ES2640091T3 - Compuestos de oligonucleótidos que comprenden excedentes no nucleótidos - Google Patents
Compuestos de oligonucleótidos que comprenden excedentes no nucleótidos Download PDFInfo
- Publication number
- ES2640091T3 ES2640091T3 ES11701706.1T ES11701706T ES2640091T3 ES 2640091 T3 ES2640091 T3 ES 2640091T3 ES 11701706 T ES11701706 T ES 11701706T ES 2640091 T3 ES2640091 T3 ES 2640091T3
- Authority
- ES
- Spain
- Prior art keywords
- structural unit
- nucleotide
- phosphate
- c3pi
- terminal
- 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.)
- Active
Links
- 125000003729 nucleotide group Chemical group 0.000 title abstract description 173
- 239000002773 nucleotide Substances 0.000 title abstract description 167
- 150000001875 compounds Chemical class 0.000 title description 49
- 108091034117 Oligonucleotide Proteins 0.000 title description 7
- 230000000692 anti-sense effect Effects 0.000 abstract description 64
- 229910019142 PO4 Inorganic materials 0.000 abstract description 40
- 239000010452 phosphate Substances 0.000 abstract description 40
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 abstract description 20
- 108020004707 nucleic acids Proteins 0.000 abstract description 17
- 102000039446 nucleic acids Human genes 0.000 abstract description 17
- 150000007523 nucleic acids Chemical class 0.000 abstract description 17
- -1 propylphosphopropyl Chemical group 0.000 abstract description 3
- YDEQDAFOTKIGKR-UHFFFAOYSA-N CCCOP(O)(=S)OCCCP(=O)=O Chemical compound CCCOP(O)(=S)OCCCP(=O)=O YDEQDAFOTKIGKR-UHFFFAOYSA-N 0.000 abstract description 2
- DWFOJHYJWHVTMI-UHFFFAOYSA-N hexan-3-yloxy-oxido-oxophosphanium Chemical compound C(CC)C(CC)OP(=O)=O DWFOJHYJWHVTMI-UHFFFAOYSA-N 0.000 abstract description 2
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 abstract 2
- QVLTXCYWHPZMCA-UHFFFAOYSA-N po4-po4 Chemical compound OP(O)(O)=O.OP(O)(O)=O QVLTXCYWHPZMCA-UHFFFAOYSA-N 0.000 abstract 1
- 125000002652 ribonucleotide group Chemical group 0.000 description 64
- 108091028664 Ribonucleotide Proteins 0.000 description 44
- 239000002336 ribonucleotide Substances 0.000 description 44
- 238000012986 modification Methods 0.000 description 34
- 230000004048 modification Effects 0.000 description 34
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 description 28
- 125000000217 alkyl group Chemical group 0.000 description 25
- 229910052757 nitrogen Inorganic materials 0.000 description 24
- 108020004459 Small interfering RNA Proteins 0.000 description 13
- 108020004999 messenger RNA Proteins 0.000 description 13
- 108090000623 proteins and genes Proteins 0.000 description 13
- 230000000694 effects Effects 0.000 description 12
- 239000004215 Carbon black (E152) Substances 0.000 description 9
- 229930195733 hydrocarbon Natural products 0.000 description 9
- 150000002430 hydrocarbons Chemical class 0.000 description 9
- 229910052816 inorganic phosphate Inorganic materials 0.000 description 9
- 150000003230 pyrimidines Chemical class 0.000 description 9
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 7
- 230000000295 complement effect Effects 0.000 description 7
- MHZDONKZSXBOGL-UHFFFAOYSA-N propyl dihydrogen phosphate Chemical compound CCCOP(O)(O)=O MHZDONKZSXBOGL-UHFFFAOYSA-N 0.000 description 7
- DNIAPMSPPWPWGF-VKHMYHEASA-N (+)-propylene glycol Chemical compound C[C@H](O)CO DNIAPMSPPWPWGF-VKHMYHEASA-N 0.000 description 6
- YPFDHNVEDLHUCE-UHFFFAOYSA-N 1,3-propanediol Substances OCCCO YPFDHNVEDLHUCE-UHFFFAOYSA-N 0.000 description 6
- 239000002253 acid Substances 0.000 description 6
- NBIIXXVUZAFLBC-UHFFFAOYSA-M dihydrogenphosphate Chemical compound OP(O)([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-M 0.000 description 6
- 239000000284 extract Substances 0.000 description 6
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 6
- 229920000166 polytrimethylene carbonate Polymers 0.000 description 6
- BDERNNFJNOPAEC-UHFFFAOYSA-N propan-1-ol Chemical compound CCCO BDERNNFJNOPAEC-UHFFFAOYSA-N 0.000 description 6
- ASJSAQIRZKANQN-CRCLSJGQSA-N 2-deoxy-D-ribose Chemical class OC[C@@H](O)[C@@H](O)CC=O ASJSAQIRZKANQN-CRCLSJGQSA-N 0.000 description 5
- 208000035657 Abasia Diseases 0.000 description 5
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 description 5
