JP2010503640A - オリゴヌクレオチド非ウイルスデリバリーシステム - Google Patents
オリゴヌクレオチド非ウイルスデリバリーシステム Download PDFInfo
- Publication number
- JP2010503640A JP2010503640A JP2009527838A JP2009527838A JP2010503640A JP 2010503640 A JP2010503640 A JP 2010503640A JP 2009527838 A JP2009527838 A JP 2009527838A JP 2009527838 A JP2009527838 A JP 2009527838A JP 2010503640 A JP2010503640 A JP 2010503640A
- Authority
- JP
- Japan
- Prior art keywords
- composition
- sirna
- molecular weight
- low molecular
- chitosan
- 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.)
- Pending
Links
- 108091034117 Oligonucleotide Proteins 0.000 title claims description 17
- 230000003612 virological effect Effects 0.000 title description 2
- 229920001661 Chitosan Polymers 0.000 claims abstract description 116
- 238000000338 in vitro Methods 0.000 claims abstract description 27
- 230000030279 gene silencing Effects 0.000 claims abstract description 24
- 238000001727 in vivo Methods 0.000 claims abstract description 9
- 239000000203 mixture Substances 0.000 claims description 72
- 238000000034 method Methods 0.000 claims description 23
- 241000124008 Mammalia Species 0.000 claims description 14
- 230000000692 anti-sense effect Effects 0.000 claims description 8
- 230000006196 deacetylation Effects 0.000 claims description 6
- 238000003381 deacetylation reaction Methods 0.000 claims description 6
- 238000006116 polymerization reaction Methods 0.000 claims description 6
- 239000000243 solution Substances 0.000 claims description 6
- 230000008685 targeting Effects 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 5
- 239000003125 aqueous solvent Substances 0.000 claims description 4
- 210000004204 blood vessel Anatomy 0.000 claims description 3
- 201000011510 cancer Diseases 0.000 claims description 3
- 201000010099 disease Diseases 0.000 claims description 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 3
- 238000007917 intracranial administration Methods 0.000 claims description 3
- 238000007918 intramuscular administration Methods 0.000 claims description 3
- 238000007913 intrathecal administration Methods 0.000 claims description 3
- 238000001990 intravenous administration Methods 0.000 claims description 3
- 238000004519 manufacturing process Methods 0.000 claims description 3
- 210000004400 mucous membrane Anatomy 0.000 claims description 3
- 108020004707 nucleic acids Proteins 0.000 claims description 3
- 102000039446 nucleic acids Human genes 0.000 claims description 3
- 150000007523 nucleic acids Chemical class 0.000 claims description 3
- 238000007920 subcutaneous administration Methods 0.000 claims description 3
- 238000001356 surgical procedure Methods 0.000 claims description 3
- 210000004876 tela submucosa Anatomy 0.000 claims description 3
- 210000001835 viscera Anatomy 0.000 claims description 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
- 208000023275 Autoimmune disease Diseases 0.000 claims description 2
- 102000053642 Catalytic RNA Human genes 0.000 claims description 2
- 108090000994 Catalytic RNA Proteins 0.000 claims description 2
- 208000026350 Inborn Genetic disease Diseases 0.000 claims description 2
- 108700011259 MicroRNAs Proteins 0.000 claims description 2
- 239000007864 aqueous solution Substances 0.000 claims description 2
- 229940039227 diagnostic agent Drugs 0.000 claims description 2
- 239000000032 diagnostic agent Substances 0.000 claims description 2
- 238000002405 diagnostic procedure Methods 0.000 claims description 2
