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公开(公告)号：US20240018515A1
公开(公告)日：2024-01-18
申请号：US17974598
申请日：2022-10-27
发明人： James D. McIninch , Adam Castoreno , Mark K. Schlegel , Elane Fishilevich , Kristina Yucius , Charalambos Kaittanis
IPC分类号： C12N15/113 , A61P43/00
CPC分类号： C12N15/113 , C12N2310/31 , C12N2310/11 , A61P43/00
摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the complement factor B (CFB) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a CFB gene and to methods of treating or preventing a CFB-associated disease in a subject.
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公开(公告)号：US11866710B2
公开(公告)日：2024-01-09
申请号：US18150827
申请日：2023-01-06
发明人： Aimee M. Deaton , John Michael Gansner , James D. McIninch , Mark K. Schlegel , Benjamin P. Garfinkel
IPC分类号： C07H21/04 , C12N15/113 , A61K47/54
CPC分类号： C12N15/1137 , A61K47/549 , C12Y304/21109 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/351
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), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, Parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.
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公开(公告)号：US11866709B2
公开(公告)日：2024-01-09
申请号：US17531913
申请日：2021-11-22
发明人： Brian Bettencourt , William Querbes , Kevin Fitzgerald , Maria Frank-Kamenetsky , Stuart Milstein , Svetlana Shulga Morskaya
IPC分类号： C12N15/113 , A61K31/713 , A61K31/7105 , C12Q1/68
CPC分类号： C12N15/1136 , A61K31/713 , A61K31/7105 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
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公开(公告)号：US20230392156A1
公开(公告)日：2023-12-07
申请号：US18129923
申请日：2023-04-03
IPC分类号： C12N15/113 , A61P3/04
CPC分类号： C12N15/1138 , A61P3/04 , C12N2310/315 , C12N2310/3515 , C12N2310/14
摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the G-protein coupled receptor 75 (GPR75) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a GPR75 gene and to methods of treating or preventing a GPR75-associated disease, such as a body weight disorder, e.g., obesity, in a subject.
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公开(公告)号：US20230310485A1
公开(公告)日：2023-10-05
申请号：US17898876
申请日：2022-08-30
发明人： Gregory Hinkle
IPC分类号： A61K31/712 , A61K31/7125 , C12N15/113 , A61P9/12 , A61K9/00 , A61K31/7115
CPC分类号： A61K31/712 , A61K9/0019 , A61K31/7115 , A61K31/7125 , A61P9/12 , C12N15/1136 , A61K2300/00 , C12N2310/11 , C12N2310/315 , C12N2310/3341 , C12N2310/351
摘要： The invention relates to polynucleotide agents, e.g., antisense polynucleotide agents, targeting an angiotensinogen (AGT) gene, and methods of using such polynucleotide agents to inhibit expression of AGT and to treat subjects having an AGT-associated disease, e.g., hypertension.
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公开(公告)号：US11725207B2
公开(公告)日：2023-08-15
申请号：US17194431
申请日：2021-03-08
发明人： Mark K. Schlegel , Maja Janas , Vasant R. Jadhav , Donald Foster , Muthiah Manoharan , Kallanthottathil G. Rajeev , Alexander V. Kel'in , Klaus Charisse , Jayaprakash K. Nair , Martin A. Maier , Shigeo Matsuda , Muthusamy Jayaraman , Alfica Sehgal , Christopher Brown , Kevin Fitzgerald , Stuart Milstein
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/351
摘要： The invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
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公开(公告)号：US11685918B2
公开(公告)日：2023-06-27
申请号：US17091693
申请日：2020-11-06
发明人： Gregory Hinkle
IPC分类号： C07H21/04 , C12N15/113 , A61K31/713 , A61K47/54 , A61K47/56 , A61P35/00
CPC分类号： C12N15/113 , A61K31/713 , A61K47/549 , A61K47/56 , A61P35/00 , C12N15/1135 , C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/335 , C12N2310/3515
摘要： The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such RNAi agents to inhibit expression of a PD-L1 gene and methods of treating subjects having a PD-L1-associated disorder.
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公开(公告)号：US20230193268A1
公开(公告)日：2023-06-22
申请号：US17974596
申请日：2022-10-27
IPC分类号： C12N15/113 , A61P25/28
CPC分类号： C12N15/113 , A61P25/28 , C12N2310/14 , C12N2310/315 , C12N2310/3515 , C12N2320/35
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an APOE gene, as well as methods of inhibiting expression of an APOE gene and methods of treating subjects having an APOE-associated neurodegenerative disease or disorder, e.g., Alzheimer's disease and Parkinson's disease, using such dsRNAi agents and compositions.
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公开(公告)号：US11679158B2
公开(公告)日：2023-06-20
申请号：US17644914
申请日：2021-12-17
发明人： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC分类号： A61K47/18 , A61K9/127 , A61K9/51 , A61K31/713 , C07D317/30 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C327/22 , C07C327/28 , C07C327/32 , C07C235/06 , C07C251/38 , C07F5/02 , C07D233/54 , C07D207/32 , C07D295/08 , C07D295/12 , C07D295/14 , C07C323/12 , C07C323/58 , C07C31/125 , A61K31/7088 , A61K31/7105
CPC分类号： A61K47/18 , A61K9/1272 , A61K9/5123 , A61K31/7088 , A61K31/713 , A61K31/7105 , C07C31/125 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C235/06 , C07C251/38 , C07C323/12 , C07C323/58 , C07C327/22 , C07C327/28 , C07C327/32 , C07D207/32 , C07D233/54 , C07D295/08 , C07D295/12 , C07D295/14 , C07D317/30 , C07F5/022
摘要： The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
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公开(公告)号：US20230183701A1
公开(公告)日：2023-06-15
申请号：US18145238
申请日：2022-12-22
发明人： Martin MAIER , Don Foster , Stuart Milstein , Satya Kuchimanchi , Vasant Jadhav , Kallanthottathil Rajeev , Muthiah Manoharan , Rubina Parmar
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/3231 , C12N2310/344 , C12N2310/14 , C12N2320/51 , C12N2310/533 , C12N2310/335 , C12N2310/313 , C12N2310/332 , C12N2320/52 , C12N2310/32 , C12N2310/346 , C12N2310/351
摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
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