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 description 5
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 description 5
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 5
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 5
- HYCSHFLKPSMPGO-UHFFFAOYSA-N 3-hydroxypropyl dihydrogen phosphate Chemical compound OCCCOP(O)(O)=O HYCSHFLKPSMPGO-UHFFFAOYSA-N 0.000 description 4
- 244000070010 Erythrina variegata Species 0.000 description 4
- ATUOYWHBWRKTHZ-UHFFFAOYSA-N Propane Chemical compound CCC ATUOYWHBWRKTHZ-UHFFFAOYSA-N 0.000 description 4
- ZEXSWWSPNTYEJC-UHFFFAOYSA-N dihydroxy-propoxy-sulfanylidene-$l^{5}-phosphane Chemical compound CCCOP(O)(O)=S ZEXSWWSPNTYEJC-UHFFFAOYSA-N 0.000 description 4
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 4
- 238000000034 method Methods 0.000 description 4
- 150000004713 phosphodiesters Chemical class 0.000 description 4
- 238000003752 polymerase chain reaction Methods 0.000 description 4
- ULWHHBHJGPPBCO-UHFFFAOYSA-N propane-1,1-diol Chemical compound CCC(O)O ULWHHBHJGPPBCO-UHFFFAOYSA-N 0.000 description 4
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 3
- LNQVTSROQXJCDD-KQYNXXCUSA-N 3'-AMP Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](OP(O)(O)=O)[C@H]1O LNQVTSROQXJCDD-KQYNXXCUSA-N 0.000 description 3
- 108020004414 DNA Proteins 0.000 description 3
- KFZMGEQAYNKOFK-UHFFFAOYSA-N Isopropanol Chemical compound CC(C)O KFZMGEQAYNKOFK-UHFFFAOYSA-N 0.000 description 3
- 101710163270 Nuclease Proteins 0.000 description 3
- LNQVTSROQXJCDD-UHFFFAOYSA-N adenosine monophosphate Natural products C1=NC=2C(N)=NC=NC=2N1C1OC(CO)C(OP(O)(O)=O)C1O LNQVTSROQXJCDD-UHFFFAOYSA-N 0.000 description 3
- 125000003545 alkoxy group Chemical group 0.000 description 3
- 230000014509 gene expression Effects 0.000 description 3
- 239000002719 pyrimidine nucleotide Substances 0.000 description 3
- UOOOPKANIPLQPU-XVFCMESISA-N 3'-CMP Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@H](OP(O)(O)=O)[C@@H](CO)O1 UOOOPKANIPLQPU-XVFCMESISA-N 0.000 description 2
- 208000009304 Acute Kidney Injury Diseases 0.000 description 2
- 102100032616 Caspase-2 Human genes 0.000 description 2
- UOOOPKANIPLQPU-UHFFFAOYSA-N Cytidylic acid B Natural products O=C1N=C(N)C=CN1C1C(O)C(OP(O)(O)=O)C(CO)O1 UOOOPKANIPLQPU-UHFFFAOYSA-N 0.000 description 2
- 208000021709 Delayed Graft Function Diseases 0.000 description 2
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 2
- 101000867612 Homo sapiens Caspase-2 Proteins 0.000 description 2
- 208000033626 Renal failure acute Diseases 0.000 description 2
- IMBYBANOLUYVCT-CRCLSJGQSA-N [(2r,3s)-2-(hydroxymethyl)oxolan-3-yl] dihydrogen phosphate Chemical compound OC[C@H]1OCC[C@@H]1OP(O)(O)=O IMBYBANOLUYVCT-CRCLSJGQSA-N 0.000 description 2
- 201000011040 acute kidney failure Diseases 0.000 description 2
- 208000012998 acute renal failure Diseases 0.000 description 2
- 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 description 2
- 229960005305 adenosine Drugs 0.000 description 2
- 229940125898 compound 5 Drugs 0.000 description 2
- 239000005547 deoxyribonucleotide Substances 0.000 description 2
- 238000010369 molecular cloning Methods 0.000 description 2
- XUYJLQHKOGNDPB-UHFFFAOYSA-N phosphonoacetic acid Chemical compound OC(=O)CP(O)(O)=O XUYJLQHKOGNDPB-UHFFFAOYSA-N 0.000 description 2
- 239000001294 propane Substances 0.000 description 2
- 210000001525 retina Anatomy 0.000 description 2
- XXYIANZGUOSQHY-XLPZGREQSA-N thymidine 3'-monophosphate Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](OP(O)(O)=O)C1 XXYIANZGUOSQHY-XLPZGREQSA-N 0.000 description 2
- WRIDQFICGBMAFQ-UHFFFAOYSA-N (E)-8-Octadecenoic acid Natural products CCCCCCCCCC=CCCCCCCC(O)=O WRIDQFICGBMAFQ-UHFFFAOYSA-N 0.000 description 1
- MXHRCPNRJAMMIM-SHYZEUOFSA-N 2'-deoxyuridine Chemical compound C1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 MXHRCPNRJAMMIM-SHYZEUOFSA-N 0.000 description 1
- LQJBNNIYVWPHFW-UHFFFAOYSA-N 20:1omega9c fatty acid Natural products CCCCCCCCCCC=CCCCCCCCC(O)=O LQJBNNIYVWPHFW-UHFFFAOYSA-N 0.000 description 1
- QSBYPNXLFMSGKH-UHFFFAOYSA-N 9-Heptadecensaeure Natural products CCCCCCCC=CCCCCCCCC(O)=O QSBYPNXLFMSGKH-UHFFFAOYSA-N 0.000 description 1
- 206010001052 Acute respiratory distress syndrome Diseases 0.000 description 1
