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.:
Date of Patent:
Jul. 13, 2021
Filed:
Apr. 17, 2017
Applicant:
Sarepta Therapeutics, Inc., Cambridge, MA (US);
Inventor:
Frederick Joseph Schnell, Cambridge, MA (US);
Assignee:
Sarepta Therapeutics, Inc., Cambridge, MA (US);
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/02 (2006.01); C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61P 3/00 (2006.01); A61K 9/00 (2006.01); A61K 31/7105 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 9/0019 (2013.01); A61K 9/0053 (2013.01); A61K 31/7105 (2013.01); A61P 3/00 (2018.01); C07H 21/02 (2013.01); C07H 21/04 (2013.01); C12N 15/1137 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3145 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/351 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/33 (2013.01); C12Y 302/0102 (2013.01);
Abstract
The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.