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公开(公告)号：US20240271136A1
公开(公告)日：2024-08-15
申请号：US18529152
申请日：2023-12-05
发明人： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin A. Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC分类号： C12N15/113 , A61K31/713 , A61K39/395 , C07K16/18
CPC分类号： C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
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公开(公告)号：US12018257B2
公开(公告)日：2024-06-25
申请号：US18296912
申请日：2023-04-06
发明人： Janne Juha Turunen , Petra Geziena De Bruijn , Bart Klein , Roxana Simona Redis , Lenka Van Sint Fiet
IPC分类号： C12N15/11 , A61K31/7125 , C12N15/10 , C12Q1/68
CPC分类号： C12N15/111 , A61K31/7125 , C12N15/102 , C12N2310/11 , C12N2310/321 , C12N2310/3521 , C12N2310/3527 , C12N2320/30
摘要： The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hairpin or stem-loop structure, and wherein said oligonucleotide comprises a cytidine (a non-complementary nucleotide) or a uridine in a position opposite to the target adenosine to be edited in the target RNA region.
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公开(公告)号：US09970011B2
公开(公告)日：2018-05-15
申请号：US15606554
申请日：2017-05-26
IPC分类号： C07H21/02 , C07H21/04 , A61K31/70 , C12N15/113 , A61K45/06 , A61K31/7088
CPC分类号： C12N15/113 , A61K31/7088 , A61K45/06 , C12N2310/113 , C12N2310/313 , C12N2310/315 , C12N2310/321 , C12N2310/3233 , C12N2310/3527
摘要： Provided herein are methods for the treatment of Alport Syndrome, using modified oligonucleotides targeted to miR-21. In certain embodiments, a modified oligonucleotide targeted to miR-21 improves kidney function and/or reduces fibrosis in subjects having Alport Syndrome. In certain embodiments, administration of a modified oligonucleotide targeted to miR-21 delays the onset of end-stage renal disease in a subject having Alport Syndrome. In certain embodiments, a modified oligonucleotide targeted to miR-21 delays the need for dialysis or kidney transplant in a subject having Alport Syndrome.
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公开(公告)号：US09850488B2
公开(公告)日：2017-12-26
申请号：US14976261
申请日：2015-12-21
发明人： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC分类号： A61K48/00 , C12N15/11 , C07H21/02 , C07H21/04 , C12N15/113 , A61K31/713 , A61K39/395 , C07K16/18
CPC分类号： C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
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公开(公告)号：US09765338B2
公开(公告)日：2017-09-19
申请号：US14814174
申请日：2015-07-30
发明人： C. Frank Bennett , Susan M. Freier , Robert A. MacLeod , Sanjay K. Pandey , Charles A. Thornton , Thurman Wheeler , Seng H. Cheng , Andrew Leger , Bruce M. Wentworth
IPC分类号： C12N15/11 , C12N15/113
CPC分类号： C12N15/1137 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525 , C12Y207/11 , C12N2310/322 , C12N2310/3533 , C12N2310/3527
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
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公开(公告)号：US09701963B2
公开(公告)日：2017-07-11
申请号：US15151568
申请日：2016-05-11
发明人： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC分类号： A61K48/00 , C07H21/02 , C07H21/04 , C12N15/11 , C12N15/113 , A61K31/713 , A61K39/395 , C07K16/18
CPC分类号： C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
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公开(公告)号：US09382537B2
公开(公告)日：2016-07-05
申请号：US14459182
申请日：2014-08-13
申请人： Asuragen, Inc.
发明人： David Brown , Lance Ford , Angie Cheng , Rich Jarvis , Mike Byrom , Dmitriy Ovcharenko , Eric Devroe , Kevin Kelnar
IPC分类号： C12N15/11 , C12N15/113 , A61K31/7105 , A61K45/06 , A61N5/10 , C12Q1/68 , A61K9/127 , A61K31/7088 , A61K31/713
CPC分类号： C12N15/113 , A61K9/127 , A61K31/7088 , A61K31/7105 , A61K31/713 , A61K45/06 , A61N5/10 , C12N15/111 , C12N15/1136 , C12N2310/14 , C12N2310/141 , C12N2310/312 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/344 , C12N2310/35 , C12N2310/351 , C12N2310/3527 , C12N2310/3535 , C12N2310/533 , C12N2320/12 , C12N2320/30 , C12N2320/50 , C12N2330/10 , C12Q1/68 , C12Q1/6876 , C12Q2600/158 , C12Q2600/178 , C12N2310/3521
摘要： The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.
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公开(公告)号：US20160108401A1
公开(公告)日：2016-04-21
申请号：US14976261
申请日：2015-12-21
发明人： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
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公开(公告)号：US20150361431A1
公开(公告)日：2015-12-17
申请号：US14841377
申请日：2015-08-31
申请人： Asuragen, Inc.
发明人： David BROWN , Lance FORD , Angie CHENG , Rich JARVIS , Mike BYROM , Dmitriy OVCHARENKO , Eric DEVROE , Kevin KELNAR
IPC分类号： C12N15/113 , A61K31/713 , A61K45/06
CPC分类号： C12N15/113 , A61K9/127 , A61K31/7088 , A61K31/7105 , A61K31/713 , A61K45/06 , A61N5/10 , C12N15/111 , C12N15/1136 , C12N2310/14 , C12N2310/141 , C12N2310/312 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/344 , C12N2310/35 , C12N2310/351 , C12N2310/3527 , C12N2310/3535 , C12N2310/533 , C12N2320/12 , C12N2320/30 , C12N2320/50 , C12N2330/10 , C12Q1/68 , C12Q1/6876 , C12Q2600/158 , C12Q2600/178 , C12N2310/3521
摘要： The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.
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公开(公告)号：US20150344883A1
公开(公告)日：2015-12-03
申请号：US14738734
申请日：2015-06-12
申请人： Asuragen, Inc.
发明人： David BROWN , Lance FORD , Angie CHENG , Rich JARVIS , Mike BYROM , Dmitriy OVCHARENKO , Eric DEVROE , Kevin KELNAR
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K9/127 , A61K31/7088 , A61K31/7105 , A61K31/713 , A61K45/06 , A61N5/10 , C12N15/111 , C12N15/1136 , C12N2310/14 , C12N2310/141 , C12N2310/312 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/344 , C12N2310/35 , C12N2310/351 , C12N2310/3527 , C12N2310/3535 , C12N2310/533 , C12N2320/12 , C12N2320/30 , C12N2320/50 , C12N2330/10 , C12Q1/68 , C12Q1/6876 , C12Q2600/158 , C12Q2600/178 , C12N2310/3521
摘要： The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.
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