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公开(公告)号：US11421211B2
公开(公告)日：2022-08-23
申请号：US16332373
申请日：2017-09-12
申请人： GENETHON , SORBONNE UNIVERSITE , DUKE UNIVERSITY
摘要： The present invention relates to variants of acid-alpha glucosidase and uses thereof. Said variants are sequence-optimized and/or are linked to a heterogenous signal peptide.
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2.发明公开公开(公告)号：US20240317814A1
公开(公告)日：2024-09-26
申请号：US18735636
申请日：2024-06-06
申请人： Genethon , INSERM (Institut National de la Santé et de la Recherche Médicale) , Universite d'Evry Val d'Essonne , Sorbonne Universite , Association Institut de Myologie
IPC分类号： C07K14/005 , A61K48/00 , A61P25/28 , C07K14/075 , C12N7/00 , C12N9/22 , C12N15/11 , C12N15/86
CPC分类号： C07K14/005 , A61P25/28 , C07K14/075 , C12N7/00 , C12N9/22 , C12N15/111 , C12N15/86 , A61K48/00 , C12N2310/20 , C12N2750/14121 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152 , C12N2750/14171
摘要： The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.
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公开(公告)号：US12037362B2
公开(公告)日：2024-07-16
申请号：US16981086
申请日：2019-04-04
申请人： Genethon , Institut National de la Sante et de la Recherche Medicale , Universite d'Evry Val d'Essonne , Sorbonne Universite , Association Institut de Myologie
IPC分类号： C07K14/005 , A61K48/00 , A61P25/28 , C07K14/075 , C12N7/00 , C12N9/22 , C12N15/11 , C12N15/86
CPC分类号： C07K14/005 , A61P25/28 , C07K14/075 , C12N7/00 , C12N9/22 , C12N15/111 , C12N15/86 , A61K48/00 , C12N2310/20 , C12N2750/14121 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152 , C12N2750/14171
摘要： The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.
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公开(公告)号：US20220380807A1
公开(公告)日：2022-12-01
申请号：US17861676
申请日：2022-07-11
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ D'EVRY-VAL-D'ESSONNE , GENETHON , FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA , FONDAZIONE TELETHON
IPC分类号： C12N15/86 , A61K45/06 , C12N15/113
摘要： The invention relates to an RNA interference (RNAi) strategy based on the use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, the invention relates to a vector that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
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公开(公告)号：US11427836B2
公开(公告)日：2022-08-30
申请号：US16604648
申请日：2018-04-11
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ D'EVRY-VAL-D'ESSONNE , GENETHON , FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA , FONDAZIONE TELETHON
IPC分类号： C12N15/113 , C12N15/86 , A61K45/06 , C12Q1/68
摘要： An RNA interference (RNAi) strategy is provided based on use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, a method is provided that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
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公开(公告)号：US11541131B2
公开(公告)日：2023-01-03
申请号：US16491623
申请日：2018-03-09
申请人： GENETHON , UNIVERSITE D'EVRY VAL D'ESSONNE , INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE , SORBONNE UNIVERSITÉ
摘要： The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.
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公开(公告)号：US10471132B2
公开(公告)日：2019-11-12
申请号：US15303834
申请日：2015-04-27
摘要： The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.
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公开(公告)号：US11376321B2
公开(公告)日：2022-07-05
申请号：US16490181
申请日：2018-03-01
申请人： GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , SORBONNE UNIVERSITÉ , ASSOCIATION INSTITUT DE MYOLOGIE
IPC分类号： A61K39/235 , A61M1/36 , C07K16/06 , C07K16/08 , C12N7/00
摘要： The present invention relates to a method for removing undesired anti-AAV antibodies from a blood-derived composition.
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公开(公告)号：US11339406B2
公开(公告)日：2022-05-24
申请号：US16332379
申请日：2017-09-12
申请人： GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY VAL D'ESSONNE
摘要： The present invention relates to variants of acid-alpha glucosidase and uses thereof.
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公开(公告)号：US20210130847A1
公开(公告)日：2021-05-06
申请号：US16604648
申请日：2018-04-11
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ D'EVRY-VAL-D'ESSONNE , GENETHON , FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA , FONDAZIONE TELETHON
IPC分类号： C12N15/86 , C12N15/113 , A61K45/06
摘要： An RNA interference (RNAi) strategy is provided based on use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, a method is provided that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
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