Title:
CUG REPEAT SEQUENCE BINDING AGENT
Document Type and Number:
WIPO Patent Application WO/2021/235293
Kind Code:
A1
Abstract:
Provided is a novel CUG repeat sequence binding agent. This CUG repeat sequence binding agent is configured so as to contain a compound A, the resonance units of which equal 10RU or higher as measured according to surface plasmon resonance at a concentration of 25nM and a (CUG)9 RNA immobilization amount of 401RU.
Inventors:
NAKATANI KAZUHIKO (JP)
MATSUMOTO JUN (JP)
OKAMOTO TATSUMASA (JP)
DOHNO CHIKARA (JP)
SEIKE ASAKO (JP)
NAKAMORI MASAYUKI (JP)
MATSUMOTO JUN (JP)
OKAMOTO TATSUMASA (JP)
DOHNO CHIKARA (JP)
SEIKE ASAKO (JP)
NAKAMORI MASAYUKI (JP)
Application Number:
PCT/JP2021/018079
Publication Date:
November 25, 2021
Filing Date:
May 12, 2021
Export Citation:
Assignee:
UNIV OSAKA (JP)
International Classes:
A61K31/496; A61K45/00; A61P21/00; C07D401/04
Foreign References:
| JP2019514938A | 2019-06-06 | |||
| US20160206753A1 | 2016-07-21 | |||
| US20140050778A1 | 2014-02-20 |
Other References:
VO TAM; PAUL ANANYA; KUMAR ARVIND; BOYKIN DAVID W; WILSON W DAVID: "Biosensor-surface plasmon resonance: A strategy to help establish a new generation RNA-specific small molecules", METHODS, vol. 167, 2019, pages 15 - 27, XP085831363, ISSN: 1046-2023, DOI: 10.1016/j.ymeth.2019.05.005
LÓPEZ-MORATÓ MARTA, BROOK JOHN DAVID, WOJCIECHOWSKA MARZENA: "Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1", FRONTIERS IN NEUROLOGY, vol. 9, 349, 18 May 2018 (2018-05-18), pages 1 - 12, XP055875495, DOI: 10.3389/fneur.2018.00349
REDDY KAALAK, JENQUIN JANA R., CLEARY JOHN D., BERGLUND J. ANDREW: "Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules", INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, vol. 20, no. 16, 4017, 17 August 2017 (2017-08-17), pages 1 - 20, XP055875496, DOI: 10.3390/ijms20164017
MATSUMOTO JUN, NAKAMORI MASAYUKI, OKAMOTO TATSUMASA, MURATA ASAKO, DOHNO CHIKARA, NAKATANI KAZUHIKO: "The Dimeric Form of 1,3- Diaminoisoquinoline Derivative Rescued the Mis- splicing of Atp2al and Clcnl Genes in Myotonic Dystrophy Type 1 Mouse Model", CHEMISTRY-A EUROPIAN JOURNAL, vol. 26, no. 63, 11 November 2020 (2020-11-11), DE, pages 14305 - 14309, XP055875497, ISSN: 0947-6539, DOI: 10.1002/chem.202001572
CHEM. EUR. J., vol. 22, 2016, pages 14881 - 14889
BIOORG. MED. CHEM. LETT., vol. 26, 2016, pages 3761 - 3764
CHEM. EUR. J., vol. 24, 2018, pages 18115 - 18122
SCIENCE, vol. 289, 2000, pages 1769 - 1772
ANN. CLIN. TRANSL. NEUROL., vol. 3, 2016, pages 42 - 54
See also references of EP 4154886A4
LÓPEZ-MORATÓ MARTA, BROOK JOHN DAVID, WOJCIECHOWSKA MARZENA: "Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1", FRONTIERS IN NEUROLOGY, vol. 9, 349, 18 May 2018 (2018-05-18), pages 1 - 12, XP055875495, DOI: 10.3389/fneur.2018.00349
REDDY KAALAK, JENQUIN JANA R., CLEARY JOHN D., BERGLUND J. ANDREW: "Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules", INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, vol. 20, no. 16, 4017, 17 August 2017 (2017-08-17), pages 1 - 20, XP055875496, DOI: 10.3390/ijms20164017
MATSUMOTO JUN, NAKAMORI MASAYUKI, OKAMOTO TATSUMASA, MURATA ASAKO, DOHNO CHIKARA, NAKATANI KAZUHIKO: "The Dimeric Form of 1,3- Diaminoisoquinoline Derivative Rescued the Mis- splicing of Atp2al and Clcnl Genes in Myotonic Dystrophy Type 1 Mouse Model", CHEMISTRY-A EUROPIAN JOURNAL, vol. 26, no. 63, 11 November 2020 (2020-11-11), DE, pages 14305 - 14309, XP055875497, ISSN: 0947-6539, DOI: 10.1002/chem.202001572
CHEM. EUR. J., vol. 22, 2016, pages 14881 - 14889
BIOORG. MED. CHEM. LETT., vol. 26, 2016, pages 3761 - 3764
CHEM. EUR. J., vol. 24, 2018, pages 18115 - 18122
SCIENCE, vol. 289, 2000, pages 1769 - 1772
ANN. CLIN. TRANSL. NEUROL., vol. 3, 2016, pages 42 - 54
See also references of EP 4154886A4
Attorney, Agent or Firm:
IWATANI Ryo (JP)
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