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:
Mar. 11, 2025
Filed:
Jul. 26, 2019
Applicants:
Regenxbio Inc., Rockville, MD (US);
The Nemours Foundation, Wilmington, DE (US);
Inventors:
Shunji Tomatsu, Wilmington, DE (US);
Kazuki Sawamoto, Tokyo, JP;
Subha Karumuthil-Melethil, Germantown, MD (US);
Olivier Danos, Princeton, NJ (US);
Assignees:
THE NEMOURS FOUNDATION, Wilmington, DE (US);
REGENXBIO INC., Rockville, MD (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); A61K 48/0058 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01); C12Y 301/06004 (2013.01);
Abstract
Provided herein are gene therapy methods for the treatment of mucopolysaccharidosis type IVA (MPS IVA) involving the use of recombinant adeno-associated viruses (rAAVs) to deliver human N-acetylgalactosamine-6-sulfate sulfatase (hGALNS) to the bone of a human subject diagnosed with MPS IVA. Also provided herein are rAAVs that can be used in the gene therapy methods and methods of making such rAAVs.