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:
Jun. 16, 2020

Filed:

May. 25, 2018
Applicant:

Genzyme Corporation, Cambridge, MA (US);

Inventors:

Carol A. Nelson, Westford, MA (US);

Bruce M. Wentworth, Northborough, MA (US);

Ronald K. Scheule, Hopkinton, MA (US);

Timothy E. Weeden, Sturbridge, MA (US);

Nicholas P. Clayton, Sudbury, MA (US);

Assignee:

Genzyme Corporation, Summit, NJ (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C07H 21/04 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
A61K 48/005 (2013.01); C12N 15/1137 (2013.01); C12Y 204/01011 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/33 (2013.01);
Abstract

The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.


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