSRHWOTWDNC-UHFFFAOYSA-N Myristic acid Chemical compound CCCCCCCCCCCCCC(O)=O TUNFSRHWOTWDNC-UHFFFAOYSA-N 0.000 claims 2
- IPCSVZSSVZVIGE-UHFFFAOYSA-N Palmitic acid Chemical compound CCCCCCCCCCCCCCCC(O)=O IPCSVZSSVZVIGE-UHFFFAOYSA-N 0.000 claims 2
- 230000036660 Plasma protein binding Effects 0.000 claims 2
- 229940113082 Thymine Drugs 0.000 claims 2
- 229940035893 Uracil Drugs 0.000 claims 2
- 150000001408 amides Chemical class 0.000 claims 2
- 125000004855 decalinyl group Chemical group C1(CCCC2CCCCC12)* 0.000 claims 2
- RTZKZFJDLAIYFH-UHFFFAOYSA-N diethyl ether Chemical compound CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 claims 2
- 150000002019 disulfides Chemical class 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 238000009472 formulation Methods 0.000 claims 2
- 125000002632 imidazolidinyl group Chemical group 0.000 claims 2
- 125000002636 imidazolinyl group Chemical group 0.000 claims 2
- 125000004628 isothiazolidinyl group Chemical group S1N(CCC1)* 0.000 claims 2
- 125000003965 isoxazolidinyl group Chemical group 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
- 125000002757 morpholinyl group Chemical group 0.000 claims 2
- QTNLALDFXILRQO-UHFFFAOYSA-N nonadecane-1,2,3-triol Chemical compound CCCCCCCCCCCCCCCCC(O)C(O)CO QTNLALDFXILRQO-UHFFFAOYSA-N 0.000 claims 2
- 125000000160 oxazolidinyl group Chemical group 0.000 claims 2
- 239000002953 phosphate buffered saline Substances 0.000 claims 2
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims 2
- 150000008298 phosphoramidates Chemical class 0.000 claims 2
- 125000004193 piperazinyl group Chemical group 0.000 claims 2
- 125000003386 piperidinyl group Chemical group 0.000 claims 2
- 238000000159 protein binding assay Methods 0.000 claims 2
- 125000003072 pyrazolidinyl group Chemical group 0.000 claims 2
- 125000002755 pyrazolinyl group Chemical group 0.000 claims 2
- 125000000719 pyrrolidinyl group Chemical group 0.000 claims 2
- 125000001567 quinoxalinyl group Chemical group N1=C(C=NC2=CC=CC=C12)* 0.000 claims 2
- 239000000243 solution Substances 0.000 claims 2
- 125000003718 tetrahydrofuranyl group Chemical group 0.000 claims 2
- 125000001984 thiazolidinyl group Chemical group 0.000 claims 2
- XSQUKJJJFZCRTK-UHFFFAOYSA-N urea Chemical compound NC(N)=O XSQUKJJJFZCRTK-UHFFFAOYSA-N 0.000 claims 2
- NOOLISFMXDJSKH-UTLUCORTSA-N (+)-Neomenthol Chemical group CC(C)[C@@H]1CC[C@@H](C)C[C@@H]1O NOOLISFMXDJSKH-UTLUCORTSA-N 0.000 claims 1
- DTGKSKDOIYIVQL-WEDXCCLWSA-N (+)-borneol Chemical compound C1C[C@@]2(C)[C@@H](O)C[C@@H]1C2(C)C DTGKSKDOIYIVQL-WEDXCCLWSA-N 0.000 claims 1
- JIUXDGYUXSBTAY-IVZWLZJFSA-N 1-[(2R,4S,5R)-4-hydroxy-5-(2-hydroxyethoxymethyl)oxolan-2-yl]-5-methylpyrimidine-2,4-dione Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](COCCO)[C@@H](O)C1 JIUXDGYUXSBTAY-IVZWLZJFSA-N 0.000 claims 1
- NVKAWKQGWWIWPM-ABEVXSGRSA-N 17-β-hydroxy-5-α-Androstan-3-one Chemical compound C1C(=O)CC[C@]2(C)[C@H]3CC[C@](C)([C@H](CC4)O)[C@@H]4[C@@H]3CC[C@H]21 NVKAWKQGWWIWPM-ABEVXSGRSA-N 0.000 claims 1
- QQMSZHORHNORLP-KVQBGUIXSA-N 2'-deoxyguanosine 3'-monophosphate Chemical group 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 1
