AU2014362262B2 - Complement component iRNA compositions and methods of use thereof - Google Patents

Complement component iRNA compositions and methods of use thereof Download PDF

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AU2014362262B2
AU2014362262B2 AU2014362262A AU2014362262A AU2014362262B2 AU 2014362262 B2 AU2014362262 B2 AU 2014362262B2 AU 2014362262 A AU2014362262 A AU 2014362262A AU 2014362262 A AU2014362262 A AU 2014362262A AU 2014362262 B2 AU2014362262 B2 AU 2014362262B2
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nucleotides
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nucleotide
subject
expression
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AU2014362262A1 (en
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Brian Bettencourt
Anna Borodovsky
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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AU2014362262A 2013-12-12 2014-12-12 Complement component iRNA compositions and methods of use thereof Active AU2014362262B2 (en)

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Application Number Priority Date Filing Date Title
AU2021204655A AU2021204655B2 (en) 2013-12-12 2021-07-02 Complement component iRNA compositions and methods of use thereof
AU2024201770A AU2024201770A1 (en) 2013-12-12 2024-03-19 Complement component iRNA compositions and methods of use thereof

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Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
KR20240170595A (ko) 2013-12-12 2024-12-03 알닐람 파마슈티칼스 인코포레이티드 보체 성분 iRNA 조성물 및 이의 이용 방법
CN106232804B (zh) 2014-05-01 2019-10-25 Ionis制药公司 用于调节补体因子b表达的组合物和方法
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
RS61297B1 (sr) * 2015-07-31 2021-02-26 Alnylam Pharmaceuticals Inc Transtiretinske (ttr) kompozicije irnk i postupci za njihovu primenu u lečenju ili prevenciji bolesti povezanih sa ttr-om
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
US11338013B2 (en) * 2016-10-17 2022-05-24 Apellis Pharmaceuticals, Inc. Combination therapy for C3 inhibition
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
WO2018145086A1 (en) 2017-02-06 2018-08-09 Mpeg La, Llc Multimeric oligonucleotides having decreased kidney clearance
PT3658184T (pt) * 2017-07-27 2023-11-29 Alexion Pharma Inc Formulações de anticorpo anti-c5 de elevada concentração
TW201909925A (zh) * 2017-08-02 2019-03-16 日商協和醱酵麒麟有限公司 核酸複合體
CN108152250B (zh) * 2017-10-27 2020-02-18 南京邮电大学 生物识别探针的构建方法及其逻辑运算方法
EP3704252A1 (en) * 2017-11-01 2020-09-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
EP3730617A1 (en) * 2019-04-26 2020-10-28 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of c3 in a cell
US20220017899A1 (en) * 2018-11-23 2022-01-20 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of c3 in a cell
KR102774146B1 (ko) 2018-12-13 2025-02-26 아르제넥스 비브이 인간 보체 인자 c2b 에 대한 항체 및 이의 사용방법
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
GB2584105B (en) * 2019-05-21 2023-08-02 Argenx Bvba Methods of treating neuropathy
WO2021026476A1 (en) * 2019-08-08 2021-02-11 Mpeg La, L.L.C. Complement targeting with multimeric oligonucleotides
MX2022002397A (es) * 2019-08-27 2022-08-25 Silence Therapeutics Gmbh Acidos nucleicos para inhibir la expresion de c3 en una celula.
