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公开(公告)号：US20230357776A1
公开(公告)日：2023-11-09
申请号：US18320948
申请日：2023-05-19
发明人： Thazha P. Prakash , Punit P. Seth , Eric E. Swayze , Tamar R. Grossman , Michael L. McCaleb , Andrew T. Watt , Susan M. Freier
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12N15/113 , C12Y304/21047 , C12N2310/11 , C12N2310/315 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/321
摘要： The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
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公开(公告)号：US20230338555A1
公开(公告)日：2023-10-26
申请号：US17998141
申请日：2021-05-18
发明人： Thazha P. Prakash , Mehran Nikan , Michael Tanowitz , Chrissa A. Dwyer , Frank Rigo , Punit P. Seth
摘要： Disclosed herein are compounds that comprise an oligonucleotide, a conjugate linker, and a conjugate moiety capable of interacting with a cell surface moiety, wherein the oligonucleotide and the conjugate moiety are connected via the conjugate linker. The conjugate moiety may comprise a cell-targeting moiety and a peptide extender. In general, peptide extenders have sufficient length and/or structure to reduce or prevent interaction between the oligonucleotide and the cell-targeting moiety. Such compounds are useful to treat, prevent or ameliorate a condition or disease in an individual with limited off-target effects.
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公开(公告)号：US11732265B2
公开(公告)日：2023-08-22
申请号：US17314537
申请日：2021-05-07
发明人： Thazha P. Prakash , Punit P. Seth , Eric E. Swayze , Tamar R. Grossman , Michael L. McCaleb , Andrew T. Watt , Susan M. Freier
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12N15/113 , C12Y304/21047 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351
摘要： The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
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公开(公告)号：US11459564B2
公开(公告)日：2022-10-04
申请号：US16956425
申请日：2018-12-21
发明人： Frank Rigo , Thazha P. Prakash , David Corey
IPC分类号： C12N15/113
摘要： Certain embodiments are directed to methods and compounds for increasing FXN. Such methods and compounds are useful for increasing expression of FXN in cells and animals.
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公开(公告)号：US20220081689A1
公开(公告)日：2022-03-17
申请号：US16951380
申请日：2020-11-18
发明人： Frank Rigo , Thazha P. Prakash , Punit P. Seth
IPC分类号： C12N15/113 , A61K9/00
摘要： Provided herein are methods, compounds, and compositions for modulation of dystrophin pre-mRNA in an animal. Such methods, compounds, and compositions are useful, for example, to treat, prevent, or ameliorate one or more symptoms of Duchenne Muscular Dystrophy disease.
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公开(公告)号：US20210395734A1
公开(公告)日：2021-12-23
申请号：US17114177
申请日：2020-12-07
IPC分类号： C12N15/113 , C07H21/04 , A61K47/54 , C12N15/11 , A61K31/7088 , A61K31/713
摘要： Provided herein are oligomeric compounds with conjugate groups targeting apolipoprotein C-III (ApoCIII). In certain embodiments, the ApoCIII targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting ApoCIII for use in decreasing ApoCIII to treat, prevent, or ameliorate diseases, disorders or conditions related to ApoCIII. Certain diseases, disorders or conditions related to ApoCIII include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
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公开(公告)号：US20210315918A1
公开(公告)日：2021-10-14
申请号：US16951449
申请日：2020-11-18
发明人： Thazha P. Prakash , Frank Rigo , Punit P. Seth
IPC分类号： A61K31/713 , A61K47/54 , A61K9/00 , C12N15/113
摘要： Provided herein are methods, compounds, and compositions for modulation of transcript processing.
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公开(公告)号：US10793862B2
公开(公告)日：2020-10-06
申请号：US16521344
申请日：2019-07-24
发明人： Thazha P. Prakash , Punit P. Seth , Eric E. Swayze , Sanjay Bhanot , Susan M. Freier , Huynh-Hoa Bui
IPC分类号： C12N15/113 , C07H21/02
摘要： The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).
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公开(公告)号：US20200123551A1
公开(公告)日：2020-04-23
申请号：US16521344
申请日：2019-07-24
发明人： Thazha P. Prakash , Punit P. Seth , Eric E. Swayze , Sanjay Bhanot , Susan M. Freier , Huynh-Hoa Bui
IPC分类号： C12N15/113
摘要： The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).
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公开(公告)号：US20170183661A1
公开(公告)日：2017-06-29
申请号：US15308027
申请日：2015-05-01
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C07H21/00 , C12N2310/315 , C12N2310/3341 , C12N2310/341 , C12N2310/351
摘要： Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.
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