- 238000006911 enzymatic reaction Methods 0.000 claims description 2
- 238000001415 gene therapy Methods 0.000 claims description 2
- 208000016361 genetic disease Diseases 0.000 claims description 2
- 208000015181 infectious disease Diseases 0.000 claims description 2
- 239000003446 ligand Substances 0.000 claims description 2
- 238000002156 mixing Methods 0.000 claims description 2
- 230000001717 pathogenic effect Effects 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 230000002685 pulmonary effect Effects 0.000 claims description 2
- 108091092562 ribozyme Proteins 0.000 claims description 2
- 239000003381 stabilizer Substances 0.000 claims description 2
- 239000000126 substance Substances 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims description 2
- 230000000699 topical effect Effects 0.000 claims description 2
- 238000002255 vaccination Methods 0.000 claims description 2
- 210000004072 lung Anatomy 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 abstract description 139
- 239000002245 particle Substances 0.000 abstract description 12
- 230000007515 enzymatic degradation Effects 0.000 abstract description 7
- 239000002105 nanoparticle Substances 0.000 abstract description 5
- 238000012226 gene silencing method Methods 0.000 abstract description 4
- 239000004055 small Interfering RNA Substances 0.000 description 131
- 108060001084 Luciferase Proteins 0.000 description 55
- 239000005089 Luciferase Substances 0.000 description 53
- 210000004027 cell Anatomy 0.000 description 53
- 238000009472 formulation Methods 0.000 description 33
- 239000012097 Lipofectamine 2000 Substances 0.000 description 31
- 229920002873 Polyethylenimine Polymers 0.000 description 29
- 238000001890 transfection Methods 0.000 description 27
- 230000000694 effects Effects 0.000 description 25
- 230000014509 gene expression Effects 0.000 description 23
- 230000037440 gene silencing effect Effects 0.000 description 14
- 230000001743 silencing effect Effects 0.000 description 14
- 230000003834 intracellular effect Effects 0.000 description 13
- 101710088194 Dehydrogenase Proteins 0.000 description 12
- 210000002966 serum Anatomy 0.000 description 11
- 231100000135 cytotoxicity Toxicity 0.000 description 10
- 230000003013 cytotoxicity Effects 0.000 description 10
- 239000000178 monomer Substances 0.000 description 10
- 230000002829 reductive effect Effects 0.000 description 8
- 238000002474 experimental method Methods 0.000 description 7
- 238000012761 co-transfection Methods 0.000 description 6
- 239000002609 medium Substances 0.000 description 6
- 230000000717 retained effect Effects 0.000 description 6
- 108020002230 Pancreatic Ribonuclease Proteins 0.000 description 5
- 102000005891 Pancreatic ribonuclease Human genes 0.000 description 5
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 5
- 238000011534 incubation Methods 0.000 description 5
- 239000011550 stock solution Substances 0.000 description 5
- 108090000331 Firefly luciferases Proteins 0.000 description 4
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 description 4
- 238000003556 assay Methods 0.000 description 4
- 229920006317 cationic polymer Polymers 0.000 description 4
- 230000004700 cellular uptake Effects 0.000 description 4
- 239000000499 gel Substances 0.000 description 4
- 230000009368 gene silencing by RNA Effects 0.000 description 4
- 229920000962 poly(amidoamine) Polymers 0.000 description 4
