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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.

Patent No.:

US 10537560 B1

PDFFull Text
Date of Patent:
Jan. 21, 2020

Filed:

Nov. 19, 2018

P38 kinase inhibitors reduce dux4 and downstream gene expression for the treatment of fshd

Applicant:

Fulcrum Therapeutics, Inc., Cambridge, MA (US);

Inventors:

Angela Marie Cacace, Haddam Neck, CT (US);

Luis Gustavo Alejandro Rojas Soto, Cambridge, MA (US);

Lorin A. Thompson, III, Cohasset, MA (US);

Owen Brendan Wallace, Brookline, MA (US);

Lucienne V. Ronco, Wellesley, MA (US);

Ning Shen, Cambridge, MA (US);

Alan Scott Robertson, Cambridge, MA (US);

Aaron Nakwon Chang, Lexington, MA (US);

Assignee:

Fulcrum Therapeutics. Inc., Cambridge, MA (US);

Attorneys:

Cooley LLP

J. Dean Farmer

Serge R. Banini

Primary Examiner:

Savitha M Rao

Int. Cl.
CPC ...
A61K 31/455 (2006.01); A61K 31/4418 (2006.01); A61K 45/06 (2006.01); A61K 31/4439 (2006.01); A61K 31/519 (2006.01); A61K 31/437 (2006.01); A61K 31/496 (2006.01); A61K 31/5377 (2006.01); A61K 31/53 (2006.01); A61K 31/416 (2006.01); A61K 31/454 (2006.01); A61K 31/513 (2006.01); A61K 31/506 (2006.01); A61P 21/00 (2006.01); C12Q 1/6827 (2018.01); C12Q 1/686 (2018.01); A61K 9/00 (2006.01); C12Q 1/6883 (2018.01);
U.S. Cl.
CPC ...
A61K 31/4418 (2013.01); A61K 31/416 (2013.01); A61K 31/437 (2013.01); A61K 31/4439 (2013.01); A61K 31/454 (2013.01); A61K 31/496 (2013.01); A61K 31/506 (2013.01); A61K 31/513 (2013.01); A61K 31/519 (2013.01); A61K 31/53 (2013.01); A61K 31/5377 (2013.01); A61P 21/00 (2018.01); C12Q 1/686 (2013.01); C12Q 1/6827 (2013.01); C12Q 1/6883 (2013.01); A61K 9/0019 (2013.01); A61K 9/0053 (2013.01); A61K 9/0073 (2013.01); C12Q 2600/106 (2013.01); C12Q 2600/156 (2013.01);
Abstract

The disclosure relates to methods and compositions including p38 kinase inhibitors and agents that regulate expression of DUX4 and downstream genes including but not restricted to ZSCAN4, LEUTX, PRAMEF2, TRIM43, MBD3L2, KHDC1L, RFPL2, CCNA1, SLC34A2, TPRX1, PRAMEF20, TRIM49, PRAMEF4, PRAME6, PRAMEF15, or ZNF280A. Methods useful for treating a disease associated with abnormal DUX4 and downstream gene expression (e.g., Fascioscapulohumeral muscular dystrophy) are disclosed.

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