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:
Jun. 17, 2025
Filed:
Feb. 15, 2023
Applicant:
Genzyme Corporation, Cambridge, MA (US);
Inventors:
Catherine R. O'Riordan, Cambridge, MA (US);
Adam Palermo, Cambridge, MA (US);
Brenda Richards, Cambridge, MA (US);
Lisa M. Stanek, Cambridge, NJ (US);
Assignee:
Genzyme Corporation, Cambridge, MA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61K 35/761 (2015.01); A61K 48/00 (2006.01); A61P 25/28 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 35/761 (2013.01); A61P 25/28 (2018.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2310/14 (2013.01); C12N 2310/531 (2013.01); C12N 2710/10023 (2013.01); C12N 2710/10043 (2013.01);
Abstract
Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.