- 230000001988 toxicity Effects 0.000 description 4
- 231100000419 toxicity Toxicity 0.000 description 4
- 229920000936 Agarose Polymers 0.000 description 3
- 239000012124 Opti-MEM Substances 0.000 description 3
- 238000002835 absorbance Methods 0.000 description 3
- 230000002776 aggregation Effects 0.000 description 3
- 230000015556 catabolic process Effects 0.000 description 3
- 238000006731 degradation reaction Methods 0.000 description 3
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 description 3
- 238000009826 distribution Methods 0.000 description 3
- 238000001476 gene delivery Methods 0.000 description 3
- 230000001771 impaired effect Effects 0.000 description 3
- 230000003993 interaction Effects 0.000 description 3
- 239000002953 phosphate buffered saline Substances 0.000 description 3
- 239000013612 plasmid Substances 0.000 description 3
- 229920000729 poly(L-lysine) polymer Polymers 0.000 description 3
- 238000011160 research Methods 0.000 description 3
- 238000012360 testing method Methods 0.000 description 3
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 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 description 2
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 description 2
- 238000012404 In vitro experiment Methods 0.000 description 2
- KFZMGEQAYNKOFK-UHFFFAOYSA-N Isopropanol Chemical compound CC(C)O KFZMGEQAYNKOFK-UHFFFAOYSA-N 0.000 description 2
- 102000006382 Ribonucleases Human genes 0.000 description 2
- 108010083644 Ribonucleases Proteins 0.000 description 2
- 238000004220 aggregation Methods 0.000 description 2
- 239000000872 buffer Substances 0.000 description 2
- 230000003833 cell viability Effects 0.000 description 2
- 230000009918 complex formation Effects 0.000 description 2
- 239000002131 composite material Substances 0.000 description 2
- 230000001419 dependent effect Effects 0.000 description 2
- 238000002296 dynamic light scattering Methods 0.000 description 2
- 229920000669 heparin Polymers 0.000 description 2
- 229960002897 heparin Drugs 0.000 description 2
- 230000005764 inhibitory process Effects 0.000 description 2
- 239000000463 material Substances 0.000 description 2
- 238000005259 measurement Methods 0.000 description 2
- 230000002085 persistent effect Effects 0.000 description 2
- BOLDJAUMGUJJKM-LSDHHAIUSA-N renifolin D Natural products CC(=C)[C@@H]1Cc2c(O)c(O)ccc2[C@H]1CC(=O)c3ccc(O)cc3O BOLDJAUMGUJJKM-LSDHHAIUSA-N 0.000 description 2
- 238000003756 stirring Methods 0.000 description 2
- 210000001519 tissue Anatomy 0.000 description 2
- 238000011282 treatment Methods 0.000 description 2
- 150000004043 trisaccharides Chemical group 0.000 description 2
- AZKSAVLVSZKNRD-UHFFFAOYSA-M 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide Chemical compound [Br-].S1C(C)=C(C)N=C1[N+]1=NC(C=2C=CC=CC=2)=NN1C1=CC=CC=C1 AZKSAVLVSZKNRD-UHFFFAOYSA-M 0.000 description 1
- 241000701022 Cytomegalovirus Species 0.000 description 1
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 1
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 1
- 229930195725 Mannitol Natural products 0.000 description 1
- OVRNDRQMDRJTHS-FMDGEEDCSA-N N-acetyl-beta-D-glucosamine Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O OVRNDRQMDRJTHS-FMDGEEDCSA-N 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 101710163270 Nuclease Proteins 0.000 description 1
- 206010033128 Ovarian cancer Diseases 0.000 description 1
- 206010061535 Ovarian neoplasm Diseases 0.000 description 1
