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Title:
READ THROUGH INDUCER, AND THERAPEUTIC AGENT FOR NONSENSE-MUTATION-TYPE GENETIC DISEASES
Document Type and Number:
WIPO Patent Application WO/2011/096484
Kind Code:
A1
Abstract:
A read through inducer for an immature termination codon formed by a nonsense mutation, which comprises a compound represented by structure formula (A); and a therapeutic agent for nonsense-mutation-type genetic diseases, which comprises the read through inducer.

Inventors:
MATSUDA, Ryoichi (3-1 Hongo 7-chome, Bunkyo-k, Tokyo 54, 〒1138654, JP)
松田 良一 (〒54 東京都文京区本郷七丁目3番1号 国立大学法人東京大学内 Tokyo, 〒1138654, JP)
SHIOZUKA, Masataka (3-1 Hongo 7-chome, Bunkyo-k, Tokyo 54, 〒1138654, JP)
塩塚 政孝 (〒54 東京都文京区本郷七丁目3番1号 国立大学法人東京大学内 Tokyo, 〒1138654, JP)
WAGATSUMA, Akira (3-1 Hongo 7-chome, Bunkyo-k, Tokyo 54, 〒1138654, JP)
我妻 玲 (〒54 東京都文京区本郷七丁目3番1号 国立大学法人東京大学内 Tokyo, 〒1138654, JP)
TAKAHASHI, Yoshikazu (14-23 Kamiosaki 3-chome, Shinagawa-k, Tokyo 21, 〒1410021, JP)
高橋 良和 (〒21 東京都品川区上大崎3丁目14番23号 財団法人微生物化学研究会内 Tokyo, 〒1410021, JP)
Application Number:
JP2011/052263
Publication Date:
August 11, 2011
Filing Date:
February 03, 2011
Export Citation:
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Assignee:
Microbial Chemistry Research Foundation (14-23, Kamiosaki 3-chome Shinagawa-k, Tokyo 21, 〒1410021, JP)
財団法人微生物化学研究会 (〒21 東京都品川区上大崎3丁目14番23号 Tokyo, 〒1410021, JP)
The University of Tokyo (3-1 Hongo 7-chome, Bunkyo-ku Tokyo, 54, 〒1138654, JP)
国立大学法人東京大学 (〒54 東京都文京区本郷七丁目3番1号 Tokyo, 〒1138654, JP)
MATSUDA, Ryoichi (3-1 Hongo 7-chome, Bunkyo-k, Tokyo 54, 〒1138654, JP)
松田 良一 (〒54 東京都文京区本郷七丁目3番1号 国立大学法人東京大学内 Tokyo, 〒1138654, JP)
SHIOZUKA, Masataka (3-1 Hongo 7-chome, Bunkyo-k, Tokyo 54, 〒1138654, JP)
塩塚 政孝 (〒54 東京都文京区本郷七丁目3番1号 国立大学法人東京大学内 Tokyo, 〒1138654, JP)
WAGATSUMA, Akira (3-1 Hongo 7-chome, Bunkyo-k, Tokyo 54, 〒1138654, JP)
我妻 玲 (〒54 東京都文京区本郷七丁目3番1号 国立大学法人東京大学内 Tokyo, 〒1138654, JP)
International Classes:
A61K31/7036; A61P1/16; A61P3/04; A61P3/06; A61P3/10; A61P5/14; A61P7/04; A61P9/02; A61P9/10; A61P11/00; A61P13/12; A61P17/00; A61P19/00; A61P19/02; A61P21/04; A61P25/00; A61P25/16; A61P25/28; A61P27/02; A61P29/00; A61P35/00; A61P37/02; A61P37/06; A61P43/00; C07H15/234
Domestic Patent References:
WO2009019504A12009-02-12
WO2008023462A12008-02-28
WO2008004610A12008-01-10
WO2002102361A12002-12-27
WO2002102361A12002-12-27
WO2008004610A12008-01-10
Foreign References:
JP2009523775A2009-06-25
JP2009532461A2009-09-10
JP2004522763A2004-07-29
JP2009255077A2009-11-05
JP2009095436A2009-05-07
JP2008161075A2008-07-17
JP2008079510A2008-04-10
Other References:
MARINA MANUVAKHOVA ET AL.: "Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system", RNA, vol. 6, 2000, pages 1044 - 1055, XP008167504
MASAYUKI ARAKAWA ET AL.: "Kin- Dystrophy ni Taisuru Yakubutsu Chiryo", IGAKU NO AYUMI, vol. 204, no. 3, 8 January 2003 (2003-01-08), pages 183 - 188, XP008167502
See also references of EP 2535051A4
ACTA. MYOL., vol. 22, 2003, pages 15 - 21
N. ENGL. J. MED., vol. 349, 2003, pages 1433 - 1441
THE JOURNAL OF ANTIBIOTICS, vol. 26, 1973, pages 412
"Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations.", J. MED. CHEM., vol. 52, 2009, pages 2836 - 2845
FUJISAWA, K.; HOSHIYA, T.; KAWAGUCHI, H.: "Aminoglycoside antibiotics. VII. Acute toxicity of aminoglycoside antibiotics.", J. ANTIBIOT., vol. 27, 1974, pages 677 - 681
KONDO, S.; HOTTA. K.: "Semisvnthetic aminoglvcoside antibiotics:Development anr! enzymatic modifications.", J. INFECT. CHEMOTHER, vol. 5, 1999, pages 1 - 9
Attorney, Agent or Firm:
HIROTA, Koichi et al. (HIROTA, NAGARE & ASSOCIATES 4th Floor, Shinjuku TR Bldg., 2-2-13, Yoyogi, Shibuya-k, Tokyo 53, 〒1510053, JP)
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