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Title:
BIS-BENZIMIDAZOLE COMPOUNDS AND METHODS OF USING SAME
Document Type and Number:
WIPO Patent Application WO/2019/099777
Kind Code:
A3
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 (492 Peacok Lane North, Jupiter, FL, 33458, US)
BLIZZARD, Timothy, Allen (43 Bertrand Drive, Princeton, NJ, 08540, US)
RZUCZEK, Suzanne (843 University Boulevard, Apartment 307Jupiter, FL, 33458, US)
NDUNGU, John (4922 Victoria Circle, West Palm Beach, FL, 33409, US)
VACCA, Joseph (733 Indian Creek Road, Telford, PA, 18969, US)
JENNINGS, Andy (4444 Mt Bigelow Drive, San Diego, CA, 92111, US)
PUSHECHNIKOV, Alexei (Inc.10996 Torreyana Road,Suite 28, San Diego CA, 92121, US)
Application Number:
US2018/061442
Publication Date:
July 18, 2019
Filing Date:
November 16, 2018
Export Citation:
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Assignee:
THE SCRIPPS RESEARCH INSTITUTE (10550 North Torrey Pines Rd, La Jolla, CA, 92037, US)
EXPANSION THERAPEUTICS, INC. (10996 Torreyana Road, Suite 280San Diego, CA, 92121, US)
International Classes:
C07D401/14; A61K31/4184; A61P25/00; C07D403/04; C07D403/10; C07D403/14; C07D407/14; C07D487/04
Domestic Patent References:
WO2017049165A12017-03-23
WO2015031819A12015-03-05
WO2003094861A22003-11-20
WO2006094235A12006-09-08
WO1993015712A11993-08-19
Foreign References:
US2488094A1949-11-15
US20140128352A12014-05-08
EP0009163A21980-04-02
Other References:
AMANDA J. GALLANT ET AL: "Mild and Selective Reduction of Imines: Formation of an Unsymmetrical Macrocycle", JOURNAL OF ORGANIC CHEMISTRY, vol. 69, no. 25, 1 December 2004 (2004-12-01), pages 8739 - 8744, XP055546311, ISSN: 0022-3263, DOI: 10.1021/jo049197u
MAURIZIO DEL POETA ET AL: "Structure-In Vitro Activity Relationships of Pentamidine Analogues and Dication-Substituted Bis-Benzimidazoles as New Antifungal Agents", ANTIMICROBIAL AGENTS AND CHEMOTHERAPY, 1 October 1998 (1998-10-01), United States, pages 2495 - 2502, XP055545945, Retrieved from the Internet DOI: 10.1128/AAC.42.10.2495
DATABASE REGISTRY [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 24 August 2006 (2006-08-24), "6,6-[1,4-phenylenebis(1H-benzimidazole-2,6-diylimino)]bis-1-hexanol", XP002788290, Database accession no. 904034-83-9
Attorney, Agent or Firm:
VOELKER, Alden, E. et al. (Marshall, Gerstein & Borun LLP233 S. Wacker Drive,6300 Willis Towe, Chicago IL, 60606-6357, US)
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