- CWOGCJRZZZRVKM-UHFFFAOYSA-N CCCC(P(=O)=O)CCOP(O)(O)=O Chemical compound CCCC(P(=O)=O)CCOP(O)(O)=O CWOGCJRZZZRVKM-UHFFFAOYSA-N 0.000 description 1
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 description 1
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 description 1
- 101100411708 Danio rerio rarga gene Proteins 0.000 description 1
- 206010011878 Deafness Diseases 0.000 description 1
- 208000003556 Dry Eye Syndromes Diseases 0.000 description 1
- 206010013774 Dry eye Diseases 0.000 description 1
- 241000196324 Embryophyta Species 0.000 description 1
- 208000010412 Glaucoma Diseases 0.000 description 1
- 206010029155 Nephropathy toxic Diseases 0.000 description 1
- ZQPPMHVWECSIRJ-UHFFFAOYSA-N Oleic acid Natural products CCCCCCCCC=CCCCCCCCC(O)=O ZQPPMHVWECSIRJ-UHFFFAOYSA-N 0.000 description 1
- 239000005642 Oleic acid Substances 0.000 description 1
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical group [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- 206010063837 Reperfusion injury Diseases 0.000 description 1
- NINIDFKCEFEMDL-UHFFFAOYSA-N Sulfur Chemical compound [S] NINIDFKCEFEMDL-UHFFFAOYSA-N 0.000 description 1
- 208000025865 Ulcer Diseases 0.000 description 1
- 208000027418 Wounds and injury Diseases 0.000 description 1
- NPRNBAPBGVERRF-ZOQUXTDFSA-N [(2r,3r,4r,5r)-5-(2,4-dioxopyrimidin-1-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-yl] dihydrogen phosphate Chemical compound CO[C@@H]1[C@H](OP(O)(O)=O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 NPRNBAPBGVERRF-ZOQUXTDFSA-N 0.000 description 1
- VXTNWQOOBHQEDS-IOSLPCCCSA-N [(2r,3r,4r,5r)-5-(6-aminopurin-9-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-yl] dihydrogen phosphate Chemical compound CO[C@@H]1[C@H](OP(O)(O)=O)[C@@H](CO)O[C@H]1N1C2=NC=NC(N)=C2N=C1 VXTNWQOOBHQEDS-IOSLPCCCSA-N 0.000 description 1
- 230000001154 acute effect Effects 0.000 description 1
- QDFHPFSBQFLLSW-KQYNXXCUSA-N adenosine 2'-phosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1OP(O)(O)=O QDFHPFSBQFLLSW-KQYNXXCUSA-N 0.000 description 1
- 150000001412 amines Chemical group 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 230000001580 bacterial effect Effects 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 210000001185 bone marrow Anatomy 0.000 description 1
- 229910052799 carbon Inorganic materials 0.000 description 1
- 238000003776 cleavage reaction Methods 0.000 description 1
- 239000011248 coating agent Substances 0.000 description 1
- 238000000576 coating method Methods 0.000 description 1
- 229940125904 compound 1 Drugs 0.000 description 1
- 229940125782 compound 2 Drugs 0.000 description 1
- 229940126214 compound 3 Drugs 0.000 description 1
- 210000004087 cornea Anatomy 0.000 description 1
- 230000006378 damage Effects 0.000 description 1
- 238000013461 design Methods 0.000 description 1
- MXHRCPNRJAMMIM-UHFFFAOYSA-N desoxyuridine Natural products C1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 MXHRCPNRJAMMIM-UHFFFAOYSA-N 0.000 description 1
- 238000001514 detection method Methods 0.000 description 1
- KQYPMOAGWMNYDC-UHFFFAOYSA-N dihydroxy-propan-2-yloxy-sulfanylidene-$l^{5}-phosphane Chemical compound CC(C)OP(O)(O)=S KQYPMOAGWMNYDC-UHFFFAOYSA-N 0.000 description 1
- 150000002148 esters Chemical class 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 238000000684 flow cytometry Methods 0.000 description 1
- MDQRDWAGHRLBPA-UHFFFAOYSA-N fluoroamine Chemical compound FN MDQRDWAGHRLBPA-UHFFFAOYSA-N 0.000 description 1
- 125000000524 functional group Chemical group 0.000 description 1
- 230000010370 hearing loss Effects 0.000 description 1
- 231100000888 hearing loss Toxicity 0.000 description 1
- 208000016354 hearing loss disease Diseases 0.000 description 1
- 210000002216 heart Anatomy 0.000 description 1
- 125000004051 hexyl group Chemical group [H]C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])* 0.000 description 1
- 230000028993 immune response Effects 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000011065 in-situ storage Methods 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 208000014674 injury Diseases 0.000 description 1
- 208000012947 ischemia reperfusion injury Diseases 0.000 description 1
- 230000000302 ischemic effect Effects 0.000 description 1
