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公开(公告)号：US20220162598A1
公开(公告)日：2022-05-26
申请号：US16869126
申请日：2020-05-07
发明人： Chandra Vargeese , Meena , Nenad Svrzikapa , Susovan Mohapatra , Christopher J. Francis , Gregory L. Verdine , Anna Sokolovska
IPC分类号： C12N15/11 , C12N15/113
摘要： Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.
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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：US20160347784A1
公开(公告)日：2016-12-01
申请号：US15167669
申请日：2016-05-27
发明人： Gregory L. Verdine , Meena , Naoki Iwamoto
IPC分类号： C07H21/00
CPC分类号： A61K31/70 , C07F9/65515 , C07H21/00
摘要： Described herein are nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties. Also described herein are methods of making and using nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties.
摘要翻译： 本文描述的是包含手性磷部分的核酸前药和核酸前药。 本文还描述了制备和使用包含手性磷部分的核酸前药和核酸前药的方法。
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公开(公告)号：US20210130821A1
公开(公告)日：2021-05-06
申请号：US16717986
申请日：2019-12-17
发明人： David Charles Donnell Butler , Naoki Iwamoto , Meena . , Nenad Svrzikapa , Gregory L. Verdine , Ivan Zlatev
IPC分类号： C12N15/113 , C07H21/00 , C07C317/28 , C07D295/088 , C12N15/11 , C12Q1/6876
摘要： The present invention relates to chirally controlled oligonucleotides, chirally controlled oligonucleotide compositions, and the method of making and using the same. The invention specifically encompasses the identification of the source of certain problems with prior methodologies for preparing chiral oligonucleotides, including problems that prohibit preparation of fully chirally controlled compositions, particularly compositions comprising a plurality of oligonucleotide types. In some embodiments, the present invention provides chirally controlled oligonucleotide compositions. In some embodiments, the present invention provides methods of making chirally controlled oligonucleotides and chirally controlled oligonucleotide compositions.
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公开(公告)号：US20200056173A1
公开(公告)日：2020-02-20
申请号：US16098836
申请日：2017-05-03
发明人： Chandra Vargeese , Meena . , Nenad Svrzikapa , Susovan Mohapatra , Christopher J. Francis , Gregory L. Verdine , Anna Sokolovska
IPC分类号： C12N15/11 , C12N15/113
摘要： Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.
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公开(公告)号：US20190390197A1
公开(公告)日：2019-12-26
申请号：US16087577
申请日：2017-10-06
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： C12N15/113 , A61P25/14 , A61P21/00 , A61K48/00 , A61K31/7125
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：US10450568B2
公开(公告)日：2019-10-22
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： A61K31/712 , A61K31/7125 , C12N15/113 , C07H21/02 , C07H21/00 , A61P25/14 , A61P21/00 , A61K31/7115 , A61K48/00 , A61P27/00 , C07F9/59
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：US10307434B2
公开(公告)日：2019-06-04
申请号：US15167669
申请日：2016-05-27
发明人： Gregory L. Verdine , Meena , Naoki Iwamoto
摘要： Described herein are nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties. Also described herein are methods of making and using nucleic acid prodrugs and nucleic acid prodrugs comprising chiral phosphorous moieties.
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公开(公告)号：US09885037B2
公开(公告)日：2018-02-06
申请号：US14414614
申请日：2013-07-12
发明人： David Butler , Naoki Iwamoto , Meena , Nenad Svrzikapa , Gregory L. Verdine , Ivan Zlatev
IPC分类号： C07H21/04 , C12N15/113 , C07H21/00 , C07C317/28 , C07D295/088 , C12Q1/68 , C12N15/11
摘要： The present invention relates to chirally controlled oligonucleotides, chirally controlled oligonucleotide compositions, and the method of making and using the same. The invention specifically encompasses the identification of the source of certain problems with prior methodologies for preparing chiral oligonucleotides, including problems that prohibit preparation of fully chirally controlled compositions, particularly compositions comprising a plurality of oligonucleotide types. In some embodiments, the present invention provides chirally controlled oligonucleotide compositions. In some embodiments, the present invention provides methods of making chirally controlled oligonucleotides and chirally controlled oligonucleotide compositions.
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公开(公告)号：US20240229026A1
公开(公告)日：2024-07-11
申请号：US18313032
申请日：2023-05-05
发明人： David Charles Donnell Butler , Naoki Iwamoto , Meena . , Nenad Svrzikapa , Gregory L. Verdine , Ivan Zlatev
IPC分类号： C12N15/113 , C07C317/28 , C07D295/088 , C07H21/00 , C12N15/11 , C12Q1/6876
CPC分类号： C12N15/113 , C07C317/28 , C07D295/088 , C07H21/00 , C12N15/11 , C12Q1/6876 , C12N15/111 , C12N2310/11 , C12N2310/14 , C12N2310/31 , C12N2310/315 , C12N2330/30
摘要： The present invention relates to chirally controlled oligonucleotides, chirally controlled oligonucleotide compositions, and the method of making and using the same. The invention specifically encompasses the identification of the source of certain problems with prior methodologies for preparing chiral oligonucleotides, including problems that prohibit preparation of fully chirally controlled compositions, particularly compositions comprising a plurality of oligonucleotide types. In some embodiments, the present invention provides chirally controlled oligonucleotide compositions. In some embodiments, the present invention provides methods of making chirally controlled oligonucleotides and chirally controlled oligonucleotide compositions.
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