公开(公告)号：US20240254487A1

公开(公告)日：2024-08-01

申请号：US18532300

申请日：2023-12-07

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： JONATHAN EDWARD FARLEY ,  MARK K. SCHLEGEL ,  JAMES D. MCININCH ,  JEFFREY ZUBER ,  ADAM CASTORENO ,  STEPHEN ABBOTT ,  JOSEPH BARRY

IPC分类号： C12N15/113 ,  A61K48/00 ,  C12N15/85

CPC分类号： C12N15/113 ,  A61K48/005 ,  C12N15/85 ,  C12N2310/14 ,  C12N2310/315

摘要： The disclosure relates to double stranded ribonucleic acid interference (dsRNAi) agents and compositions targeting a microtubule-associated protein tau (MAPT) gene, as well as methods of inhibiting expression of a MAPT gene and methods of treating subjects having a MAPT-associated disease or disorder, e.g., Alzheimer's disease, frontotemporal dementia, progressive supranuclear palsy, or other tauopathies, using such dsRNAi agents and compositions.

公开(公告)号：US20240301426A1

公开(公告)日：2024-09-12

申请号：US18575157

申请日：2022-06-29

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： LAN THI HOANG DANG ,  JAMES D. MCININCH ,  MARK K. SCHLEGEL ,  ADAM CASTORENO ,  TUYEN M. NGUYEN ,  JOSEPH BARRY ,  MATTHEW STRICOS ,  SARAH LEBLANC

IPC分类号： C12N15/113 ,  G01N33/573

CPC分类号： C12N15/1137 ,  C12Y207/11001 ,  G01N33/573 ,  C12N2310/14 ,  C12N2310/3515 ,  G01N2333/912 ,  G01N2800/2835

摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a leucine-rich repeat kinase 2 (LRRK2) gene, as well as methods of inhibiting expression of a LRRK2 gene and methods of treating subjects having a LRRK2-associated disease or disorder, e.g., Parkinson's disease, using such dsRNAi agents and compositions.