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公开(公告)号：US20210332365A1
公开(公告)日：2021-10-28
申请号：US17268324
申请日：2019-08-12
IPC分类号： C12N15/113 , A61P31/20 , A61K47/54
摘要： The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.
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公开(公告)号：US20220125823A1
公开(公告)日：2022-04-28
申请号：US17052945
申请日：2019-05-07
发明人： Jayaprakash K. NAIR , Martin MAIER , Vasant JADHAV , Stuart MILSTEIN , Kirk BROWN , Rubina G. PARMAR , Kallanthottathil G. RAJEEV , Muthiah MANOHARAN , Alexander V. KEL'IN , Muthusamy JAYARAMAN , Klaus CHARISSE , Adam CASTORENO , Christopher S. THEILE , Kevin FITZGERALD
IPC分类号： A61K31/713 , C12N15/113 , A61K47/54 , A61K9/00
摘要： The invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs at one or more internal positions on at least one strand, optionally via a linker or carrier.
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公开(公告)号：US20220002727A1
公开(公告)日：2022-01-06
申请号：US17479617
申请日：2021-09-20
发明人： Martin MAIER , Don FOSTER , Stuart MILSTEIN , Satya KUCHIMANCHI , Vasant JADHAV , Kallanthottathil RAJEEV , Muthiah MANOHARAN , Rubina PARMAR
IPC分类号： C12N15/113 , C12N15/11
摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises. at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
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公开(公告)号：US20240009225A1
公开(公告)日：2024-01-11
申请号：US18311484
申请日：2023-05-03
发明人： Jayaprakash K. NAIR , Martin MAIER , Vasant JADHAV , Stuart MILSTEIN , Kirk BROWN , Rubina G. PARMAR , Kallanthottathil G. RAJEEV , Muthiah MANOHARAN , Alexander V. KEL'IN , Muthusamy JAYARAMAN , Klaus CHARISSE , Adam CASTORENO , Christopher THEILE , Kevin FITZGERALD
IPC分类号： A61K31/713 , A61K47/54 , A61K9/00 , C12N15/113
CPC分类号： A61K31/713 , A61K47/543 , A61K9/0019 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2310/3515
摘要： One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.
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公开(公告)号：US20220389424A1
公开(公告)日：2022-12-08
申请号：US17847550
申请日：2022-06-23
发明人： Martin MAIER , Don FOSTER , Stuart MILSTEIN , Satya KUCHIMANCHI , Vasant JADHAV , Kallanthottathil RAJEEV , Muthiah MANOHARAN , Rubina PARMAR
IPC分类号： C12N15/113 , C12N15/11
摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises. at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
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公开(公告)号：US20220056448A1
公开(公告)日：2022-02-24
申请号：US17523240
申请日：2021-11-10
发明人： Martin MAIER , Don FOSTER , Stuart MILSTEIN , Satya KUCHIMANCHI , Vasant JADHAV , Kallanthottathil RAJEEV , Muthiah MANOHARAN , Rubina PARMAR
IPC分类号： C12N15/113 , C12N15/11
摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
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公开(公告)号：US20240358738A1
公开(公告)日：2024-10-31
申请号：US18651230
申请日：2024-04-30
发明人： Jayaprakash K. NAIR , Martin MAIER , Vasant JADHAV , Stuart MILSTEIN , Kirk BROWN , Rubina G. PARMAR , Kallanthottathil G. RAJEEV , Muthiah MANOHARAN , Alexander V. KEL'IN , Muthusamy JAYARAMAN , Klaus CHARISSE , Adam CASTORENO , Christopher S. THEILE , Kevin FITZGERALD
IPC分类号： A61K31/713 , A61K9/00 , A61K47/54 , C12N15/113
CPC分类号： A61K31/713 , A61K9/0019 , A61K47/543 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2310/3515
摘要： One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.
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公开(公告)号：US20230256001A1
公开(公告)日：2023-08-17
申请号：US17937256
申请日：2022-09-30
发明人： Mark K. SCHLEGEL , Maja JANAS , Vasant R. JADHAV , Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Muthusamy JAYARAMAN , Alexander V. KEL'IN , Shigeo MATSUDA , Klaus CHARISSE , Jayaprakash K. NAIR , Martin A. MAIER , Alfica SEHGAL , Christopher BROWN , Christopher THEILE , Stuart MILSTEIN
IPC分类号： A61K31/713 , C12N15/113
CPC分类号： A61K31/713 , C12N15/113 , C12N2310/14 , C12N2310/31 , C12N2310/351
摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2′-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
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公开(公告)号：US20230128522A1
公开(公告)日：2023-04-27
申请号：US17936258
申请日：2022-09-28
IPC分类号： C12N15/113 , A61K47/54 , A61P31/20
摘要： The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.
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公开(公告)号：US20190241893A1
公开(公告)日：2019-08-08
申请号：US16384644
申请日：2019-04-15
发明人： Martin MAIER , Don FOSTER , Stuart MILSTEIN , Satya KUCHIMANCHI , Vasant JADHAV , Kallanthottathil RAJEEV , Muthiah MANOHARAN , Rubina PARMAR
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/14 , C12N2310/313 , C12N2310/32 , C12N2310/3231 , C12N2310/332 , C12N2310/335 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2310/533 , C12N2320/51 , C12N2320/52
摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
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