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公开(公告)号：US11597927B2
公开(公告)日：2023-03-07
申请号：US16618001
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 , Luciano Henrique Apponi , Hanna Maria Wisniewska , Xiayun Cheng , Young Jin Cho
IPC分类号： C12N15/113 , A61K47/54 , A61K47/55 , A61K45/06
摘要： Among other things, the present disclosure provides designed PNPLA3 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 N 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：US10724035B2
公开(公告)日：2020-07-28
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： A61K31/712 , A61K31/7125 , 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：US20190127733A1
公开(公告)日：2019-05-02
申请号：US15766578
申请日：2016-10-07
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
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：US20240026358A1
公开(公告)日：2024-01-25
申请号：US18252029
申请日：2022-03-11
发明人： Prashant Monian , Chikdu Shakti Shivalila , Subramanian Marappan , Chandra Vargeese , Pachamuthu Kandasamy , Genliang Lu , Hui Yu , David Charles Donnell Butler , Luciano Henrique Apponi , Mamoru Shimizu , Stephany Michelle Standley , David John Boulay , Andrew Guzior Hoss , Jigar Desai , Jack David Godfrey , Hailin Yang , Naoki Iwamoto , Jayakanthan Kumarasamy , Anthony Lamattina , Tom Liantang Pu
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/322 , C12N2310/321 , C12N2310/315 , C12N2320/34
摘要： Among other things, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders or diseases that can benefit from adenosine modification.
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公开(公告)号：US20230392137A1
公开(公告)日：2023-12-07
申请号：US17953292
申请日：2022-09-26
发明人： Prashant Monian , Chikdu Shakti Shivalila , Subramanian Marappan , Chandra Vargeese , Pachamuthu Kandasamy , Genliang Lu , Hui Yu , David Charles Donnell Butler , Luciano Henrique Apponi , Mamoru Shimizu , Stephany Michelle Standley , David John Boulay , Andrew Guzior Hoss , Jigar Desai , Jack David Godfrey , Hailin Yang , Naoki Iwamoto , Jayakanthan Kumarasamy , Anthony Lamattina , Tom Liantang Pu
IPC分类号： C12N15/10 , C12N15/113
CPC分类号： C12N15/102 , C12N15/1135
摘要： Among other things, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders or diseases that can benefit from adenosine modification.
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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：US20220401467A1
公开(公告)日：2022-12-22
申请号：US17177111
申请日：2021-02-16
发明人： Jason Jingxin Zhang , Naoki Iwamoto , Christopher J. Francis , Chandra Vargeese , David Charles Donnell Butler , Sethumadhavan Divakaramenon , Genliang Lu , Subramanian Marappan
IPC分类号： A61K31/7125 , A61P21/00 , C12N15/117 , A61K31/7088
摘要： The present disclosure pertains to the recognition that immune responses mediated by CpG oligonucleotides can be affected by the stereochemistry of modified internucleotidic linkages such as phosphorothioates. In some embodiments, the present disclosure relates to chirally controlled CpG oligonucleotide compositions comprising CpG oligonucleotides comprising multiple modified internucleotidic linkages such as phosphorothioate linkages, wherein the oligonucleotides comprise one or more CpG region motifs having defined stereochemistry patterns of chiral internucleotidic linkages. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are capable of agonizing an immune response. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are antagonistic. Methods for making and using chirally controlled CpG oligonucleotide compositions are also described. In some embodiments, no immune modulation is desired, and the present disclosure provides methods of identifying chirally controlled oligonucleotide compositions which have decreased immune modulation.
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公开(公告)号：US20210228615A1
公开(公告)日：2021-07-29
申请号：US16755544
申请日：2018-10-12
IPC分类号： A61K31/7125 , C12N15/113
摘要： Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).
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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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公开(公告)号：US20200299692A1
公开(公告)日：2020-09-24
申请号：US16782021
申请日：2020-02-04
发明人： Maria David Frank-Kamenetsky , Hailin Yang , Aaron Jay Morris , Chandra Vargeese , Christopher J. Francis
IPC分类号： C12N15/113 , C12N15/115 , A61P25/28
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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