- QXJSBBXBKPUZAA-UHFFFAOYSA-N isooleic acid Natural products CCCCCCCC=CCCCCCCCCC(O)=O QXJSBBXBKPUZAA-UHFFFAOYSA-N 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 210000004185 liver Anatomy 0.000 description 1
- 210000004072 lung Anatomy 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 230000007694 nephrotoxicity Effects 0.000 description 1
- 231100000417 nephrotoxicity Toxicity 0.000 description 1
- 201000001119 neuropathy Diseases 0.000 description 1
- 230000007823 neuropathy Effects 0.000 description 1
- ZQPPMHVWECSIRJ-KTKRTIGZSA-N oleic acid Chemical compound CCCCCCCC\C=C/CCCCCCCC(O)=O ZQPPMHVWECSIRJ-KTKRTIGZSA-N 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 125000004430 oxygen atom Chemical group O* 0.000 description 1
- 210000000496 pancreas Anatomy 0.000 description 1
- 208000033808 peripheral neuropathy Diseases 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- UEZVMMHDMIWARA-UHFFFAOYSA-M phosphonate Chemical compound [O-]P(=O)=O UEZVMMHDMIWARA-UHFFFAOYSA-M 0.000 description 1
- 229910052698 phosphorus Inorganic materials 0.000 description 1
- 239000011574 phosphorus Substances 0.000 description 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 1
- 230000002685 pulmonary effect Effects 0.000 description 1
- 208000023504 respiratory system disease Diseases 0.000 description 1
- 238000003757 reverse transcription PCR Methods 0.000 description 1
- 230000007017 scission Effects 0.000 description 1
- 208000020431 spinal cord injury Diseases 0.000 description 1
- 230000000087 stabilizing effect Effects 0.000 description 1
- 230000003068 static effect Effects 0.000 description 1
- 208000003265 stomatitis Diseases 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 229910052717 sulfur Inorganic materials 0.000 description 1
- 239000011593 sulfur Substances 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 238000002054 transplantation Methods 0.000 description 1
- 231100000397 ulcer Toxicity 0.000 description 1
Classifications
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/02—Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/06—Antiglaucoma agents or miotics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3527—Other alkyl chain
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Ophthalmology & Optometry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Cardiology (AREA)
- Epidemiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Urology & Nephrology (AREA)
- Vascular Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Dermatology (AREA)
- Transplantation (AREA)
- Neurosurgery (AREA)
- Pulmonology (AREA)
- Neurology (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US29287810P | 2010-01-07 | 2010-01-07 | |
| US292878P | 2010-01-07 | ||
| WOPCT/US2010/049047 | 2010-09-16 | ||
| PCT/US2010/049047 WO2011084193A1 (en) | 2010-01-07 | 2010-09-16 | Oligonucleotide compounds comprising non-nucleotide overhangs |
| PCT/US2011/020298 WO2011085056A1 (en) | 2010-01-07 | 2011-01-06 | Oligonucleotide compounds comprising non-nucleotide overhangs |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2640091T3 true ES2640091T3 (es) | 2017-10-31 |
Family
ID=44305677
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES11701706.1T Active ES2640091T3 (es) | 2010-01-07 | 2011-01-06 | Compuestos de oligonucleótidos que comprenden excedentes no nucleótidos |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US8859751B2 (enExample) |
| EP (1) | EP2521783B1 (enExample) |
| JP (1) | JP5824463B2 (enExample) |
| CN (1) | CN102770541B (enExample) |
| ES (1) | ES2640091T3 (enExample) |
| WO (2) | WO2011084193A1 (enExample) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5685267B2 (ja) | 2009-12-09 | 2015-03-18 | 日東電工株式会社 | Ｈｓｐ４７発現の調節 |
| RU2012153703A (ru) * | 2010-06-24 | 2014-07-27 | Кварк Фармасьютикалс, Инк. | Соединения двухцепочечных рнк к rhoa и их применение |
| EP2631291B1 (en) | 2010-10-22 | 2019-05-15 | OliX Pharmaceuticals, Inc. | Nucleic acid molecules inducing rna interference, and uses thereof |
| US20130324591A1 (en) | 2010-12-06 | 2013-12-05 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising positional modifications |
| WO2012118911A1 (en) | 2011-03-03 | 2012-09-07 | Quark Pharmaceuticals, Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| US9796979B2 (en) | 2011-03-03 | 2017-10-24 | Quark Pharmaceuticals Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| EP2773758B1 (en) | 2011-11-03 | 2017-06-07 | Quark Pharmaceuticals, Inc. | Compositions for use in neuroprotection |
