-
公开(公告)号：US20240285767A1
公开(公告)日：2024-08-29
申请号：US18589636
申请日：2024-02-28
发明人： Martin MAIER , Muthusamy JAYARAMAN , Akin AKINC , Shigeo MATSUDA , Pachamuthu KANDASAMY , Kallanthottathil G. RAJEEV , Muthiah MANOHARAN , Jayaprakash K. NAIR , Thomas A. BAILLIE
IPC分类号： A61K47/18 , A61K9/127 , A61K9/51 , A61K31/7088 , A61K31/7105 , A61K31/713 , 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 , C07F5/02
CPC分类号： A61K47/18 , A61K9/1272 , A61K9/5123 , A61K31/7088 , A61K31/7105 , A61K31/713 , 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.
-
公开(公告)号：US20210207144A1
公开(公告)日：2021-07-08
申请号：US16956271
申请日：2018-12-21
发明人： Muthiah MANOHARAN , Nate TANEJA , Hartmut Ingo JAHNS , Shigeo MATSUDA , Klaus CHARISSE , Guo HE , Jayaprakash K. NAIR , Christopher BROWN , Mark K. SCHLEGEL , Vasant JADHAV , Martin MAIER
IPC分类号： C12N15/113
摘要： The present invention relates to a chirally-modified dsRNA agent capable of inhibiting the expression of a target gene. The sense and antisense strands of chirally-modified dsRNA agent independently or in combination comprises one or more site specific-site specific/position specific, chirally-modified internucleotide linkages.
-
公开(公告)号：US20200297853A1
公开(公告)日：2020-09-24
申请号：US16861494
申请日：2020-04-29
IPC分类号： A61K47/28 , A61K31/7004 , A61K31/7052 , A61K31/70 , A61K47/60 , A61K47/54 , C07H21/02 , A61K31/7088 , A61K31/713 , A61K47/16 , A61K47/22
摘要： The present invention provides a phosphorothioate-modified oligonucleotide comprising a structure shown below: The present invention also provides a phosphorothioate-modified oligonucleotide comprising a structure having formula (CIII):
-
公开(公告)号：US20190184018A1
公开(公告)日：2019-06-20
申请号：US16196628
申请日：2018-11-20
IPC分类号： A61K47/28 , A61K31/70 , A61K47/60 , A61K31/7004 , A61K31/7052 , A61K47/54 , C07H21/02 , A61K31/7088 , A61K47/22 , A61K47/16 , A61K31/713
摘要： The present invention provides a phosphorothioate-modified oligonucleotide comprising a structure shown below: The present invention also provides a phosphorothioate-modified oligonucleotide comprising a structure having formula (CIII):
-
公开(公告)号：US20180326070A1
公开(公告)日：2018-11-15
申请号：US15817473
申请日：2017-11-20
IPC分类号： A61K47/28 , C07H21/02 , A61K31/7052 , A61K47/54 , A61K47/22 , A61K47/16 , A61K31/713 , A61K31/7088 , A61K31/7004 , A61K31/70 , A61K47/60 , A61K48/00
CPC分类号： A61K47/28 , A61K31/70 , A61K31/7004 , A61K31/7052 , A61K31/7088 , A61K31/713 , A61K47/16 , A61K47/22 , A61K47/543 , A61K47/549 , A61K47/60 , A61K48/00 , C07H21/02 , Y02A50/385 , Y02A50/411 , Y02A50/467 , Y02P20/55
摘要： The present invention provides iRNA agents comprising at least one subunit of the formula (I): wherein: A and B are each independently for each occurrence O, N(RN) or S; X and Y are each independently for each occurrence H, OH, a hydroxyl protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z′)(Z″)O-nucleoside, —P(Z′)(Z″)O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, —P(Z′)(Z″)O-Linker-OP(Z′″)(Z″″)O-oligonucleotide, a nucleotide, an oligonucleotide, —P(Z′)(Z″)-formula (I), —P(Z′)(Z″)— or -Linker-R; R is LG, -Linker-LG, or has the structure shown below: LG is independently for each occurrence a carbohydrate, e.g., monosaccharide, disaccharide, trisaccharide, tetrasaccharide, oligosaccharide, polysaccharide; RN is independently for each occurrence H, methyl, ethyl, propyl, isopropyl, butyl, or benzyl; and Z′, Z″, Z′″ and Z″″ are each independently for each occurrence O or S.
-
公开(公告)号：US20180208927A1
公开(公告)日：2018-07-26
申请号：US15745591
申请日：2016-07-15
发明人： Vasant JADHAV , Martin MAIER , Ivan ZLATEV , Akin AKINC , Kallanthottathil G. RAJEEV , Jayaprakash K. NAIR , Pachamuthu KANDASAMY , Muthiah MANOHARAN
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , A61K47/549 , A61K47/55 , C07H21/00 , C12N15/111 , C12N2310/14 , C12N2310/20 , C12N2310/351 , C12N2310/3519 , C12N2310/51 , C12N2320/31
摘要： The present invention relates, in general to, compounds, compositions and methods useful for modulating gene expression of multiple target nucleic acids by a single chemical entity.
-
公开(公告)号：US20230256098A1
公开(公告)日：2023-08-17
申请号：US18304097
申请日：2023-04-20
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 , A61K31/713 , C07D317/30 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C327/22 , C07C327/28 , C07C327/32 , C07C235/06 , C07C251/38 , C07F5/022 , C07D233/54 , C07D207/32 , C07D295/08 , C07D295/12 , C07D295/14 , C07C323/12 , C07C323/58 , C07C31/125 , A61K31/7088 , A61K31/7105
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.
-
公开(公告)号：US20210238595A1
公开(公告)日：2021-08-05
申请号：US17055710
申请日：2019-05-16
发明人： Shigeo MATSUDA , Mark K. SCHLEGEL , Maja JANAS , Vasant R. JADHAV , Martin MAIER , Klaus CHARISSE , Muthiah MANOHARAN , Kallathotathil G. RAJEEV , Jayaprakash K. NAIR
IPC分类号： 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 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.
-
公开(公告)号：US20210017519A1
公开(公告)日：2021-01-21
申请号：US16693683
申请日：2019-11-25
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
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.
-
公开(公告)号：US20170275626A1
公开(公告)日：2017-09-28
申请号：US15504855
申请日：2015-08-14
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
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/14 , C12N2310/313 , C12N2310/32 , C12N2310/3231 , C12N2310/332 , C12N2310/335 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2310/533 , C12N2320/51 , C12N2320/52
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.
-
-
-
-
-
-
-
-
-
搜索结果
48 条记录 (耗时 0.021 秒)
