JP2016533765A5 - - Google Patents
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- 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
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- 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 (https=) | 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)
| Country | Link |
|---|---|
| US (5) | US20160222389A1 (https=) |
| EP (2) | EP3043827B1 (https=) |
| JP (3) | JP6666250B2 (https=) |
| KR (1) | KR102381790B1 (https=) |
| CN (2) | CN105744959B (https=) |
| AU (2) | AU2014318580B2 (https=) |
| BR (1) | BR112016004671B1 (https=) |
| CA (1) | CA2921842A1 (https=) |
| CL (2) | CL2016000606A1 (https=) |
| CR (1) | CR20160170A (https=) |
| DK (1) | DK3043827T3 (https=) |
| DO (1) | DOP2016000063A (https=) |
| EA (1) | EA035433B1 (https=) |
| ES (1) | ES2745758T3 (https=) |
| HR (1) | HRP20191600T1 (https=) |
| HU (1) | HUE045109T2 (https=) |
| IL (1) | IL244095A0 (https=) |
| LT (1) | LT3043827T (https=) |
| MA (1) | MA38959A1 (https=) |
| MX (2) | MX371518B (https=) |
| MY (1) | MY181251A (https=) |
| PE (2) | PE20160500A1 (https=) |
| PH (1) | PH12016500443A1 (https=) |
| PL (1) | PL3043827T3 (https=) |
| PT (1) | PT3043827T (https=) |
| RS (1) | RS59252B1 (https=) |
| RU (1) | RU2662967C2 (https=) |
| SG (2) | SG11201601445XA (https=) |
| SI (1) | SI3043827T1 (https=) |
| UA (1) | UA119046C2 (https=) |
| WO (1) | WO2015038939A2 (https=) |
| ZA (1) | ZA201601236B (https=) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2161038E (pt) | 2006-01-26 | 2014-03-10 | Isis Pharmaceuticals Inc | Composições e suas utilizações dirigidas à huntingtina |
| WO2008049085A1 (en) | 2006-10-18 | 2008-04-24 | Isis Pharmaceuticals, Inc. | Antisense compounds |
| EP3626823A1 (en) | 2009-09-11 | 2020-03-25 | Ionis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
| US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| DK3043827T3 (da) * | 2013-09-13 | 2019-08-26 | Ionis Pharmaceuticals Inc | Modulatorer af komplement faktor b |
| SG10201804960RA (en) * | 2013-12-12 | 2018-07-30 | Alnylam Pharmaceuticals Inc | Complement component irna compositions and methods of use thereof |
| SG11201608502TA (en) | 2014-05-01 | 2016-11-29 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
| WO2017135397A1 (ja) * | 2016-02-05 | 2017-08-10 | 協和発酵キリン株式会社 | 補体ｂ因子の発現を抑制するアンチセンスオリゴヌクレオチド |
| RU2018145364A (ru) | 2016-06-27 | 2020-07-28 | Ачиллион Фармасьютикалс, Инк. | Хиназолиновые и индольные соединения для лечения медицинских нарушений |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| 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 |
| TW202134435A (zh) | 2019-10-22 | 2021-09-16 | 美商阿尼拉製藥公司 | 補體成分ｃ３ ｉｒｎａ組成物及其使用方法 |
| MX2022013606A (es) * | 2020-04-30 | 2023-01-16 | Alnylam Pharmaceuticals Inc | Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas. |
| CN114057758A (zh) | 2020-08-07 | 2022-02-18 | 上海美悦生物科技发展有限公司 | 补体因子b抑制剂及其药物组合物、制备方法和用途 |
| EP4194449A4 (en) | 2020-08-07 | 2024-03-20 | Shanghai Meiyue Biotech Development Co., Ltd. | Heterocyclic compound, 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 |
| KR20240051221A (ko) | 2021-09-02 | 2024-04-19 | 사일런스 테라퓨틱스 게엠베하 | 세포에서 보체 인자 b (cfb)의 발현을 억제하기 위한 핵산 |
| TW202333749A (zh) | 2021-10-29 | 2023-09-01 | 美商艾拉倫製藥股份有限公司 | 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法 |
| PE20241340A1 (es) | 2021-11-24 | 2024-07-03 | Adarx Pharmaceuticals Inc | Composiciones moduladoras de complemento del factor b y metodos de uso de estas |
| WO2023129496A2 (en) * | 2021-12-27 | 2023-07-06 | Apellis Pharmaceuticals, Inc. | Rnas for complement inhibition |
| CA3242815A1 (en) | 2022-01-26 | 2023-08-03 | Shanghai Meiyue Biotech Development Co., Ltd. | Salt type and crystal form of complement factor b inhibitor, and preparation method therefor and application thereof |
| CA3266935A1 (en) | 2022-09-10 | 2024-03-14 | Jiangsu Hansoh Pharmaceutical Group Co., Ltd. | Piperidine derivatives substituted at position 2, their preparation methods and medicinal uses |
| AU2023372971A1 (en) * | 2022-11-02 | 2025-05-15 | Ionis Pharmaceuticals, Inc. | Methods for modulating complement factor b expression |
| KR20260004425A (ko) | 2023-04-27 | 2026-01-08 | 상하이 한서 바이오메디컬 컴퍼니 리미티드 | 피페리딜인돌 화합물의 염 및 이의 제조 방법 |
| TW202513799A (zh) * | 2023-06-02 | 2025-04-01 | 大陸商維亞臻生物技術（蘇州）有限公司 | 用於免疫類疾病的雙鏈核苷酸化合物及其用途 |
| 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 |
| AU2024307421A1 (en) * | 2023-06-28 | 2026-02-12 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of complement factor b (cfb) |
| 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 |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
