JP2011511004A5 - - Google Patents
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- JP2011511004A5 JP2011511004A5 JP2010545236A JP2010545236A JP2011511004A5 JP 2011511004 A5 JP2011511004 A5 JP 2011511004A5 JP 2010545236 A JP2010545236 A JP 2010545236A JP 2010545236 A JP2010545236 A JP 2010545236A JP 2011511004 A5 JP2011511004 A5 JP 2011511004A5
- Authority
- JP
- Japan
- Prior art keywords
- dsrna
- nucleic acid
- lipid particle
- composition
- item
- 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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- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 46
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims description 46
- 125000003729 nucleotide group Chemical group 0.000 claims description 31
- 239000000203 mixture Substances 0.000 claims description 29
- 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 description 19
- 239000002773 nucleotide Substances 0.000 claims description 16
- 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 description 9
- 239000003795 chemical substances by application Substances 0.000 claims description 7
- 210000002966 serum Anatomy 0.000 claims description 7
- 238000008214 LDL Cholesterol Methods 0.000 claims description 6
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims description 6
- 238000009472 formulation Methods 0.000 claims description 6
- 150000002632 lipids Chemical class 0.000 claims description 6
- 101150094724 PCSK9 gene Proteins 0.000 claims description 5
- 108091081021 Sense strand Proteins 0.000 claims description 5
- 230000000692 anti-sense effect Effects 0.000 claims description 5
- 235000012000 cholesterol Nutrition 0.000 claims description 5
- 150000001875 compounds Chemical class 0.000 claims description 5
- 102000000853 LDL receptors Human genes 0.000 claims description 4
- 108010001831 LDL receptors Proteins 0.000 claims description 4
- 230000000295 complement effect Effects 0.000 claims description 4
- 210000004185 liver Anatomy 0.000 claims description 4
- 210000005229 liver cell Anatomy 0.000 claims description 4
- 208000035657 Abasia Diseases 0.000 claims description 3
- 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 description 3
- 230000014509 gene expression Effects 0.000 claims description 3
- 239000003446 ligand Substances 0.000 claims description 3
- 125000001921 locked nucleotide group Chemical group 0.000 claims description 3
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 3
- 150000008298 phosphoramidates Chemical class 0.000 claims description 3
- UFTFJSFQGQCHQW-UHFFFAOYSA-N triformin Chemical compound O=COCC(OC=O)COC=O UFTFJSFQGQCHQW-UHFFFAOYSA-N 0.000 claims description 3
- 201000001320 Atherosclerosis Diseases 0.000 claims description 2
- 208000032928 Dyslipidaemia Diseases 0.000 claims description 2
- 229940121710 HMGCoA reductase inhibitor Drugs 0.000 claims description 2
- 208000035150 Hypercholesterolemia Diseases 0.000 claims description 2
- 208000017170 Lipid metabolism disease Diseases 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 229940079593 drug Drugs 0.000 claims description 2
- 230000002401 inhibitory effect Effects 0.000 claims description 2
- 239000002245 particle Substances 0.000 claims 12
- LRFJOIPOPUJUMI-KWXKLSQISA-N 2-[2,2-bis[(9z,12z)-octadeca-9,12-dienyl]-1,3-dioxolan-4-yl]-n,n-dimethylethanamine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCCC1(CCCCCCCC\C=C/C\C=C/CCCCC)OCC(CCN(C)C)O1 LRFJOIPOPUJUMI-KWXKLSQISA-N 0.000 claims 2
- XVUQPECVOGMPRU-ZPPAUJSGSA-N n,n-dimethyl-1,2-bis[(9z,12z)-octadeca-9,12-dienoxy]propan-1-amine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCCOC(C)C(N(C)C)OCCCCCCCC\C=C/C\C=C/CCCCC XVUQPECVOGMPRU-ZPPAUJSGSA-N 0.000 claims 2
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 1
- 208000031226 Hyperlipidaemia Diseases 0.000 claims 1
- 239000002471 hydroxymethylglutaryl coenzyme A reductase inhibitor Substances 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 238000000034 method Methods 0.000 description 54
- 101001098868 Homo sapiens Proprotein convertase subtilisin/kexin type 9 Proteins 0.000 description 4
- 102100038955 Proprotein convertase subtilisin/kexin type 9 Human genes 0.000 description 4
- 108020004999 messenger RNA Proteins 0.000 description 2
- RZVAJINKPMORJF-UHFFFAOYSA-N Acetaminophen Chemical compound CC(=O)NC1=CC=C(O)C=C1 RZVAJINKPMORJF-UHFFFAOYSA-N 0.000 description 1
- 102000018616 Apolipoproteins B Human genes 0.000 description 1
- 108010027006 Apolipoproteins B Proteins 0.000 description 1
- 241000288906 Primates Species 0.000 description 1
- FYPMFJGVHOHGLL-UHFFFAOYSA-N probucol Chemical compound C=1C(C(C)(C)C)=C(O)C(C(C)(C)C)=CC=1SC(C)(C)SC1=CC(C(C)(C)C)=C(O)C(C(C)(C)C)=C1 FYPMFJGVHOHGLL-UHFFFAOYSA-N 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US2496808P | 2008-01-31 | 2008-01-31 | |
| US3908308P | 2008-03-24 | 2008-03-24 | |
| US7654808P | 2008-06-27 | 2008-06-27 | |
| US18876508P | 2008-08-11 | 2008-08-11 | |
| PCT/US2009/032743 WO2009134487A2 (en) | 2008-01-31 | 2009-01-30 | Optimized methods for delivery of dsrna targeting the pcsk9 gene |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2011511004A JP2011511004A (ja) | 2011-04-07 |
| JP2011511004A5 true JP2011511004A5 (enExample) | 2013-03-14 |
Family
ID=41255636
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010545236A Pending JP2011511004A (ja) | 2008-01-31 | 2009-01-30 | ＰＣＳＫ９遺伝子を標的とするｄｓＲＮＡを送達するための最適化された方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US20100010066A1 (enExample) |
| EP (1) | EP2245039A4 (enExample) |
| JP (1) | JP2011511004A (enExample) |
| AU (1) | AU2009241591A1 (enExample) |
| BR (1) | BRPI0907008A2 (enExample) |
| CA (1) | CA2713379A1 (enExample) |
| MX (1) | MX2010008394A (enExample) |
| WO (1) | WO2009134487A2 (enExample) |
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-
2009
- 2009-01-30 EP EP09739290A patent/EP2245039A4/en not_active Withdrawn
- 2009-01-30 CA CA2713379A patent/CA2713379A1/en not_active Abandoned
- 2009-01-30 WO PCT/US2009/032743 patent/WO2009134487A2/en not_active Ceased
- 2009-01-30 AU AU2009241591A patent/AU2009241591A1/en not_active Abandoned
- 2009-01-30 JP JP2010545236A patent/JP2011511004A/ja active Pending
- 2009-01-30 MX MX2010008394A patent/MX2010008394A/es active IP Right Grant
- 2009-01-30 BR BRPI0907008-7A patent/BRPI0907008A2/pt not_active IP Right Cessation
- 2009-06-04 US US12/478,452 patent/US20100010066A1/en not_active Abandoned
-
2011
- 2011-09-26 US US13/245,730 patent/US20120016009A1/en not_active Abandoned
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