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公开(公告)号：US20220347302A1
公开(公告)日：2022-11-03
申请号：US17302310
申请日：2021-04-29
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
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：US20220331446A1
公开(公告)日：2022-10-20
申请号：US17848850
申请日：2022-06-24
发明人： Kallanthottathil G. RAJEEV , Tracy ZIMMERMANN , Muthiah MANOHARAN , Martin MAIER , Satyanarayana KUCHIMANCHI , Klaus CHARISSE
IPC分类号： A61K48/00 , C12N15/113 , C07H21/02 , C12N15/85
摘要： One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. 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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公开(公告)号：US20220307024A1
公开(公告)日：2022-09-29
申请号：US17619633
申请日：2020-06-16
发明人： Jayaprakash K. Nair , Martin Maier , Vasant Jadhav , Stuart Milstein , Kirk Brown , Rubina G. Parmar , Kallanthottathil G. Rajeev , Muthiah Manoharan , Alexander V. Kel'in , Muthusamy Jayaraman , Klaus Charisse , Adam Castoreno , Christopher S. Theile , Kevin Fitzgerald
IPC分类号： C12N15/113 , A61K9/00 , A61P35/00
摘要： One aspect of the present invention relates to a double stranded iRNA agent 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 moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier, which provides for targeting to, and uptake by, tissues and cells of the CNS, and in particular the striatum. Another aspect of the invention relates to a method of gene silencing in tissues and cells of the CNS, and in particular the striatum, that includes administering to a tissue/cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.
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公开(公告)号：US20220290152A1
公开(公告)日：2022-09-15
申请号：US17639881
申请日：2020-09-02
发明人： HO-CHOU TU , JAMES MCININCH
IPC分类号： C12N15/113 , A61K31/713
摘要： The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.
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公开(公告)号：US20220290145A1
公开(公告)日：2022-09-15
申请号：US17743663
申请日：2022-05-13
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.
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公开(公告)号：US11382979B2
公开(公告)日：2022-07-12
申请号：US17644907
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 , C07F5/02 , C07C327/32 , C07C211/11 , C07D233/54 , C07C229/12 , C07C327/28 , C07C31/125 , A61K31/713 , C07C323/58 , C07C251/38 , C07C211/10 , C07D295/08 , C07C327/22 , C07C323/12 , C07D207/32 , C07D317/30 , C07C211/09 , C07C217/08 , C07D295/14 , A61K31/7088 , C07C235/06 , A61K9/51 , C07D295/12 , 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.
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公开(公告)号：US20220211743A1
公开(公告)日：2022-07-07
申请号：US17611761
申请日：2020-05-15
发明人： Jayaprakash K. NAIR , Xiaojun QIN , Mikyung YU , Vasant JADHAV , Martin MAIER , Diane RAMSDEN
IPC分类号： A61K31/713 , C12N15/113 , A61K9/00
摘要： One aspect of the present invention relates to an oral formulation for reducing or inhibiting the expression of a target gene in a subject, comprising a) double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; 2′-OMe modifications to more than fifteen, more than twenty, more than twenty-five, or more than thirty nucleotides; and a carbohydrate-based ligand conjugated to at least one strand, optionally via a linker or carrier; and b) a penetration enhancer. Another aspect of the invention relates to a method of gene silencing, comprising orally administering to a subject in need thereof the oral formulation.
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公开(公告)号：US20220177885A1
公开(公告)日：2022-06-09
申请号：US17668917
申请日：2022-02-10
发明人： Jayaprakash K. NAIR , Kallanthottathil G. RAJEEV , Scott LENTINI , Christopher S. THEILE , Martin MAIER , Ivan ZLATEV
IPC分类号： C12N15/113
摘要： The present disclosure relates generally to cleavable linkers and uses thereof.
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公开(公告)号：US20220143189A1
公开(公告)日：2022-05-12
申请号：US17533559
申请日：2021-11-23
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 modified RNAi agent comprising a sense strand and an antisense strand, wherein the antisense strand targets an HBV mRNA; all of the nucleotide sugars of the RNAi agent are 2′-modified; and at least one of the sense and antisense strands is conjugated to at least one targeting ligand that comprises two or more building blocks connected to a biantennary or triantennary branched linker, optionally via one or more linking groups.
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公开(公告)号：US11326166B1
公开(公告)日：2022-05-10
申请号：US17505732
申请日：2021-10-20
IPC分类号： C12N15/113 , A61K47/54 , A61K31/713
摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the xanthine dehydrogenase (XDH) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an XDH gene and to methods of treating or preventing an XDH-associated disease in a subject.
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