- 239000008049 TAE buffer Substances 0.000 description 1
- 208000036142 Viral infection Diseases 0.000 description 1
- HGEVZDLYZYVYHD-UHFFFAOYSA-N acetic acid;2-amino-2-(hydroxymethyl)propane-1,3-diol;2-[2-[bis(carboxymethyl)amino]ethyl-(carboxymethyl)amino]acetic acid Chemical compound CC(O)=O.OCC(N)(CO)CO.OC(=O)CN(CC(O)=O)CCN(CC(O)=O)CC(O)=O HGEVZDLYZYVYHD-UHFFFAOYSA-N 0.000 description 1
- 230000021736 acetylation Effects 0.000 description 1
- 238000006640 acetylation reaction Methods 0.000 description 1
- 231100000131 acute cytotoxicity Toxicity 0.000 description 1
- 239000011543 agarose gel Substances 0.000 description 1
- 238000000246 agarose gel electrophoresis Methods 0.000 description 1
- 238000005054 agglomeration Methods 0.000 description 1
- 238000000540 analysis of variance Methods 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 238000003705 background correction Methods 0.000 description 1
- MSWZFWKMSRAUBD-QZABAPFNSA-N beta-D-glucosamine Chemical compound N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O MSWZFWKMSRAUBD-QZABAPFNSA-N 0.000 description 1
- -1 cationic lipid Chemical class 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 230000000536 complexating effect Effects 0.000 description 1
- 238000010668 complexation reaction Methods 0.000 description 1
- 230000009133 cooperative interaction Effects 0.000 description 1
- 230000002596 correlated effect Effects 0.000 description 1
- 239000013078 crystal Substances 0.000 description 1
- 238000007405 data analysis Methods 0.000 description 1
- 230000003111 delayed effect Effects 0.000 description 1
- 239000000412 dendrimer Substances 0.000 description 1
- 229920000736 dendritic polymer Polymers 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 206010012601 diabetes mellitus Diseases 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 230000003828 downregulation Effects 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 238000001962 electrophoresis Methods 0.000 description 1
- 238000005516 engineering process Methods 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 239000012737 fresh medium Substances 0.000 description 1
- 150000004676 glycans Chemical class 0.000 description 1
- 239000005090 green fluorescent protein Substances 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 210000003292 kidney cell Anatomy 0.000 description 1
- 238000002356 laser light scattering Methods 0.000 description 1
- 230000000670 limiting effect Effects 0.000 description 1
- 150000002632 lipids Chemical class 0.000 description 1
- 239000012139 lysis buffer Substances 0.000 description 1
- 229920002521 macromolecule Polymers 0.000 description 1
- 239000000594 mannitol Substances 0.000 description 1
- 235000010355 mannitol Nutrition 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- VMGAPWLDMVPYIA-HIDZBRGKSA-N n'-amino-n-iminomethanimidamide Chemical compound N\N=C\N=N VMGAPWLDMVPYIA-HIDZBRGKSA-N 0.000 description 1
- 231100000252 nontoxic Toxicity 0.000 description 1
- 230000003000 nontoxic effect Effects 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 230000000704 physical effect Effects 0.000 description 1
- 229920001282 polysaccharide Polymers 0.000 description 1
- 239000005017 polysaccharide Substances 0.000 description 1
- 239000013641 positive control Substances 0.000 description 1
- 230000008092 positive effect Effects 0.000 description 1
- 230000002265 prevention Effects 0.000 description 1