| WO2013070821A1 (en) | 2011-11-08 | 2013-05-16 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the nervous system |
| CN104080480A (zh) | 2012-01-01 | 2014-10-01 | 奇比艾企业有限公司 | 用于选择性递送治疗剂和诊断剂的endo180靶向微粒 |
| US9382542B2 (en) * | 2012-01-04 | 2016-07-05 | Quark Pharmaceuticals, Inc. | Double-stranded RNA compounds to CASP2 and uses thereof |
| US9707235B1 (en) * | 2012-01-13 | 2017-07-18 | University Of Kentucky Research Foundation | Protection of cells from degeneration and treatment of geographic atrophy |
| DK2853597T3 (en) | 2012-05-22 | 2019-04-08 | Olix Pharmaceuticals Inc | RNA INTERFERENCE-INducing NUCLEIC ACID MOLECULES WITH CELL PENETENING EQUIPMENT AND USE THEREOF |
| AU2013315524B2 (en) | 2012-09-12 | 2019-01-31 | Quark Pharmaceuticals, Inc. | Double-stranded oligonucleotide molecules to p53 and methods of use thereof |
| DK2895607T3 (da) | 2012-09-12 | 2021-05-25 | Quark Pharmaceuticals Inc | Dobbeltstrengede oligonukleotidmolekyler til ddit4 og fremgangsmåder til anvendelse deraf |
| US10006024B2 (en) * | 2012-11-29 | 2018-06-26 | Gifu University | RNA interference agent, method for producing same, and use therefor |
| EP3027223A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| US9889200B2 (en) | 2013-07-31 | 2018-02-13 | Qbi Enterprises Ltd. | Sphingolipid-polyalkylamine-oligonucleotide compounds |
| KR101696704B1 (ko) | 2013-12-17 | 2017-01-16 | 주식회사 인코드젠 | 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도 |
| LT3149172T (lt) | 2014-05-29 | 2018-09-10 | Quark Pharmaceuticals, Inc. | Būdai ir kompozicijos, skirti organų išeminės reperfuzijos pažeidimo prevencijai |
| WO2016149020A1 (en) * | 2015-03-17 | 2016-09-22 | Arrowhead Research Corporation | Rna interference agents |
| SI3350333T1 (sl) | 2015-09-17 | 2022-04-29 | ModernaTX,Inc. | Polinukleotidi, ki zajemajo stabilizacijsko repno regijo |
| EP3377630A4 (en) | 2015-11-16 | 2020-01-01 | Olix Pharmaceuticals, Inc. | Treatment of age-related macular degeneration using rna complexes that target myd88 or tlr3 |
| JP6944942B2 (ja) | 2016-02-02 | 2021-10-06 | オリックス ファーマシューティカルズ，インコーポレーテッド | ＩＬ４Ｒα、ＴＲＰＡ１、またはＦ２ＲＬ１を標的とするＲＮＡ複合体を用いたアトピー性皮膚炎および喘息の治療 |
| EP3411481A4 (en) | 2016-02-02 | 2020-02-26 | Olix Pharmaceuticals, Inc. | TREATMENT OF DISEASES ASSOCIATED WITH ANGIOGENESIS USING ANGPT2 AND PDGFB TARGETED RNA COMPLEXES |
| WO2017178883A2 (en) | 2016-04-11 | 2017-10-19 | Olix Pharmaceuticals, Inc. | Treatment of idiopathic pulmonary fibrosis using rna complexes that target connective tissue growth factor |
| KR101916652B1 (ko) | 2016-06-29 | 2018-11-08 | 올릭스 주식회사 | 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도 |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| EP3580339A4 (en) | 2017-02-10 | 2020-12-23 | Research & Business Foundation Sungkyunkwan University | LONG DOUBLE STRAND RNA FOR RNA INTERFERENCE |
| WO2020191177A1 (en) * | 2019-03-21 | 2020-09-24 | Sudhir Agrawal | Antisense oligonucleotides for allele specificity |
| AU2020450890B2 (en) * | 2020-05-26 | 2025-04-10 | Olix Pharmaceuticals, Inc. | RNAi agent targeting MyD88 and use thereof |
| JP2024504503A (ja) * | 2021-01-30 | 2024-01-31 | イー セラピューティクス パブリック リミテッド カンパニー | 脱塩基ヌクレオチドを含有する核酸 |
| WO2022162155A1 (en) * | 2021-01-30 | 2022-08-04 | E-Therapeutics Plc | Nucleic acids containing abasic nucleotides |
| WO2023059948A1 (en) * | 2021-10-08 | 2023-04-13 | E-Therapeutics Plc | Nucleic acids containing abasic nucleosides |
| EP4562148A1 (en) * | 2022-07-27 | 2025-06-04 | E-Therapeutics plc | Nucleic acid compounds |
| EP4562147A1 (en) * | 2022-07-27 | 2025-06-04 | E-Therapeutics plc | Nucleic acid compounds |
| KR20250043473A (ko) * | 2022-07-27 | 2025-03-28 | 이-테라퓨틱스 피엘씨 | 핵산 화합물 |
| WO2024165571A2 (en) * | 2023-02-06 | 2024-08-15 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| WO2025125576A2 (en) * | 2023-12-15 | 2025-06-19 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| EP4686757A1 (en) * | 2024-07-31 | 2026-02-04 | e-therapeutics PLC | Inhibitors of expression and/or function |
