Invention Grant
- Patent Title: Modified siRNA and pharmaceutical composition
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Application No.: US15749191Application Date: 2016-07-29
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Publication No.: US10548990B2Publication Date: 2020-02-04
- Inventor: Kazunobu Futami , Yasuhiro Furuichi , Satoshi Kaneto
- Applicant: GeneCare Research Institute Co., Ltd.
- Applicant Address: JP Kamakura
- Assignee: GeneCare Research Institute Co., Ltd.
- Current Assignee: GeneCare Research Institute Co., Ltd.
- Current Assignee Address: JP Kamakura
- Agency: DLA Piper LLP (US)
- Priority: JP2015-151974 20150731
- International Application: PCT/JP2016/072261 WO 20160729
- International Announcement: WO2017/022650 WO 20170209
- Main IPC: C07H21/04
- IPC: C07H21/04 ; A61K48/00 ; C12N15/113 ; A61K31/713 ; A61P35/00

Abstract:
Double-stranded modified siRNA targeting a RecQL1 helicase gene includes a sense strand including the nucleotide sequence shown in SEQ ID NO: 1, and an antisense strand including the nucleotide sequence shown in SEQ ID NO: 2, wherein the sense strand includes 2′-substituted nucleotides at positions 2, 3, 4 and 13 in the nucleotide sequence shown in SEQ ID NO: 1, the sense strand further includes a 2′-substituted nucleotide(s) at one or more positions selected from the group consisting of positions 12, 14, 17, 18 and 19 in the nucleotide sequence shown in SEQ ID NO: 1, wherein the position 2′ of the 2′-substituted nucleotides is —R1, —OR1, —R2OR1, —OR2OR1 or —R3OR2OR1, wherein R1 represents a C1-4 alkyl group, and R2 and R3 independently represent a C1-3 alkylene group.
Public/Granted literature
- US20180214574A1 MODIFIED siRNA AND PHARMACEUTICAL COMPOSITION Public/Granted day:2018-08-02
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