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公开(公告)号：US20190249173A1
公开(公告)日：2019-08-15
申请号：US16098658
申请日：2017-05-03
申请人： Chandra VARGEESE , Jason Jingxin ZHANG , Sethumadhavan DIVAKARAMENON , David Charles Donnell BUTLER , Genliang LU , Naoki IWAMOTO , Hailin YANG , Maria David FRANK-KAMENETSKY , Subramanian MARAPPAN , WAVE LIFE SCIENCES LTD.
发明人： Chandra Vargeese , Jason Jingxin Zhang , Sethumadhavan Divakaramenon , David Charles Donnell Butler , Genliang Lu , Naoki Iwamoto , Hailin Yang , Maria David Frank-Kamenetsky , Subramanian Marappan
IPC分类号： C12N15/11 , A61K31/7088 , A61K47/54
CPC分类号： C12N15/111 , A61K31/7088 , A61K47/542 , C12N2310/11 , C12N2310/14 , C12N2310/141 , C12N2310/16 , C12N2310/20 , C12N2310/315 , C12N2310/3515 , C12N2320/32
摘要： In some embodiments, the present disclosure pertains to compositions and methods related to delivery of a biologically active agent, wherein the compositions comprise a biologically active agent and a lipid. In various embodiments, the lipid is selected from: lauric acid, myristic acid, palmitic acid, stearic acid, oleic acid, linoleic acid, alpha-linolenic acid, gamma-linolenic acid, docosa-hexaenoic acid (cis-DHA), turbinaric acid and dilinoleyl. In some embodiments, a composition and method are useful for delivery of a biologically active agent to a particular cell or tissue, e.g., a muscle cell or tissue.
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公开(公告)号：US20200157545A1
公开(公告)日：2020-05-21
申请号：US16618010
申请日：2018-06-01
申请人： Chandra VARGEESE , Naoki IWAMOTO , David Charles Donnell BUTLER , Subramanian MARAPPAN , Genliang LU , Jason Jingxin ZHANG , Vinod VATHIPADIEKAL , Maria David FRANK-KAMENETSKY , Luciano Henrique APPONI , WAVE LIFE SCIENCES LTD.
发明人： Chandra Vargeese , Naoki Iwamoto , David Charles Donnell Butler , Subramanian Marappan , Genliang Lu , Jason Jingxin Zhang , Vinod Vathipadiekal , Maria David Frank-Kamenetsky , Luciano Henrique Apponi , Young Jin Cho
IPC分类号： C12N15/113 , A61K31/712
摘要： Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
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公开(公告)号：US20180216107A1
公开(公告)日：2018-08-02
申请号：US15746199
申请日：2016-07-22
申请人： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Morris , Chandra Vargeese , Christopher J. Francis
发明人： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Morris , Chandra Vargeese , Christopher J. Francis
IPC分类号： C12N15/113 , C12N15/115
摘要： Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved up-take. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
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公开(公告)号：US20190008986A1
公开(公告)日：2019-01-10
申请号：US16139091
申请日：2018-09-23
发明人： David Charles Donnell Butler , Sethumadhavan Divakaramenon , Christopher J. Francis , Maria David Frank-Kamenetsky , Naoki Iwamoto , Genliang Lu , Subramanian Marappan , Meena . , Chandra Vargeese , Gregory L. Verdine , Hailin Yang , Jason Jingxin Zhang
IPC分类号： A61K48/00 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61P21/00 , A61P25/14
CPC分类号： C12N15/113 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K48/0016 , A61K48/0066 , A61K48/0083 , A61P21/00 , A61P25/14 , A61P27/00 , C07F9/59 , C07H21/00 , C07H21/02
摘要： Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
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公开(公告)号：US20240132894A1
公开(公告)日：2024-04-25
申请号：US17956741
申请日：2022-09-29
发明人： Chandra Vargeese , Naoki Iwamoto , David Charles Donnell Butler , Subramanian Marappan , Genliang Lu , Jason Jingxin Zhang , Vinod Vathipadiekal , Maria David Frank-Kamenetsky , Luciano Henrique Apponi , Young Jin Cho
IPC分类号： C12N15/113 , A61K31/712
CPC分类号： C12N15/1137 , A61K31/712 , C12N2310/14 , C12N2310/315 , C12N2310/343
摘要： Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
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公开(公告)号：US20220098585A1
公开(公告)日：2022-03-31
申请号：US17426511
申请日：2020-01-30
发明人： Jeffrey Matthew Brown , Shaunna Syu-Mei Berkovitch , Naoki Iwamoto , Chandra Vargeese , Kidist M. Aklilu , Maria David Frank-Kamenetsky , Duncan Parley Brown
IPC分类号： C12N15/113
摘要： Among other things, the present disclosure provides oligonucleotides, compositions, and methods for preventing and/or treating various conditions, disorders or diseases. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides oligonucleotides, compositions and methods for HTT-related conditions, disorders or diseases, such as Huntington's disease.
