公开(公告)号：US20220155716A1

公开(公告)日：2022-05-19

申请号：US17494039

申请日：2021-10-05

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Alfica SEHGAL ,  David BUMCROT ,  Brian BETTENCOURT

IPC分类号： G03G15/00 ,  C12N15/113 ,  G03G15/08

摘要： The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina1.

公开(公告)号：US20230256001A1

公开(公告)日：2023-08-17

申请号：US17937256

申请日：2022-09-30

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Mark K. SCHLEGEL ,  Maja JANAS ,  Vasant R. JADHAV ,  Muthiah MANOHARAN ,  Kallanthottathil G. RAJEEV ,  Muthusamy JAYARAMAN ,  Alexander V. KEL'IN ,  Shigeo MATSUDA ,  Klaus CHARISSE ,  Jayaprakash K. NAIR ,  Martin A. MAIER ,  Alfica SEHGAL ,  Christopher BROWN ,  Christopher THEILE ,  Stuart MILSTEIN

IPC分类号： A61K31/713 ,  C12N15/113

CPC分类号： A61K31/713 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/351

摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2′-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.

公开(公告)号：US20160244752A1

公开(公告)日：2016-08-25

申请号：US14974650

申请日：2015-12-18

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Alfica SEHGAL ,  David BUMCROT ,  Brian BETTENCOURT

IPC分类号： C12N15/113

CPC分类号： G03G15/556 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2310/3521 ,  G03G15/0853 ,  G03G15/086 ,  G03G2215/0888

摘要： The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina1.

摘要翻译： 本文描述的技术涉及靶向Serpina1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Serpina1表达的方法。

公开(公告)号：US20210041823A1

公开(公告)日：2021-02-11

申请号：US17079699

申请日：2020-10-26

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Alfica SEHGAL ,  David BUMCROT ,  Brian BETTENCOURT

IPC分类号： G03G15/00 ,  C12N15/113 ,  G03G15/08

摘要： The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina1.

公开(公告)号：US20180120749A1

公开(公告)日：2018-05-03

申请号：US15843656

申请日：2017-12-15

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Alfica SEHGAL ,  David BUMCROT ,  Brian BETTENCOURT

IPC分类号： G03G15/00 ,  G03G15/08

CPC分类号： G03G15/556 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2310/3521 ,  G03G15/0853 ,  G03G15/086 ,  G03G2215/0888

摘要： The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina1.