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公开(公告)号：US20240285669A1
公开(公告)日：2024-08-29
申请号：US18243156
申请日：2023-09-07
发明人： Berit Elissa Powers , Frank Rigo
IPC分类号： A61K31/712 , A61K31/7125 , A61P25/28 , C12N15/113
CPC分类号： A61K31/712 , A61K31/7125 , A61P25/28 , C12N15/113
摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include motor delays, cognitive delays, paroxysmal deterioration, seizures, vomiting, swallowing difficulties, ataxic gait, palatal myoclonus, autonomic dysfunction, and presence of intra-astrocytic inclusions called Rosenthal fibers. Such leukodystrophies include Alexander Disease.
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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：US11786546B2
公开(公告)日：2023-10-17
申请号：US16938826
申请日：2020-07-24
发明人： Berit Elissa Powers , Frank Rigo
IPC分类号： A61K31/712 , A61K31/7125 , A61P25/28 , C12N15/113
CPC分类号： A61K31/712 , A61K31/7125 , A61P25/28 , C12N15/113
摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include motor delays, cognitive delays, paroxysmal deterioration, seizures, vomiting, swallowing difficulties, ataxic gait, palatal myoclonus, autonomic dysfunction, and presence of intra-astrocytic inclusions called Rosenthal fibers. Such leukodystrophies include Alexander Disease.
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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：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：US20210277397A1
公开(公告)日：2021-09-09
申请号：US17236671
申请日：2021-04-21
IPC分类号： C12N15/113
摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject.
Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.-
公开(公告)号：US20200239882A1
公开(公告)日：2020-07-30
申请号：US16546283
申请日：2019-08-20
发明人： C. Frank Bennett , Susan M. Freier , Frank Rigo , Eric E. Swayze
IPC分类号： C12N15/113
摘要： Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C9ORF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).
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公开(公告)号：US10590412B2
公开(公告)日：2020-03-17
申请号：US14785411
申请日：2014-04-21
发明人： Amanda Ward , Frank Rigo
IPC分类号： C12N15/113
摘要： Disclosed herein are compounds, compositions and methods for modulating the amount or activity of a target nucleic acid. In certain embodiments, the amount or activity of a target nucleic acid is modulated through nonsense mediated decay.
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公开(公告)号：US20190382760A1
公开(公告)日：2019-12-19
申请号：US16398037
申请日：2019-04-29
发明人： Susan M. Freier , Frank Rigo
IPC分类号： C12N15/113 , C07H21/00
摘要： The present invention provides compounds comprising oligonucleotides complementary to a fibronectin transcript. Certain such compounds are useful for hybridizing to a fibronectin transcript, including but not limited to a fibronectin transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the fibronectin transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with fibrosis. In certain embodiments, such compounds are used to treat one or more symptoms associated with renal fibrosis.
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