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公开(公告)号：US08344127B2
公开(公告)日：2013-01-01
申请号：US13090580
申请日：2011-04-20
Applicant: Antonin De Fougerolles , John L. Diener , Emma Hickman , Gregory Hinkle , Stuart Milstein , Anne-Marie Pulichino , Andrew Sprague
Inventor： Antonin De Fougerolles , John L. Diener , Emma Hickman , Gregory Hinkle , Stuart Milstein , Anne-Marie Pulichino , Andrew Sprague
CPC classification number: C12N15/1138 , A61K31/713 , C12N2310/111 , C12N2310/14 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2310/351 , C12N2310/3513 , C12N2310/3515 , C12N2310/3517 , C12N2310/3521
Abstract: The present disclosure relates to RNAi agents useful in methods of treating Beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC.
Abstract translation: 本公开涉及可用于使用治疗有效量治疗β-ENAC相关疾病如囊性纤维化，1型假性醛固酮增多症（1例（PHA1），跛行综合征，高血压，碱中毒，低钾血症和肥胖相关性高血压）的方法中的RNAi剂 的RNAi试剂到β-ENaC。
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公开(公告)号：US20110166058A1
公开(公告)日：2011-07-07
申请号：US12970268
申请日：2010-12-16
Applicant: Gregory HINKLE , Satyanarayana KUCHIMANCHI , Stuart MILSTEIN , Markus WARMUTH , Wenlai ZHOU , Ping ZHU , Tracy S. ZIMMERMANN
Inventor： Gregory HINKLE , Satyanarayana KUCHIMANCHI , Stuart MILSTEIN , Markus WARMUTH , Wenlai ZHOU , Ping ZHU , Tracy S. ZIMMERMANN
IPC: A61K38/00 , C07H21/02 , A61K31/7105 , C12N5/071 , A61P35/00 , A61P37/02 , A61P31/12 , A61P35/04
CPC classification number: C12N15/113 , A61K31/713 , A61K45/06 , C07H21/02 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2320/31 , C12N2320/51 , C12N2320/53 , Y02A50/467 , C12N2310/3521
Abstract: The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.
Abstract translation: 本公开涉及使用治疗有效量的RNAi剂对HSF治疗热休克因子1（HSF1）相关疾病如癌症和病毒性疾病的方法。
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13.发明授权Compositions and methods for inhibiting expression of klf-1 and bcl11a genes 有权用于抑制klf-1和bcl11a基因表达的组合物和方法公开(公告)号：US09127275B2
公开(公告)日：2015-09-08
申请号：US13992669
申请日：2011-12-09
Applicant: Tatiana Novobrantseva , Brian Bettencourt , Stuart Milstein , Anna Borodovsky
Inventor： Tatiana Novobrantseva , Brian Bettencourt , Stuart Milstein , Anna Borodovsky
IPC: C12N15/11 , C12N15/113
CPC classification number: C12N15/113 , C12N2310/14 , C12N2310/344 , C12N2310/321 , C12N2310/3521
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the KLF1 gene and the BCL11A gene, and methods of using such dsRNA compositions to inhibit expression of KLF1 and BCL11 A, respectively.
Abstract translation: 本发明涉及靶向KLF1基因和BCL11A基因的双链核糖核酸（dsRNA）组合物，以及分别使用这些dsRNA组合物抑制KLF1和BCL11A表达的方法。
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14.发明申请Compositions and Methods for Inhibition of Expression of Protein C (PROC) Genes 有权抑制蛋白C（PROC）基因表达的组成和方法公开(公告)号：US20140142162A1
公开(公告)日：2014-05-22
申请号：US14127090
申请日：2012-06-21
Applicant: Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
Inventor： Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
IPC: C12N15/113
CPC classification number: C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
Abstract: 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 of the 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.
Abstract translation: 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2-0-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸，以及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。
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公开(公告)号：US20130012571A1
公开(公告)日：2013-01-10
申请号：US13614836
申请日：2012-09-13
Applicant: Antonin DE FOUGEROLLES , John L. DIENER , Emma HICKMAN , Gregory HINKLE , Stuart MILSTEIN , Anne-Marie PULICHINO , Andrew SPRAGUE
Inventor： Antonin DE FOUGEROLLES , John L. DIENER , Emma HICKMAN , Gregory HINKLE , Stuart MILSTEIN , Anne-Marie PULICHINO , Andrew SPRAGUE
CPC classification number: C12N15/1138 , A61K31/713 , C12N2310/111 , C12N2310/14 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2310/351 , C12N2310/3513 , C12N2310/3515 , C12N2310/3517 , C12N2310/3521
Abstract: The present disclosure relates to RNAi agents useful in methods of treating Beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC.
