JP2016533765A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2016533765A5 JP2016533765A5 JP2016542824A JP2016542824A JP2016533765A5 JP 2016533765 A5 JP2016533765 A5 JP 2016533765A5 JP 2016542824 A JP2016542824 A JP 2016542824A JP 2016542824 A JP2016542824 A JP 2016542824A JP 2016533765 A5 JP2016533765 A5 JP 2016533765A5
- Authority
- JP
- Japan
- Prior art keywords
- wing segment
- linked nucleosides
- modified oligonucleotide
- segment
- compound
- 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.)
- Granted
Links
- 150000001875 compounds Chemical class 0.000 claims description 67
- 239000002777 nucleoside Substances 0.000 claims description 55
- 108091034117 Oligonucleotide Proteins 0.000 claims description 49
- 125000003835 nucleoside group Chemical group 0.000 claims description 45
- 230000000295 complement effect Effects 0.000 claims description 27
- 208000002780 macular degeneration Diseases 0.000 claims description 19
- 208000017169 kidney disease Diseases 0.000 claims description 18
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 15
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 14
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 14
- 201000010099 disease Diseases 0.000 claims description 12
- 230000037361 pathway Effects 0.000 claims description 12
- 208000035913 Atypical hemolytic uremic syndrome Diseases 0.000 claims description 11
- 208000016323 C3 glomerulonephritis Diseases 0.000 claims description 10
- 208000004451 Membranoproliferative Glomerulonephritis Diseases 0.000 claims description 10
- 208000022401 dense deposit disease Diseases 0.000 claims description 10
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 10
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 8
- 229940104302 cytosine Drugs 0.000 claims description 7
- 230000008482 dysregulation Effects 0.000 claims description 7
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 7
- 102100035325 Complement factor H-related protein 5 Human genes 0.000 claims description 5
- 101000878134 Homo sapiens Complement factor H-related protein 5 Proteins 0.000 claims description 5
- 208000005777 Lupus Nephritis Diseases 0.000 claims description 5
- 239000000203 mixture Substances 0.000 claims description 5
- 201000000596 systemic lupus erythematosus Diseases 0.000 claims description 4
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 3
- 206010003694 Atrophy Diseases 0.000 claims description 2
- 230000037444 atrophy Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- -1 methoxyethyl Chemical group 0.000 claims description 2
- 150000003839 salts Chemical class 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 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 claims 1
- 230000024203 complement activation Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 54
- 102000003712 Complement factor B Human genes 0.000 description 21
- 108090000056 Complement factor B Proteins 0.000 description 21
- 239000003112 inhibitor Substances 0.000 description 13
- 230000000692 anti-sense effect Effects 0.000 description 12
- 238000009825 accumulation Methods 0.000 description 8
- 230000002401 inhibitory effect Effects 0.000 description 6
- 210000003734 kidney Anatomy 0.000 description 5
- 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 4
- 230000008685 targeting Effects 0.000 description 4
