公开(公告)号：WO2022087329A1

公开(公告)日：2022-04-28

申请号：PCT/US2021/056131

申请日：2021-10-22

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： NOETZLI, Leila ,  CASTORENO, Adam ,  PARKER, Margaret ,  SCHLEGEL, Mark, K. ,  MCININCH, James, D. ,  ZUBER, Jeffrey

IPC分类号： C12N15/113

摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the mucin 5B (MUC5B) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a MUC5B gene and to methods of treating or preventing a MUC5B-associated disease, such as a lung disease, e.g., cystic fibrosis, chronic obstructive pulmonary disease (COPD), and/or pulmonary fibrosis, e.g., idiopathic pulmonary fibrosis (IPF), in a subject.

公开(公告)号：WO2021237097A1

公开(公告)日：2021-11-25

申请号：PCT/US2021/033663

申请日：2021-05-21

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： TREMBLAY, Frederic ,  MCININCH, James, D.

IPC分类号： C12N15/113

摘要： The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the MARC1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of MARC1.

公开(公告)号：WO2021076828A1

公开(公告)日：2021-04-22

申请号：PCT/US2020/055877

申请日：2020-10-16

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： WARD, Lucas, D. ,  TU, Ho-Chou ,  MCININCH, James, D. ,  NIOI, Paul

IPC分类号： C12N15/113

摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting a solute carrier family member gene, e.g., SLC30A10, or SLC39A8. The invention also relates to methods of using such RNAi agents to inhibit expression of a solute carrier family member gene, e.g., an SLC30A10 gene, or an SLC39A8 gene, and to methods of preventing and treating a solute carrier family member-associated disorder, e.g., a hypermanganesemia.

公开(公告)号：WO2023014677A1

公开(公告)日：2023-02-09

申请号：PCT/US2022/039111

申请日：2022-08-02

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： SCHLEGEL, Mark, K. ,  CASTORENO, Adam ,  MCININCH, James, D.

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/61 ,  C07H21/02

摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the transthyretin (TTR) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an TTR gene and to methods of preventing and treating an TTR-associated disorder, e.g., senile systemic amyloidosis (SSA), systemic familial amyloidosis, familial amyloidotic polyneuropathy (FAP), familial amyloidotic cardiomyopathy (FAC), leptomeningeal/Central Nervous System (CNS) amyloidosis, and hyperthyroxinemia.

公开(公告)号：WO2022231999A1

公开(公告)日：2022-11-03

申请号：PCT/US2022/026097

申请日：2022-04-25

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  GARFINKEL, Benjamin, P.

发明人： DEATON, Aimee, M. ,  GANSNER, John, Michael ,  MCININCH, James, D. ,  SCHLEGEL, Mark, K.

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/54 ,  A61P7/00

摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, β-thalassemia(e.g., β-thalassemia major and β-thalassemia intermedia), polycthemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.

公开(公告)号：WO2022212153A1

公开(公告)日：2022-10-06

申请号：PCT/US2022/021635

申请日：2022-03-24

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： ERBE, David ,  MCININCH, James, D.

IPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/351 ,  C12Y105/05

摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the proline dehydrogenase 2 (PRODH2) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a PRODH2 gene and to methods of preventing and treating a PRODH2-associated disorder, e.g., primary hyperoxalurias and/or kidney stone diseases.

公开(公告)号：WO2022182574A1

公开(公告)日：2022-09-01

申请号：PCT/US2022/016890

申请日：2022-02-18

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  NOETZLI, Leila ,  MCININCH, James, D. ,  TREMBLAY, Frederic ,  SCHLEGEL, Mark, K. ,  CASTORENO, Adam

发明人： NOETZLI, Leila ,  MCININCH, James, D. ,  TREMBLAY, Frederic ,  SCHLEGEL, Mark, K. ,  CASTORENO, Adam

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P3/06 ,  A61P3/08 ,  A61P13/12

摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KHK gene and to methods of treating or preventing a KHK-associated disorder in a subject.

公开(公告)号：WO2022103999A1

公开(公告)日：2022-05-19

申请号：PCT/US2021/059047

申请日：2021-11-12

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： KEATING, Mark ,  MCININCH, James, D. ,  SCHLEGEL, Mark, K.

IPC分类号： C12N15/113

摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the Coagulation Factor V (F5) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an F5 gene and to methods of treating or preventing an F5-associated disease, e.g., a disorder associated with thrombosis, in a subject.

公开(公告)号：WO2021206922A1

公开(公告)日：2021-10-14

申请号：PCT/US2021/024076

申请日：2021-03-25

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： AKINC, Akin ,  MCININCH, James, D. ,  ANGLERO-RODRIGUEZ, Yesseinia ,  SCHLEGEL, Mark, K.

IPC分类号： C12N15/113

摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the transmembrane serine protein 2 (TMPRSS2) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS2 gene and to methods of treating or preventing a TMPRSS2-associated disease, e.g., COVID-19, in a subject.

公开(公告)号：WO2021119226A1

公开(公告)日：2021-06-17

申请号：PCT/US2020/064159

申请日：2020-12-10

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  REGENERON PHARMACEUTICALS, INC.

发明人： FISHILEVICH, Elane ,  MILSTEIN, Stuart ,  BROWN, Kirk ,  ZIMMERMANN, Tracy ,  MCININCH, James, D. ,  FRENDEWEY, David ,  CHIAO, Eric ,  SHARMA-KANNING, Aarti ,  GAGLIARDI, Anthony ,  DROGUETT, Gustavo ,  DUBOSE, Brittany ,  ZAMBROWICZ, Brian

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

摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disorder, e.g., C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.