KR102369736B1 - 보체 인자 b 발현을 조절하기 위한 조성물 및 방법 - Google Patents
보체 인자 b 발현을 조절하기 위한 조성물 및 방법 Download PDFInfo
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- KR102369736B1 KR102369736B1 KR1020167032652A KR20167032652A KR102369736B1 KR 102369736 B1 KR102369736 B1 KR 102369736B1 KR 1020167032652 A KR1020167032652 A KR 1020167032652A KR 20167032652 A KR20167032652 A KR 20167032652A KR 102369736 B1 KR102369736 B1 KR 102369736B1
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- 0 *[C@@](CC1)[C@@]1N Chemical compound *[C@@](CC1)[C@@]1N 0.000 description 68
- ZEVOCXOZYFLVKN-UHFFFAOYSA-N CC(NC(C1O)C(OC)OC(CO)C1O)=O Chemical compound CC(NC(C1O)C(OC)OC(CO)C1O)=O ZEVOCXOZYFLVKN-UHFFFAOYSA-N 0.000 description 2
- ZAJXBYWHQAQBBZ-UHFFFAOYSA-N CC(C(CC(CCNC(CCC=N)=O)=C)NC)=O Chemical compound CC(C(CC(CCNC(CCC=N)=O)=C)NC)=O ZAJXBYWHQAQBBZ-UHFFFAOYSA-N 0.000 description 1
- RDXDXJMEOWEVHH-KDXUFGMBSA-N CC(N[C@@H](CO[C@H](CCO)[C@@H]1O)[C@H]1O)=O Chemical compound CC(N[C@@H](CO[C@H](CCO)[C@@H]1O)[C@H]1O)=O RDXDXJMEOWEVHH-KDXUFGMBSA-N 0.000 description 1
- BEXGMGPZUNBHOF-UHFFFAOYSA-N CCC(C(C)C(C1NC(C)=O)O)OC1NC Chemical compound CCC(C(C)C(C1NC(C)=O)O)OC1NC BEXGMGPZUNBHOF-UHFFFAOYSA-N 0.000 description 1
- CHVCCUQAHLZBTL-UHFFFAOYSA-N CNC(COC)(CON)CON=C Chemical compound CNC(COC)(CON)CON=C CHVCCUQAHLZBTL-UHFFFAOYSA-N 0.000 description 1
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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
- C12N15/1137—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 enzymes
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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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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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- 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
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- 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
- A61K47/555—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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21047—Alternative-complement-pathway C3/C5 convertase (3.4.21.47), i.e. properdin factor B
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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
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- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Ophthalmology & Optometry (AREA)
- Urology & Nephrology (AREA)
- Cell Biology (AREA)
- Analytical Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461987471P | 2014-05-01 | 2014-05-01 | |
| US61/987,471 | 2014-05-01 | ||
| US201462076273P | 2014-11-06 | 2014-11-06 | |
| US62/076,273 | 2014-11-06 | ||
| PCT/US2015/028916 WO2015168635A2 (en) | 2014-05-01 | 2015-05-01 | Compositions and methods for modulating complement factor b expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20160147892A KR20160147892A (ko) | 2016-12-23 |
| KR102369736B1 true KR102369736B1 (ko) | 2022-03-02 |
Family
ID=54359510
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020167032652A Active KR102369736B1 (ko) | 2014-05-01 | 2015-05-01 | 보체 인자 b 발현을 조절하기 위한 조성물 및 방법 |
Country Status (29)
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013159108A2 (en) * | 2012-04-20 | 2013-10-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| KR102138781B1 (ko) | 2013-05-01 | 2020-07-28 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포지질단백질 c-iii 발현을 조절하는 조성물 및 방법 |