- 208000035475 disorder Diseases 0.000 description 3
- 108020004707 nucleic acids Proteins 0.000 description 3
- 102000039446 nucleic acids Human genes 0.000 description 3
- 150000007523 nucleic acids Chemical class 0.000 description 3
- 101710131943 40S ribosomal protein S3a Proteins 0.000 description 2
- HEDRZPFGACZZDS-UHFFFAOYSA-N Chloroform Chemical compound ClC(Cl)Cl HEDRZPFGACZZDS-UHFFFAOYSA-N 0.000 description 2
- 125000002619 bicyclic group Chemical group 0.000 description 2
- 239000007791 liquid phase Substances 0.000 description 2
- 210000005228 liver tissue Anatomy 0.000 description 2
- 239000002773 nucleotide Substances 0.000 description 2
- 125000003729 nucleotide group Chemical group 0.000 description 2
- 210000002966 serum Anatomy 0.000 description 2
- 210000001519 tissue Anatomy 0.000 description 2
- 238000009736 wetting Methods 0.000 description 2
- 108010034753 Complement Membrane Attack Complex Proteins 0.000 description 1
- 206010064571 Gene mutation Diseases 0.000 description 1
- ISWSIDIOOBJBQZ-UHFFFAOYSA-N Phenol Chemical compound OC1=CC=CC=C1 ISWSIDIOOBJBQZ-UHFFFAOYSA-N 0.000 description 1
- 108091028664 Ribonucleotide Proteins 0.000 description 1
- 108091036066 Three prime untranslated region Proteins 0.000 description 1
- 208000034841 Thrombotic Microangiopathies Diseases 0.000 description 1
- 230000001668 ameliorated effect Effects 0.000 description 1
- 238000011088 calibration curve Methods 0.000 description 1
- 239000005547 deoxyribonucleotide Substances 0.000 description 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 1
- 238000000605 extraction Methods 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000002414 normal-phase solid-phase extraction Methods 0.000 description 1
- 238000011002 quantification Methods 0.000 description 1
- 210000005084 renal tissue Anatomy 0.000 description 1
- 239000002336 ribonucleotide Substances 0.000 description 1
- 125000002652 ribonucleotide group Chemical group 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361877624P | 2013-09-13 | 2013-09-13 | |
| US61/877,624 | 2013-09-13 | ||
| PCT/US2014/055458 WO2015038939A2 (en) | 2013-09-13 | 2014-09-12 | Modulators of complement factor b |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019201990A Division JP7158363B2 (ja) | 2013-09-13 | 2019-11-07 | 補体ｂ因子の調節薬 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016533765A JP2016533765A (ja) | 2016-11-04 |
| JP2016533765A5 true JP2016533765A5 (enExample) | 2017-10-19 |
| JP6666250B2 JP6666250B2 (ja) | 2020-03-13 |
Family
ID=52666522
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016542824A Active JP6666250B2 (ja) | 2013-09-13 | 2014-09-12 | 補体ｂ因子の調節薬 |
| JP2019201990A Active JP7158363B2 (ja) | 2013-09-13 | 2019-11-07 | 補体ｂ因子の調節薬 |
| JP2022011551A Active JP7297112B2 (ja) | 2013-09-13 | 2022-01-28 | 補体ｂ因子の調節薬 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019201990A Active JP7158363B2 (ja) | 2013-09-13 | 2019-11-07 | 補体ｂ因子の調節薬 |
| JP2022011551A Active JP7297112B2 (ja) | 2013-09-13 | 2022-01-28 | 補体ｂ因子の調節薬 |
Country Status (32)
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2377327T5 (es) | 2006-10-18 | 2020-04-28 | Ionis Pharmaceuticals Inc | Compuestos antisentido |
| EP3626823A1 (en) | 2009-09-11 | 2020-03-25 | Ionis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
