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1. 发明申请

WO2017156015A3 MICRORNAS AND METHODS OF THEIR USE 审中-公开
公开(公告)号：WO2017156015A3
公开(公告)日：2017-09-14
申请号：PCT/US2017/021178
申请日：2017-03-07
申请人： THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES , MIRECULE, INC.
发明人： SALEH, Anthony, D. , VAN WAES, Carter , CHEN, Zhong , CHENG, Hui
IPC分类号： C12N15/113 , A61K31/7088 , A61P35/00
摘要： Disclosed herein are methods of treating a tumor in a subject, including administering to the subject one or more miRNA nucleic acids or variants (such as mimics or mimetics) thereof with altered expression in the tumor. Also disclosed herein are compositions including one or more miRNA nucleic acids. In some examples, the miRNA nucleic acids are modified miRNAs, for example, and miRNA nucleic acid including one or more modified nucleotides and/or a 5'-end and/or 3'-end modification. In particular examples, the modified miRNA nucleic acid is an miR-30a nucleic acid. Further disclosed herein are methods of diagnosing a subject as having a tumor with altered expression of one or more miRNA nucleic acids. In some embodiments, the methods include detecting expression of one or more miRNAs in a sample from the subject and comparing the expression in the sample from the subject to a control.

2. 发明申请

WO2021203043A2 TARGETED INHIBITION USING ENGINEERED OLIGONUCLEOTIDES 审中-公开
公开(公告)号：WO2021203043A2
公开(公告)日：2021-10-07
申请号：PCT/US2021/025639
申请日：2021-04-02
申请人： MIRECULE, INC.
发明人： PLACE, Robert , SALEH, Anthony , WILLIAMS, Tishan
IPC分类号： C12N15/113 , A61K31/7088 , A61K31/7105 , A61K31/711 , A61K31/7115 , A61K31/712 , A61K31/7125 , C12N2310/141 , C12N2310/316 , C12N2310/531 , G16B15/30
摘要： Disclosed herein are engineered oligonucleotides for selective inhibition of polypeptide expression and activity. Also disclosed herein are methods of selectively inhibiting polypeptide expression and activity contacting an engineered oligonucleotide with a polynucleotide encoding the polypeptide.

3. 发明申请

WO2017156015A2 MICRORNAS AND METHODS OF THEIR USE 审中-公开
标题翻译： MICRORNAS及其使用方法
公开(公告)号：WO2017156015A2
公开(公告)日：2017-09-14
申请号：PCT/US2017/021178
申请日：2017-03-07
申请人： THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES , MIRECULE, INC.
发明人： SALEH, Anthony, D. , VAN WAES, Carter , CHEN, Zhong , CHENG, Hui
IPC分类号： C12N15/113 , A61K31/7088
摘要： Disclosed herein are methods of treating a tumor in a subject, including administering to the subject one or more miRNA nucleic acids or variants (such as mimics or mimetics) thereof with altered expression in the tumor. Also disclosed herein are compositions including one or more miRNA nucleic acids. In some examples, the miRNA nucleic acids are modified miRNAs, for example, and miRNA nucleic acid including one or more modified nucleotides and/or a 5'-end and/or 3'-end modification. In particular examples, the modified miRNA nucleic acid is an miR-30a nucleic acid. Further disclosed herein are methods of diagnosing a subject as having a tumor with altered expression of one or more miRNA nucleic acids. In some embodiments, the methods include detecting expression of one or more miRNAs in a sample from the subject and comparing the expression in the sample from the subject to a control.
摘要翻译：本文公开了治疗受试者的肿瘤的方法，包括向受试者施用一种或多种在肿瘤中具有改变的表达的miRNA核酸或变体（例如模拟物或模拟物）。本文还公开了包含一种或多种miRNA核酸的组合物。在一些实例中，miRNA核酸是例如修饰的miRNA，和包含一个或多个修饰的核苷酸和/或5'-端和/或3'-端修饰的miRNA核酸。在特定的例子中，修饰的miRNA核酸是miR-30a核酸。本文进一步公开了诊断受试者患有具有改变的一种或多种miRNA核酸表达的肿瘤的方法。在一些实施方案中，所述方法包括检测来自受试者的样品中一种或多种miRNA的表达，并将来自受试者的样品中的表达与对照进行比较。

4. 发明申请

WO2023288289A2 OLIGONUCLEOTIDES AND COMPOSITIONS THEREOF FOR NEUROMUSCULAR DISORDERS 审中-公开
公开(公告)号：WO2023288289A2
公开(公告)日：2023-01-19
申请号：PCT/US2022/073754
申请日：2022-07-14
申请人： MIRECULE, INC.
发明人： SALEH, Anthony , BELGARD, Grant , MUNZ, Marton , MARUSAK, Charles , PLACE, Robert
IPC分类号： C12N15/113 , A61K31/7125 , A61K31/712 , G16B30/00 , C12N2310/11 , C12N2310/3125 , C12N2310/315 , C12N2310/3231 , C12N2310/341 , C12N2310/3513 , C12N2320/11
摘要： Disclosed herein are engineered DUX4-targeting oligonucleotides for selective inhibition of RNA transcripts associated with a neuromuscular disease such as facioscapulohumeral muscular dystrophy. Also disclosed are vectors containing any of these, pharmaceutical formulations containing any of the these, and kits containing any of the these. Also disclosed herein are methods of selectively inhibiting polypeptide expression and activity by contacting a DUX4-targeting oligonucleotide with an RNA transcript associated with a neuromuscular disease such as facioscapulohumeral muscular dystrophy.

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