Title:
ANTISENSE OLIGOMER
Document Type and Number:
WIPO Patent Application WO/2022/239863
Kind Code:
A1
Abstract:
The present invention addresses the problem of providing an antisense oligomer capable of treating Werner's syndrome without direct mutated gene repair, or a pharmacologically acceptable salt of the antisense oligomer. The present invention provides an antisense oligomer that is composed of a base sequence complementary to the base sequence of (i) or (ii) and that enables skipping of the 27th exon of the human WRN gene, or a pharmacologically acceptable salt of the antisense oligomer. (i) A base sequence of 15 or more contiguous bases of a base sequence represented by SEQ ID NO: 1. (ii) A base sequence in which one or a plurality of bases have been deleted, substituted, or inserted in a base sequence of 15 or more contiguous bases of a base sequence represented by SEQ ID NO: 1.
Inventors:
YOKOTE KOUTARO (JP)
OUCHI YASUO (JP)
KATO HISAYA (JP)
ETO KOJI (JP)
MAEZAWA YOSHIRO (JP)
OUCHI YASUO (JP)
KATO HISAYA (JP)
ETO KOJI (JP)
MAEZAWA YOSHIRO (JP)
Application Number:
PCT/JP2022/020224
Publication Date:
November 17, 2022
Filing Date:
May 13, 2022
Export Citation:
Assignee:
UNIV CHIBA NAT UNIV CORP (JP)
International Classes:
A61K48/00; A61K31/7088; A61K35/76; A61P3/06; A61P3/10; A61P9/10; A61P17/00; A61P17/14; A61P27/12; A61P43/00; C12N15/113; C12N15/86
Domestic Patent References:
| WO2010048586A1 | 2010-04-29 | |||
| WO1991009033A1 | 1991-06-27 | |||
| WO2009064471A1 | 2009-05-22 | |||
| WO2013100190A1 | 2013-07-04 |
Other References:
YOKOTE KOUTARO, CHANPRASERT SIRISAK, LEE LIN, EIRICH KATHARINA, TAKEMOTO MINORU, WATANABE AKI, KOIZUMI NAOKO, LESSEL DAVOR, MORI T: "WRN Mutation Update: Mutation Spectrum, Patient Registries, and Translational Prospects", HUMAN MUTATION, vol. 38, no. 1, 1 January 2017 (2017-01-01), US , pages 7 - 15, XP093003516, ISSN: 1059-7794, DOI: 10.1002/humu.23128
KATO HISAYA; MAEZAWA YOSHIRO; OUCHI YASUO; TAKAYAMA NAOYA; SONE MASAMITSU; SONE KANAKO; TAKADA-WATANABE AKI; TSUJIMURA KYOKO; KOSH: "Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome", STEM CELL RESEARCH, vol. 53, 23 April 2021 (2021-04-23), NL , pages 1 - 5, XP086583333, ISSN: 1873-5061, DOI: 10.1016/j.scr.2021.102360
AARTSMA-RUS ANNEMIEKE; VAN OMMEN GERT-JAN B: "Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications", RNA, vol. 13, no. 10, 1 October 2007 (2007-10-01), US , pages 1609 - 1624, XP009144451, ISSN: 1355-8382
HARDING P L; FALL A M; HONEYMAN K; FLETCHER S; WILTON S D: "The influence of antisense oligonucleotide length on dystrophin exon skipping.", MOLECULAR THERAPY, vol. 15, no. 1, 1 January 2007 (2007-01-01), US , pages 157 - 166, XP009101408, ISSN: 1525-0016, DOI: 10.1038/sj.mt.6300006
YOKOTE K ET AL., HUM MUTAT., vol. 38, no. 1, January 2017 (2017-01-01), pages 7 - 15
KATO H ET AL., STEM CELL RESEARCH., vol. 53, May 2021 (2021-05-01), pages 102360
XIE J ET AL., FRONT PHARMACOL., vol. 11, 20 May 2020 (2020-05-20), pages 697
GE SX ET AL., BMC BIOINFORMATICS, vol. 19, 2018, pages 534
KATO HISAYA; MAEZAWA YOSHIRO; OUCHI YASUO; TAKAYAMA NAOYA; SONE MASAMITSU; SONE KANAKO; TAKADA-WATANABE AKI; TSUJIMURA KYOKO; KOSH: "Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome", STEM CELL RESEARCH, vol. 53, 23 April 2021 (2021-04-23), NL , pages 1 - 5, XP086583333, ISSN: 1873-5061, DOI: 10.1016/j.scr.2021.102360
AARTSMA-RUS ANNEMIEKE; VAN OMMEN GERT-JAN B: "Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications", RNA, vol. 13, no. 10, 1 October 2007 (2007-10-01), US , pages 1609 - 1624, XP009144451, ISSN: 1355-8382
HARDING P L; FALL A M; HONEYMAN K; FLETCHER S; WILTON S D: "The influence of antisense oligonucleotide length on dystrophin exon skipping.", MOLECULAR THERAPY, vol. 15, no. 1, 1 January 2007 (2007-01-01), US , pages 157 - 166, XP009101408, ISSN: 1525-0016, DOI: 10.1038/sj.mt.6300006
YOKOTE K ET AL., HUM MUTAT., vol. 38, no. 1, January 2017 (2017-01-01), pages 7 - 15
KATO H ET AL., STEM CELL RESEARCH., vol. 53, May 2021 (2021-05-01), pages 102360
XIE J ET AL., FRONT PHARMACOL., vol. 11, 20 May 2020 (2020-05-20), pages 697
GE SX ET AL., BMC BIOINFORMATICS, vol. 19, 2018, pages 534
Attorney, Agent or Firm:
OHTANI PATENT OFFICE (JP)
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