公开(公告)号：US20240254493A1

公开(公告)日：2024-08-01

申请号：US18562787

申请日：2022-05-27

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： JEFFREY ZUBER ,  JAMES D. MCININCH ,  MARK K. SCHLEGEL ,  ADAM CASTORENO

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  C12Y402/01001 ,  C12N2310/14 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/3515

摘要： Carbonic anhydrase inhibitors have been shown to reduce aqueous humor production and thereby reduce intraocular pressure in the eye. Accordingly, there is a need for agents that can selectively and efficiently inhibit expression of the CA2 gene such that subjects having a CA2-associated disorder, such as glaucoma, can be effectively treated. The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting carbonic anhydrase 2 (CA2), and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of carbonic anhydrase 2.

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：US20230203486A1

公开(公告)日：2023-06-29

申请号：US17995035

申请日：2021-03-30

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： MANGALA MEENAKSHI SOUNDARAPANDIAN ,  JAMES D. MCININCH ,  ELANE FISHILEVICH ,  ADAM CASTORENO ,  CHARALAMBOS KAITTANIS ,  MARK K. SCHLEGEL ,  JONATHAN EDWARD FARLEY ,  JEFFREY ZUBER

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/312 ,  C12N2310/315 ,  C12N2310/3183

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.