| CN105744959B (zh) * | 2013-09-13 | 2020-12-01 | Ionis制药公司 | 补体因子b的调节剂 |
| PT3137596T (pt) | 2014-05-01 | 2019-09-27 | Ionis Pharmaceuticals Inc | Composições e métodos para modulação da expressão do factor b do complemento |
| US9994855B2 (en) | 2014-05-01 | 2018-06-12 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating growth hormone receptor expression |
| RU2017114156A (ru) | 2014-10-10 | 2018-11-15 | Ф. Хоффманн-Ля Рош Аг | N-ацетилгалактозаминовые (galnac) фосфорамитиды, их конъюгаты с нуклеиновыми кислотами и их применение |
| KR20250078597A (ko) * | 2015-11-06 | 2025-06-02 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포리포프로테인 (a) 발현 조정 |
| IL291323B2 (en) | 2016-07-15 | 2023-09-01 | Am Chemicals Llc | Solid non-nucleoside supports and phosphoramidite building blocks for oligonucleotide synthesis |
| JOP20190215A1 (ar) * | 2017-03-24 | 2019-09-19 | Ionis Pharmaceuticals Inc | مُعدّلات التعبير الوراثي عن pcsk9 |
| WO2020109343A1 (en) | 2018-11-29 | 2020-06-04 | F. Hoffmann-La Roche Ag | Combination therapy for treatment of macular degeneration |
| EP3990465A1 (en) * | 2019-06-25 | 2022-05-04 | Amgen Inc. | Purification methods for carbohydrate-linked oligonucleotides |
| US12290567B2 (en) | 2019-09-03 | 2025-05-06 | Arcturus Therapeutics, Inc. | Asialoglycoprotein Receptor mediated delivery of therapeutically active conjugates |
| CN115176004A (zh) | 2019-10-22 | 2022-10-11 | 阿尔尼拉姆医药品有限公司 | 补体成分C3 iRNA组合物及其使用方法 |
| US12447173B2 (en) | 2019-12-26 | 2025-10-21 | Eisai R&D Management Co., Ltd. | Pharmaceutical composition containing double-stranded ribonucleic acid inhibiting expression of complement C5 |
| KR20230017789A (ko) * | 2020-04-30 | 2023-02-06 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 인자 B (CFB) iRNA 조성물 및 이의 사용 방법 |
| IL310180A (en) * | 2021-07-17 | 2024-03-01 | Sirnaomics Inc | products and preparations |
| IL311146A (en) | 2021-09-02 | 2024-04-01 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell |
| CA3234636A1 (en) * | 2021-10-29 | 2023-05-04 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| EP4611768A1 (en) * | 2022-11-02 | 2025-09-10 | Ionis Pharmaceuticals, Inc. | Methods for modulating complement factor b expression |
| EP4638743A2 (en) * | 2022-12-19 | 2025-10-29 | Sanegene Bio USA Inc. | Small interfering rna targeting cfb and uses thereof |
| AU2024240837A1 (en) * | 2023-03-21 | 2025-09-11 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of complement factor b (cfb) pharmaceutical compositions thereof, and methods of use |
| WO2024245424A1 (zh) * | 2023-06-02 | 2024-12-05 | 维亚臻生物技术(苏州)有限公司 | 一种用于免疫类疾病的双链核苷酸化合物及其应用 |
| CN118620964A (zh) * | 2023-06-28 | 2024-09-10 | 百奥赛图(北京)医药科技股份有限公司 | 一种cfb基因修饰的非人动物 |
| TW202519657A (zh) * | 2023-07-28 | 2025-05-16 | 大陸商蘇州炫景生物科技有限公司 | Cfb抑制劑組合物及其應用 |
| WO2025151250A2 (en) * | 2024-01-09 | 2025-07-17 | Adarx Pharmaceuticals, Inc. | Complement factor b-modulating compositions and methods of use thereof |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040249178A1 (en) | 2001-05-18 | 2004-12-09 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| WO2013166121A1 (en) | 2012-05-02 | 2013-11-07 | Merck Sharp & Dohme Corp. | Novel tetragalnac containing conjugates and methods for delivery of oligonucleotides |
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