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1. 发明申请

WO2012135246A3 COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE 审中-公开
标题翻译：抑制TMPRSS6基因表达的组合物和方法
公开(公告)号：WO2012135246A3
公开(公告)日：2014-05-01
申请号：PCT/US2012030786
申请日：2012-03-28
申请人： ALNYLAM PHARMACEUTICALS INC , BUMCROT DAVID , BETTENCOURT BRIAN , TOUDJARSKA IVANKA
发明人： BUMCROT DAVID , BETTENCOURT BRIAN , TOUDJARSKA IVANKA
IPC分类号： A61K48/00 , A61K9/127 , C12N15/11
CPC分类号： C12N15/1138 , A61K31/713 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2320/30 , C12Y304/21 , C12N2310/3521
摘要： The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 (Transmembrane Protease, Serine 6) gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6. Further disclosed are dsRNA sequences and pharmaceutical compositions comprising lipid formulation of the dsRNA and methods of using the dsRNA compositions for treating disorders associated with an increased expression of TMPRSS6.
摘要翻译：本公开涉及靶向TMPRSS6（跨膜蛋白酶，丝氨酸6）基因的双链核糖核酸（dsRNA）组合物，以及使用这种dsRNA组合物抑制TMPRSS6表达的方法。还公开了dsRNA序列和包含dsRNA的脂质制剂的药物组合物，以及使用dsRNA组合物治疗与TMPRSS6表达增加相关的疾病的方法。

2. 发明申请

WO2012177949A3 COMPOSITIONS AND METHODS FOR INHIBITION OF EXPRESSION OF PROTEIN C (PROC) GENES 审中-公开
标题翻译：用于抑制蛋白C（PROC）基因表达的组合物和方法
公开(公告)号：WO2012177949A3
公开(公告)日：2013-05-30
申请号：PCT/US2012043644
申请日：2012-06-21
申请人： ALNYLAM PHARMACEUTICALS INC , TOUDJARSKA IVANKA , MARAGANORE JOHN M , BETTENCOURT BRIAN , MILSTEIN STUART , MAIER MARTIN , CHARISSE KLAUS , KALLANTHOTTATHIL RAJEEV , KUCHIMANCHI SATYANARAYANA
发明人： TOUDJARSKA IVANKA , MARAGANORE JOHN M , BETTENCOURT BRIAN , MILSTEIN STUART , MAIER MARTIN , CHARISSE KLAUS , KALLANTHOTTATHIL RAJEEV , KUCHIMANCHI SATYANARAYANA
IPC分类号： A61K31/713
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC. At least one nucleotide ofthe dsRNA can be a modified nucleotide, e.g., a 2 -0-methyl modified nucleotide, a nucleotide comprising a 5'-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative or dodecanoic acid bisdecylamide group. Other examples of modified nucleotides include a 2'-deoxy-2'-fluoro modified nucleotide, a 2'-deoxymodified nucleotide, a locked nucleotide, an abasic nucleotide, 2'-amino-modified nucleotide, 2' -alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural base comprising nucleotide. A dsRNA of the invention can include one or more of any of these modified nucleotides.
摘要翻译：本发明涉及靶向PROC基因的双链核糖核酸（dsRNA）以及使用dsRNA抑制PROC表达的方法。 dsRNA的至少一个核苷酸可以是修饰的核苷酸，例如2-O-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸和与胆固醇衍生物或十二烷酸双癸酰胺基团连接的末端核苷酸。修饰的核苷酸的其它实例包括2'-脱氧-2'-氟修饰的核苷酸，2'-脱氧改性的核苷酸，锁定的核苷酸，脱碱核苷酸，2'-氨基修饰的核苷酸，2'-烷基修饰的核苷酸，吗啉代核苷酸，氨基磷酸酯和包含核苷酸的非天然碱基。本发明的dsRNA可以包括这些修饰的核苷酸中的一种或多种。

3. 发明专利

CA2746514C GNAQ TARGETED DSRNA COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION 未知
公开(公告)号：CA2746514C
公开(公告)日：2018-11-27
申请号：CA2746514
申请日：2009-12-10
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： GOLLOB JARED , HINKLE GREGORY , TOUDJARSKA IVANKA , BUMCROT DAVID
IPC分类号： A61K31/713 , A61P35/00 , C12N5/10 , C12N15/113
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.

4. 发明专利

CL2016000188A1 Composiciones de arní de serpinc1 y métodos de uso de las mismas 未知
公开(公告)号：CL2016000188A1
公开(公告)日：2016-08-19
申请号：CL2016000188
申请日：2016-01-25
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： MANOHARAN MUTHIAH , MAIER MARTIN , KUCHIMANCHI SATYANARAYANA , CHARISSE KLAUS , BETTENCOURT BRIAN , AKINC AKIN , SEHGAL ALFICA , TOUDJARSKA IVANKA , FOSTER DONALD , MILSTEIN STUART , RAJEV KALLANTHOTTATHIL G
IPC分类号： C12N15/113
摘要： ÁCIDO RIBONUCLEICO DE DOBLE HEBRA (ARNdh) PARA INHIBIR LA EXPRESIÓN DE SERPINC1; CÉLULA QUE LO CONTIENE; VECTOR QUE LO CODIFICA; CÉLULA HUÉSPED;COMPOSICIÓN FARMACÉUTICA; Y USO EN PREVENCIÓN O TRATAMIENTO DE TRASTORNOS QUE SE BENEFICIAN DE REDUCCIÓN DE EXPRESIÓN DE SERPINC1, TAL COMO HEMOFILIA (DIVISIONAL DE SOL. N°2869-14).