- AOTQGWFNFTVXNQ-UHFFFAOYSA-N 2-(1-adamantyl)acetic acid Chemical compound C1C(C2)CC3CC2CC1(CC(=O)O)C3 AOTQGWFNFTVXNQ-UHFFFAOYSA-N 0.000 claims 1
- KJJPLEZQSCZCKE-UHFFFAOYSA-N 2-aminopropane-1,3-diol Chemical group OCC(N)CO KJJPLEZQSCZCKE-UHFFFAOYSA-N 0.000 claims 1
- KRKNYBCHXYNGOX-UHFFFAOYSA-K 2qpq Chemical compound [O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O KRKNYBCHXYNGOX-UHFFFAOYSA-K 0.000 claims 1
- QXYRRCOJHNZVDJ-UHFFFAOYSA-N 4-pyren-1-ylbutanoic acid Chemical compound C1=C2C(CCCC(=O)O)=CC=C(C=C3)C2=C2C3=CC=CC2=C1 QXYRRCOJHNZVDJ-UHFFFAOYSA-N 0.000 claims 1
- 101700077116 AVE3 Proteins 0.000 claims 1
- 101700006934 AVEA Proteins 0.000 claims 1
- 101700044096 AVEE Proteins 0.000 claims 1
- 101700047979 AVEF Proteins 0.000 claims 1
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Natural products NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 1
- 229960000643 Adenine Drugs 0.000 claims 1
- 229940116229 Borneol Drugs 0.000 claims 1
- 241000282693 Cercopithecidae Species 0.000 claims 1
- 210000001638 Cerebellum Anatomy 0.000 claims 1
- 229940107161 Cholesterol Drugs 0.000 claims 1
- BHQCQFFYRZLCQQ-OELDTZBJSA-N Cholic acid Chemical compound C([C@H]1C[C@H]2O)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 BHQCQFFYRZLCQQ-OELDTZBJSA-N 0.000 claims 1
- 239000004380 Cholic acid Substances 0.000 claims 1
- 229940104302 Cytosine Drugs 0.000 claims 1
- OPTASPLRGRRNAP-UHFFFAOYSA-N Cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 1
- GZCGUPFRVQAUEE-KCDKBNATSA-N D-(+)-Galactose Natural products OC[C@@H](O)[C@H](O)[C@H](O)[C@@H](O)C=O GZCGUPFRVQAUEE-KCDKBNATSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-GASJEMHNSA-N D-Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-QTVWNMPRSA-N D-mannopyranose Chemical compound OC[C@H]1OC(O)[C@@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-QTVWNMPRSA-N 0.000 claims 1
- ZBCBWPMODOFKDW-UHFFFAOYSA-N Diethanolamine Chemical group OCCNCCO ZBCBWPMODOFKDW-UHFFFAOYSA-N 0.000 claims 1
- 101700064290 GDA0 Proteins 0.000 claims 1
- 101700058641 GDA2 Proteins 0.000 claims 1
- 101700033989 GDA3 Proteins 0.000 claims 1
- 101700012011 GDA4 Proteins 0.000 claims 1
- 101700031401 GDA5 Proteins 0.000 claims 1
- 101700036788 GDA6 Proteins 0.000 claims 1
- 101700014913 GDA7 Proteins 0.000 claims 1
- 101700083010 GDA8 Proteins 0.000 claims 1
- 101700061165 GDA9 Proteins 0.000 claims 1
- UYTPUPDQBNUYGX-UHFFFAOYSA-N Guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 1
- 102000008100 Human Serum Albumin Human genes 0.000 claims 1
- 108091006822 Human Serum Albumin Proteins 0.000 claims 1
- 229960004873 LEVOMENTHOL Drugs 0.000 claims 1
- 101710024851 LNPK Proteins 0.000 claims 1
- SMEROWZSTRWXGI-HVATVPOCSA-N Lithocholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)CC1 SMEROWZSTRWXGI-HVATVPOCSA-N 0.000 claims 1
- 210000004705 Lumbosacral Region Anatomy 0.000 claims 1
- 241000282560 Macaca mulatta Species 0.000 claims 1
- 229940041616 Menthol Drugs 0.000 claims 1
- 241000699670 Mus sp. Species 0.000 claims 1
- 235000021360 Myristic acid Nutrition 0.000 claims 1
