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. 12, 2024

Filed:

Sep. 07, 2021
Applicant:

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

Inventors:

Louise Rodino-Klapac, Cambridge, MA (US);

Eric Pozsgai, Cambridge, MA (US);

Assignee:

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

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
A61P 21/00 (2006.01); A61K 38/17 (2006.01); A61K 48/00 (2006.01); C07K 14/705 (2006.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
A61K 38/177 (2013.01); A61K 48/0058 (2013.01); A61K 48/0066 (2013.01); A61P 21/00 (2018.01); C07K 14/705 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01);
Abstract

Described herein are methods of treating muscular dystrophy comprising administering to a subject a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCG vector, methods of expressing gamma-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV scAAVrh74.MHCK7.hSGCG for usage in such methods, the doses and the ranges thereof for such methods, and methods of generating and producing the rAAV scAAVrh74.MHCK7.hSGCG.


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