| EP3067421B1 (en) | 2011-02-08 | 2018-10-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| RU2670614C9 (ru) | 2013-05-01 | 2018-11-23 | Ионис Фармасьютикалз, Инк. | Композиции и способы модулирования экспрессии hbv и ttr |
| HRP20191600T1 (hr) | 2013-09-13 | 2019-11-29 | Ionis Pharmaceuticals Inc | Modulatori faktora b komplementa |
| KR102736538B1 (ko) * | 2013-12-12 | 2024-12-03 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법 |
| CA2943894A1 (en) * | 2014-05-01 | 2015-11-05 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating complement factor b expression |
| TW201730341A (zh) * | 2016-02-05 | 2017-09-01 | Kyowa Hakko Kirin Co Ltd | 抑制補體b因子之表現之反義寡核苷酸 |
| EP3448389B1 (en) | 2016-06-27 | 2021-09-29 | Achillion Pharmaceuticals, Inc. | Quinazoline and indole compounds to treat medical disorders |
| JP2019535839A (ja) | 2016-11-29 | 2019-12-12 | ピュアテック ヘルス エルエルシー | 治療剤の送達のためのエクソソーム |
| TW201827596A (zh) * | 2016-12-23 | 2018-08-01 | 日商協和醱酵麒麟有限公司 | 抑制補體b因子之表現之核酸 |
| WO2019027015A1 (ja) * | 2017-08-02 | 2019-02-07 | 協和発酵キリン株式会社 | 核酸複合体 |
| AU2018360697A1 (en) | 2017-11-01 | 2020-05-14 | Alnylam Pharmaceuticals, Inc. | Complement component C3 iRNA compositions and methods of use thereof |
| WO2020109343A1 (en) | 2018-11-29 | 2020-06-04 | F. Hoffmann-La Roche Ag | Combination therapy for treatment of macular degeneration |
| AR120267A1 (es) | 2019-10-22 | 2022-02-09 | Alnylam Pharmaceuticals Inc | COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C3 DEL COMPLEMENTO Y SUS MÉTODOS DE USO |
| EP4142738A1 (en) * | 2020-04-30 | 2023-03-08 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| US20230331710A1 (en) | 2020-08-07 | 2023-10-19 | Shanghai Meiyue Biotech Development Co. Ltd | Heterocyclic compound, preparation method and use thereof |
| EP4282486A3 (en) | 2020-08-07 | 2024-03-06 | Shanghai Meiyue Biotech Development Co., Ltd. | Complement factor b inhibitor, and pharmaceutical composition thereof, preparation method therefor and use thereof |
| JP2024525868A (ja) * | 2021-07-17 | 2024-07-12 | サーナオミクス インコーポレイテッド | 産物及び組成物 |
| WO2023031359A1 (en) | 2021-09-02 | 2023-03-09 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell |
| KR20240095325A (ko) * | 2021-10-29 | 2024-06-25 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 인자 b (cfb) irna 조성물 및 이의 사용 방법 |
| US20250075209A1 (en) * | 2021-12-27 | 2025-03-06 | Apellis Pharmaceuticals, Inc. | Rnas for complement inhibition |
| AU2023210941A1 (en) | 2022-01-26 | 2024-07-11 | Shanghai Meiyue Biotech Development Co., Ltd. | Salt type and crystal form of complement factor b inhibitor, and preparation method therefor and application thereof |
| WO2024051849A1 (en) | 2022-09-10 | 2024-03-14 | Jiangsu Hansoh Pharmaceutical Group Co., Ltd. | 2-substituted piperidine derivatives, preparation methods and medicinal uses thereof |
| IL320604A (en) * | 2022-11-02 | 2025-07-01 | Ionis Pharmaceuticals Inc | Methods for modulating complement factor B expression |
| 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 |
| TW202500169A (zh) * | 2023-06-28 | 2025-01-01 | 大陸商上海舶望製藥有限公司 | 用於抑制補體因子b(cfb)表達的組合物和方法 |
| 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 |
| WO2025122669A1 (en) * | 2023-12-05 | 2025-06-12 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a humanized complement factor b gene |
Family Cites Families (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
