Voyager Therapeutics, Inc.

Hongxing Wang

Dinah Wen-Yee Sah

Holger Patzke

Martin Goulet

Yanqun Shu

Jinzhao Hou

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.

Growing community of inventors

Lexington, MA, United States of America

Hongxing Wang