-
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.
-
公开(公告)号：US20230022185A1
公开(公告)日：2023-01-26
申请号：US17584463
申请日：2022-01-26
IPC分类号： C12N15/113
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 diseases.
-
公开(公告)号：US11492623B2
公开(公告)日：2022-11-08
申请号：US17268324
申请日：2019-08-12
IPC分类号： C12N15/113 , A61P31/20 , A61K47/54
摘要： The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.
-
公开(公告)号：US20190316127A1
公开(公告)日：2019-10-17
申请号：US16420263
申请日：2019-05-23
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
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.
-
5.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.
-
6.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.
-
公开(公告)号：US20210269796A1
公开(公告)日：2021-09-02
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
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.
-
公开(公告)号：US20200291405A1
公开(公告)日：2020-09-17
申请号：US16801373
申请日：2020-02-26
发明人： Gregory Hinkle , Laura Sepp-Lorenzino , Vasant R. Jadhav , Martin A. Maier , Muthiah Manoharan , Stuart Milstein , Svetlana Shulga Morskaya , Kallanthottathil G. Rajeev , Huilei Xu
IPC分类号： C12N15/113 , A61K31/713 , A61K45/06
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the hepatitis D virus (HDV) genome, and methods of using such RNAi agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HDV infection and/or HDV-associated disorder.
-
公开(公告)号：US11725209B2
公开(公告)日：2023-08-15
申请号：US17062748
申请日：2020-10-05
发明人： James Butler , Martin A. Maier , Kevin Fitzgerald , Shannon Fishman , Donald Foster , Vasant R. Jadhav , Stuart Milstein
IPC分类号： C07H21/04 , C07H21/02 , C12N15/113 , A61K31/713 , A61K45/06
CPC分类号： C12N15/1137 , A61K31/713 , A61K45/06 , C12N15/1138 , C12Y304/21 , C12N2310/14 , C12N2310/322 , C12N2310/351 , C12N2310/353 , C12N2310/3533 , C12N2320/31
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
-
公开(公告)号：US11504391B1
公开(公告)日：2022-11-22
申请号：US16461523
申请日：2017-11-22
发明人： Mark K. Schlegel , Maja Janas , Vasant R. Jadhav , Donald Foster , Muthiah Manoharan , Kallanthottathil G. Rajeev , Muthusamy Jayaraman , Alexander V. Kel'in , Shigeo Matsuda , Klaus Charisse , Jayaprakash K. Nair , Martin A. Maier , Alfica Sehgal , Christopher Brown , Christopher Theile , Stuart Milstein
IPC分类号： A61K31/713 , C12N15/113
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 antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2′-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
-
-
-
-
-
-
-
-
-
搜索结果
22 条记录 (耗时 0.025 秒)