| EP0497875B1 (en) | 1989-10-24 | 2000-03-22 | Isis Pharmaceuticals, Inc. | 2' modified oligonucleotides |
| US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
| US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
| GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
| ATE167523T1 (de) | 1990-05-11 | 1998-07-15 | Microprobe Corp | Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden |
| DE59208572D1 (de) | 1991-10-17 | 1997-07-10 | Ciba Geigy Ag | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
| EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
| JPH08504559A (ja) | 1992-12-14 | 1996-05-14 | ハネウエル・インコーポレーテッド | 個別に制御される冗長巻線を有するモータシステム |
| ATE155467T1 (de) | 1993-03-30 | 1997-08-15 | Sanofi Sa | Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen |
| US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
| US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
| US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
| US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
| 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 |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| ATE293123T1 (de) | 1997-09-12 | 2005-04-15 | Exiqon As | Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US6043352A (en) | 1998-08-07 | 2000-03-28 | Isis Pharmaceuticals, Inc. | 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides |
| AU1705100A (en) * | 1998-10-09 | 2000-05-01 | Musc Foundation For Research Development | Blocking factor b to treat complement-mediated immune disease |
| IL145778A0 (en) | 1999-04-21 | 2002-07-25 | American Home Prod | Methods and compositions for inhibiting the function of polynucleotide sequences |
| CA2372085C (en) | 1999-05-04 | 2009-10-27 | Exiqon A/S | L-ribo-lna analogues |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| DE60027203T2 (de) | 1999-12-30 | 2007-01-04 | K.U. Leuven Research & Development | Cyclohexennukleinsäuren |
| CA2452458A1 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| US6964950B2 (en) * | 2001-07-25 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of C-reactive protein expression |
| EP2325303B1 (en) * | 2002-09-13 | 2013-11-06 | Life Technologies Corporation | Thermostable reverse transcriptases and uses thereof |
| CA2504694C (en) | 2002-11-05 | 2013-10-01 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| US8604183B2 (en) | 2002-11-05 | 2013-12-10 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
| EP2213738B1 (en) | 2002-11-14 | 2012-10-10 | Dharmacon, Inc. | siRNA molecules targeting Bcl-2 |
| US6673661B1 (en) | 2002-12-20 | 2004-01-06 | Taiwan Semiconductor Manufacturing Co., Ltd. | Self-aligned method for forming dual gate thin film transistor (TFT) device |
| WO2004103288A2 (en) * | 2003-05-13 | 2004-12-02 | New York Society For The Ruptured And Crippled Maintaining The Hospital For Special Surgery | Method of preventing recurrent miscarriages |
| JP4731324B2 (ja) | 2003-08-28 | 2011-07-20 | 武 今西 | Ｎ−ｏ結合性架橋構造型新規人工核酸 |
| WO2005027962A1 (en) | 2003-09-18 | 2005-03-31 | Isis Pharmaceuticals, Inc. | 4’-thionucleosides and oligomeric compounds |
| US7959919B2 (en) * | 2003-11-19 | 2011-06-14 | Novelmed Therapeutics, Inc. | Method of inhibiting factor B-mediated complement activation |
| BRPI0506629A (pt) * | 2004-02-10 | 2007-05-02 | Univ Colorado | inibição do fator b, a via alternativa do sistema complemento e métodos relacionados |
