BR112019021359A2 - moléculas, composições, compostos, métodos para administrar um sirna, para preparar um composto e para tratar uma infecção, composto ou sal, conjugado de galnac e uso de um composto - Google Patents
moléculas, composições, compostos, métodos para administrar um sirna, para preparar um composto e para tratar uma infecção, composto ou sal, conjugado de galnac e uso de um composto Download PDFInfo
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- BR112019021359A2 BR112019021359A2 BR112019021359-7A BR112019021359A BR112019021359A2 BR 112019021359 A2 BR112019021359 A2 BR 112019021359A2 BR 112019021359 A BR112019021359 A BR 112019021359A BR 112019021359 A2 BR112019021359 A2 BR 112019021359A2
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- compound
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- 0 C*(CCC1)C1NC Chemical compound C*(CCC1)C1NC 0.000 description 46
- GIJYOKFIUTWACT-UHFFFAOYSA-N CC(C(C(OCC(C)(C)COCC(C)(C)CN)OC1COC(C)=O)NC(C)=O)C1OC(C)=O Chemical compound CC(C(C(OCC(C)(C)COCC(C)(C)CN)OC1COC(C)=O)NC(C)=O)C1OC(C)=O GIJYOKFIUTWACT-UHFFFAOYSA-N 0.000 description 1
- RFURTYMUXRORJS-SLZIBOOSSA-N CC(C)(CN)COCC(C)(C)OC([C@@H](C1)NC(C)=O)O[C@H](COC(C)=O)[C@@H]1OC(C)=O Chemical compound CC(C)(CN)COCC(C)(C)OC([C@@H](C1)NC(C)=O)O[C@H](COC(C)=O)[C@@H]1OC(C)=O RFURTYMUXRORJS-SLZIBOOSSA-N 0.000 description 1
- FGFVVIGVZTTXAG-UHFFFAOYSA-N CC(NC(C1OC(C)=O)C(OCCOCCOCCOCCNN)OC(COC(C)=O)C1OC(C)=O)=O Chemical compound CC(NC(C1OC(C)=O)C(OCCOCCOCCOCCNN)OC(COC(C)=O)C1OC(C)=O)=O FGFVVIGVZTTXAG-UHFFFAOYSA-N 0.000 description 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N CC(N[C@H]([C@H]1O)C(O)O[C@H](CO)[C@@H]1O)=O Chemical compound CC(N[C@H]([C@H]1O)C(O)O[C@H](CO)[C@@H]1O)=O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 description 1
- HUQNDNAQVPCTFV-RQICVUQASA-N CC(N[C@H]([C@H]1OC(C)=O)C(O)O[C@H](COC(C)=O)[C@@H]1OC(C)=O)=O Chemical compound CC(N[C@H]([C@H]1OC(C)=O)C(O)O[C@H](COC(C)=O)[C@@H]1OC(C)=O)=O HUQNDNAQVPCTFV-RQICVUQASA-N 0.000 description 1
- JDVWBJYXWBLBIY-UHFFFAOYSA-N CCCC(NCCOCCOCCOCCC(C)C)=O Chemical compound CCCC(NCCOCCOCCOCCC(C)C)=O JDVWBJYXWBLBIY-UHFFFAOYSA-N 0.000 description 1
- FZMVESZPIWQXHB-PFGBXZAXSA-N CC[C@H]([C@@H](C[C@H]1NC(C)=O)OC(C)=O)OC1OC(C)=O Chemical compound CC[C@H]([C@@H](C[C@H]1NC(C)=O)OC(C)=O)OC1OC(C)=O FZMVESZPIWQXHB-PFGBXZAXSA-N 0.000 description 1
- KJJFOZSLZICFGY-NSHDSACASA-N CN[C@@H](Cc(cc1)ccc1O)C(NCCO)=O Chemical compound CN[C@@H](Cc(cc1)ccc1O)C(NCCO)=O KJJFOZSLZICFGY-NSHDSACASA-N 0.000 description 1
- BAUKSGYHLZQETQ-UHFFFAOYSA-N COCC(C(C(C1)O)C(F)(F)F)N1I Chemical compound COCC(C(C(C1)O)C(F)(F)F)N1I BAUKSGYHLZQETQ-UHFFFAOYSA-N 0.000 description 1
- BVRAEWOKXYTDPM-UHFFFAOYSA-N COCC(C(CC1)CN1I)O Chemical compound COCC(C(CC1)CN1I)O BVRAEWOKXYTDPM-UHFFFAOYSA-N 0.000 description 1
- JOBMHODEQYWEDF-UHFFFAOYSA-N COCC(C(CCC1)N1I)O Chemical compound COCC(C(CCC1)N1I)O JOBMHODEQYWEDF-UHFFFAOYSA-N 0.000 description 1
- QLDWNTKBKWBOIY-UHFFFAOYSA-N COCC(CC1NI)CC1O Chemical compound COCC(CC1NI)CC1O QLDWNTKBKWBOIY-UHFFFAOYSA-N 0.000 description 1
- OOIPYWZLUNCIIQ-UHFFFAOYSA-N COCCC(CC1)(CC1O)NI Chemical compound COCCC(CC1)(CC1O)NI OOIPYWZLUNCIIQ-UHFFFAOYSA-N 0.000 description 1
- TXDUEWONXCEAKV-RQJHMYQMSA-N C[C@H]([C@@H](C(NCCOC)=O)NC)O Chemical compound C[C@H]([C@@H](C(NCCOC)=O)NC)O TXDUEWONXCEAKV-RQJHMYQMSA-N 0.000 description 1
- ZTFLEUHOAYGPJO-HFRXGUQJSA-N C[C@H]([C@@H]([C@@H]([C@H]1NC(C)=O)OC(C)=O)OC(C)=O)OC1OC(C)(C)CCO Chemical compound C[C@H]([C@@H]([C@@H]([C@H]1NC(C)=O)OC(C)=O)OC(C)=O)OC1OC(C)(C)CCO ZTFLEUHOAYGPJO-HFRXGUQJSA-N 0.000 description 1
- RIHQRXXGQXZUCM-UHFFFAOYSA-N NC1OC1C1CC1 Chemical compound NC1OC1C1CC1 RIHQRXXGQXZUCM-UHFFFAOYSA-N 0.000 description 1
- QFHKFGOUFKUPNX-UHFFFAOYSA-N OCC(C1O)OCCC1O Chemical compound OCC(C1O)OCCC1O QFHKFGOUFKUPNX-UHFFFAOYSA-N 0.000 description 1
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- C—CHEMISTRY; METALLURGY