Family Cites Families (84)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4475196A (en) | 1981-03-06 | 1984-10-02 | Zor Clair G | Instrument for locating faults in aircraft passenger reading light and attendant call control system |
| US4447233A (en) | 1981-04-10 | 1984-05-08 | Parker-Hannifin Corporation | Medication infusion pump |
| US4439196A (en) | 1982-03-18 | 1984-03-27 | Merck & Co., Inc. | Osmotic drug delivery system |
| US4447224A (en) | 1982-09-20 | 1984-05-08 | Infusaid Corporation | Variable flow implantable infusion apparatus |
| US4487603A (en) | 1982-11-26 | 1984-12-11 | Cordis Corporation | Implantable microinfusion pump system |
| US4486194A (en) | 1983-06-08 | 1984-12-04 | James Ferrara | Therapeutic device for administering medicaments through the skin |
| US4666828A (en) | 1984-08-15 | 1987-05-19 | The General Hospital Corporation | Test for Huntington's disease |
| US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
| US4801531A (en) | 1985-04-17 | 1989-01-31 | Biotechnology Research Partners, Ltd. | Apo AI/CIII genomic polymorphisms predictive of atherosclerosis |
| US4959217A (en) | 1986-05-22 | 1990-09-25 | Syntex (U.S.A.) Inc. | Delayed/sustained release of macromolecules |
| US4925678A (en) | 1987-04-01 | 1990-05-15 | Ranney David F | Endothelial envelopment drug carriers |
| US5080646A (en) | 1988-10-03 | 1992-01-14 | Alza Corporation | Membrane for electrotransport transdermal drug delivery |
| US5272057A (en) | 1988-10-14 | 1993-12-21 | Georgetown University | Method of detecting a predisposition to cancer by the use of restriction fragment length polymorphism of the gene for human poly (ADP-ribose) polymerase |
| US5270030A (en) | 1988-12-29 | 1993-12-14 | Bio-Technology General Corp. | Fibrin binding domain polypeptide and method of producing |
| US5703055A (en) | 1989-03-21 | 1997-12-30 | Wisconsin Alumni Research Foundation | Generation of antibodies through lipid mediated DNA delivery |
| US5192659A (en) | 1989-08-25 | 1993-03-09 | Genetype Ag | Intron sequence analysis method for detection of adjacent and remote locus alleles as haplotypes |
| US5167616A (en) | 1989-12-14 | 1992-12-01 | Alza Corporation | Iontophoretic delivery method |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| US5225182A (en) | 1991-10-31 | 1993-07-06 | Sharma Yash P | Vectored drug delivery system using a cephaloplastin linking agent and a methed of using the system |
| KR940703846A (ko) | 1991-12-24 | 1994-12-12 | 비. 린네 파샬 | 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides) |
| WO1993023569A1 (en) | 1992-05-11 | 1993-11-25 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for inhibiting viral replication |
| US6235886B1 (en) | 1993-09-03 | 2001-05-22 | Isis Pharmaceuticals, Inc. | Methods of synthesis and use |
| US5593972A (en) | 1993-01-26 | 1997-01-14 | The Wistar Institute | Genetic immunization |
| WO1995006731A2 (en) | 1993-09-02 | 1995-03-09 | Ribozyme Pharmaceuticals, Inc. | Non-nucleotide containing enzymatic nucleic acid |
| US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
| US20050042647A1 (en) | 1996-06-06 | 2005-02-24 | Baker Brenda F. | Phosphorous-linked oligomeric compounds and their use in gene modulation |
| US20080119427A1 (en) | 1996-06-06 | 2008-05-22 | Isis Pharmaceuticals, Inc. | Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation |
| US5898031A (en) | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
| US20050032067A1 (en) | 2002-11-05 | 2005-02-10 | Prakash Thazha P. | Non-phosphorous-linked oligomeric compounds and their use in gene modulation |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6251666B1 (en) | 1997-03-31 | 2001-06-26 | Ribozyme Pharmaceuticals, Inc. | Nucleic acid catalysts comprising L-nucleotide analogs |
| CA2303299C (en) | 1997-09-12 | 2016-02-23 | Exiqon A/S | Oligonucleotide analogues |
| US6506559B1 (en) | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
| WO2000044914A1 (en) | 1999-01-28 | 2000-08-03 | Medical College Of Georgia Research Institute, Inc. | Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna |
| DE19956568A1 (de) | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
| PT1152009E (pt) | 1999-02-12 | 2005-03-31 | Sankyo Co | Novos analogos de nucleosidos e oligonucleotidos |
| WO2000049035A1 (en) | 1999-02-19 | 2000-08-24 | The General Hospital Corporation | Gene silencing |