JP7676377B2 (ja) * 2019-10-22 2025-05-14 アルナイラム ファーマシューティカルズ, インコーポレイテッド 補体成分C3 iRNA組成物およびその使用方法
EP4114947A1 (en) * 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
WO2021222549A1 (en) * 2020-04-30 2021-11-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
EP4274896A1 (en) * 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
US20230135763A1 (en) * 2021-07-17 2023-05-04 Sirnaomics, Inc. 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
KR20240095325A (ko) 2021-10-29 2024-06-25 알닐람 파마슈티칼스 인코포레이티드 보체 인자 b (cfb) irna 조성물 및 이의 사용 방법
WO2023088455A1 (zh) * 2021-11-22 2023-05-25 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
IL312928A (en) * 2021-11-24 2024-07-01 Adarx Pharmaceuticals Inc Complement Factor B Modulating Preparations and Methods for Using Them
UY40118A (es) * 2022-01-20 2023-08-15 Alexion Pharma Inc Composiciones y métodos para inhibir el factor b del complemento
CN114783511B (zh) * 2022-02-25 2025-03-25 深圳市硬核酸生物科技有限公司 核酸分子溶液浓度推荐方法及装置、计算机设备
TW202405170A (zh) * 2022-04-02 2024-02-01 大陸商上海舶望製藥有限公司 用於抑制補體成分c3蛋白表達的組合物和方法
CN116622710A (zh) * 2022-05-27 2023-08-22 北京福元医药股份有限公司 用于抑制c3基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2024008114A1 (zh) * 2022-07-08 2024-01-11 北京福元医药股份有限公司 用于抑制补体因子B表达的siRNA、其缀合物和药物组合物及其用途
AU2023367106A1 (en) 2022-10-27 2025-05-01 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement component c3 (c3), pharmaceutical compositions thereof, and methods of use
JP2025542234A (ja) * 2022-12-19 2025-12-25 セーンジーン バイオ ユーエスエー インコーポレイティド 低分子干渉rna標的化cfb及びその使用
WO2024169907A1 (zh) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 一种调节补体C3表达的siRNA、其缀合物和药物组合物及用途
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
WO2025002299A1 (en) * 2023-06-28 2025-01-02 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of complement factor b (cfb)
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
CN117563009B (zh) 2023-09-01 2025-07-01 杭州天龙药业有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物
WO2025113592A1 (zh) * 2023-12-01 2025-06-05 厦门甘宝利生物医药有限公司 一种抑制补体系统基因表达的rna抑制剂及其应用
WO2025151250A2 (en) * 2024-01-09 2025-07-17 Adarx Pharmaceuticals, Inc. Complement factor b-modulating compositions and methods of use thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070088154A1 (en) * 2002-11-14 2007-04-19 Dharmacon Inc. siRNA targeting complement factor B
WO2008036841A2 (en) * 2006-09-22 2008-03-27 Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
WO2013074974A2 (en) * 2011-11-18 2013-05-23 Alnylam Pharmaceuticals, Inc. Modified rnai agents

Family Cites Families (277)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US513030A (en) 1894-01-16 Machine for waxing or coating paper
US39464A (en) 1863-08-11 Improvement in grain-forks
US564562A (en) 1896-07-21 Joseph p
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
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
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
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
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
CA1340032C (en) 1987-06-24 1998-09-08 Jim Haralambidis Lucleoside derivatives
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
EP0406309A4 (en) 1988-03-25 1992-08-19 The University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
JPH05504552A (ja) 1989-10-24 1993-07-15 ギリアド サイエンシズ,インコーポレイテッド 2’位が改変されたオリゴヌクレオチド
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
CA2029273A1 (en) 1989-12-04 1991-06-05 Christine L. Brakel Modified nucleotide compounds
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US7037646B1 (en) 1990-01-11 2006-05-02 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US5852188A (en) 1990-01-11 1998-12-22 Isis Pharmaceuticals, Inc. Oligonucleotides having chiral phosphorus linkages
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US6783931B1 (en) 1990-01-11 2004-08-31 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
WO1991013080A1 (en) 1990-02-20 1991-09-05 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
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
EP0745689A3 (en) 1990-05-11 1996-12-11 Microprobe Corporation A dipstick for a nucleic acid hybridization assay
US5981276A (en) 1990-06-20 1999-11-09 Dana-Farber Cancer Institute Vectors containing HIV packaging sequences, packaging defective HIV vectors, and uses thereof
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
ATE154246T1 (de) 1990-07-27 1997-06-15 Isis Pharmaceuticals Inc Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
FI930455A7 (fi) 1990-08-03 1993-03-24 Sanofi Sa Yhdisteitä ja menetelmiä geeniekspression inhiboimiseksi
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
EP0549686A4 (en) 1990-09-20 1995-01-18 Gilead Sciences Inc Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
DE69132510T2 (de) 1990-11-08 2001-05-03 Hybridon, Inc. Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden
GB9100304D0 (en) 1991-01-08 1991-02-20 Ici Plc Compound
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
EP0538194B1 (de) 1991-10-17 1997-06-04 Novartis AG Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
ATE262374T1 (de) 1991-11-22 2004-04-15 Affymetrix Inc Kombinatorische strategien für polymersynthese
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US6235887B1 (en) 1991-11-26 2001-05-22 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US6277603B1 (en) 1991-12-24 2001-08-21 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
DE4203923A1 (de) 1992-02-11 1993-08-12 Henkel Kgaa Verfahren zur herstellung von polycarboxylaten auf polysaccharid-basis
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
AU4528493A (en) 1992-06-04 1994-01-04 Regents Of The University Of California, The In vivo gene therapy with intron-free sequence of interest
WO1994000569A1 (en) 1992-06-18 1994-01-06 Genpharm International, Inc. Methods for producing transgenic non-human animals harboring a yeast artificial chromosome
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
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
AU4769893A (en) 1992-07-17 1994-02-14 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
US5783701A (en) 1992-09-08 1998-07-21 Vertex Pharmaceuticals, Incorporated Sulfonamide inhibitors of aspartyl protease
AU680459B2 (en) 1992-12-03 1997-07-31 Genzyme Corporation Gene therapy for cystic fibrosis
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
ES2142934T3 (es) 1993-02-19 2000-05-01 Nippon Shinyaku Co Ltd Derivado del glicerol, dispositivo y composicion farmaceutica.
