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公开(公告)号：US20220364088A1
公开(公告)日：2022-11-17
申请号：US17721530
申请日：2022-04-15
发明人： Mark Keating , James D. McIninch , Elane Fishilevich , Kristina Yucius , Sarah Solomon , Mark K. Schlegel , Adam Castoreno , Charalambos Kaittanis
IPC分类号： C12N15/113 , C12N15/66
摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, pemphigus, e.g., pemphigus vulgaris (PV) and pemphigus foliaceus (PF), and C3 glomerulopathy.
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公开(公告)号：US20200339991A1
公开(公告)日：2020-10-29
申请号：US16925286
申请日：2020-07-09
发明人： Stuart Milstein , Kirk Brown , Jayaprakash Nair , Martin Maier , Vasant Jadhav , Mark Keating , Adam Castoreno , Patrick Haslett , Mangala Meenakshi Soundarapandian , Kevin Fitzgerald
IPC分类号： C12N15/113 , A61K9/00
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions.
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公开(公告)号：US20240209369A1
公开(公告)日：2024-06-27
申请号：US18594132
申请日：2024-03-04
发明人： Mark Keating , James D. McIninch , Elane Fishilevich , Kristina Yucius , Sarah Solomon , Mark K. Schlegel , Adam Castoreno , Charalambos Kaittanis
IPC分类号： C12N15/113 , C12N15/66
CPC分类号： C12N15/113 , C12N15/66 , C12N2310/3125 , C12N2310/315 , C12N2310/321
摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, pemphigus, e.g., pemphigus vulgaris (PV) and pemphigus foliaceus (PF), and C3 glomerulopathy.
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公开(公告)号：US20240218366A1
公开(公告)日：2024-07-04
申请号：US18586689
申请日：2024-02-26
IPC分类号： C12N15/113 , A61K9/00 , A61K45/06 , A61K47/54 , A61P7/02
CPC分类号： C12N15/113 , A61K9/0019 , A61K45/06 , A61K47/549 , A61P7/02 , C12N2310/11 , C12N2310/3125 , C12N2310/313
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.
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公开(公告)号：US20220002724A1
公开(公告)日：2022-01-06
申请号：US17314909
申请日：2021-05-07
发明人： Stuart Milstein , Kirk Brown , Jayaprakash Nair , Martin Maier , Vasant Jadhav , Mark Keating , Adam Castoreno , Patrick Haslett , Mangala Meenakshi Soundarapandian , Kevin Fitzgerald
IPC分类号： C12N15/113 , A61K9/00
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions.
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6.发明公开公开(公告)号：US20240200061A1
公开(公告)日：2024-06-20
申请号：US18225919
申请日：2023-07-25
发明人： Jayaprakash K. Nair , Martin A. Maier , Vasant R. Jadhav , Mark Keating , Kevin Fitzgerald , Stuart Milstein , Kirk Brown , Muthiah Manoharan
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2320/34
摘要： The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated ocular diseases.
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7.发明公开公开(公告)号：US20230304002A1
公开(公告)日：2023-09-28
申请号：US17736251
申请日：2022-05-04
发明人： Jayaprakash K. Nair , Martin A. Maier , Vasant R. Jadhav , Mark Keating , Kevin Fitzgerald , Stuart Milstein , John R. Petrulis
IPC分类号： C12N15/113 , A61K47/54
CPC分类号： C12N15/113 , A61K47/543 , A61K47/549 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3513 , C12N2310/3515 , C12N2320/32
摘要： The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated ocular diseases.
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公开(公告)号：US20230272382A1
公开(公告)日：2023-08-31
申请号：US17900919
申请日：2022-09-01
发明人： Mark Keating , James D. McIninch
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/3515 , C12N2310/321
摘要： The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the complement component C3 gene and methods of using such iRNA agents for treating or preventing C3-associated ocular diseases or C3-associated neurodegenerative diseases.
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9.发明公开公开(公告)号：US20240294906A1
公开(公告)日：2024-09-05
申请号：US18007007
申请日：2021-07-28
发明人： Elane Fishilevich , Mark Keating , Stuart Milstein , Mark Schlegel , Adam Castoreno , Charalambos Kaittanis , Sarah Hyde , Alex Eaton , James McIninch , Jason Gilbert
IPC分类号： C12N15/113 , A61K45/06 , A61P25/28
CPC分类号： C12N15/113 , A61K45/06 , A61P25/28 , C12N2310/11 , C12N2310/3515 , C12N2320/32
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an ATXN2 gene, as well as methods of inhibiting expression of an ATXN2 gene and methods of treating subjects having an ATXN2-associated neurodegenerative disease or disorder, e.g., SCAs and ALS, using such dsRNAi agents and compositions.
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公开(公告)号：US20240209358A1
公开(公告)日：2024-06-27
申请号：US18144276
申请日：2023-05-08
IPC分类号： C12N15/113 , A61K9/00 , A61K45/06 , A61K47/54 , A61P7/02
CPC分类号： C12N15/113 , A61K9/0019 , A61K45/06 , A61K47/549 , A61P7/02 , C12N2310/11 , C12N2310/3125 , C12N2310/313
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.
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