| KR20070085113A (ko) * | 2004-05-11 | 2007-08-27 | 가부시키가이샤 알파젠 | Rna간섭을 생기게 하는 폴리뉴클레오티드, 및 이를 이용한유전자발현억제 방법 |
| CA2569419A1 (en) | 2004-06-03 | 2005-12-22 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| WO2006034348A2 (en) | 2004-09-17 | 2006-03-30 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| US20080261905A1 (en) | 2004-11-08 | 2008-10-23 | K.U. Leuven Research And Development | Modified Nucleosides for Rna Interference |
| GB0521716D0 (en) * | 2005-10-25 | 2005-11-30 | Genomica Sau | Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases |
| EP1984381B1 (en) | 2006-01-27 | 2010-09-29 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| DK2066684T3 (da) | 2006-05-11 | 2012-10-22 | Isis Pharmaceuticals Inc | 5´-Modificerede bicycliske nukleinsyreanaloge |
| WO2008036841A2 (en) * | 2006-09-22 | 2008-03-27 | Dharmacon, Inc. | Tripartite oligonucleotide complexes and methods for gene silencing by rna interference |
| CN101589143A (zh) * | 2006-11-27 | 2009-11-25 | Isis药物公司 | 用于治疗高胆固醇血症的方法 |
| EP2125852B1 (en) | 2007-02-15 | 2016-04-06 | Ionis Pharmaceuticals, Inc. | 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom |
| CA2678774A1 (en) * | 2007-03-01 | 2008-09-04 | Advanced Vision Therapies, Inc. | Treatment of diseases characterized by inflammation |
| AU2008260277C1 (en) | 2007-05-30 | 2014-04-17 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| DK2173760T4 (en) | 2007-06-08 | 2016-02-08 | Isis Pharmaceuticals Inc | Carbocyclic bicyclic nukleinsyreanaloge |
| CN101796062B (zh) | 2007-07-05 | 2014-07-30 | Isis制药公司 | 6-双取代双环核酸类似物 |
| CN102149390A (zh) * | 2008-07-10 | 2011-08-10 | 阿姆斯特丹大学附属医院 | 补体拮抗剂及其应用 |
| EP2361256B1 (en) | 2008-09-24 | 2013-04-10 | Isis Pharmaceuticals, Inc. | Cyclohexenyl nucleic acid analogs |
| US8993738B2 (en) | 2010-04-28 | 2015-03-31 | Isis Pharmaceuticals, Inc. | Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom |
| DK3031920T3 (da) * | 2010-07-19 | 2019-10-14 | Ionis Pharmaceuticals Inc | Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression |
| KR101839177B1 (ko) * | 2011-04-13 | 2018-03-15 | 아이오니스 파마수티컬즈, 인코포레이티드 | Ｐｔｐｉｂ 발현의 안티센스 조절 |
| HRP20191600T1 (hr) | 2013-09-13 | 2019-11-29 | Ionis Pharmaceuticals Inc | Modulatori faktora b komplementa |
| CA2943894A1 (en) * | 2014-05-01 | 2015-11-05 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating complement factor b expression |
-
2014
- 2014-09-12 HR HRP20191600 patent/HRP20191600T1/hr unknown
- 2014-09-12 EP EP14844168.6A patent/EP3043827B1/en active Active
- 2014-09-12 PT PT14844168T patent/PT3043827T/pt unknown
- 2014-09-12 EP EP19182415.0A patent/EP3603677A1/en active Pending
- 2014-09-12 SI SI201431272T patent/SI3043827T1/sl unknown
- 2014-09-12 PE PE2018003111A patent/PE20190354A1/es unknown
- 2014-09-12 BR BR112016004671-4A patent/BR112016004671B1/pt active IP Right Grant
- 2014-09-12 ES ES14844168T patent/ES2745758T3/es active Active
- 2014-09-12 JP JP2016542824A patent/JP6666250B2/ja active Active
- 2014-09-12 CA CA2921842A patent/CA2921842A1/en not_active Abandoned
- 2014-09-12 US US15/021,651 patent/US20160222389A1/en not_active Abandoned
- 2014-09-12 RU RU2016113763A patent/RU2662967C2/ru active
- 2014-09-12 MY MYPI2016700852A patent/MY181251A/en unknown
- 2014-09-12 PL PL14844168T patent/PL3043827T3/pl unknown
