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公开(公告)号：US20240318175A1
公开(公告)日：2024-09-26
申请号：US18572486
申请日：2022-06-28
发明人： ADAM CASTORENO , BHAUMIK A. PANDYA , ELENA CASTELLANOS-RIZALDOS , MARK K. SCHLEGEL , VASANT R. JADHAV
IPC分类号： C12N15/113 , A61K9/00 , A61K45/06 , A61P27/06
CPC分类号： C12N15/113 , A61K9/0048 , A61K45/06 , A61P27/06 , C12N2310/11 , C12N2310/3125 , C12N2310/313 , C12N2310/3513 , C12N2310/3515 , C12N2310/533 , C12N2320/32
摘要： The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting MYOC, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of MYOC.
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2.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：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：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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公开(公告)号：US20230159933A1
公开(公告)日：2023-05-25
申请号：US17995699
申请日：2021-04-06
发明人： WILLIAM CANTLEY , JAMES D. MCININCH , ADAM CASTORENO , CHARALAMBOS KAITTANIS , MARK K. SCHLEGEL
IPC分类号： C12N15/113 , A61K31/713 , A61K45/06
CPC分类号： C12N15/1138 , A61K31/713 , A61K45/06 , C12N2310/14 , C12N2310/3515 , C12N2310/321 , C12N2310/3525 , C12N2320/31 , C12N2310/315 , C12N2310/3125
摘要： The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting SCN9A, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of SCN9A.
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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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公开(公告)号：US20240301418A1
公开(公告)日：2024-09-12
申请号：US18572553
申请日：2022-06-23
IPC分类号： C12N15/113 , A61K31/713
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/11 , C12N2310/315 , C12N2310/343 , C12N2310/3515 , C12N2310/352
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Filamin A (FLNA) gene, as well as methods of inhibiting expression of an FLNA gene and methods of treating subjects having an FLNA-associated N disease or disorder, e.g., Alzheimer's disease, using such dsRNAi agents and compositions.
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公开(公告)号：US20240254493A1
公开(公告)日：2024-08-01
申请号：US18562787
申请日：2022-05-27
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.
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10.发明公开公开(公告)号：US20240254487A1
公开(公告)日：2024-08-01
申请号：US18532300
申请日：2023-12-07
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.
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