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- 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
- C12N15/1131—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 against viruses
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- A—HUMAN NECESSITIES
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- 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
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- 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
- A61K47/54—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 compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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- 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
- A61K47/54—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 compound
- A61K47/554—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 compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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- 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
-
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
- C07H15/02—Acyclic radicals, not substituted by cyclic structures
- C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
- C07H15/08—Polyoxyalkylene derivatives
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J63/00—Steroids in which the cyclopenta(a)hydrophenanthrene skeleton has been modified by expansion of only one ring by one or two atoms
- C07J63/008—Expansion of ring D by one atom, e.g. D homo steroids
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- 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
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- 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
- C12N2310/3521—Methyl
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- Wood Science & Technology (AREA)
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762484247P | 2017-04-11 | 2017-04-11 | |
| US62/484,247 | 2017-04-11 | ||
| US201762525071P | 2017-06-26 | 2017-06-26 | |
| US62/525,071 | 2017-06-26 | ||
| PCT/US2018/026918 WO2018191278A2 (en) | 2017-04-11 | 2018-04-10 | Targeted compositions |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112019021359A2 true BR112019021359A2 (pt) | 2020-05-05 |
Family
ID=62063656
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112019021359-7A BR112019021359A2 (pt) | 2017-04-11 | 2018-04-10 | moléculas, composições, compostos, métodos para administrar um sirna, para preparar um composto e para tratar uma infecção, composto ou sal, conjugado de galnac e uso de um composto |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US11427823B2 (enExample) |
| EP (2) | EP3610017A2 (enExample) |
| JP (3) | JP2020516296A (enExample) |
| KR (3) | KR20250145702A (enExample) |
| CN (2) | CN110741087B (enExample) |
| AU (2) | AU2018250553B2 (enExample) |
| BR (1) | BR112019021359A2 (enExample) |
| CA (1) | CA3059426A1 (enExample) |
| MA (1) | MA52546A (enExample) |
| MX (3) | MX2019012280A (enExample) |
| NZ (1) | NZ757927A (enExample) |
| PH (1) | PH12019502333A1 (enExample) |
| SG (1) | SG11201909433XA (enExample) |
| TW (2) | TW202413641A (enExample) |
| WO (1) | WO2018191278A2 (enExample) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA45478A (fr) * | 2016-04-11 | 2019-02-20 | Arbutus Biopharma Corp | Compositions de conjugués d'acides nucléiques ciblés |
| NZ757927A (en) | 2017-04-11 | 2023-07-28 | Arbutus Biopharma Corp | Targeted compositions |
| JP7360716B2 (ja) | 2017-12-01 | 2023-10-13 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、当該核酸を含む組成物および複合体ならびに調製方法と使用 |
| EP3719128B1 (en) | 2017-12-01 | 2025-01-15 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof |
| EP3718572B1 (en) | 2017-12-01 | 2024-07-31 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method and use |
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