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公开(公告)号：US11590229B2
公开(公告)日：2023-02-28
申请号：US17651029
申请日：2022-02-14
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC: 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
Abstract: 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：US20220175929A1
公开(公告)日：2022-06-09
申请号：US17651029
申请日：2022-02-14
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC: 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
Abstract: 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：US11246933B1
公开(公告)日：2022-02-15
申请号：US17302311
申请日：2021-04-29
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC: A61K47/18 , A61K48/00 , 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
Abstract: 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：US10995336B2
公开(公告)日：2021-05-04
申请号：US16420263
申请日：2019-05-23
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： 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: C12N15/113
Abstract: 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.
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公开(公告)号：US11597932B2
公开(公告)日：2023-03-07
申请号：US16956271
申请日：2018-12-21
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： 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: C12N15/113
Abstract: 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.
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公开(公告)号：US20220175930A1
公开(公告)日：2022-06-09
申请号：US17651038
申请日：2022-02-14
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC: 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
Abstract: 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：US20220175928A1
公开(公告)日：2022-06-09
申请号：US17651023
申请日：2022-02-14
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC: 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
Abstract: 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：US20220125927A1
公开(公告)日：2022-04-28
申请号：US17573878
申请日：2022-01-12
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G Rajeev , Muthiah Manoharan
IPC: A61K47/18 , C07C323/58 , C07C327/28 , A61K31/713 , C07C323/12 , C07D233/54 , C07D295/12 , C07D295/14 , A61K9/51 , C07C31/125 , C07C211/09 , C07C235/06 , A61K31/7088 , C07C211/10 , C07C217/08 , C07D295/08 , C07D317/30 , A61K31/7105 , C07C327/22 , C07C327/32 , C07F5/02 , C07C211/11 , C07C251/38 , A61K9/127 , C07C229/12 , C07D207/32
Abstract: 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：US20210269796A1
公开(公告)日：2021-09-02
申请号：US17194431
申请日：2021-03-08
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： 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: C12N15/113
Abstract: 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.
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公开(公告)号：US20190048344A1
公开(公告)日：2019-02-14
申请号：US16104045
申请日：2018-08-16
Applicant: ALNYLAM PHARMACEUTICALS, INC.
Inventor： Muthiah Manoharan , Kallanthottathil G. Rajeev , Takeshi Yamada , David Butler , Jayaprakash K. Nair , Muthusamy Jayaraman , Shigeo Matsuda , Rajendra K. Pandey , Chang Geng Peng
IPC: C12N15/113 , H04L29/08 , A61K31/713 , C07H21/00 , H04L29/06 , A61K47/54 , A61K47/64
Abstract: The invention features compounds of formula I or II: In one embodiment, the invention relates compounds and processes for conjugating ligand to oligonucleotide. The invention further relates to methods for treating various disorders and diseases such as viral infections, bacterial infections, parasitic infections, cancers, allergies, autoimmune diseases, immunodeficiencies and immunosuppression.