- ONKSSDKXDIVIHK-UHFFFAOYSA-N N,N-didecyldodecanamide Chemical group CCCCCCCCCCCC(=O)N(CCCCCCCCCC)CCCCCCCCCC ONKSSDKXDIVIHK-UHFFFAOYSA-N 0.000 claims 1
- 102100016150 NUSAP1 Human genes 0.000 claims 1
- 101710011271 NUSAP1 Proteins 0.000 claims 1
- 210000001577 Neostriatum Anatomy 0.000 claims 1
- 235000021314 Palmitic acid Nutrition 0.000 claims 1
- TZMSYXZUNZXBOL-UHFFFAOYSA-N Phenoxazine Chemical compound C1=CC=C2NC3=CC=CC=C3OC2=C1 TZMSYXZUNZXBOL-UHFFFAOYSA-N 0.000 claims 1
- 241000700159 Rattus Species 0.000 claims 1
- FDDDEECHVMSUSB-UHFFFAOYSA-N Sulfanilamide Chemical compound NC1=CC=C(S(N)(=O)=O)C=C1 FDDDEECHVMSUSB-UHFFFAOYSA-N 0.000 claims 1
- LSNNMFCWUKXFEE-UHFFFAOYSA-L Sulphite Chemical group [O-]S([O-])=O LSNNMFCWUKXFEE-UHFFFAOYSA-L 0.000 claims 1
- SHGAZHPCJJPHSC-NWVFGJFESA-N Tretinoin Chemical compound OC(=O)/C=C(\C)/C=C/C=C(C)C=CC1=C(C)CCCC1(C)C SHGAZHPCJJPHSC-NWVFGJFESA-N 0.000 claims 1
- 229960001727 Tretinoin Drugs 0.000 claims 1
- 230000036743 UNBOUND FRACTION Effects 0.000 claims 1
- JCAQMQLAHNGVPY-UUOKFMHZSA-N [(2R,3S,4R,5R)-3,4-dihydroxy-5-(2,2,4-trioxo-1H-imidazo[4,5-c][1,2,6]thiadiazin-7-yl)oxolan-2-yl]methyl dihydrogen phosphate Chemical class O[C@@H]1[C@H](O)[C@@H](COP(O)(O)=O)O[C@H]1N1C(NS(=O)(=O)NC2=O)=C2N=C1 JCAQMQLAHNGVPY-UUOKFMHZSA-N 0.000 claims 1
- QTBSBXVTEAMEQO-UHFFFAOYSA-M acetate Chemical compound CC([O-])=O QTBSBXVTEAMEQO-UHFFFAOYSA-M 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 125000002015 acyclic group Chemical group 0.000 claims 1
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 1
- 125000001931 aliphatic group Chemical group 0.000 claims 1
- 150000001345 alkine derivatives Chemical group 0.000 claims 1
- 229960003473 androstanolone Drugs 0.000 claims 1
- IVRMZWNICZWHMI-UHFFFAOYSA-N azide Chemical group [N-]=[N+]=[N-] IVRMZWNICZWHMI-UHFFFAOYSA-N 0.000 claims 1
- 230000015572 biosynthetic process Effects 0.000 claims 1
- 229930006709 borneol Natural products 0.000 claims 1
- KXDHJXZQYSOELW-UHFFFAOYSA-M carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims 1
- 239000004202 carbamide Substances 0.000 claims 1
- 150000001732 carboxylic acid derivatives Chemical group 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 239000007795 chemical reaction product Substances 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 229960002471 cholic acid Drugs 0.000 claims 1
- 235000019416 cholic acid Nutrition 0.000 claims 1
- 230000004059 degradation Effects 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims 1
- 230000000368 destabilizing Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 238000002337 electrophoretic mobility shift assay Methods 0.000 claims 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 238000005755 formation reaction Methods 0.000 claims 1
- 125000000524 functional group Chemical group 0.000 claims 1
- 150000008275 galactosamines Chemical class 0.000 claims 1
- 101700085810 gda1 Proteins 0.000 claims 1
- 150000002302 glucosamines Chemical class 0.000 claims 1
- 239000008103 glucose Substances 0.000 claims 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 1
- 230000000640 hydroxylating Effects 0.000 claims 1
