Title:
SPINOCEREBELLAR ATAXIA TYPE 31 INHIBITOR
Document Type and Number:
WIPO Patent Application WO/2019/194024
Kind Code:
A1
Abstract:
The purpose of the present invention is to provide a spinocerebellar ataxia type 31 inhibitor in which an active ingredient is a small-molecule compound that directly acts on, and thereby mitigates the toxicity of, the repeat RNA that is a cause of spinocerebellar ataxia type 31. The spinocerebellar ataxia type 31 inhibitor according to the present invention is characterized by comprising a compound represented by formula (I) as an active ingredient. [In the formula, X and Y independently represent, for example, a =N- group, Z represents an m-valent linker group that bonds m structures in the brackets to each other, m represents an integer of 2-4, α represents a substituent, and l represents an integer of 0-4.]
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Inventors:
NAKATANI KAZUHIKO (JP)
SHIBATA TOMONORI (JP)
NAGAI YOSHITAKA (JP)
UEYAMA MORIO (JP)
SHIBATA TOMONORI (JP)
NAGAI YOSHITAKA (JP)
UEYAMA MORIO (JP)
Application Number:
PCT/JP2019/012776
Publication Date:
October 10, 2019
Filing Date:
March 26, 2019
Export Citation:
Assignee:
UNIV OSAKA (JP)
International Classes:
A61K31/4375; A61P25/28
Domestic Patent References:
| WO2018029660A1 | 2018-02-15 |
Other References:
YAMADA, T. ET AL.: "Synthesis of naphthyridine carbamate dimer (NCD) derivatives modified with alkanethiol and binding properties of G-G mismatch DNA", ORGANIC LETTERS, vol. 19, no. 16, 2017, pages 4163 - 4166, XP055643086
NAGANO, KURUMI ET AL.: "Analysis of the interaction between RNA containing UGGAA repeat sequence and the low-molecular compound naphthyridine carbamate dimer", ABSTRACTS OF THE 91ST ANNUAL MEETING OF THE JAPANESE BIOCHEMICAL SOCIETY, vol. 91, no. 1P-351, October 2018 (2018-10-01)
NAGANO, KURUMI ET AL.: "Analysis of the interaction between RNA containing UGGAA repeat sequence and the low-molecular compound naphthyridine carbamate dimer", ABSTRACTS OF THE 91ST ANNUAL MEETING OF THE JAPANESE BIOCHEMICAL SOCIETY, vol. 91, no. 1P-351, October 2018 (2018-10-01)
Attorney, Agent or Firm:
USFI PATENT ATTORNEYS INTERNATIONAL OFFICE (JP)
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