- 238000012216 screening Methods 0.000 description 1
- 239000012679 serum free medium Substances 0.000 description 1
- 238000001542 size-exclusion chromatography Methods 0.000 description 1
- 238000011146 sterile filtration Methods 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 230000002459 sustained effect Effects 0.000 description 1
- 230000036962 time dependent Effects 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 231100000027 toxicology Toxicity 0.000 description 1
- 239000013598 vector Substances 0.000 description 1
- 238000012795 verification Methods 0.000 description 1
- 230000009385 viral infection Effects 0.000 description 1
Images
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
- A61K31/716—Glucans
- A61K31/722—Chitin, chitosan
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/61—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule the organic macromolecular compound being a polysaccharide or a derivative thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/513—Organic macromolecular compounds; Dendrimers
- A61K9/5161—Polysaccharides, e.g. alginate, chitosan, cellulose derivatives; Cyclodextrin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US84472906P | 2006-09-15 | 2006-09-15 | |
| PCT/EP2007/059740 WO2008031899A2 (en) | 2006-09-15 | 2007-09-14 | Oligonucleotide non-viral delivery systems |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2010503640A true JP2010503640A (ja) | 2010-02-04 |
| JP2010503640A5 JP2010503640A5 (enExample) | 2010-07-29 |
Family
ID=39092312
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009527838A Pending JP2010503640A (ja) | 2006-09-15 | 2007-09-14 | オリゴヌクレオチド非ウイルスデリバリーシステム |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US7875449B2 (enExample) |
| EP (1) | EP2061516A2 (enExample) |
| JP (1) | JP2010503640A (enExample) |
| KR (1) | KR20090058562A (enExample) |
| CN (1) | CN101588821A (enExample) |
| BR (1) | BRPI0716925A2 (enExample) |
| CA (1) | CA2662560A1 (enExample) |
| IL (1) | IL197497A (enExample) |
| WO (1) | WO2008031899A2 (enExample) |
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2016511255A (ja) * | 2013-02-28 | 2016-04-14 | チョン クン ダン ファーマシューティカル コーポレーション | キトサンおよび液晶形成物質を含む遺伝子伝達用組成物 |
| WO2018216792A1 (ja) * | 2017-05-26 | 2018-11-29 | 公益財団法人川崎市産業振興財団 | 血中におけるｒｎａの安定性の改善剤および投与方法 |
| US10646579B2 (en) | 2015-11-04 | 2020-05-12 | Tme Therapeutics Inc. | Complex comprising RNAi molecule and N-acetylated chitosan |
| JP2023519754A (ja) * | 2020-06-24 | 2023-05-12 | 珠海舒桐医療科技有限公司 | 膣ゲル製剤及びその調製方法 |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2362376T3 (es) * | 2006-07-07 | 2011-07-04 | Aarhus Universitet | Nanopartículas para la administración de ácido nucleico. |
| WO2010021720A1 (en) | 2008-08-19 | 2010-02-25 | Nektar Therapeutics | Conjugates of small-interfering nucleic acids |
| WO2013059617A1 (en) * | 2011-10-21 | 2013-04-25 | Ndsu Research Foundation | Liposome compositions and methods of use |
| DK2806896T3 (en) | 2012-01-27 | 2018-03-05 | Hoffmann La Roche | Chitosan covalently bound to small molecule integrin antagonist for targeted delivery |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2005533016A (ja) * | 2002-05-03 | 2005-11-04 | エフエムシー バイオポリマー エイエス | 非ウイルス性遺伝子送達システム |
| JP2005533078A (ja) * | 2002-06-20 | 2005-11-04 | バイオオーリアンス ファーマ | 少なくとも１つのポリマー及び少なくとも１つの正帯電多糖類の均一サイズのナノ粒子を含むベクター化システム及びその調製方法 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3922260A (en) * | 1973-08-24 | 1975-11-25 | Quintin P Peniston | Process for depolymerization of chitosan |