| JP2000290185A (ja) * | 1999-04-08 | 2000-10-17 | Sankyo Co Ltd | Ｔｇｇｇ配列を持つ修飾オリゴデオキシリボヌクレオチドを含有する抗エイズ剤 |
| CA2370628A1 (en) | 1999-04-21 | 2000-10-26 | American Home Products Corporation | Methods and compositions for inhibiting the function of polynucleotide sequences |
| EP1235842A4 (en) | 1999-10-15 | 2003-04-23 | Univ Massachusetts | Rna interference pathway genes as tools for targeted genetic interference |
| GB9925459D0 (en) | 1999-10-27 | 1999-12-29 | Plant Bioscience Ltd | Gene silencing |
| DE60041452D1 (de) | 1999-11-02 | 2009-03-12 | Novartis Vaccines & Diagnostic | CpG REZEPTOR (CpG-R) UND DARAUF BEZOGENE METHODEN |
| GB9927444D0 (en) | 1999-11-19 | 2000-01-19 | Cancer Res Campaign Tech | Inhibiting gene expression |
| US7829693B2 (en) | 1999-11-24 | 2010-11-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| US20050020525A1 (en) | 2002-02-20 | 2005-01-27 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| WO2003070918A2 (en) | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | Rna interference by modified short interfering nucleic acid |
| US8202979B2 (en) | 2002-02-20 | 2012-06-19 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid |
| WO2001068836A2 (en) | 2000-03-16 | 2001-09-20 | Genetica, Inc. | Methods and compositions for rna interference |
| ATE450621T2 (de) | 2000-03-30 | 2009-12-15 | Whitehead Biomedical Inst | Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind |
| US6693187B1 (en) | 2000-10-17 | 2004-02-17 | Lievre Cornu Llc | Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge |
| KR100872437B1 (ko) | 2000-12-01 | 2008-12-05 | 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. | Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자 |
| US20040019001A1 (en) | 2002-02-20 | 2004-01-29 | Mcswiggen James A. | RNA interference mediated inhibition of protein typrosine phosphatase-1B (PTP-1B) gene expression using short interfering RNA |
| US20070032441A1 (en) | 2001-05-18 | 2007-02-08 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina) |
| ES2401326T3 (es) | 2001-11-21 | 2013-04-18 | Astellas Pharma Inc. | Procedimiento para inhibir la expresión génica |
| WO2003064621A2 (en) | 2002-02-01 | 2003-08-07 | Ambion, Inc. | HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES |
| US20050096289A1 (en) * | 2002-02-07 | 2005-05-05 | Hans Prydz | Methods and compositions for modulating tissue factor |
| ATE449846T1 (de) * | 2002-02-07 | 2009-12-15 | Sirnasense As | Post-transcriptional silencing der gewebefaktor expression durch kurze interferierende rnas |
| AU2003207708A1 (en) | 2002-02-20 | 2003-09-09 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of map kinase genes |
| ES2280826T5 (es) | 2002-08-05 | 2017-08-03 | Silence Therapeutics Gmbh | Nuevas formas adicionales de moléculas de ARN de interferencia |
| US9150605B2 (en) | 2002-11-05 | 2015-10-06 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
| JP2006507841A (ja) | 2002-11-14 | 2006-03-09 | ダーマコン， インコーポレイテッド | 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ |
| US20050233342A1 (en) | 2003-03-07 | 2005-10-20 | Muthiah Manoharan | Methods of preventing off-target gene silencing |
| US8653252B2 (en) | 2003-03-21 | 2014-02-18 | Santaris Pharma A/S | Short interfering RNA (siRNA) analogues |
| AU2004252442B2 (en) | 2003-06-02 | 2010-04-08 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of FNAi |
| ES2712695T3 (es) | 2003-06-02 | 2019-05-14 | Univ Massachusetts | Métodos y composiciones para controlar la eficacia de la silenciación del ARN |
| US20060241072A1 (en) | 2003-06-20 | 2006-10-26 | Isis Pharmaceuticals, Inc. | Oligomeric compounds for use in gene modulation |
| KR101147147B1 (ko) | 2004-04-01 | 2012-05-25 | 머크 샤프 앤드 돔 코포레이션 | Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드 |
| JP5192234B2 (ja) | 2004-08-10 | 2013-05-08 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 化学修飾オリゴヌクレオチド |
| JP4468989B2 (ja) | 2004-08-16 | 2010-05-26 | クアーク・ファーマスーティカルス、インコーポレイテッド | Ｒｔｐ８０１阻害剤の治療への使用 |
| US20110313024A1 (en) | 2004-08-20 | 2011-12-22 | Leonid Beigelman | RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| EP1812569A2 (en) | 2004-11-08 | 2007-08-01 | K.U. Leuven Research and Development | Modified nucleosides for rna interference |
