JP6637442B2 - 補体b因子発現を調節するための組成物及び方法 - Google Patents
補体b因子発現を調節するための組成物及び方法 Download PDFInfo
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- JP6637442B2 JP6637442B2 JP2016565465A JP2016565465A JP6637442B2 JP 6637442 B2 JP6637442 B2 JP 6637442B2 JP 2016565465 A JP2016565465 A JP 2016565465A JP 2016565465 A JP2016565465 A JP 2016565465A JP 6637442 B2 JP6637442 B2 JP 6637442B2
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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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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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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
- 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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- 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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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
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- 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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- C12N2310/00—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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- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
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- Biotechnology (AREA)
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- Animal Behavior & Ethology (AREA)
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- 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)
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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 |
|---|---|---|---|
| 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019228789A Division JP7001663B2 (ja) | 2014-05-01 | 2019-12-19 | 補体b因子発現を調節するための組成物及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017514489A JP2017514489A (ja) | 2017-06-08 |
| JP2017514489A5 JP2017514489A5 (enExample) | 2018-06-14 |
| JP6637442B2 true JP6637442B2 (ja) | 2020-01-29 |
Family
ID=54359510
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016565465A Active JP6637442B2 (ja) | 2014-05-01 | 2015-05-01 | 補体b因子発現を調節するための組成物及び方法 |
| JP2019228789A Active JP7001663B2 (ja) | 2014-05-01 | 2019-12-19 | 補体b因子発現を調節するための組成物及び方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019228789A Active JP7001663B2 (ja) | 2014-05-01 | 2019-12-19 | 補体b因子発現を調節するための組成物及び方法 |
Country Status (29)
| Country | Link |
|---|---|
| US (4) | US10280423B2 (enExample) |
| EP (3) | EP3608406B1 (enExample) |
| JP (2) | JP6637442B2 (enExample) |
| KR (1) | KR102369736B1 (enExample) |
| CN (2) | CN106232804B (enExample) |
| AU (1) | AU2015252858C1 (enExample) |
| BR (1) | BR112016022593B1 (enExample) |
| CA (1) | CA2943894A1 (enExample) |
| CL (2) | CL2016002764A1 (enExample) |
| CR (2) | CR20200228A (enExample) |
| DK (1) | DK3137596T3 (enExample) |
| DO (1) | DOP2016000291A (enExample) |
| EA (1) | EA036496B1 (enExample) |
| ES (2) | ES2941742T3 (enExample) |
| HR (1) | HRP20191664T1 (enExample) |
| HU (1) | HUE046272T2 (enExample) |
| IL (2) | IL247883B (enExample) |
| LT (1) | LT3137596T (enExample) |
| MX (1) | MX380866B (enExample) |
| PE (2) | PE20190626A1 (enExample) |
| PH (1) | PH12016502062B1 (enExample) |
| PL (2) | PL3608406T3 (enExample) |
| PT (1) | PT3137596T (enExample) |
| RS (1) | RS59182B1 (enExample) |
| RU (1) | RU2701645C2 (enExample) |
| SG (2) | SG11201608502TA (enExample) |
| SI (1) | SI3137596T1 (enExample) |
| UA (1) | UA121656C2 (enExample) |
| WO (1) | WO2015168635A2 (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3336189A1 (en) * | 2012-04-20 | 2018-06-20 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| PL2992098T3 (pl) | 2013-05-01 | 2019-09-30 | Ionis Pharmaceuticals, Inc. | Kompozycje i sposoby modulowania ekspresji hbv i ttr |
| HRP20191600T1 (hr) * | 2013-09-13 | 2019-11-29 | Ionis Pharmaceuticals Inc | Modulatori faktora b komplementa |
| KR102369736B1 (ko) | 2014-05-01 | 2022-03-02 | 아이오니스 파마수티컬즈, 인코포레이티드 | 보체 인자 b 발현을 조절하기 위한 조성물 및 방법 |
| ES2812099T3 (es) | 2014-05-01 | 2021-03-16 | Ionis Pharmaceuticals Inc | Composiciones y métodos para modular la expresión del receptor de la hormona del crecimiento |
| MX2017004039A (es) | 2014-10-10 | 2017-07-24 | Hoffmann La Roche | Fosforamiditas de n-acetilgalactosamina (galnac), conjugados de acido nucleico de las mismas y su uso. |
| MY203086A (en) * | 2015-11-06 | 2024-06-07 | Ionis Pharmaceuticals Inc | Modulating apolipoprotein (a) expression |
| WO2018013999A1 (en) | 2016-07-15 | 2018-01-18 | Am Chemicals Llc | Non-nucleosidic solid 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 |
| JP7720788B2 (ja) * | 2019-06-25 | 2025-08-08 | アムジエン・インコーポレーテツド | 炭水化物に連結されたオリゴヌクレオチドのための精製方法 |
| WO2021046260A1 (en) | 2019-09-03 | 2021-03-11 | Arcturus Therapeutics, Inc. | Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates |
| IL292360A (en) | 2019-10-22 | 2022-06-01 | Alnylam Pharmaceuticals Inc | IRNA components c3 supplementary component and methods of using them |
| MX2022006514A (es) | 2019-12-26 | 2022-07-11 | Eisai R&D Man Co Ltd | Composicion farmaceutica que contiene un acido ribonucleico de doble hebra que inhibe la expresion de c5 de complemento. |
| CA3181198A1 (en) * | 2020-04-30 | 2021-11-04 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| CA3225937A1 (en) * | 2021-07-17 | 2023-02-16 | Dmitry Samarsky | Products and compositions |
| WO2023031359A1 (en) | 2021-09-02 | 2023-03-09 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell |
| CN118302525A (zh) | 2021-10-29 | 2024-07-05 | 阿尔尼拉姆医药品有限公司 | 补体因子B(CFB)iRNA组合物及其使用方法 |
| JP2025537139A (ja) * | 2022-11-02 | 2025-11-14 | アイオーニス ファーマシューティカルズ, インコーポレーテッド | 補体因子bの発現を調節するための方法 |
| EP4638743A2 (en) * | 2022-12-19 | 2025-10-29 | Sanegene Bio USA Inc. | Small interfering rna targeting cfb and uses thereof |
| KR20250159727A (ko) * | 2023-03-21 | 2025-11-11 | 애로우헤드 파마슈티컬스 인코포레이티드 | 보체 인자 B (CFB)의 발현을 억제하기 위한 RNAi 작용제, 그의 제약 조성물 및 사용 방법 |
| 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 |
| WO2025002401A1 (zh) * | 2023-06-28 | 2025-01-02 | 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 |
| WO2025026228A1 (zh) * | 2023-07-28 | 2025-02-06 | 苏州炫景生物科技有限公司 | Cfb抑制剂组合物及其应用 |
| WO2025151250A2 (en) * | 2024-01-09 | 2025-07-17 | Adarx Pharmaceuticals, Inc. | Complement factor b-modulating compositions and methods of use thereof |
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| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
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