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
EP0691977B1 (en) 1993-03-31 1997-11-26 Sanofi Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US6191105B1 (en) 1993-05-10 2001-02-20 Protein Delivery, Inc. Hydrophilic and lipophilic balanced microemulsion formulations of free-form and/or conjugation-stabilized therapeutic agents such as insulin
US5955591A (en) 1993-05-12 1999-09-21 Imbach; Jean-Louis Phosphotriester oligonucleotides, amidites and method of preparation
US6015886A (en) 1993-05-24 2000-01-18 Chemgenes Corporation Oligonucleotide phosphate esters
US6294664B1 (en) 1993-07-29 2001-09-25 Isis Pharmaceuticals, Inc. Synthesis of oligonucleotides
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
KR960705837A (ko) 1993-11-16 1996-11-08 라이오넬 엔. 사이몬 비포스포네이트 뉴클레오시드간 연결기와 혼합된 비대칭적으로 순수한 포스포네이트 뉴클레오시드간 연결기를 갖는 합성 올리고머(Synthetic Oligomers Having Chirally Pure Phosphonate Internucleosidyl Linkages Mixed with Non-Phosphonate Internucleosidyl Linkages)
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
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
US5599922A (en) 1994-03-18 1997-02-04 Lynx Therapeutics, Inc. Oligonucleotide N3'-P5' phosphoramidates: hybridization and nuclease resistance properties
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US6054299A (en) 1994-04-29 2000-04-25 Conrad; Charles A. Stem-loop cloning vector and method
US6074642A (en) 1994-05-02 2000-06-13 Alexion Pharmaceuticals, Inc. Use of antibodies specific to human complement component C5 for the treatment of glomerulonephritis
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5543152A (en) 1994-06-20 1996-08-06 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5556752A (en) 1994-10-24 1996-09-17 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US6608035B1 (en) 1994-10-25 2003-08-19 Hybridon, Inc. Method of down-regulating gene expression
US5665557A (en) 1994-11-14 1997-09-09 Systemix, Inc. Method of purifying a population of cells enriched for hematopoietic stem cells populations of cells obtained thereby and methods of use thereof
JP3269301B2 (ja) 1994-12-28 2002-03-25 豊田合成株式会社 ガラスラン用ゴム配合物
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
AU5359496A (en) 1995-03-06 1996-09-23 Isis Pharmaceuticals, Inc. Improved process for the synthesis of 2'-o-substituted pyrimidines and oligomeric compounds therefrom
JPH11508231A (ja) 1995-05-26 1999-07-21 ソマティックス セラピー コーポレイション 安定な脂質/核酸複合体を含む送達ビヒクル
DE69634084T2 (de) 1995-06-07 2005-12-08 Inex Pharmaceuticals Corp. Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5858397A (en) 1995-10-11 1999-01-12 University Of British Columbia Liposomal formulations of mitoxantrone
CA2234931C (en) 1995-10-16 2010-01-19 Dana-Farber Cancer Institute Novel expression vectors and methods of use
US6160109A (en) 1995-10-20 2000-12-12 Isis Pharmaceuticals, Inc. Preparation of phosphorothioate and boranophosphate oligomers
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US5858401A (en) 1996-01-22 1999-01-12 Sidmak Laboratories, Inc. Pharmaceutical composition for cyclosporines
US5994316A (en) 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US6444423B1 (en) 1996-06-07 2002-09-03 Molecular Dynamics, Inc. Nucleosides comprising polydentate ligands
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
US6576752B1 (en) 1997-02-14 2003-06-10 Isis Pharmaceuticals, Inc. Aminooxy functionalized oligomers
