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:
Nov. 03, 2020
Filed:
May. 01, 2013
Applicant:
Genzyme Corporation, Cambridge, MA (US);
Inventors:
Marco A. Passini, Northborough, MA (US);
Lamya S. Shihabuddin, West Newton, MA (US);
Catherine R. O'Riordan, Waban, MA (US);
Seng H. Cheng, Natick, MA (US);
Assignee:
Genzyme Corporation, Cambridge, MA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 38/17 (2006.01); A61K 48/00 (2006.01); A61K 9/00 (2006.01);
U.S. Cl.
CPC ...
A61K 38/1709 (2013.01); A61K 9/0019 (2013.01); A61K 9/0085 (2013.01); A61K 48/0058 (2013.01); A61K 48/0075 (2013.01); A61K 48/00 (2013.01); C12N 2750/14141 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01);
Abstract
The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.