公开(公告)号：US08598139B2

公开(公告)日：2013-12-03

申请号：US13568898

申请日：2012-08-07

Applicant: Kevin Fitzgerald ,  Gregory Hinkle ,  Akin Akinc ,  Stuart Milstein

Inventor： Kevin Fitzgerald ,  Gregory Hinkle ,  Akin Akinc ,  Stuart Milstein

IPC: A61K48/00 ,  C12N15/85 ,  C07H21/04

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。

公开(公告)号：US20120115934A1

公开(公告)日：2012-05-10

申请号：US13355930

申请日：2012-01-23

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

IPC: A61K31/713 ,  C07K14/00 ,  A61P7/00 ,  A61P3/00 ,  A61P9/12 ,  A61P13/12 ,  C07H21/02 ,  C07K14/79

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。

公开(公告)号：US20120115933A1

公开(公告)日：2012-05-10

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 SPRAGUE

Inventor： Antonin DE FOUGEROLLES ,  John L. DIENER ,  Emma HICKMAN ,  Gregory HINKLE ,  Stuart MILSTEIN ,  Anne-Marie PULICHINO ,  Andrew SPRAGUE

IPC: A61K31/713 ,  C07K14/42 ,  A61P9/12 ,  A61P5/40 ,  A61P3/00 ,  C07H21/02 ,  C07K2/00

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.

公开(公告)号：US08168775B2

公开(公告)日：2012-05-01

申请号：US12582669

申请日：2009-10-20

Applicant: Dinah Wen-Yee Sah ,  Gregory Hinkle ,  Rene Alvarez ,  Stuart Milstein ,  Qingmin Chen

Inventor： Dinah Wen-Yee Sah ,  Gregory Hinkle ,  Rene Alvarez ,  Stuart Milstein ,  Qingmin Chen

IPC: C07H21/04 ,  C07H21/02 ,  C12N15/63 ,  C12N15/85 ,  A61K48/00

CPC classification number: C12N15/113 ,  A61K9/0019 ,  A61K31/713 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/335 ,  C12N2310/3515 ,  C12N2320/30 ,  C12N2320/32 ,  C12N2310/3521

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.

Abstract translation: 本发明涉及靶向转甲状腺素蛋白（TTR）基因的双链核糖核酸（dsRNA）以及使用dsRNA抑制TTR表达的方法。