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公开(公告)号：US20210332365A1
公开(公告)日：2021-10-28
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.
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公开(公告)号：US20240336914A1
公开(公告)日：2024-10-10
申请号：US18259887
申请日：2021-12-30
发明人： Shigeo MATSUDA , Jayaprakash K. NAIR , Martin A. MAIER , Michelle H. JUNG , Anna BISBE , Ivan ZLATEV , Mark K. SCHLEGEL , Christopher BROWN , Vasant R. JADHAV
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/351
摘要： This invention relates to an oligonucleotide comprising one or more 2′-modified nucleosides, wherein the 2′-position of the nucleoside has a structure of formula (I). The invention also relates to a pharmaceutical composition comprising the oligonucleotide described herein and a method of reducing or inhibiting the expression of a target gene by administering to the subject a therapeutically effective amount of the oligonucleotide described herein. The invention also relates to a method of bioactivating an oligonucleotide comprising one or more 2′-modified nucleosides, wherein the 2′-position of the nucleoside is modified by a bio-cleavable linking group, wherein the bio-cleavable linking group comprises acetal, disulfide, carbamate, amide, sulfonamide, a biocleavable carbohydrate linker, or combinations thereof, said method comprising the step of: exposing the oligonucleotide to a physiological condition that causes the bio-cleavable linking group to be cleaved from the 2′-modified nucleoside, thereby regenerating the 2′-OH group of the nucleoside.
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公开(公告)号：US20230256001A1
公开(公告)日：2023-08-17
申请号：US17937256
申请日：2022-09-30
发明人： Mark K. SCHLEGEL , Maja JANAS , Vasant R. JADHAV , 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
CPC分类号： A61K31/713 , C12N15/113 , C12N2310/14 , C12N2310/31 , 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 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 relate 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.
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公开(公告)号：US20230128522A1
公开(公告)日：2023-04-27
申请号：US17936258
申请日：2022-09-28
IPC分类号： C12N15/113 , A61K47/54 , A61P31/20
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.
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公开(公告)号：US20230068339A1
公开(公告)日：2023-03-02
申请号：US17741550
申请日：2022-05-11
发明人： Kevin FITZGERALD , William QUERBES , James BUTLER , Stephanie WILLIAMS , Abigail LIEBOW , Gregory HINKLE , Martin A. MAIER , Stuart MILSTEIN , Satyanarayana KUCHIMANCHI , Muthiah MANOHARAN
IPC分类号： C12N15/113 , A61P3/04 , A61P43/00 , A61P13/12 , A61P1/18 , A61P3/10 , A61P9/12 , A61P15/08 , A61P9/10 , A61P1/16 , A61P3/06 , A61K31/713
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
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公开(公告)号：US20230016929A1
公开(公告)日：2023-01-19
申请号：US17774477
申请日：2020-11-06
发明人： Jayaprakash K. NAIR , Martin A. MAIER , Juan C. SALINAS , Shigeo MATSUDA , Alexander V. KEL'IN , Scott P. LENTINI , Guo HE , Michelle H. JUNG , Justin M. PIERSON , Muthiah MANOHARAN , Dale C. GUENTHER , Ivan ZLATEV , Christopher S. THEILE , Vasant R. JADHAV , Stuart MILSTEIN , Maja JANAS , Dhrubajyoti DATTA
IPC分类号： A61K31/713 , A61K48/00 , C12N15/113 , A61P25/28
摘要： One aspect of the present invention relates to a compound comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic monomers, containing one or more lipophilic moieties, conjugated to one or more positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic monomer-conjugated compound.
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公开(公告)号：US20240343746A1
公开(公告)日：2024-10-17
申请号：US18259884
申请日：2021-12-30
发明人： Alexander V. KEL’IN , Justin M. PIERSON , Jayaprakash k. NAIR , Martin A. MAIER , Anna BISBE , Cheng TANG
IPC分类号： C07F9/6553 , C07F9/6558 , C07F9/6561 , C07H21/02
CPC分类号： C07F9/655345 , C07F9/655363 , C07F9/655381 , C07F9/65586 , C07F9/6561 , C07H21/02
摘要： This invention relates to a compound comprising a structure of formula (I): cyclic disulfide moiety-phosphorus coupling group (I). The cyclic disulfide moiety has the structure of (C-I), (C-II), or (C-III). This invention also relates to an oligonucleotide comprising one or more compounds that comprise the structure of formula (I), wherein at least one phosphorus coupling group contains a nucleoside or oligonucleotide. The invention also relates to a pharmaceutical composition comprising the oligonucleotide described herein and a method of reducing or inhibiting the expression of a target gene by administering to the subject a therapeutically effective amount of the oligonucleotide described herein.
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公开(公告)号：US20230021879A1
公开(公告)日：2023-01-26
申请号：US17779706
申请日：2020-11-23
发明人： Jayaprakash K. NAIR , Juan C. SALINAS , John Frederick BRIONES , Mark K. SCHLEGEL , Shigeo MATSUDA , Alexander V. KEL'IN , Ligang ZHANG , Martin A. MAIER
摘要： Provided is a method of treating cancer cells localized in the lung by administering to such patients a therapeutically effective amount of a liposomal annamycin formulation (L-Ann).
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