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1.发明公开公开(公告)号：US20230203496A1
公开(公告)日：2023-06-29
申请号：US18001065
申请日：2021-06-09
发明人： JAMES D. MCININCH , LUCAS BONDURANT , AIMEE DEATON
IPC分类号： C12N15/113 , C12N15/63 , A61P1/16
CPC分类号： C12N15/1137 , C12N15/63 , A61P1/16 , C12N2310/11 , C12N2310/32 , C12N2310/315 , C12N2310/3125
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the GPAM gene, as well as methods of inhibiting expression of GPAM, and methods of treating subjects that would benefit from reduction in expression of GPAM, such as subjects having a GPAM-associated disease, disorder, or condition, using such dsRNA compositions.
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2.发明申请公开(公告)号：US20230126881A1
公开(公告)日：2023-04-27
申请号：US17907366
申请日：2021-03-26
IPC分类号： C12N15/113
摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting a Fc fragment of IgG receptor and transporter (FCGRT) gene encoding neonatal Fc receptor (FcRn). The invention also relates to methods of using such RNAi agents to inhibit expression of the FCGRT gene or production of the FcRn protein and to methods of preventing and treating a hepatotoxicity-associated disorder, e.g., alcoholic hepatitis, iron overload, and hepatocellular carcinoma.
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公开(公告)号：US20240200077A1
公开(公告)日：2024-06-20
申请号：US18550669
申请日：2022-03-17
IPC分类号： C12N15/113 , A61M5/142
CPC分类号： C12N15/1137 , A61M5/14212 , C12N2310/14 , C12N2310/3515
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Stearoyl-CoA Desaturase 5 (SCD5) gene, as well as methods of inhibiting expression of an SCD5 gene and methods of treating subjects having an SCD5-associated disease or disorder, e.g., Parkinson's disease, using such dsRNAi agents and compositions.
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公开(公告)号：US20230126233A1
公开(公告)日：2023-04-27
申请号：US17794637
申请日：2021-01-22
发明人： JAMES D. MCININCH , JASON A. GILBERT , ADAM CASTORENO , LAN THI HOANG DANG , SARAH LEBLANC , HAIYAN PENG , CHARALAMBOS KAITTANIS , MANGALA MEENAKSHI SOUNDARAPANDIAN
IPC分类号： C12N15/113 , A61K31/713
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.
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5.发明公开公开(公告)号：US20240301426A1
公开(公告)日：2024-09-12
申请号：US18575157
申请日：2022-06-29
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.
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公开(公告)号：US20230340485A1
公开(公告)日：2023-10-26
申请号：US18001813
申请日：2021-06-15
IPC分类号： C12N15/113 , A61P3/00
CPC分类号： C12N15/1137 , A61P3/00 , C12N2310/315 , C12N2310/32 , C12N2310/351
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an anaplastic lymphoma kinase (ALK) gene, as well as methods of inhibiting expression of an ALK gene and methods of treating subjects having an ALK-associated disease or disorder, e.g., type 2 diabetes, obesity, or an obesity-associated disorder, using such dsRNAi agents and compositions.
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公开(公告)号：US20230190785A1
公开(公告)日：2023-06-22
申请号：US17907664
申请日：2021-03-30
IPC分类号： A61K31/713 , A61K31/7115 , A61K31/712 , A61K31/7125
CPC分类号： A61K31/713 , A61K31/712 , A61K31/7115 , A61K31/7125
摘要： The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the DNAJC15 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of DNAJC15.
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公开(公告)号：US20230138507A1
公开(公告)日：2023-05-04
申请号：US17907741
申请日：2021-04-01
IPC分类号： C12N15/113 , A61K47/54
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an alpha-2A adrenergic receptor (ADRA2A) gene, as well as methods of inhibiting expression of an ADRA2A gene and methods of treating subjects having an ADRA2A-associated disease or disorder, e.g., a primary tauopathy or Alzheimer's disease, using such dsRNAi agents and compositions.
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公开(公告)号：US20240279653A1
公开(公告)日：2024-08-22
申请号：US18547102
申请日：2022-02-22
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/351
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the FLCN gene, as well as methods of inhibiting expression of FLCN, and methods of treating subjects that would benefit from reduction in expression of FLCN, such as subjects having a FLCN-associated disease, disorder, or condition, using such dsRNA compositions.
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公开(公告)号：US20230332147A1
公开(公告)日：2023-10-19
申请号：US18044401
申请日：2021-09-09
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/351 , C12N2310/315 , C12N2310/3515
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the GRB 10 or GRB 14 gene, as well as methods of inhibiting expression of GRB 10 or GRB 14, and methods of treating subjects that would benefit from reduction in expression of GRB 10 or GRB 14, such as subjects having a GRB 10- or GRB 14-associated disease, disorder, or condition, such as diabetes, using such dsRNA compositions.
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