| US20090176725A1 (en) * | 2005-08-17 | 2009-07-09 | Sirna Therapeutics Inc. | Chemically modified short interfering nucleic acid molecules that mediate rna interference |
| WO2007031877A2 (en) * | 2005-09-16 | 2007-03-22 | Coley Pharmaceutical Gmbh | Modulation of immunostimulatory properties of short interfering ribonucleic acid (sirna) by nucleotide modification |
| WO2007087451A2 (en) | 2006-01-25 | 2007-08-02 | University Of Massachusetts | Compositions and methods for enhancing discriminatory rna interference |
| GB0608838D0 (en) | 2006-05-04 | 2006-06-14 | Novartis Ag | Organic compounds |
| CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| JP2010507387A (ja) | 2006-10-25 | 2010-03-11 | クアーク・ファーマスーティカルス、インコーポレイテッド | 新規のｓｉＲＮＡおよびその使用方法 |
| WO2008104978A2 (en) | 2007-02-28 | 2008-09-04 | Quark Pharmaceuticals, Inc. | Novel sirna structures |
| US7812002B2 (en) | 2007-03-21 | 2010-10-12 | Quark Pharmaceuticals, Inc. | Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer |
| CN103898110A (zh) | 2007-10-03 | 2014-07-02 | 夸克制药公司 | 新siRNA结构 |
| US8188060B2 (en) * | 2008-02-11 | 2012-05-29 | Dharmacon, Inc. | Duplex oligonucleotides with enhanced functionality in gene regulation |
| DK2504435T3 (da) | 2009-11-26 | 2019-12-09 | Quark Pharmaceuticals Inc | Sirna-forbindelser omfattende terminale substitutioner |
| JP5685267B2 (ja) | 2009-12-09 | 2015-03-18 | 日東電工株式会社 | Ｈｓｐ４７発現の調節 |
| US20130324591A1 (en) | 2010-12-06 | 2013-12-05 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising positional modifications |
-
2010
- 2010-09-16 WO PCT/US2010/049047 patent/WO2011084193A1/en not_active Ceased
-
2011
- 2011-01-06 EP EP11701706.1A patent/EP2521783B1/en not_active Not-in-force
- 2011-01-06 CN CN201180011234.0A patent/CN102770541B/zh not_active Expired - Fee Related
- 2011-01-06 ES ES11701706.1T patent/ES2640091T3/es active Active
- 2011-01-06 JP JP2012548108A patent/JP5824463B2/ja not_active Expired - Fee Related
- 2011-01-06 WO PCT/US2011/020298 patent/WO2011085056A1/en not_active Ceased
- 2011-01-06 US US13/521,033 patent/US8859751B2/en active Active
-
2014
- 2014-09-15 US US14/485,828 patent/US9249414B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| US20150159154A1 (en) | 2015-06-11 |
| CN102770541A (zh) | 2012-11-07 |
| HK1178566A1 (zh) | 2013-09-13 |
| WO2011085056A1 (en) | 2011-07-14 |
| WO2011084193A1 (en) | 2011-07-14 |
| US8859751B2 (en) | 2014-10-14 |
| US20130035368A1 (en) | 2013-02-07 |
| US9249414B2 (en) | 2016-02-02 |
| EP2521783B1 (en) | 2017-08-09 |
| JP2013516190A (ja) | 2013-05-13 |
| CN102770541B (zh) | 2016-03-09 |
| EP2521783A1 (en) | 2012-11-14 |
| JP5824463B2 (ja) | 2015-11-25 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7660154B2 (ja) | アンジオポエチン様３（ＡＮＧＰＴＬ３）の発現を阻害するためのＲＮＡｉ剤および組成物、ならびに使用方法 | |
| JP7245254B2 (ja) | 細胞におけるｌｐａの発現を抑制するための核酸 | |
| ES2562499T3 (es) | Modulación de la expresión de HSP47 | |
| JP7245328B2 (ja) | 細胞におけるｌｐａの発現を抑制するための核酸 | |
| ES2690538T3 (es) | Composiciones y métodos para inhibir la expresión génica del virus de la hepatitis B | |
| CA3163322A1 (en) | Rnai constructs and methods for inhibiting lpa expression | |
| BR112021011043A2 (pt) | Construtos de rnai quimicamente modificados e seus usos | |
| ES2875558T3 (es) | Métodos y composiciones para la inhibición específica de glicolato oxidasa (HAO1) por ARN de doble cadena | |
| JP2023545502A (ja) | リポタンパク質（ａ）を阻害するためのｒｎａ組成物および方法 | |
| CN104411831B (zh) | 寡核苷酸类似物的硼酸缀合物 | |
| JP2016513976A5 (enExample) | ||
| CN109415732B (zh) | 用于调节htra1表达的反义寡核苷酸 | |
| KR20220069103A (ko) | 최소 플루오린 함량을 갖는 작은 간섭 rna의 화학적 변형 | |
| CN113166760A (zh) | 用于抑制angptl8的新型rna组合物和方法 | |
| CN110832077A (zh) | 用于抑制α-ENaC表达的RNAi剂及使用方法 | |
| CN102089429A (zh) | 用于抑制TGF-β受体基因表达的组合物和方法 | |
| JPWO2017068790A1 (ja) | 核酸複合体 | |
| TW202003842A (zh) | 髮夾型單股ｒｎａ分子之製造方法 | |
| WO2019092282A1 (en) | Nucleic acids for inhibiting expression of aldh2 in a cell | |
| US20240392299A1 (en) | Treatment of mst1r related diseases and disorders | |
| TW201119681A (en) | Compositions and methods for inhibiting expression of KIF10 genes | |
| EP4396347B1 (en) | Chimeric complex and therapeutic uses thereof | |
| RU2822093C1 (ru) | Нуклеиновые кислоты для ингибирования экспрессии lpa в клетке | |
| WO2026002138A1 (zh) | 特定修饰的双链寡核苷酸及其组合物 | |
| JP2022120380A (ja) | ｍｉＲＮＡ１３３－ｂ誘導体及びその利用 |