| US6184037B1 (en) | 1996-05-17 | 2001-02-06 | Genemedicine, Inc. | Chitosan related compositions and methods for delivery of nucleic acids and oligonucleotides into a cell |
| SE9904475D0 (sv) | 1999-12-08 | 1999-12-08 | Artursson | Nucleic acid delivery system |
| US20030134810A1 (en) | 2001-10-09 | 2003-07-17 | Chris Springate | Methods and compositions comprising biocompatible materials useful for the administration of therapeutic agents |
| NO317654B1 (no) | 2002-05-03 | 2004-11-29 | Stiftelsen Biopolymer | Formulering som inneholder en nukleinsyre og et kitosan, fremgangsmate for fremstilling av formuleringen, samt anvendelser derav. |
| WO2004074314A2 (en) | 2003-02-14 | 2004-09-02 | University Of South Florida | Chistosan-microparticles for ifn gene delivery |
| WO2005113770A1 (en) | 2004-05-13 | 2005-12-01 | Institut Gustave Roussy | Anti-rhoa and -rhoc sirnas and therapeutic compositions comprising them. |
| US8399025B2 (en) * | 2004-06-04 | 2013-03-19 | Board Of Regents, The University Of Texas System | Polyamine modified particles |
| CA2628313A1 (en) | 2005-11-04 | 2007-05-31 | Bio Syntech Canada Inc. | Composition and method for efficient delivery of nucleic acids to cells using chitosan |
| US8846102B2 (en) * | 2006-03-30 | 2014-09-30 | Engene, Inc. | Non-viral compositions and methods for transfecting gut cells in vivo |
| ES2362376T3 (es) * | 2006-07-07 | 2011-07-04 | Aarhus Universitet | Nanopartículas para la administración de ácido nucleico. |
-
2007
- 2007-09-14 US US11/855,193 patent/US7875449B2/en not_active Expired - Fee Related
- 2007-09-14 BR BRPI0716925-6A2A patent/BRPI0716925A2/pt not_active IP Right Cessation
- 2007-09-14 CA CA002662560A patent/CA2662560A1/en not_active Abandoned
- 2007-09-14 KR KR1020097007646A patent/KR20090058562A/ko not_active Ceased
- 2007-09-14 JP JP2009527838A patent/JP2010503640A/ja active Pending
- 2007-09-14 WO PCT/EP2007/059740 patent/WO2008031899A2/en not_active Ceased
- 2007-09-14 EP EP07803506A patent/EP2061516A2/en not_active Withdrawn
- 2007-09-14 CN CNA2007800343442A patent/CN101588821A/zh active Pending
-
2009
- 2009-03-09 IL IL197497A patent/IL197497A/en not_active IP Right Cessation
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2005533016A (ja) * | 2002-05-03 | 2005-11-04 | エフエムシー バイオポリマー エイエス | 非ウイルス性遺伝子送達システム |
| JP2005533078A (ja) * | 2002-06-20 | 2005-11-04 | バイオオーリアンス ファーマ | 少なくとも１つのポリマー及び少なくとも１つの正帯電多糖類の均一サイズのナノ粒子を含むベクター化システム及びその調製方法 |
Non-Patent Citations (1)
| Title |
|---|
| JPN6012052387; ＧＡＯ Ｓ ＥＴ ＡＬ: 'Ｔａｒｇｅｔｉｎｇ ｄｅｌｉｖｅｒｙ ｏｆ ｏｌｉｇｏｎｕｃｌｅｏｔｉｄｅ ａｎｄ ｐｌａｓｍｉｄ ＤＮＡ ｔｏ ｈｅｐａｔｏｃｙｔｅ ｖｉａ ｇａｌａｃｔｏｓｙｌａｔｅｄ ｃｈｉｔｏｓａｎ ｖｅｃｔ' ＥＵＲＯＰＥＡＮ ＪＯＵＲＮＡＬ ＯＦ ＰＨＡＲＭＡＣＥＵＴＩＣＳ ＡＮＤ ＢＩＯＰＨＡＲＭＡＣＥＵＴＩＣＳ ｖｏｌ． ６０， ｎｏ． ３, 200508, ｐａｇｅｓ ３２７-３３４ * |
Cited By (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2016511255A (ja) * | 2013-02-28 | 2016-04-14 | チョン クン ダン ファーマシューティカル コーポレーション | キトサンおよび液晶形成物質を含む遺伝子伝達用組成物 |
| US11318215B2 (en) | 2013-02-28 | 2022-05-03 | Chong Kun Dang Pharmaceutical Corp. | Composition for gene delivery comprising chitosan and liquid crystal formation material |
| US10646579B2 (en) | 2015-11-04 | 2020-05-12 | Tme Therapeutics Inc. | Complex comprising RNAi molecule and N-acetylated chitosan |
| WO2018216792A1 (ja) * | 2017-05-26 | 2018-11-29 | 公益財団法人川崎市産業振興財団 | 血中におけるｒｎａの安定性の改善剤および投与方法 |
| JPWO2018216792A1 (ja) * | 2017-05-26 | 2020-03-26 | 公益財団法人川崎市産業振興財団 | 血中におけるｒｎａの安定性の改善剤および投与方法 |
