VY-FXN01

VY-FXN01 is an investigational adeno-associated virus (AAV) gene therapy candidate being developed for the treatment of Friedreich's ataxia (FA), a rare, progressive neurological disorder. The therapy aims to address the underlying cause of FA—a deficiency in the frataxin protein due to mutations in the FXN gene—by delivering a functional version of the gene to target tissues. Originally part of a broad collaboration between Voyager Therapeutics and Sanofi Genzyme established in 2015, the program's rights were restructured in 2019, leading to a new strategic partnership between Voyager and Neurocrine Biosciences. Under the current agreement, Neurocrine funds the development activities, while Voyager retains the option to co-develop and co-commercialize the product in the United States.

Other names

FA Program