公开(公告)号：US20240116973A1

公开(公告)日：2024-04-11

申请号：US18272448

申请日：2022-01-14

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Muthiah MANOHARAN ,  Dhrubajyoti DATTA

IPC分类号： C07H19/16 ,  C12N15/113

CPC分类号： C07H19/16 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/333 ,  C12N2310/344

摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) molecules comprising a 6-methyladenine nucleobase and capable of inhibiting the expression of a target gene. 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：US20230016929A1

公开(公告)日：2023-01-19

申请号：US17774477

申请日：2020-11-06

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Jayaprakash K. NAIR ,  Martin A. MAIER ,  Juan C. SALINAS ,  Shigeo MATSUDA ,  Alexander V. KEL'IN ,  Scott P. LENTINI ,  Guo HE ,  Michelle H. JUNG ,  Justin M. PIERSON ,  Muthiah MANOHARAN ,  Dale C. GUENTHER ,  Ivan ZLATEV ,  Christopher S. THEILE ,  Vasant R. JADHAV ,  Stuart MILSTEIN ,  Maja JANAS ,  Dhrubajyoti DATTA

IPC分类号： A61K31/713 ,  A61K48/00 ,  C12N15/113 ,  A61P25/28

摘要： One aspect of the present invention relates to a compound comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic monomers, containing one or more lipophilic moieties, conjugated to one or more positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic monomer-conjugated compound.

公开(公告)号：US20220389419A1

公开(公告)日：2022-12-08

申请号：US17767190

申请日：2020-10-13

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Muthiah MANOHARAN ,  Pawan KUMAR ,  Dhrubajyoti DATTA

IPC分类号： C12N15/11 ,  A61K31/713 ,  C12N15/113

摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. 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.