Abstract translation: 本公开涉及可用于使用治疗有效量治疗β-ENAC相关疾病如囊性纤维化，1型假性醛固酮增多症（1例（PHA1），跛行综合征，高血压，碱中毒，低钾血症和肥胖相关性高血压）的方法中的RNAi剂 的RNAi试剂到β-ENaC。
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Patent Agency Ranking
公开(公告)号：US08344130B2
公开(公告)日：2013-01-01
申请号：US13355903
申请日：2012-01-23
Applicant: Antonin De Fougerolles , John L. Diener , Emma Hickman , Gregory Hinkle , Stuart Milstein , Anne-Marie Pulichino , Andrew Griffin Sprague
Inventor： Antonin De Fougerolles , John L. Diener , Emma Hickman , Gregory Hinkle , Stuart Milstein , Anne-Marie Pulichino , Andrew Griffin Sprague
CPC classification number: C12N15/1138 , A61K31/713 , C12N2310/111 , C12N2310/14 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2310/351 , C12N2310/3513 , C12N2310/3515 , C12N2310/3517 , C12N2310/3521
Abstract: The present disclosure relates to RNAi agents useful in methods of treating Beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC.
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公开(公告)号：US20110015252A1
公开(公告)日：2011-01-20
申请号：US12816207
申请日：2010-06-15
Applicant: Kevin Fitzgerald , Gregory Hinkle , Akin Akinc , Stuart Milstein
Inventor： Kevin Fitzgerald , Gregory Hinkle , Akin Akinc , Stuart Milstein
IPC: A61K31/713 , A61P9/10
CPC classification number: A61K31/713 , A61K9/0019 , A61K9/1272 , A61K9/1273 , A61K38/1709 , A61K47/545 , A61K47/55 , A61K47/6911 , C12N15/1137 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515
Abstract: This invention relates to composition and methods using lipid formulated siRNA targeted to a PCSK9 gene.
Abstract translation: 本发明涉及使用针对PCSK9基因的脂质配制的siRNA的组合物和方法。
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公开(公告)号：US10023862B2
公开(公告)日：2018-07-17
申请号：US14371131
申请日：2013-01-08
Applicant: Dieter Huesken , Brian Bettencourt , David Bumcrot , Satyanarayana Kuchimanchi , Stuart Milstein , Michael Schlabach, Jr. , Frank P. Stegmeier , Markus Warmuth , Jan Weiler , NOVARTIS AG
Inventor： Brian Richard Bettencourt , David Anton Bumcrot , Dieter Huesken , Satyanarayana Kuchimanchi , Stuart Milstein , Michael Ray Schlabach, Jr. , Frank Peter Stegmeier , Markus Warmuth , Jan Weiler
IPC: A61P35/00 , A61P17/02 , C12N15/113 , A61K31/713
Abstract: The present disclosure relates to RNAi agents useful in methods of treating Beta-Catenin-related diseases such as adenomatous polyposis of the colon, colorectal cancer, basal cell carcinoma, breast cancer, kidney cancer, Wilms tumors, medulloblastoma, ovarian cancer, adrenocortical tumors, gastric cancer, liver cancer, melanoma, pancreatic cancers, prostate cancer, renal cancer, ectopic teeth and taste papillae, skin cancer, pilomatrixoma, anaplastic thyroid carcinoma, and uterine carcinosarcoma, oligodontia, osteoporosis, ageing, degenerative diseases, bedsores, chronic wounds and impaired wound healing, and similar and related diseases, using a therapeutically effective amount of a RNAi agent to Beta-Catenin.
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19.发明授权Compositions and methods for inhibition of expression of protein C (PROC) genes 有权抑制蛋白C（PROC）基因表达的组合物和方法公开(公告)号：US09068184B2
公开(公告)日：2015-06-30
申请号：US14127090
申请日：2012-06-21
Applicant: Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
Inventor： Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
IPC: A61K48/00 , C12N15/11 , C07H21/02 , C07H21/04 , C12N15/113
CPC classification number: C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
Abstract: 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 of the 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.
Abstract translation: 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2-0-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸，以及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。
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公开(公告)号：US20140039039A1
公开(公告)日：2014-02-06
申请号：US14054166
申请日：2013-10-15
Applicant: Gregory HINKLE , Satyanarayana KUCHIMANCHI , Stuart MILSTEIN , Markus WARMUTH , Wenlai ZHOU , Ping ZHU , Tracy S. ZIMMERMANN
Inventor： Gregory HINKLE , Satyanarayana KUCHIMANCHI , Stuart MILSTEIN , Markus WARMUTH , Wenlai ZHOU , Ping ZHU , Tracy S. ZIMMERMANN
IPC: C07H21/02 , A61K31/713 , A61K45/06 , A61K47/48
CPC classification number: C12N15/113 , A61K31/713 , A61K45/06 , C07H21/02 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2320/31 , C12N2320/51 , C12N2320/53 , Y02A50/467 , C12N2310/3521
Abstract: The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.
Abstract translation: 本公开涉及使用治疗有效量的RNAi剂对HSF治疗热休克因子1（HSF1）相关疾病如癌症和病毒性疾病的方法。
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