- 125000002960 margaryl 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])C([H])([H])[H] 0.000 claims 1
- 230000003278 mimic Effects 0.000 claims 1
- 150000002772 monosaccharides Chemical class 0.000 claims 1
- 229920001542 oligosaccharide Polymers 0.000 claims 1
- 150000002482 oligosaccharides Polymers 0.000 claims 1
- 150000004713 phosphodiesters Chemical class 0.000 claims 1
- 150000003013 phosphoric acid derivatives Chemical group 0.000 claims 1
- 229920000166 polytrimethylene carbonate Chemical group 0.000 claims 1
- OZAIFHULBGXAKX-UHFFFAOYSA-N precursor Substances N#CC(C)(C)N=NC(C)(C)C#N OZAIFHULBGXAKX-UHFFFAOYSA-N 0.000 claims 1
- 229930002330 retinoic acid Natural products 0.000 claims 1
- 230000001568 sexual Effects 0.000 claims 1
- FAPWRFPIFSIZLT-UHFFFAOYSA-M sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 1
- 239000011780 sodium chloride Substances 0.000 claims 1
- 229960001663 sulfanilamide Drugs 0.000 claims 1
- 150000003568 thioethers Chemical class 0.000 claims 1
- 150000003573 thiols Chemical group 0.000 claims 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-PHYPRBDBSA-N α-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
- WQZGKKKJIJFFOK-VFUOTHLCSA-N β-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862781774P | 2018-12-19 | 2018-12-19 | |
| US62/781,774 | 2018-12-19 | ||
| US201962862472P | 2019-06-17 | 2019-06-17 | |
| US62/862,472 | 2019-06-17 | ||
| US201962928795P | 2019-10-31 | 2019-10-31 | |
| US62/928,795 | 2019-10-31 | ||
| PCT/US2019/067449 WO2020132227A2 (en) | 2018-12-19 | 2019-12-19 | AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022515193A JP2022515193A (ja) | 2022-02-17 |
| JPWO2020132227A5 true JPWO2020132227A5 (ru) | 2022-12-26 |
Family
ID=71101909
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021536024A Pending JP2022515193A (ja) | 2018-12-19 | 2019-12-19 | アミロイド前駆体タンパク質（ＡＰＰ）ＲＮＡｉ薬剤組成物およびその使用方法 |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US11034957B2 (ru) |
| EP (1) | EP3898979A4 (ru) |
| JP (1) | JP2022515193A (ru) |
| KR (1) | KR20210111780A (ru) |
| CN (1) | CN113454222A (ru) |
| AU (1) | AU2019405783A1 (ru) |
| BR (1) | BR112021011895A2 (ru) |
| CA (1) | CA3124090A1 (ru) |
| CL (1) | CL2021001605A1 (ru) |
| CO (1) | CO2021009163A2 (ru) |
| CR (1) | CR20210393A (ru) |
| DO (1) | DOP2021000126A (ru) |
| IL (1) | IL283852A (ru) |
| MX (1) | MX2021007570A (ru) |
| PE (1) | PE20211420A1 (ru) |
| SG (1) | SG11202105886QA (ru) |
| TW (1) | TW202039844A (ru) |
| WO (1) | WO2020132227A2 (ru) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112014029833B1 (pt) | 2012-05-29 | 2019-10-01 | 3M Innovative Properties Company | Artigo absorvente que compreende espuma polimérica e intermediários |
| US11732260B2 (en) | 2018-03-02 | 2023-08-22 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of amyloid-β precursor protein |
| EP4189090A1 (en) * | 2020-07-28 | 2023-06-07 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing app expression |
| WO2022165172A1 (en) * | 2021-01-29 | 2022-08-04 | Alnylam Pharmaceuticals, Inc. | Irna compositions and methods for silencing amyloid precursor protein (app) |