- 2014-09-12 CN CN201480056788.6A patent/CN105744959B/zh active Active
- 2014-09-12 KR KR1020167009610A patent/KR102381790B1/ko active Active
- 2014-09-12 LT LTEP14844168.6T patent/LT3043827T/lt unknown
- 2014-09-12 MX MX2016003263A patent/MX371518B/es active IP Right Grant
- 2014-09-12 AU AU2014318580A patent/AU2014318580B2/en active Active
- 2014-09-12 PE PE2016000376A patent/PE20160500A1/es unknown
- 2014-09-12 CN CN202011263979.3A patent/CN112359042A/zh active Pending
- 2014-09-12 WO PCT/US2014/055458 patent/WO2015038939A2/en not_active Ceased
- 2014-09-12 RS RSP20191160 patent/RS59252B1/sr unknown
- 2014-09-12 DK DK14844168.6T patent/DK3043827T3/da active
- 2014-09-12 SG SG10201806787VA patent/SG10201806787VA/en unknown
- 2014-09-12 SG SG11201601445XA patent/SG11201601445XA/en unknown
- 2014-09-12 MA MA38959A patent/MA38959A1/fr unknown
- 2014-09-12 HU HUE14844168A patent/HUE045109T2/hu unknown
- 2014-09-12 EA EA201690582A patent/EA035433B1/ru unknown
- 2014-12-09 UA UAA201603969A patent/UA119046C2/uk unknown
-
2016
- 2016-02-09 IL IL244095A patent/IL244095A0/en unknown
- 2016-02-22 ZA ZA2016/01236A patent/ZA201601236B/en unknown
- 2016-03-08 PH PH12016500443A patent/PH12016500443A1/en unknown
- 2016-03-11 MX MX2020001258A patent/MX2020001258A/es unknown
- 2016-03-11 DO DO2016000063A patent/DOP2016000063A/es unknown
- 2016-03-14 CL CL2016000606A patent/CL2016000606A1/es unknown
- 2016-04-12 CR CR20160170A patent/CR20160170A/es unknown
-
2018
- 2018-03-05 US US15/912,386 patent/US20190048351A1/en not_active Abandoned
- 2018-08-14 CL CL2018002334A patent/CL2018002334A1/es unknown
-
2019
- 2019-06-12 US US16/439,317 patent/US20200149047A1/en not_active Abandoned
- 2019-11-07 JP JP2019201990A patent/JP7158363B2/ja active Active
-
2020
- 2020-03-11 AU AU2020201763A patent/AU2020201763A1/en not_active Abandoned
-
2021
- 2021-09-02 US US17/465,035 patent/US11926830B2/en active Active
-
2022
- 2022-01-28 JP JP2022011551A patent/JP7297112B2/ja active Active
-
2024
- 2024-02-02 US US18/431,253 patent/US20240247268A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016533765A5 (enExample) | ||
| JP7297112B2 (ja) | 補体ｂ因子の調節薬 | |
| US12110491B2 (en) | Selective reduction of allelic variants | |
| CA2918600C (en) | Compositions for modulating tau expression | |
| JP7007304B2 (ja) | Ｂ型肝炎感染症治療用のＰＡＰＤ５又はＰＡＰＤ７のｍＲＮＡ減少用核酸分子 | |
| JP6606557B2 (ja) | Ｃ９ｏｒｆ７２発現を調節するための組成物 | |
| US9752142B2 (en) | Gapped oligomeric compounds comprising 5′-modified deoxyribonucleosides in the gap and uses thereof | |
| CN110724687B (zh) | 用于调节补体因子b表达的组合物和方法 | |
| EP3208347B1 (en) | Selective reduction of allelic variants | |
| JP2013226147A5 (enExample) | ||
| CN102482672B (zh) | 通过抑制唐氏综合征基因的天然反义转录物治疗唐氏综合征基因相关疾病 | |
| JP2016116520A5 (enExample) | ||
| CN105452464A (zh) | 用于调节肌强直性营养不良蛋白激酶(dmpk)表达的化合物和方法 | |
| JP2016513976A5 (enExample) | ||
| TW202039842A (zh) | 治療黃斑退化之組合治療 | |
| AU2022203490A1 (en) | Organic compositions to treat epas1-related diseases | |
| KR20150004414A (ko) | Kras-관련 질환을 치료하기 위한 유기 조성물 | |
| EP2683411A1 (en) | Methods of using microrna-26a to promote angiogenesis | |
| WO2013013165A2 (en) | Use of lna compounds for therapeutic inhibition of mir-21 in systemic lupus erythematosus | |
| CN114340678A (zh) | 用于诊断或治疗与elf4e活性增加相关的病症的包含elf4e抑制剂的组合物 | |
| RU2018127385A (ru) | Модуляторы фактора в комплемента |