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公开(公告)号：US11529428B2
公开(公告)日：2022-12-20
申请号：US16910237
申请日：2020-06-24
发明人： Marija Tadin-Strapps , Tayeba Khan , Walter Richard Strapps , Laura Sepp-Lorenzino , Vasant R. Jadhav , Duncan Brown
IPC分类号： C12N15/113 , A61K48/00 , A61K31/713 , A61K47/54
摘要： The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
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2.发明申请公开(公告)号：US20220195433A1
公开(公告)日：2022-06-23
申请号：US17510277
申请日：2021-10-25
发明人： Mark Cancilla , James John Cunningham , William Michael Flanagan , Henry J. Haringsma , Denise M. Kenski , Matthew G. Stanton , Steven M. Stirdivant , Aarron T. Willingham
IPC分类号： C12N15/113 , C12N15/11 , A61K31/713 , C07H21/02
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (MAO against target gene expression.
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3.发明授权公开(公告)号：US11193126B2
公开(公告)日：2021-12-07
申请号：US15947248
申请日：2018-04-06
发明人： Mark Cancilla , James John Cunningham , William Michael Flanagan , Henry J. Haringsma , Denise M. Kenski , Matthew G. Stanton , Steven M. Stirdivant , Aarron T. Willingham
IPC分类号： C07H21/02 , C07H21/04 , C12N15/113 , C12N15/11 , A61K31/713
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
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4.发明授权公开(公告)号：US11117917B2
公开(公告)日：2021-09-14
申请号：US16268262
申请日：2019-02-05
发明人： David Tellers , Steven L. Colletti , Vadim Dudkin , Jeffrey Aaronson , Aaron Momose , Thomas Joseph Tucker , Yu Yuan , Kathleen B. Calati , Lu Tian , Rubina G. Parmar , Anthony W. Shaw , Weimin Wang , Rachel Anne Storr , Marina Busuek , Robert A. Kowtoniuk
IPC分类号： A61K31/7056 , A61K31/713 , C12N15/113 , C07H15/26 , A61K47/54
摘要： Disclosed herein is a modular composition comprising 1) an oligonucleotide; 2) one or more tetraGalNAc ligands of Formula (I), which may be the same or different; optionally, 3) one or more linkers, which may be the same or different; 4) one or more peptides independently selected from Table 3, which may be the same or different; and optionally, 5) one or more targeting ligands, solubilizing agents, pharmacokinetics enhancing agents, lipids, and/or masking agents.
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公开(公告)号：US10793860B2
公开(公告)日：2020-10-06
申请号：US16532245
申请日：2019-08-05
发明人： Steven Bartz , Duncan Brown , Michael Robinson
IPC分类号： C12N15/113 , A61K31/713 , A61K47/34 , A61K47/28 , A61K47/24 , A61K9/51 , A61K47/18
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of HBV gene expression and/or activity, and/or modulate a HBV gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against HBV gene expression.
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公开(公告)号：US10584335B2
公开(公告)日：2020-03-10
申请号：US15809777
申请日：2017-11-10
发明人： Lee Lim , Guillaume Chorn , Aarron T. Willingham , Lihong Zhao
IPC分类号： C12N15/113 , C12N15/11
摘要： The instant disclosure features single-stranded RNA molecules comprising one or more internal, non-nucleotide spacers. A non-nucleotide spacer covalently links nucleotide portions of the molecule. The single-stranded RNA molecules function as guide or antisense strands that are capable of inhibiting gene expression via an RNA interference mechanism and, thus, represent single-stranded RNAi agents. The single-stranded RNAi molecules can be used in methods for a variety of therapeutic, diagnostic, target validation, genomic discovery, genetic engineering, and pharmacogenomic applications.
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7.发明申请公开(公告)号：US20180193279A1
公开(公告)日：2018-07-12
申请号：US15827162
申请日：2017-11-30
IPC分类号： A61K9/51 , C07F7/18 , C07C229/12 , A61K31/713
CPC分类号： A61K9/5123 , A61K9/51 , A61K9/5146 , A61K31/713 , A61K47/60 , C07C229/12 , C07C229/28 , C07C229/30 , C07C2601/02 , C07F7/1804 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/32 , C12N2320/51
摘要： The instant invention provides for novel cationic lipids of Formula A that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhances efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain coupled with inclusion of hydrolysable functionality in the lipid chains to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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公开(公告)号：US10000754B2
公开(公告)日：2018-06-19
申请号：US15637579
申请日：2017-06-29
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C07H21/02 , C12N15/111 , C12N15/1131 , C12N15/1137 , C12N15/1138 , C12N15/8218 , C12N2310/14 , C12N2310/141 , C12N2310/315 , C12N2310/317 , C12N2310/322 , C12N2310/3231 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2310/53 , C12N2320/32 , C12N2320/51 , C12Y304/21021
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
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公开(公告)号：US09732342B2
公开(公告)日：2017-08-15
申请号：US14747264
申请日：2015-06-23
发明人： Guillaume Chorn , Lee Lim , Lihong Zhao
IPC分类号： C12N15/11 , C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1138 , C12N2310/113 , C12N2310/141 , C12N2310/3231 , C12N2310/33 , C12N2310/332 , C12N2310/3517 , C12N2310/533 , C12N2320/51 , C12N2320/52
摘要： This invention relates generally to segmented oligonucleotides capable of modulating gene expression. Specifically, the instant invention relates to segmented microRNA (miRNA) oligonucleotides, including segmented miRNA precursors and segmented pre-microRNAs. The invention also relates to compositions comprising such segmented oligonucleotides, as well as to methods of making and using such oligonucleotides for diagnosis and treatment of diseases associated or causally linked to aberrant levels or activities of gene expression, including aberrant levels of coding and/or non-coding RNA.
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公开(公告)号：US20170135962A1
公开(公告)日：2017-05-18
申请号：US15198271
申请日：2016-06-30
IPC分类号： A61K9/51 , C07C229/12 , C07F7/18 , A61K31/713
CPC分类号： A61K9/5123 , A61K9/51 , A61K9/5146 , A61K31/713 , A61K47/60 , C07C229/12 , C07C229/28 , C07C229/30 , C07C2101/02 , C07C2601/02 , C07F7/1804 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/32 , C12N2320/51
摘要： The instant invention provides for novel cationic lipids of Formula A that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain coupled with inclusion of hydrolysable functionality in the lipid chains to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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