| JP7237825B2 (ja) | 2017-05-26 | 2023-03-13 | 公益財団法人川崎市産業振興財団 | 血中におけるｒｎａの安定性の改善剤および投与方法 |
| JP2023519754A (ja) * | 2020-06-24 | 2023-05-12 | 珠海舒桐医療科技有限公司 | 膣ゲル製剤及びその調製方法 |
| AU2020455080B2 (en) * | 2020-06-24 | 2024-05-23 | Zhuhai Shu Tong Medical Technology Co., Ltd. | Vaginal gel preparation and preparation method therefor |
| JP7612228B2 (ja) | 2020-06-24 | 2025-01-14 | 珠海舒桐医療科技有限公司 | 膣ゲル製剤及びその調製方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| KR20090058562A (ko) | 2009-06-09 |
| US20080131371A1 (en) | 2008-06-05 |
| IL197497A (en) | 2013-06-27 |
| CA2662560A1 (en) | 2008-03-20 |
| IL197497A0 (en) | 2009-12-24 |
| CN101588821A (zh) | 2009-11-25 |
| EP2061516A2 (en) | 2009-05-27 |
| BRPI0716925A2 (pt) | 2013-09-17 |
| WO2008031899A3 (en) | 2008-05-08 |
| US7875449B2 (en) | 2011-01-25 |
| WO2008031899A2 (en) | 2008-03-20 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7256824B2 (ja) | 粒子状製剤用の凍結保護剤 | |
| Matsumoto et al. | Environment-responsive block copolymer micelles with a disulfide cross-linked core for enhanced siRNA delivery | |
| US7875449B2 (en) | Oligonucleotide non-viral delivery systems | |
| US11007279B2 (en) | Highly stable biodegradable gene vector platforms for overcoming biological barriers | |
| AU2014300980B2 (en) | Compositions for introducing RNA into cells | |
| Nimesh | Polyethylenimine as a promising vector for targeted siRNA delivery | |
| CN103255174B (zh) | 以聚乙二醇接枝的透明质酸为外层的三元复合物及三元复合物的液体与应用 | |
| Nikitenko et al. | Non-viral delivery and therapeutic application of small interfering RNAs | |
| Jiang et al. | Nanotechnology-enabled gene delivery for cancer and other genetic diseases | |
| CN112972703B (zh) | 基因编辑纳米胶囊及其制备方法与应用 | |
| Khurana et al. | siRNA delivery using nanocarriers-an efficient tool for gene silencing | |
| JP2020172534A (ja) | 合成脳浸透遺伝子ベクターの操作 | |
| US20120076853A1 (en) | Composition for use in gene therapy | |
| US20020016304A1 (en) | Carrier for stabilizing nucleic acid | |
| Vader et al. | Polymeric carrier systems for siRNA delivery | |
| Opanasopit et al. | Nucleic acid delivery with chitosan hydroxybenzotriazole | |
| Alvarez et al. | Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment | |
| Zhang et al. | Effective mRNA Inhibition in PANC-1 Cells in Vitro Mediated via an mPEG–SeSe–PEI Delivery System | |
| CN114829615B (zh) | 包含聚丙烯亚胺的非病毒载体 | |
| RU2820713C2 (ru) | Криопротективные агенты для содержащих частицы составов | |
| TÜZMEN et al. | Attractive approaches in siRNA delivery using polymer bio-based carrier systems. | |
| Yalınca et al. | ATTRACTIVE APPROACHES in DRUG DELIVERY: BIO-BASED CARRIERS for siRNA TARGETING | |
| Mouro | Identification of critical points in colloidal delivery systems for intravenous administration and intracellular delivery of nucleic acids as therapeutic agents | |
| Hetti | Development of novel biodegradable core-shell nanoparticles as carriers for gene delivery | |
| Merkel | Delivery of Nucleic Acids Using Polymers |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20100611 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20100611 |
|
| RD03 | Notification of appointment of power of attorney |
Free format text: JAPANESE INTERMEDIATE CODE: A7423 Effective date: 20120119 |
|
| A977 | Report on retrieval |
Free format text: JAPANESE INTERMEDIATE CODE: A971007 Effective date: 20120928 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20121009 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20130312 |