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1.发明申请公开(公告)号：US20170218366A1
公开(公告)日：2017-08-03
申请号：US15502702
申请日：2015-08-07
Inventor： Kevin Flanigan , Nicolas Wein , Steven Wilton
IPC: C12N15/113 , A61K9/00 , A61K31/58 , A61K31/712 , A61K31/573
CPC classification number: C12N15/113 , A61K9/0019 , A61K31/573 , A61K31/58 , A61K31/712 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2310/3233 , C12N2310/346 , C12N2310/3513 , C12N2310/3519 , C12N2320/31 , C12N2320/33 , C12N2330/51 , C12N2750/14143 , C12N2840/203 , C12N2310/321 , C12N2310/3521
Abstract: The present invention relates to the delivery of oligomers for treating patients with a 5′ mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulting in translation of a functional truncated isoform of dystrophin. The methods and materials can be used for the treatment of muscular dystrophies arising from 5′ mutations in the DMD gene such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
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2.发明申请RECOMBINANT ADENO-ASSOCIATED VIRUS DELIVERY OF EXON 2-TARGETED U7SNRNA POLYNUCLEOTIDE CONSTRUCTS 有权重组腺病毒相关的外显子2对靶向U7SNRNA多核苷酸构建体的病毒公开(公告)号：US20160076028A1
公开(公告)日：2016-03-17
申请号：US14785769
申请日：2014-04-18
Inventor： Kevin Flanigan , Aneline Vulin-Chaffiol , Nicolas Wein
IPC: C12N15/113 , C12N7/00
CPC classification number: C12N15/113 , A61K48/005 , C07K14/4708 , C12N7/00 , C12N15/111 , C12N15/86 , C12N2310/11 , C12N2320/32 , C12N2320/33 , C12N2330/51 , C12N2750/14132 , C12N2750/14143 , C12N2750/14171 , C12N2750/14321 , C12N2750/14333
Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
Abstract translation: 本发明涉及重组腺相关病毒（rAAV）递送多核苷酸，用于治疗由DMD外显子2复制而产生的Duchenne肌营养不良症。本发明提供rAAV产品和使用rAAV治疗杜氏肌营养不良症的方法。
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3.发明授权公开(公告)号：US11230707B2
公开(公告)日：2022-01-25
申请号：US16032744
申请日：2018-07-11
Inventor： Kevin Flanigan , Adeline Vulin-Chaffiol , Nicolas Wein
IPC: C12N15/113 , A61K48/00 , C12N15/86 , C12N15/11 , C12N15/864 , C12N7/00 , C07K14/47
Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
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4.发明授权公开(公告)号：US11053494B2
公开(公告)日：2021-07-06
申请号：US15502702
申请日：2015-08-07
Applicant: The Research Institute at Nationwide Children's Hospital , The University of Western Australia
Inventor： Kevin Flanigan , Nicolas Wein , Stephen Wilton
IPC: C12N15/113 , A61K9/00 , A61K31/573 , A61K31/58 , A61K31/712
Abstract: The present invention relates to the delivery of oligomers for treating patients with a 5′ mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulting in translation of a functional truncated isoform of dystrophin. The methods and materials can be used for the treatment of muscular dystrophies arising from 5′ mutations in the DMD gene such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
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Patent Agency Ranking
公开(公告)号：US20240398861A1
公开(公告)日：2024-12-05
申请号：US18689800
申请日：2022-09-06
Inventor： Kevin A. Cassady , Uksha Saini , Dawn Chandler , Ruoning Wang , Nicolas Wein
Abstract: This disclosure relates to populations and compositions of purified cancer cell-derived vesicles and uses thereof.
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6.发明授权公开(公告)号：US09862945B2
公开(公告)日：2018-01-09
申请号：US14785769
申请日：2014-04-18
Inventor： Kevin Flanigan , Adeline Vulin-Chaffiol , Nicolas Wein
CPC classification number: C12N15/113 , A61K48/005 , C07K14/4708 , C12N7/00 , C12N15/111 , C12N15/86 , C12N2310/11 , C12N2320/32 , C12N2320/33 , C12N2330/51 , C12N2750/14132 , C12N2750/14143 , C12N2750/14171 , C12N2750/14321 , C12N2750/14333
Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
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7.发明申请公开(公告)号：US20230025574A1
公开(公告)日：2023-01-26
申请号：US17546428
申请日：2021-12-09
Inventor： Kevin Flanigan , Adeline Vulin-Chaffiol , Nicolas Wein
IPC: C12N15/113 , A61K48/00 , C07K14/47 , C12N15/86 , C12N15/11 , C12N15/864 , C12N7/00
Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
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公开(公告)号：US11180755B2
公开(公告)日：2021-11-23
申请号：US16079426
申请日：2017-02-24
Inventor： Scott Quenton Harper , Nicolas Wein
IPC: C12N15/113 , C12Q1/68 , A61P21/00 , C12N15/864
Abstract: The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nuclear RNAs to induce DUX4 exon-skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
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9.发明申请公开(公告)号：US20190276822A1
公开(公告)日：2019-09-12
申请号：US16032744
申请日：2018-07-11
Inventor： Kevin Flanigan , Adeline Vulin-Chaffiol , Nicolas Wein
Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
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