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公开(公告)号：US20240150756A1
公开(公告)日：2024-05-09
申请号：US18305195
申请日：2023-04-21
发明人： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Jay Morris , Chandra Vargeese , Christopher J. Francis
IPC分类号： C12N15/113 , A61P25/28 , C12N15/115
CPC分类号： C12N15/113 , A61P25/28 , C12N15/115 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/346
摘要： Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
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公开(公告)号：US20230295617A1
公开(公告)日：2023-09-21
申请号：US18072296
申请日：2022-11-30
发明人： Chandra Vargeese , Jason Jingxin Zhang , Sethumadhavan Divakaramenon , David Charles Donnell Butler , Genliang Lu , Naoki Iwamoto , Hailin Yang , Maria David Frank-Kamenetsky , Subramanian Marappan
IPC分类号： C12N15/11 , A61K47/54 , A61K31/7088
CPC分类号： C12N15/111 , A61K47/542 , A61K31/7088 , C12N2310/141 , C12N2310/14 , C12N2310/20 , C12N2320/32 , C12N2310/11 , C12N2310/16 , C12N2310/315 , C12N2310/3515
摘要： In some embodiments, the present disclosure pertains to compositions and methods related to delivery of a biologically active agent, wherein the compositions comprise a biologically active agent and a lipid. In various embodiments, the lipid is selected from: lauric acid, myristic acid, palmitic acid, stearic acid, oleic acid, linoleic acid, alpha-linolenic acid, gamma-linolenic acid, docosahexaenoic acid (cis-DHA), turbinaric acid and dilinoleyl. In some embodiments, a composition and method are useful for delivery of a biologically active agent to a particular cell or tissue, e.g., a muscle cell or tissue.
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公开(公告)号：US11603532B2
公开(公告)日：2023-03-14
申请号：US16618010
申请日：2018-06-01
发明人： Chandra Vargeese , Naoki Iwamoto , David Charles Donnell Butler , Subramanian Marappan , Genliang Lu , Jason Jingxin Zhang , Vinod Vathipadiekal , Maria David Frank-Kamenetsky , Luciano Henrique Apponi , Young Jin Cho
IPC分类号： C12N15/11 , C12N15/113 , A61K31/712
摘要： Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
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公开(公告)号：US20210198305A1
公开(公告)日：2021-07-01
申请号：US16618003
申请日：2018-06-01
发明人： Chandra Vargeese , Naoki Iwamoto , David Charles Donnell Butler , Subramanian Marappan , Genliang Lu , Jason Jingxin Zhang , Vinod Vathipadiekal , Maria David Frank-Kamenetsky , Luciano Henrique Apponi , Hanna Maria Wisniewska , Xiayun Cheng , Young Jin Cho
IPC分类号： C07H17/04 , C07H19/04 , C12N15/113
摘要： Among other things, the present disclosure provides designed APOC3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
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