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1.发明公开
公开(公告)号：EP4410979A3
公开(公告)日：2024-11-06
申请号：EP24157430.0
申请日：2017-04-14
IPC分类号： A61K48/00 , C12N15/11 , C12N15/113
摘要： The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from dystrophinopathy or muscular dystrophy. The invention also provides for combination therapies comprising expressing both miR-29 and micro-dystrophin to reduce and prevent fibrosis in patients suffering from dystrophinopathy or muscular dystrophy.
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2.发明公开
公开(公告)号：EP4407035A3
公开(公告)日：2024-10-30
申请号：EP24163078.9
申请日：2017-04-14
摘要： Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
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公开(公告)号：EP4335502A3
公开(公告)日：2024-07-03
申请号：EP23207092.0
申请日：2017-03-31
IPC分类号： C12N15/113 , A61K35/76 , A61P21/00
CPC分类号： C07H21/02 , C12Q1/70 , C12N2310/14120130101 , C12N2330/5120130101 , C12N15/113 , C12N2320/3220130101 , Y02A50/30
摘要： The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.
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4.发明公开
公开(公告)号：EP4301462A1
公开(公告)日：2024-01-10
申请号：EP22712160.5
申请日：2022-03-04
IPC分类号： A61P21/04 , C07K14/47 , C12N15/864
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公开(公告)号：EP4295864A2
公开(公告)日：2023-12-27
申请号：EP23209820.2
申请日：2019-09-30
IPC分类号： A61K45/06
摘要： The present disclosure relates to a specific dibenzoazepine compound for use in inhibiting or treating an infection of a subject with a pathogen that interacts with a complement receptor 3 (CR3) polypeptide-expressing cell, wherein the pathogen is selected from a Neisseria species and a retrovirus.
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公开(公告)号：EP4219727A3
公开(公告)日：2023-09-06
申请号：EP22215568.1
申请日：2014-08-26
IPC分类号： C12N15/86 , A61P25/28 , C12N9/02 , C12N15/113
摘要： The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.
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公开(公告)号：EP4214317A2
公开(公告)日：2023-07-26
申请号：EP21789957.4
申请日：2021-09-15
IPC分类号： C12N15/113 , C12N15/10 , C12N9/22 , C12N15/90 , A61K31/7088 , A61P21/00
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公开(公告)号：EP4178359A2
公开(公告)日：2023-05-17
申请号：EP21837938.6
申请日：2021-07-06
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公开(公告)号：EP4164668A1
公开(公告)日：2023-04-19
申请号：EP21746204.3
申请日：2021-06-15
发明人： RODINO-KLAPAC, Louise , MENDELL, Jerry, R. , PETERSON, Ellyn , POTTER, Rachael , GRIFFIN, Danielle
IPC分类号： A61K35/761 , A61P21/00
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