Title:
BIS-BENZIMIDAZOLE COMPOUNDS AND METHODS OF USING THE SAME
Document Type and Number:
WIPO Patent Application WO/2019/099777
Kind Code:
A8
Abstract:
Provided herein are compounds and methods for modulating abnormal repeat expansions of gene sequences. More particularly, provided are inhibitors of RNA and the uses of such inhibitors in regulating nucleotide repeat expansions, e.g., to treat Myotonic Dystrophy Type 1 (DM1 ), Myotonic Dystrophy Type 2 (DM2), Fuchs dystrophy, Huntington Disease, Amyotrophic Lateral Sclerosis, or Frontotemporal Dementia.
Inventors:
DISNEY MATTHEW (US)
BLIZZARD TIMOTHY (US)
RZUCZEK SUZANNE (US)
NDUNGU JOHN (US)
VACCA JOSEPH (US)
JENNINGS ANDY (US)
PUSHECHNIKOV ALEXEI (US)
BLIZZARD TIMOTHY (US)
RZUCZEK SUZANNE (US)
NDUNGU JOHN (US)
VACCA JOSEPH (US)
JENNINGS ANDY (US)
PUSHECHNIKOV ALEXEI (US)
Application Number:
PCT/US2018/061442
Publication Date:
December 26, 2019
Filing Date:
November 16, 2018
Export Citation:
Assignee:
SCRIPPS RESEARCH INST (US)
EXPANSION THERAPEUTICS INC (US)
EXPANSION THERAPEUTICS INC (US)
International Classes:
C07D401/14; A61K31/4184; A61P25/00; C07D403/04; C07D403/10; C07D403/14; C07D407/14; C07D487/04
Attorney, Agent or Firm:
VOELKER, Alden, E. et al. (US)
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