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
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
CA2294988C (en) 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
EP2253639A1 (en) 1997-09-12 2010-11-24 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6528640B1 (en) 1997-11-05 2003-03-04 Ribozyme Pharmaceuticals, Incorporated Synthetic ribonucleic acids with RNAse activity
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
AU2092599A (en) 1997-12-24 1999-07-19 Vertex Pharmaceuticals Incorporated Prodrugs of aspartyl protease inhibitors
US6436989B1 (en) 1997-12-24 2002-08-20 Vertex Pharmaceuticals, Incorporated Prodrugs of aspartyl protease inhibitors
US7273933B1 (en) 1998-02-26 2007-09-25 Isis Pharmaceuticals, Inc. Methods for synthesis of oligonucleotides
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US6531590B1 (en) 1998-04-24 2003-03-11 Isis Pharmaceuticals, Inc. Processes for the synthesis of oligonucleotide compounds
US20030096775A1 (en) * 2001-10-23 2003-05-22 Isis Pharmaceuticals Inc. Antisense modulation of complement component C3 expression
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
ATE237312T1 (de) 1998-07-20 2003-05-15 Protiva Biotherapeutics Inc In liposomen verkapselte nukleinsäurekomplexe
AU6430599A (en) 1998-10-09 2000-05-01 Cytogenix, Inc. Enzymatic synthesis of ssdna
BR9914773A (pt) 1998-10-09 2002-02-05 Ingene Inc Conjunto de elementos genéricos, método para a produção de dna de cordão único, transcrição de mrna, construção de ácido nucléico, transcrição de ssdna, vetor, sistema vetor, célula hospedeira, conjunto para a produção de uma sequência de ácido nucléico de cordão único, método para a produção in vivo ou in vitro de uma sequência de ácido nucléico de cordão único, transcrição de cdna de cordão único, ácido nucléico inibidor, molécula heteroduplex, e composição farmacêutica
US6465628B1 (en) 1999-02-04 2002-10-15 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
TWI260322B (en) 1999-02-12 2006-08-21 Vertex Pharma Inhibitors of aspartyl protease
JP2002537343A (ja) 1999-02-23 2002-11-05 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 多重粒子製剤
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6593466B1 (en) 1999-07-07 2003-07-15 Isis Pharmaceuticals, Inc. Guanidinium functionalized nucleotides and precursors thereof
US6147200A (en) 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
WO2001053307A1 (en) 2000-01-21 2001-07-26 Geron Corporation 2'-arabino-fluorooligonucleotide n3'→p5'phosphoramidates: their synthesis and use
IT1318539B1 (it) 2000-05-26 2003-08-27 Italfarmaco Spa Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente
ATE325806T1 (de) 2000-10-04 2006-06-15 Santaris Pharma As Verbesserte synthese von purin-blockierten nukleinsäure-analoga
US6558313B1 (en) * 2000-11-17 2003-05-06 Embro Corporation Vein harvesting system and method
WO2003015698A2 (en) 2001-08-13 2003-02-27 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
EP2314690A1 (en) 2002-07-10 2011-04-27 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. RNA-interference by single-stranded RNA molecules
US6878805B2 (en) 2002-08-16 2005-04-12 Isis Pharmaceuticals, Inc. Peptide-conjugated oligomeric compounds
ES2373649T3 (es) 2002-09-20 2012-02-07 The Trustees Of The University Of Pennsylvania Análogos de compstatina con actividad mejorada.
