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51. 发明专利

CA2658183A1 RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEICACID (SINA) 未知
公开(公告)号：CA2658183A1
公开(公告)日：2008-01-24
申请号：CA2658183
申请日：2007-07-17
申请人： SIRNA THERAPEUTICS INC
发明人： GUERCIOLINI ROBERTO , VARGEESE CHANDRA , JADHAV VASANT , VAISH NARENDRA K , MCSWIGGEN JAMES
IPC分类号： A61K48/00 , C12N15/113
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of Proprotein Convertase Subtilisin Kexin 9 (PCSK 9) gene expression and/or activity. The present invention is also directed t o compounds, compositions, and methods relating to traits, diseases and cond itions that respond to the modulation of expression and/or activity of genes involved in Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expressio n pathways or other cellular processes that mediate the maintenance or devel opment of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic ac id molecules, such as short interfering nucleic acid (siNA), short interferi ng RNA (siRNA), double-stranded RNA (dsRNA), micro- RNA (miRNA), and short h airpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) ag ainst Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression, incl uding cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present inventi on also relates to small nucleic acid molecules, such as siNA, siRNA, and ot hers that can inhibit the function of endogenous RNA molecules, such as endo genous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfe ring RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nu cleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, f or example, in providing compositions to prevent, inhibit, or reduce metabol ic diseases, traits and conditions, including but not limited to hyperlipide mia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hyperten sion, diabetis (e.g., type I and/or type II diabetis), insulin resistance, o besity and/or other disease states, conditions, or traits that are associate d with gene expression or activity in a subject or organism.

52. 发明专利

CA2492208A1 DEPROTECTION AND PURIFICATION OF OLIGONUCLEOTIDES AND THEIR DERIVATIVES 未知
公开(公告)号：CA2492208A1
公开(公告)日：2004-01-22
申请号：CA2492208
申请日：2003-07-14
申请人： SIRNA THERAPEUTICS INC
发明人： VARGEESE CHANDRA , SHAFFER CHRISTOPHER , BOWMAN KEITH
IPC分类号： C12N15/09 , C07H21/00 , C07H21/02 , C12N15/10 , C07H1/02
摘要： Method for synthesis, deprotection, and/or purification of nucleic acid molecules, such as oligonucleotides comprising one or more ribonucleotides. Such nucleic acid molecules include siRNA, dsRNA, ribozymes, antisense, and aptamers.

53. 发明专利

CA2447161A1 CONJUGATES AND COMPOSITIONS FOR CELLULAR DELIVERY 未知
公开(公告)号：CA2447161A1
公开(公告)日：2002-11-28
申请号：CA2447161
申请日：2002-05-20
申请人： SIRNA THERAPEUTICS INC
发明人： SHAFFER CHRISTOPHER , BLATT LAWRENCE , KARPEISKY ALEXANDER , BEIGELMAN LEONID , MATULIC-ADAMIC JASENKA , VARGEESE CHANDRA
IPC分类号： G01N33/53 , A61K20060101 , A61K6/00 , A61K31/519 , A61K31/52 , A61K31/7048 , A61K31/7052 , A61K31/706 , A61K31/7088 , A61K31/712 , A61P31/12 , A61P31/14 , A61P31/16 , A61P31/18 , A61P31/20 , A61P35/00 , A61P35/02 , A61P43/00 , C07D475/00 , C07D475/04 , C07H19/16 , C07H19/167 , C07H19/207 , C07H19/22 , C07H21/00 , C07H21/04 , C12P17/18 , C12Q1/68
摘要： This invention features conjugates, degradable linkers, compositions, method s of synthesis, and applications thereof, including folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HAS) derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys and aptamers.

54. 发明公开

EP1622572A4 CONJUGATES AND COMPOSITIONS FOR CELLULAR DELIVERY 有权转让
标题翻译：赞助商ZUSAMMENSETZUNGENFÜRDIEZELLULÄREABGABE
公开(公告)号：EP1622572A4
公开(公告)日：2011-04-06
申请号：EP04775924
申请日：2004-04-30
申请人： SIRNA THERAPEUTICS INC
发明人： VARGEESE CHANDRA , HAEBERLI PETER , WANG WEIMIN , CHEN TONGQIAN
IPC分类号： A61K38/00 , A61K47/48 , C12N1/15 , C12N5/02 , C12N5/22 , C12N15/113 , C12N15/12 , C12N15/51
CPC分类号： C12N15/1138 , A61K38/00 , A61K47/544 , A61K47/549 , A61K47/551 , A61K47/554 , A61K47/60 , A61K47/64 , C12N15/111 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12Y103/01022 , C12Y604/01002 , C12N2310/3521
摘要： The present invention relates to specific short interfering nucleic acid (siNA) conjugates and pharmaceutical compositions comprising the same.
摘要翻译：本发明的特征在于缀合物，可降解接头，组合物，合成方法及其应用，包括胆固醇，叶酸，半乳糖，半乳糖胺，N-乙酰半乳糖胺，PEG，磷脂，肽和人血清白蛋白（HSA）衍生的生物活性化合物包括抗体，抗病毒剂，化学治疗剂，肽，蛋白质，激素，核苷，核苷酸，非核苷和核酸，包括酶核酸，DNA酶，同源酶，反义，dsRNA，siNA，siRNA，三重寡核苷酸，2,5-A 嵌合体，诱饵和适体。

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