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公开(公告)号：US20190241911A1
公开(公告)日：2019-08-08
申请号：US16269021
申请日：2019-02-06
Applicant: Ohio State Innovation Foundation
Inventor： Yizhou Dong , Bin Li
IPC: C12N15/90 , C12N15/85 , C12N9/22 , A61K31/712 , A61K31/7125 , A61K31/7105
CPC classification number: C12N15/907 , A61K31/7105 , A61K31/712 , A61K31/7125 , C12N9/22 , C12N15/85 , C12N2310/122 , C12N2310/20 , C12N2310/315 , C12N2310/3235 , C12N2310/333 , C12N2310/3341 , C12N2310/335
Abstract: The present disclosure generally relates to systems, methods and compositions for use in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cpf1 genome editing systems.
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公开(公告)号：US20170145409A1
公开(公告)日：2017-05-25
申请号：US15125634
申请日：2015-03-17
Applicant: Ionis Pharmaceuticals, Inc.
Inventor： Punit P. Seth , Eric E. Swayze
IPC: C12N15/113 , C07F9/6512
CPC classification number: C12N15/113 , C07D239/54 , C07D473/34 , C07F9/6512 , C07H19/06 , C07H19/16 , C07H21/00 , C07H23/00 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3235 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3525 , C12N2320/51 , C12N2320/52
Abstract: The present invention provides novel bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom. Incorporation of one or more of the bicyclic carbocyclic nucleosides into an oligomeric compound is expected to enhance one or more properties of the oligomeric compound. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in modulation of normal function of the target RNA. In certain embodiments, bicyclic carbocyclic nucleosides are provided as monomers for use as antivirals.
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3.发明申请Oligonucleotide Derivative, Oligonucleotide Derivative-Containing Pharmaceutical Composition for Treatment and Pharmaceutical Composition for Diagnosis, and Oligonucleotide Derivative for Regulation of miRNA Function 有权用于治疗的寡核苷酸衍生物，含寡核苷酸衍生物的药物组合物和用于诊断的药物组合物和用于调节miRNA功能的寡核苷酸衍生物公开(公告)号：US20140128347A1
公开(公告)日：2014-05-08
申请号：US14123562
申请日：2012-06-01
Applicant: Akira Matsuda , Mayumi Takahashi
Inventor： Akira Matsuda , Mayumi Takahashi
IPC: C12N15/113
CPC classification number: C12N15/113 , A61K31/7088 , A61K31/713 , C12N2310/113 , C12N2310/315 , C12N2310/3235 , C12N2310/351 , C12N2310/321 , C12N2310/3521
Abstract: An oligonucleotide derivative comprises repeating structural units represented by the following general formula (wherein B represents adenine, guanine, cytosine, or uracil; X represents a sulfur atom or an oxygen atom; n represents an integer of 6 to 60; and B and X are independently represented in each of the repeating structural units), wherein X is a sulfur atom in at least one of the repeating structural units represented by the general formula:
Abstract translation: 寡核苷酸衍生物包含由以下通式表示的重复结构单元（其中B表示腺嘌呤，鸟嘌呤，胞嘧啶或尿嘧啶; X表示硫原子或氧原子; n表示6〜60的整数，B和X为 在每个重复结构单元中独立地表示），其中X是至少一个由以下通式表示的重复结构单元中的硫原子：
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公开(公告)号：US10036019B2
公开(公告)日：2018-07-31
申请号：US15125634
申请日：2015-03-17
Applicant: Ionis Pharmaceuticals, Inc.
Inventor： Punit P. Seth , Eric E. Swayze
IPC: C12N15/113 , C07F9/6512
CPC classification number: C12N15/113 , C07D239/54 , C07D473/34 , C07F9/6512 , C07H19/06 , C07H19/16 , C07H21/00 , C07H23/00 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3235 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3525 , C12N2320/51 , C12N2320/52
Abstract: The present invention provides novel bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom. Incorporation of one or more of the bicyclic carbocyclic nucleosides into an oligomeric compound is expected to enhance one or more properties of the oligomeric compound. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in modulation of normal function of the target RNA. In certain embodiments, bicyclic carbocyclic nucleosides are provided as monomers for use as antivirals.
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公开(公告)号：US20140343127A1
公开(公告)日：2014-11-20
申请号：US14357385
申请日：2012-11-08
Applicant: Santaris Pharma A/S
Inventor： Susanne Kammler
IPC: C12N15/113
CPC classification number: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2310/3235 , C12N2320/33
Abstract: The present invention relates to oligomer compounds (oligomers) which target nucleic acids encoding human SMN2 in a cell, leading to modulation of SMN2 mRNA splicing which favors full length SMN2 mRNA rather than the poorly functional truncated transcript, SMN2 Δ7. Reduction of SMNA7 mRNA expression and/or increase in full length SMN2 mRNA expression are beneficial for the treatment of diseases or disorders associated with overexpression or undesirably high levels of aberrant forms of SMN2, particularly SMN2 Δ7, such as spinal muscular atrophy (SMA).
