The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Mar. 30, 2021

Filed:

Nov. 16, 2018
Applicants:

The Scripps Research Institute, La Jolla, CA (US);

Expansion Therapeutics, Inc., San Diego, CA (US);

Inventors:

Matthew Disney, Jupiter, FL (US);

Timothy Allen Blizzard, Princeton, NJ (US);

Suzanne Rzuczek, Jupiter, FL (US);

John Ndungu, West Palm Beach, FL (US);

Joseph Vacca, Telford, PA (US);

Andy Jennings, San Diego, CA (US);

Alexei Pushechnikov, San Diego, CA (US);

Assignees:

THE SCRIPPS RESEARCH INSTITUTE, La Jolla, CA (US);

EXPANSION THERAPEUTICS, INC., San Diego, CA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07D 235/20 (2006.01); C07D 403/10 (2006.01); C07D 401/14 (2006.01); C07D 487/04 (2006.01); A61P 25/00 (2006.01); C07D 403/14 (2006.01); C07D 403/04 (2006.01); A61P 25/28 (2006.01); C07D 407/14 (2006.01);
U.S. Cl.
CPC ...
C07D 235/20 (2013.01); A61P 25/00 (2018.01); A61P 25/28 (2018.01); C07D 401/14 (2013.01); C07D 403/04 (2013.01); C07D 403/10 (2013.01); C07D 403/14 (2013.01); C07D 407/14 (2013.01); C07D 487/04 (2013.01);
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.


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