| ATE269870T1 (de) | 1989-10-24 | 2004-07-15 | Isis Pharmaceuticals Inc | 2'-modifizierte oligonukleotide |
| US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
| US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
| 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 |
| ES2116977T3 (es) | 1990-05-11 | 1998-08-01 | Microprobe Corp | Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente. |
| EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis 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 |
| EP0673559A1 (en) | 1992-12-14 | 1995-09-27 | Honeywell Inc. | Motor system with individually controlled redundant windings |
| AU6449394A (en) | 1993-03-30 | 1994-10-24 | Sterling Winthrop Inc. | Acyclic nucleoside analogs and oligonucleotide sequences containing them |
| 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 |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| AU9063398A (en) | 1997-09-12 | 1999-04-05 | Exiqon A/S | Oligonucleotide analogues |
| US6043352A (en) | 1998-08-07 | 2000-03-28 | Isis Pharmaceuticals, Inc. | 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides |
| WO2000021559A2 (en) * | 1998-10-09 | 2000-04-20 | Musc Foundation For Research Development | Blocking factor b to treat complement-mediated immune disease |
| WO2000063364A2 (en) | 1999-04-21 | 2000-10-26 | American Home Products Corporation | Methods and compositions for inhibiting the function of polynucleotide sequences |
| EP1178999B1 (en) | 1999-05-04 | 2007-03-14 | Santaris Pharma A/S | L-ribo-lna analogues |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| ES2261270T3 (es) | 1999-12-30 | 2006-11-16 | K.U. LEUVEN RESEARCH & DEVELOPMENT | Acidos nucleicos que contienen ciclohexeno. |
| JP2005504020A (ja) | 2001-07-03 | 2005-02-10 | アイシス・ファーマシューティカルス・インコーポレーテッド | ヌクレアーゼ耐性キメラオリゴヌクレオチド |
| US6964950B2 (en) * | 2001-07-25 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of C-reactive protein expression |
| JP4634799B2 (ja) * | 2002-09-13 | 2011-02-16 | ライフ テクノロジーズ コーポレーション | 耐熱性逆転写酵素およびその使用法 |
| 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 |
| EP2305812A3 (en) | 2002-11-14 | 2012-06-06 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| 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 |
| US20070123466A1 (en) * | 2003-05-13 | 2007-05-31 | New York Society For The Ruptured And Crippled Maintaining The Hospital For Special Surgery | Method of treating recurrent miscarriages |
| EP1661905B9 (en) | 2003-08-28 | 2012-12-19 | IMANISHI, Takeshi | Novel artificial nucleic acids of n-o bond crosslinkage type |
| 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 |
| ES2432112T3 (es) * | 2004-02-10 | 2013-11-29 | The Regents Of The University Of Colorado, A Body Corporate | Inhibición del factor B, de la vía alternativa del complemento y métodos relacionados |
| EP1752536A4 (en) * | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
| AU2005252663B2 (en) | 2004-06-03 | 2011-07-07 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| EP2397563A3 (en) | 2004-09-17 | 2012-07-18 | 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 |
| JP5342881B2 (ja) | 2006-01-27 | 2013-11-13 | アイシス ファーマシューティカルズ， インコーポレーテッド | ６−修飾された二環式核酸類似体 |
| AU2007249349B2 (en) | 2006-05-11 | 2012-03-08 | Isis Pharmaceuticals, Inc. | 5'-Modified bicyclic nucleic acid analogs |
| 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 |
| US20100190837A1 (en) | 2007-02-15 | 2010-07-29 | Isis 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 |
| EP2170917B1 (en) | 2007-05-30 | 2012-06-27 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| CA2692579C (en) | 2007-07-05 | 2016-05-03 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| PL2320925T3 (pl) * | 2008-07-10 | 2016-06-30 | Regenesance B V | Antagoniści dopełniacza i ich zastosowania |
| US8604192B2 (en) | 2008-09-24 | 2013-12-10 | Isis Pharmaceuticals, Inc. | Cyclohexenyl nucleic acids analogs |
| KR101869570B1 (ko) | 2010-04-28 | 2018-06-20 | 아이오니스 파마수티컬즈, 인코포레이티드 | 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물 |
| WO2012012467A2 (en) * | 2010-07-19 | 2012-01-26 | Isis Pharmaceuticals, Inc. | Modulation of nuclear-retained rna |
| WO2012142458A1 (en) * | 2011-04-13 | 2012-10-18 | Isis Pharmaceuticals, Inc. | Antisense modulation of ptp1b expression |
| DK3043827T3 (da) | 2013-09-13 | 2019-08-26 | Ionis Pharmaceuticals Inc | Modulatorer af komplement faktor b |
| SG11201608502TA (en) * | 2014-05-01 | 2016-11-29 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
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