5. 发明专利

SG10201602369PA Double-Stranded RNA (dsRNA) For Inhibiting Expression Of TMPRSS6 未知
公开(公告)号：SG10201602369PA
公开(公告)日：2016-05-30
申请号：SG10201602369P
申请日：2012-03-28
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： BUMCROT DAVID , BETTENCOURT BRIAN , TOUDJARSKA IVANKA
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6.

6. 发明专利

CO7240361A2 Composiciones de arni de serpinc1 y métodos de uso de las mismas 未知
公开(公告)号：CO7240361A2
公开(公告)日：2015-04-17
申请号：CO14259376
申请日：2014-11-25
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： AKINC AKIN , SEHGAL ALFICA , TOUDJARSKA IVANKA , FOSTER DONALD , MILSTEIN STUART , BETTENCOURT BRIAN , MAIER MARTIN , CHARISSE KLAUS , KUCHIMANCHI SATYANARAYANA , RAJEEV KALLANTHOTTATHILL G , MANOHARAN MUTHIAH
IPC分类号： C07H21/04 , C12N15/113
摘要： la invención se refiere a ARNi, por ejemplo ácido ribonucleico de doble hebra (ARNdh), composiciones que se dirigen al gen serpinc1 y a métodos para utilizar dicho ARNi, por ejemplo ARNdh, composiciones para inhibir la expresión de Serpinc1 y métodos para tratar sujetos que tienen un transtorno hemorrágico como hemofilia.

7. 发明专利

CA2869922A1 SERPINC1 IRNA COMPOSITIONS AND METHODS OF USE THEREOF 未知
公开(公告)号：CA2869922A1
公开(公告)日：2013-10-31
申请号：CA2869922
申请日：2013-04-25
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： AKINC AKIN , SEHGAL ALFICA , TOUDJARSKA IVANKA , FOSTER DONALD , MILSTEIN STUART , BETTENCOURT BRIAN , MAIER MARTIN , CHARISSE KLAUS , KUCHIMANCHI SATYANARAYANA , RAJEEV KALLANTHOTTATHIL G , MANOHARAN MUTHIAH
IPC分类号： C07H21/04
摘要： The invention relates to iRNA, e.g., double- stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.

8. 发明专利

AU2012236700A1 Compositions and methods for inhibiting expression of TMPRSS6 gene 未知
公开(公告)号：AU2012236700A1
公开(公告)日：2013-10-03
申请号：AU2012236700
申请日：2012-03-28
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： BUMCROT DAVID , BETTENCOURT BRIAN , TOUDJARSKA IVANKA
IPC分类号： A61K48/00
摘要： The invention relates to double- stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6.

9. 发明专利

NZ588280A COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF EG5 AND VEGF GENES 未知
公开(公告)号：NZ588280A
公开(公告)日：2012-11-30
申请号：NZ58828009
申请日：2009-03-05
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： BUMCROT DAVID , SAH DINAH WEN-YEE , TOUDJARSKA IVANKA
IPC分类号： A61K31/713 , C07H21/00 , C12N15/113
摘要： Disclosed is a composition comprising a first double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a human kinesin family member 11 (Eg5/KSP) gene in a cell and a second dsRNA for inhibiting expression of a human VEGF in a cell, wherein: both said first and said second dsRNA are formulated in a stable nucleic acid lipid particle (SNALP) comprising 1,2,-dilinoleyloxy-N,N-dimethyl-3-aminopropane (DLinDMA), cholesterol, dipalmitoyl phosphatidylcholine (DPPC), and (3-N-[ù-Methoxy poly(ethylene glycol)2000)carbamoyl]-1,2-dimyristyloxy-propylamine) (PEG2000-C-DMA); said first dsRNA consists of a first sense strand and a first antisense strand, and said first sense strand comprises a first sequence and said first antisense strand comprises a second sequence complementary to at least 15 contiguous nucleotides of SEQ ID NO:1311 (5'- UCGAGAAUCUAAACUAACU-3'), wherein said first sequence is complementary to said second sequence and wherein said first dsRNA is between 15 and 30 base pairs in length; and said second dsRNA consists of a second sense strand and a second antisense strand, said second sense strand comprising a third sequence and said second antisense strand comprising a fourth sequence complementary to at least 15 contiguous nucleotides of SEQ ID NO:1538 (5'- GCACAUAGGAGAGAUGAGCUU-3'), wherein said third sequence is complementary to said fourth sequence and wherein each strand is between 15 and 30 base pairs in length.

10. 发明专利

AU2009221775B2 Compositions and methods for inhibiting expression of Eg5 and VEGF genes 未知
公开(公告)号：AU2009221775B2
公开(公告)日：2015-05-07
申请号：AU2009221775
申请日：2009-03-05
申请人： ALNYLAM PHARMACEUTICALS INC
发明人： BUMCROT DAVID , SAH DINAH WEN-YEE , TOUDJARSKA IVANKA
IPC分类号： A61K31/713 , A61K48/00 , C07H21/00 , C12N15/113
摘要： This invention relates to compositions containing double-stranded ribonucleic acid (dsRNA) in a SNALP formulation, and methods of using the compositions to inhibit the expression of the Eg5 and Vascular Endothelial Growth Factor (VEGF), and methods of using the compositions to treat pathological processes mediated by Eg5 and VEGF expression, such as cancer.

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