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公开(公告)号:US20240352464A1
公开(公告)日:2024-10-24
申请号:US18599052
申请日:2024-03-07
发明人: Leonid Beigelman , Vivek Kumar Rajwanshi , Xuan Luong , Kellan Passow , Aneerban Bhattacharya , Saul Martinez Montero , Jerome Deval
IPC分类号: C12N15/113
CPC分类号: C12N15/1137 , C12Y101/01062 , C12N2310/14
摘要: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2′-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising additional modification including a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap. The siNA molecules may reduce or inhibit the production of hydroxysteroid dehydrogenase.
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公开(公告)号:US20230365970A1
公开(公告)日:2023-11-16
申请号:US18361363
申请日:2023-07-28
发明人: Leonid Beigelman , Vivek Kumar Rajwanshi , Markus Hossbach , Rajendra K. Pandey , Jin Hong , Laxman Eltepu , Saul Martinez Montero , N. Tilani S. De Costa
IPC分类号: C12N15/113 , A61K31/713 , A61K45/06 , A61P31/20
CPC分类号: C12N15/1131 , A61K31/713 , A61K45/06 , A61P31/20 , C12N2310/351 , C12N2310/315 , C12N2310/14 , C12N2320/31 , C12N2310/321 , C12N2310/3231 , C12N2310/322
摘要: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2′-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap; and (b) a short interfering nucleic acid (siNA).
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公开(公告)号:US11549110B2
公开(公告)日:2023-01-10
申请号:US17672268
申请日:2022-02-15
发明人: Leonid Beigelman , Vivek Kumar Rajwanshi , Markus Hossbach , Rajendra K. Pandey , Jin Hong , Laxman Eltepu , Saul Martinez Montero , N. Tilani S. De Costa
IPC分类号: C12N15/113 , A61P31/20 , A61K31/713 , A61K45/06
摘要: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2′-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap; and (b) a short interfering nucleic acid (siNA).
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公开(公告)号:US20210189392A1
公开(公告)日:2021-06-24
申请号:US17117494
申请日:2020-12-10
发明人: Leonid Beigelman , Rajendra Pandey , Vivek Kumar Rajwanshi , David Bernard Smith , Lawrence M. Blatt , Jin Hong , Megan Elizabeth Fitzgerald
IPC分类号: C12N15/113
摘要: Various embodiments provide STOPS™ polymers that are S-antigen transport inhibiting oligonucleotide polymers, processes for making them and methods of using them to treat diseases and conditions. In some embodiments the STOPS™ modified oligonucleotides include an at least partially phosphorothioated sequence of alternating A and C units having modifications as described herein. The sequence independent antiviral activity against hepatitis B of embodiments of STOPS™ modified oligonucleotides, as determined by HBsAg Secretion Assay, is an EC50 that is less than 100 nM.
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公开(公告)号:US12129469B2
公开(公告)日:2024-10-29
申请号:US18361363
申请日:2023-07-28
发明人: Leonid Beigelman , Vivek Kumar Rajwanshi , Markus Hossbach , Rajendra K. Pandey , Jin Hong , Laxman Eltepu , Saul Martinez Montero , N. Tilani S. De Costa
IPC分类号: C12N15/113 , A61K31/713 , A61K45/06 , A61P31/20
CPC分类号: C12N15/1131 , A61K31/713 , A61K45/06 , A61P31/20 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/351 , C12N2320/31
摘要: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2′-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap; and (b) a short interfering nucleic acid (siNA).
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公开(公告)号:US20230332153A1
公开(公告)日:2023-10-19
申请号:US18059561
申请日:2022-11-29
发明人: Leonid Beigelman , Vivek Kumar Rajwanshi , Markus Hossbach , Rajendra K. Pandey , Jin Hong , Laxman Eltepu , Saul Martinez Montero , N. Tilani S. De Costa
IPC分类号: C12N15/113 , A61K31/713 , A61K45/06 , A61P31/20
CPC分类号: C12N15/1131 , A61K31/713 , A61K45/06 , A61P31/20 , C12N2310/351 , C12N2310/315 , C12N2310/14 , C12N2320/31 , C12N2310/321 , C12N2310/3231 , C12N2310/322
摘要: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2′-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap; and (b) a short interfering nucleic acid (siNA).
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公开(公告)号:US20220364096A1
公开(公告)日:2022-11-17
申请号:US17194079
申请日:2021-03-05
发明人: Leonid Beigelman , Vivek Kumar Rajwanshi , Markus Hossbach , Rajendra K. Pandey , Jin Hong , Laxman Eltepu , Saul Martinez Montero , N. Tilani S. De Costa
IPC分类号: C12N15/113 , A61K45/06 , A61K31/713 , A61P31/20
摘要: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2′-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap; and (b) a short interfering nucleic acid (siNA).
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公开(公告)号:US11466274B2
公开(公告)日:2022-10-11
申请号:US16887063
申请日:2020-05-29
IPC分类号: C12N15/11 , C12N15/113 , A61P31/20 , A61K31/7125
摘要: The disclosure includes antisense oligonucleotides, including gapmer antisense oligonucleotides, along with methods of making and use, e.g., against HBV.
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公开(公告)号:US20230383296A1
公开(公告)日:2023-11-30
申请号:US18122433
申请日:2023-03-16
IPC分类号: C12N15/113 , A61P31/20
CPC分类号: C12N15/1131 , A61P31/20 , C12N15/1137 , C12N15/1136 , C12N2310/11 , C12N2310/315 , C12N2310/314 , C12N2310/3231 , C12N2310/341 , C12N2320/32 , C12N2320/51 , C12N2320/53 , C12N2310/351
摘要: The disclosure includes antisense oligonucleotides (ASOs), including gapmer ASOs, and methods of making and using the same.
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公开(公告)号:US11166976B2
公开(公告)日:2021-11-09
申请号:US17018822
申请日:2020-09-11
发明人: Leonid Beigelman , Rajendra Pandey , Vivek Kumar Rajwanshi , David Bernard Smith , Lawrence M. Blatt , Jin Hong
IPC分类号: A61K31/7125 , A61K9/00 , A61P31/20 , C12N15/113 , C07H21/02
摘要: Various embodiments provide STOPS™ polymers that are S-antigen transport inhibiting oligonucleotide polymers, processes for making them and methods of using them to treat diseases and conditions. In some embodiments the STOPS™ modified oligonucleotides include an at least partially phosphorothioated sequence of alternating A and C units having modifications as described herein. The sequence independent antiviral activity against hepatitis B of embodiments of STOPS™ modified oligonucleotides, as determined by HBsAg Secretion Assay, is greater than that of a reference compound.
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