WO2004044139A2 (en) 2002-11-05 2004-05-27 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
CA2554212A1 (en) 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using multifunctional short interfering nucleic acid (multifunctional sina)
CN101052717A (zh) * 2004-05-11 2007-10-10 α基因株式会社 诱发rna干扰的多核苷酸以及使用该多核苷酸的基因表达抑制方法
JP2008501694A (ja) * 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク. 遺伝子調節の使用のために個別に修飾された鎖を有する二本鎖組成物
US7919094B2 (en) 2004-06-10 2011-04-05 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
SG156690A1 (en) * 2004-10-27 2009-11-26 Univ Vanderbilt Mammalian genes involved in infection
JP2008537551A (ja) 2005-03-31 2008-09-18 カランド ファーマシューティカルズ, インコーポレイテッド リボヌクレオチドレダクターゼサブユニット2の阻害剤およびその使用
US7794019B2 (en) * 2005-07-08 2010-09-14 Charles Hoberman Folding structures made of thick hinged sheets
JP5302004B2 (ja) 2005-11-28 2013-10-02 ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア 強力なコンプスタチン類似体
EP1963508A2 (en) * 2005-11-30 2008-09-03 Intradigm Corporation COMPOSITIONS AND METHODS OF USING siRNA TO KNOCKDOWN GENE EXPRESSION AND TO IMPROVE SOLID ORGAN AND CELL TRANSPLANTATION
DE602006014026D1 (de) * 2005-12-22 2010-06-10 Opko Ophthalmics Llc Zusammensetzungen und verfahren zur regulierung eines komplementsystems
CN102908630B (zh) 2006-01-27 2014-11-19 Isis制药公司 6-修饰的双环核酸类似物
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
US8362229B2 (en) 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
KR20120115412A (ko) 2006-04-07 2012-10-17 이데라 파마슈티칼즈, 인코포레이티드 Tlr7 및 tlr8에 대한 안정화된 면역 조절성 rna〔simra〕 화합물
JP5441688B2 (ja) 2006-05-11 2014-03-12 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 5’修飾二環式核酸類似体
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
US20100105134A1 (en) 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
NZ580712A (en) 2007-05-22 2011-12-22 Marina Biotech Inc Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
US8442776B2 (en) 2007-06-08 2013-05-14 The Trustees Of The University Of Pennsylvania Structure of compstatin-C3 complex and use for rational drug design
ES2376507T5 (es) 2007-07-05 2015-08-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos 6-disustituidos
WO2009014887A2 (en) 2007-07-09 2009-01-29 Idera Pharmaceuticals, Inc. Stabilized immune modulatory rna (simra) compounds
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
BRPI0921237A2 (pt) 2008-11-10 2015-09-22 Alexion Pharma Inc métodos e composições para o tratamento de distúrbios associados ao complemento
CA2744093A1 (en) 2008-12-03 2010-06-10 Marina Biotech, Inc. Una oligomer structures for therapeutic agents
CA2760839C (en) 2009-05-01 2019-02-12 The Trustees Of The University Of Pennsylvania Modified compstatin with peptide backbone and c-terminal modifications
CA2764158A1 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
LT2440183T (lt) 2009-06-10 2018-08-10 Arbutus Biopharma Corporation Patobulinta lipido kompozicija
WO2011005861A1 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
CN104673795A (zh) 2009-08-27 2015-06-03 艾德拉药物股份有限公司 用于抑制基因表达的组合物及其用途
US9358266B2 (en) 2010-02-25 2016-06-07 The Trustees Of The University Of Pennsylvania Treatment of sepsis using complement inhibitors
US20130190383A1 (en) 2010-04-26 2013-07-25 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2012037254A1 (en) * 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. MODIFIED iRNA AGENTS
WO2012174055A1 (en) 2011-06-13 2012-12-20 The Trustees Of The University Of Pennsylvania Wound healing using complement inhibitors
BR112013033272A2 (pt) 2011-06-22 2020-11-10 Apellis Pharmaceuticals, Inc. composição farmaceutica compreendendo um inibidor do complemento e um agente anti-th17, e usos dos mesmos
CN104136451A (zh) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 具有构象限制的单体的核酸化合物的合成和用途
AU2012304442B2 (en) 2011-09-07 2017-05-25 The Trustees Of The University Of Pennsylvania Compstatin analogs with improved pharmacokinetic properties
CN103917647B (zh) * 2011-11-03 2020-07-10 夸克制药公司 用于神经保护的方法和组合物
AU2012340159B2 (en) * 2011-11-18 2017-09-07 Alnylam Pharmaceuticals, Inc. RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
KR20150103280A (ko) * 2013-01-08 2015-09-09 베니텍 바이오파마 리미티드 연령-관련 황반 변성 치료
LT3043827T (lt) * 2013-09-13 2019-08-12 Ionis Pharmaceuticals, Inc. Komplemento faktoriaus b moduliatoriai
KR20240170595A (ko) 2013-12-12 2024-12-03 알닐람 파마슈티칼스 인코포레이티드 보체 성분 iRNA 조성물 및 이의 이용 방법
KR20160012758A (ko) * 2014-07-25 2016-02-03 삼성전자주식회사 영상 진단 보조 장치 및 방법
CN114525280A (zh) 2015-09-02 2022-05-24 阿尔尼拉姆医药品有限公司 程序性细胞死亡1配体1(PD-L1)的iRNA组合物及其使用方法
EP3704252A1 (en) 2017-11-01 2020-09-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070088154A1 (en) * 2002-11-14 2007-04-19 Dharmacon Inc. siRNA targeting complement factor B
WO2008036841A2 (en) * 2006-09-22 2008-03-27 Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
WO2013074974A2 (en) * 2011-11-18 2013-05-23 Alnylam Pharmaceuticals, Inc. Modified rnai agents

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