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公开(公告)号：US20210363205A1
公开(公告)日：2021-11-25
申请号：US17246549
申请日：2021-04-30
发明人： Joan David Bettoun
IPC分类号： C07K14/47
摘要： The present disclosure provides methods for treating or ameliorating a myelin associated disease or a mitochondria associated disease that comprising administering to a subject in need thereof a frataxin replacement therapeutic compound, e.g., a fusion protein comprising frataxin.
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公开(公告)号：US12091437B2
公开(公告)日：2024-09-17
申请号：US18390884
申请日：2023-12-20
IPC分类号： C07K14/47 , A61K38/00 , A61K47/64 , C07K14/005 , C12N9/10
CPC分类号： C07K14/47 , A61K47/64 , C07K14/005 , C12N9/104 , C12Y203/02 , A61K38/00 , C07K2319/02 , C07K2319/07 , C07K2319/50 , C12N2740/16322
摘要： The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia. The fusion protein may comprise a protein of interest to be delivered to a non-nuclear organelle; an organelle targeting sequence (OTS); a cell penetrating peptide (CPP); and a target enhancing sequence (TES); wherein the CPP is capable of interference with delivery of the protein of interest to the non-nuclear organelle; and wherein the TES prevents said interference by the CPP. The fusion protein may also comprise a protein of interest to be delivered to a non-nuclear organelle; a CPP and a TES. The present disclosure also provides methods for treating a non-nuclear organelle associated disorder by administering the fusion protein to a subject in need thereof.
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公开(公告)号：US20240254176A1
公开(公告)日：2024-08-01
申请号：US18432505
申请日：2024-02-05
IPC分类号： C07K14/47 , A61K38/00 , A61K47/64 , C07K14/005 , C12N9/10
CPC分类号： C07K14/47 , A61K47/64 , C07K14/005 , C12N9/104 , C12Y203/02 , A61K38/00 , C07K2319/02 , C07K2319/07 , C07K2319/50 , C12N2740/16322
摘要： The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia. The fusion protein may comprise a protein of interest to be delivered to a non-nuclear organelle; an organelle targeting sequence (OTS); a cell penetrating peptide (CPP); and a target enhancing sequence (TES); wherein the CPP is capable of interference with delivery of the protein of interest to the non-nuclear organelle; and wherein the TES prevents said interference by the CPP. The fusion protein may also comprise a protein of interest to be delivered to a non-nuclear organelle; a CPP and a TES. The present disclosure also provides methods for treating a non-nuclear organelle associated disorder by administering the fusion protein to a subject in need thereof.
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公开(公告)号：US11988675B2
公开(公告)日：2024-05-21
申请号：US17105149
申请日：2020-11-25
发明人： Joan David Bettoun
CPC分类号： G01N33/6893 , G01N33/5306 , G01N2333/902
摘要： The present disclosure provides methods, compositions and kits for measuring activity of a frataxin (FXN) protein, e.g., an FXN fusion protein. The present invention also provides methods for identifying compounds capable of modulating activity of an FXN protein, e.g., an FXN fusion protein.
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公开(公告)号：US20230357851A1
公开(公告)日：2023-11-09
申请号：US18296966
申请日：2023-04-06
IPC分类号： C12Q1/6883
CPC分类号： C12Q1/6883 , C12Q2600/106 , C12Q2600/158
摘要： The present disclosure is based, at least in part, on providing a set of markers, also referred to herein as FXN-sensitive genomic markers (or FSGMs), the respective expression levels of which are positively or negatively correlated to frataxin (FXN) levels in a cell. Therefore, these FSGMs can be used to determine, evaluate, and/or monitor the effectiveness of FXN replacement therapy in a subject.
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公开(公告)号：US20220378869A1
公开(公告)日：2022-12-01
申请号：US17627638
申请日：2020-07-17
发明人： Joan David Bettoun
摘要： The present disclosure provides a method for treatment of a subject suffering from Leigh Syndrome French Canadian Type (LSFC), the method comprising administering to the subject a therapeutically effective amount of a frataxin (FXN) therapeutic compound.
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公开(公告)号：US20220193190A1
公开(公告)日：2022-06-23
申请号：US17549770
申请日：2021-12-13
发明人： Denis Boyle , Thomas Ransohoff
摘要： Pharmaceutical compositions comprising a TAT-FXN fusion polypeptide are disclosed, as are methods of use of the pharmaceutical compositions to treat subjects diagnosed with Friedrich's Ataxia and/or hypertrophic cardiomyopathy.
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公开(公告)号：US20240343766A1
公开(公告)日：2024-10-17
申请号：US18433336
申请日：2024-02-05
发明人： Joan David BETTOUN
CPC分类号： C07K14/4702 , A61K38/00 , C07K2319/095 , C07K2319/10 , C07K2319/50
摘要： The present disclosure provides methods for treating or ameliorating a myelin associated disease or a mitochondria associated disease that comprising administering to a subject in need thereof a frataxin replacement therapeutic compound, e.g., a fusion protein comprising frataxin.
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公开(公告)号：US20240228557A1
公开(公告)日：2024-07-11
申请号：US18390884
申请日：2023-12-20
IPC分类号： C07K14/47 , A61K47/64 , C07K14/005 , C12N9/10 , A61K38/00
CPC分类号： C07K14/47 , A61K47/64 , C07K14/005 , C12N9/104 , C12Y203/02 , A61K38/00 , C07K2319/02 , C07K2319/07 , C07K2319/50 , C12N2740/16322
摘要： The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia. The fusion protein may comprise a protein of interest to be delivered to a non-nuclear organelle; an organelle targeting sequence (OTS); a cell penetrating peptide (CPP); and a target enhancing sequence (TES); wherein the CPP is capable of interference with delivery of the protein of interest to the non-nuclear organelle; and wherein the TES prevents said interference by the CPP. The fusion protein may also comprise a protein of interest to be delivered to a non-nuclear organelle; a CPP and a TES. The present disclosure also provides methods for treating a non-nuclear organelle associated disorder by administering the fusion protein to a subject in need thereof.
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公开(公告)号：US11891420B2
公开(公告)日：2024-02-06
申请号：US17214757
申请日：2021-03-26
IPC分类号： C07K14/47 , A61K47/64 , C07K14/005 , C12N9/10 , A61K38/00
CPC分类号： C07K14/47 , A61K47/64 , C07K14/005 , C12N9/104 , C12Y203/02 , A61K38/00 , C07K2319/02 , C07K2319/07 , C07K2319/50 , C12N2740/16322
摘要： The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia. The fusion protein may comprise a protein of interest to be delivered to a non-nuclear organelle; an organelle targeting sequence (OTS); a cell penetrating peptide (CPP); and a target enhancing sequence (TES); wherein the CPP is capable of interference with delivery of the protein of interest to the non-nuclear organelle; and wherein the TES prevents the interference by the CPP. The fusion protein may also comprise a protein of interest to be delivered to a non-nuclear organelle; a CPP and a TES. The present disclosure also provides methods for treating a non-nuclear organelle associated disorder by administering the fusion protein to a subject in need thereof.
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