| WO2023039503A2 (en) * | 2021-09-10 | 2023-03-16 | Alnylam Pharmaceuticals, Inc. | App irna compositions and methods of use thereof for treating or preventing diseases characterized by enlarged endosomes |
| WO2023081500A2 (en) * | 2021-11-08 | 2023-05-11 | Dicerna Pharmaceuticals, Inc. | RNAi OLIGONUCLEOTIDE CONJUGATES |
| WO2023173061A2 (en) * | 2022-03-11 | 2023-09-14 | University Of Massachusetts | Oligonucleotides for app modulation |
| WO2023207615A1 (zh) * | 2022-04-26 | 2023-11-02 | 南京明德新药研发有限公司 | 一类含由天然核苷酸组成突出端的双链RNAi化合物 |
| US20230399645A1 (en) * | 2022-05-16 | 2023-12-14 | University Of Massachusetts | Optimized sirna scaffolds |
| WO2023230465A1 (en) * | 2022-05-23 | 2023-11-30 | Switch Therapeutics Inc. | Antisense oligonucleotides |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050209179A1 (en) * | 2000-08-30 | 2005-09-22 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA) |
| US20080032942A1 (en) * | 2000-08-30 | 2008-02-07 | Mcswiggen James | RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA) |
| WO2005003350A2 (en) * | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| EP2333062A1 (en) | 2002-07-10 | 2011-06-15 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA-interference by single-stranded RNA molecules |
| US7851615B2 (en) * | 2003-04-17 | 2010-12-14 | Alnylam Pharmaceuticals, Inc. | Lipophilic conjugated iRNA agents |
| GB0324854D0 (en) * | 2003-10-24 | 2003-11-26 | Expresson Biosystems Ltd | App/ena antisense |
| WO2009113579A1 (ja) * | 2008-03-11 | 2009-09-17 | 学校法人埼玉医科大学 | 癌の予防乃至治療に好適な二本鎖核酸分子、癌細胞増殖抑制剤、並びに医薬 |
| EP2510098B1 (en) * | 2009-12-09 | 2015-02-11 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the cns |
| WO2012030683A2 (en) * | 2010-08-31 | 2012-03-08 | Merck Sharp & Dohme Corp. | Novel single chemical entities and methods for delivery of oligonucleotides |
| US9127274B2 (en) * | 2012-04-26 | 2015-09-08 | Alnylam Pharmaceuticals, Inc. | Serpinc1 iRNA compositions and methods of use thereof |
| EP2857513A4 (en) * | 2012-05-26 | 2016-05-25 | Bonac Corp | SINGLE STRANDED NUCLEIC ACID MOLECULE FOR CONTROLLING THE EXPRESSION OF A GENE HAVING AN ADMINISTRATION FUNCTION |
| HUE035887T2 (en) * | 2012-12-05 | 2018-05-28 | Alnylam Pharmaceuticals Inc | PCSK9 iRNA preparations and methods for their use |
| CN114181942A (zh) * | 2014-08-20 | 2022-03-15 | 阿尔尼拉姆医药品有限公司 | 经修饰的双链rna试剂 |
| JOP20200115A1 (ar) * | 2014-10-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) |
| AU2016337034A1 (en) * | 2015-10-16 | 2018-05-10 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for use in treating Alzheimer's disease |
| JP7353301B2 (ja) * | 2018-05-07 | 2023-09-29 | アルニラム ファーマスーティカルズ インコーポレイテッド | 肝臓外送達 |
| US20220380773A1 (en) * | 2019-01-29 | 2022-12-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing app expression |
| WO2020257194A1 (en) * | 2019-06-17 | 2020-12-24 | Alnylam Pharmaceuticals, Inc. | Delivery of oligonucleotides to the striatum |
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2019
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- 2019-12-19 TW TW108146697A patent/TW202039844A/zh unknown
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