Abstract translation: 本发明涉及靶向细胞中编码人SMN2的核酸的低聚物化合物（低聚物），导致SMN2 mRNA剪接的调节，其有利于全长SMN2 mRNA而不是功能不良的截短的转录物SMN2和Dgr; 7。 SMNA7 mRNA表达的减少和/或全长SMN2 mRNA表达的增加有益于治疗与过度表达或不期望的高水平的SMN2异常形式（特别是SMN2和Dgr）7相关的疾病或病症，例如脊髓性肌萎缩（SMA） ）。
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Patent Agency Ranking
公开(公告)号：US11866701B2
公开(公告)日：2024-01-09
申请号：US16760593
申请日：2018-11-01
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Gregory Hinkle , Anna Borodovsky
IPC: C12N15/113 , A61K31/7115
CPC classification number: C12N15/113 , A61K31/7115 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3235 , C12N2310/351
Abstract: The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.
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公开(公告)号：US20180112213A1
公开(公告)日：2018-04-26
申请号：US15560968
申请日：2016-03-25
Applicant: Editas Medicine, Inc.
Inventor： Gordon Grant Welstead , Jennifer Leah Gori , Jack Michael Heath , David A. Bumcrot
CPC classification number: C12N15/11 , A61K35/17 , A61K48/00 , A61P7/00 , C12N5/0636 , C12N9/22 , C12N9/96 , C12N15/111 , C12N15/113 , C12N2310/20 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/3231 , C12N2310/3235 , C12N2310/334 , C12N2310/335 , C12N2310/336 , C12N2310/344 , C12N2320/51
Abstract: CRISPR/Cas-related compositions and methods which provide for efficient gene editing of eukaryotic cells using modified gRNAs.
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公开(公告)号：US20240294912A1
公开(公告)日：2024-09-05
申请号：US18519295
申请日：2023-11-27
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Gregory Hinkle , Anna Borodovsky
IPC: C12N15/113 , A61K31/7115
CPC classification number: C12N15/113 , A61K31/7115 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3235 , C12N2310/351
Abstract: The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.
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公开(公告)号：US20230203491A1
公开(公告)日：2023-06-29
申请号：US17999399
申请日：2021-05-21
Applicant: BIONEER CORPORATION , SIRNAGEN THERAPEUTICS CORPORATION
Inventor： Han-Oh PARK , Jangseon KIM , Mi Sun LEE , Soonja CHOI , Eun-Kwang LEE , Sang Jin BYUN
IPC: C12N15/113 , A61K9/51 , A61P31/14 , A61P11/00 , A61K31/713
CPC classification number: C12N15/1131 , A61K9/51 , A61P31/14 , A61P11/00 , A61K31/713 , C12N2310/3235 , C12N2310/3231 , C12N2310/315 , C12N2310/312 , C12N2310/14 , C12N2320/32
Abstract: The present invention relates to: a double-stranded oligonucleotide which can highly specifically and efficiently inhibit the proliferation of Severe acute respiratory syndrome coronavirus 2 (SARS-Cov-2), preferably a double-stranded oligonucleotide comprising a sequence in the form of RNA/RNA, DNA/DNA or a DNA/RNA hybrid; a double-stranded oligonucleotide structure and nanoparticles comprising the double-stranded oligonucleotide; and a use thereof for treating COVID-19.
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10.发明授权Oligonucleotide derivative, oligonucleotide derivative-containing pharmaceutical composition for treatment and pharmaceutical composition for diagnosis, and oligonucleotide derivative for regulation of miRNA function 有权用于治疗的寡核苷酸衍生物，含寡核苷酸衍生物的药物组合物和用于诊断的药物组合物和用于调节miRNA功能的寡核苷酸衍生物公开(公告)号：US09315810B2
公开(公告)日：2016-04-19
申请号：US14123562
申请日：2012-06-01
Applicant: Akira Matsuda , Mayumi Takahashi
Inventor： Akira Matsuda , Mayumi Takahashi
IPC: C12N15/113 , A61K31/7088 , A61K31/713
CPC classification number: C12N15/113 , A61K31/7088 , A61K31/713 , C12N2310/113 , C12N2310/315 , C12N2310/3235 , C12N2310/351 , C12N2310/321 , C12N2310/3521
Abstract: An oligonucleotide derivative comprises repeating structural units represented by the following general formula (wherein B represents adenine, guanine, cytosine, or uracil; X represents a sulfur atom or an oxygen atom; n represents an integer of 6 to 60; and B and X are independently represented in each of the repeating structural units), wherein X is a sulfur atom in at least one of the repeating structural units represented by the general formula:
Abstract translation: 寡核苷酸衍生物包含由以下通式表示的重复结构单元（其中B表示腺嘌呤，鸟嘌呤，胞嘧啶或尿嘧啶; X表示硫原子或氧原子; n表示6〜60的整数，B和X为 在每个重复结构单元中独立地表示），其中X是至少一个由以下通式表示的重复结构单元中的硫原子：
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