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公开(公告)号:US20240301416A1
公开(公告)日:2024-09-12
申请号:US18570778
申请日:2022-06-23
发明人: Yuichiro Tone , Yoshitsugu Aoki , Norio Motohashi
IPC分类号: C12N15/113 , A61P21/00
CPC分类号: C12N15/113 , A61P21/00 , C12N2310/11 , C12N2310/314 , C12N2310/321 , C12N2310/3233 , C12N2320/33
摘要: Herein, a combination of antisense oligomers or pharmaceutically acceptable salts thereof, or hydrates thereof which cause simultaneous skipping of any two or more numerically consecutive exons selected from the group consisting of the 45th exon to the 55th exon in human dystrophin pre-mRNA is provided.
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2.
公开(公告)号:US20240158792A1
公开(公告)日:2024-05-16
申请号:US18504781
申请日:2023-11-08
发明人: Tomonori UNO , Takashi NATSUKAWA , Youichi EGAWA , Youhei SATOU
IPC分类号: C12N15/113 , A61P21/00
CPC分类号: C12N15/113 , A61P21/00 , C12N2310/11 , C12N2320/35
摘要: The present invention relates to a composition containing an antisense oligonucleotide, and the use thereof to treat Duchenne muscular dystrophy. The present invention particularly relates to the above-described composition that is effective for the treatment of Duchenne muscular dystrophy when it is administered at a dose for the treatment, and the use thereof.
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公开(公告)号:US11560401B2
公开(公告)日:2023-01-24
申请号:US17053448
申请日:2019-05-10
发明人: Yutaro Kotobuki , Fumiya Urabe
摘要: A method for subjecting a compound [A] having a hydroxyl group or a primary or secondary amino group and a compound [B] having a substituent containing a phosphorus atom to a condensation reaction to prepare a compound [C] represented by the following general formula [C]: wherein the condensation reaction is carried out in a mixed solvent containing a polar solvent and a halogen solvent as a reaction solvent.
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公开(公告)号:US20230014840A1
公开(公告)日:2023-01-19
申请号:US17776356
申请日:2020-11-13
发明人: Yutaro KOTOBUKI , Koki KATO
摘要: The present invention relates to a method for producing a compound represented by general formula [C-1-1], which comprises: a step for reacting a compound represented by general formula [B-1-1] with a compound represented by general formula [P] to form a compound represented by general formula [B-1-2]; and a step for reacting the compound represented by general formula [B-1-2] with a compound represented by general formula [A-1] to form the compound represented by general formula [C-1-1].
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5.
公开(公告)号:US20230000840A1
公开(公告)日:2023-01-05
申请号:US17775345
申请日:2020-11-12
发明人: Ryohei YOSHINAGA , Maki KURITA , Yasunori ASAO , Tomomi FUKUI
IPC分类号: A61K31/437 , A61K31/496 , A61P1/00
摘要: A novel prophylactic agent or therapeutic agent for functional gastrointestinal disorders or xerostomia is provided. The present invention is a therapeutic agent or prophylactic agent for functional gastrointestinal disorders, containing an azabenzimidazole compound represented by the following formula [1] (each symbol in the formula is as described in the specification), or a pharmaceutically acceptable salt thereof, or a solvate thereof, as an active ingredient.
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公开(公告)号:US20210261963A1
公开(公告)日:2021-08-26
申请号:US17253760
申请日:2019-06-26
发明人: Tomonori UNO , Takashi NATSUKAWA , Youichi EGAWA , Youhei SATOU
IPC分类号: C12N15/113 , A61P21/00
摘要: The present invention relates to a composition containing an antisense oligonucleotide, and the use thereof to treat Duchenne muscular dystrophy. The present invention particularly relates to the above-described composition that is effective for the treatment of Duchenne muscular dystrophy when it is administered at a dose for the treatment, and the use thereof.
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公开(公告)号:US20200149040A1
公开(公告)日:2020-05-14
申请号:US16437130
申请日:2019-06-11
IPC分类号: C12N15/113 , C12N15/11 , A61K31/713
摘要: Provided is a drug that allows highly-efficient skipping of exon 51 in the human dystrophin gene. The present invention provides an antisense oligomer which enables exon 51 in the human dystrophin gene to be skipped.
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公开(公告)号:US10385092B2
公开(公告)日:2019-08-20
申请号:US16359213
申请日:2019-03-20
发明人: Naoki Watanabe , Youhei Satou , Shin'ichi Takeda , Tetsuya Nagata
IPC分类号: C07H21/04 , C12N15/113 , C12N15/11 , C07H21/00 , C12N5/00
摘要: The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene. Also provided is a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.
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公开(公告)号:US10329319B2
公开(公告)日:2019-06-25
申请号:US15619996
申请日:2017-06-12
发明人: Naoki Watanabe , Youhei Satou , Shin'ichi Takeda , Tetsuya Nagata
摘要: The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene. Also provided is a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.
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公开(公告)号:US20190048023A1
公开(公告)日:2019-02-14
申请号:US16077889
申请日:2017-02-28
发明人: Fumi HIGUCHI
IPC分类号: C07D513/04
摘要: An object of the present invention is to provide a compound with an excellent JAK1 inhibitory activity.The compound of the invention has JAK1 inhibitory activity, and thus, immunosuppressive effect, anti-inflammatory effect, anti-proliferative effect and so on, and is useful in the treatment of the diseases, for example, rheumatoid arthritis, inflammatory bowel disease, psoriasis, vasculitis, bronchial asthma, chronic obstructive pulmonary disease, eosinophilic sinusitis and nasal polyp.
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