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:
Apr. 21, 2025
Filed:
Sep. 26, 2019
Voyager Therapeutics, Inc., Lexington, MA (US);
Holger Patzke, Lexington, MA (US);
Jinzhao Hou, Lexington, MA (US);
Hongxing Wang, Lexington, MA (US);
Yanqun Shu, Lexington, MA (US);
Martin Goulet, Lexington, MA (US);
Dinah Wen-Yee Sah, Lexington, MA (US);
Voyager Therapeutics, Inc., Lexington, MA (US);
Abstract
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.