Title:
ANTISENSE OLIGONUCLEOTIDE WITH REDUCED SIDE EFFECTS
Document Type and Number:
WIPO Patent Application WO/2018/051762
Kind Code:
A1
Abstract:
The present invention addresses the problem of providing an antisense oligonucleotide (ASO) with reduced side effects. To solve this problem, provided is an ASO having a structure, wherein phosphodiester bonds occurring naturally between oligonucleotides and phosphorothioate bonds obtained by substituting one of oxygen atoms in a phosphate group by a sulfur atom are alternately positioned, as an antisense activity site.
Inventors:
YANO JUNICHI (JP)
TANIGAWARA KAZUAKI (JP)
TANIGAWARA KAZUAKI (JP)
Application Number:
PCT/JP2017/030447
Publication Date:
March 22, 2018
Filing Date:
August 25, 2017
Export Citation:
Assignee:
RENA THERAPEUTICS INC (JP)
International Classes:
C12N15/113; A61K31/7125; A61K48/00
Domestic Patent References:
| WO2000003720A1 | 2000-01-27 | |||
| WO1999050409A1 | 1999-10-07 | |||
| WO2015017675A2 | 2015-02-05 |
Foreign References:
| JPH09506248A | 1997-06-24 | |||
| US20090203896A1 | 2009-08-13 |
Other References:
ZHOU W. ET AL.: "Mixed-backbone oligonucleotides as second-generation antisense agents with reduced phosphorothioate-related side effects", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, vol. 8, no. 22, 1998, pages 3269 - 3274, XP004143740
SCHMAJUK G. ET AL.: "Antisense Oligonucleotides with Different Backbones", J. BIOL. CHEM., vol. 274, no. 31, 1999, pages 21783 - 21789, XP055200366
PATIL S.V. ET AL.: "Syntheses and properties of oligothymidylate analogs containing stereoregulated phosphorothioate and phosphodiester linkages in an alternating manner", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, vol. 4, no. 22, 1994, pages 2663 - 2666, XP002924967
LI SONG ET AL.: "Folate-Mediated Targeting of Antisense Oligodeoxynucleotides to Ovarian Cancer Cells", PHARMACEUTICAL RESEARCH, vol. 15, no. 10, 1998, pages 1540 - 1545, XP002567483
JEONG J.H. ET AL.: "In vivo tumor targeting of ODN-PEG-folic acid/PEI polyelectrolyte complex micelles", J. BIOMATER. SCI. POLYMER EDN, vol. 16, no. 11, 2005, pages 1409 - 1419, XP055498605
SCHMAJUK G. ET AL.: "Antisense Oligonucleotides with Different Backbones", J. BIOL. CHEM., vol. 274, no. 31, 1999, pages 21783 - 21789, XP055200366
PATIL S.V. ET AL.: "Syntheses and properties of oligothymidylate analogs containing stereoregulated phosphorothioate and phosphodiester linkages in an alternating manner", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, vol. 4, no. 22, 1994, pages 2663 - 2666, XP002924967
LI SONG ET AL.: "Folate-Mediated Targeting of Antisense Oligodeoxynucleotides to Ovarian Cancer Cells", PHARMACEUTICAL RESEARCH, vol. 15, no. 10, 1998, pages 1540 - 1545, XP002567483
JEONG J.H. ET AL.: "In vivo tumor targeting of ODN-PEG-folic acid/PEI polyelectrolyte complex micelles", J. BIOMATER. SCI. POLYMER EDN, vol. 16, no. 11, 2005, pages 1409 - 1419, XP055498605
Attorney, Agent or Firm:
ESAKI Koushi et al. (JP)
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