Delivering on HopeTM

Additional Pipeline Programs

Scientist

Our research scientists possess the skills and experience necessary to bring compounds from basic research concepts to drug candidate stage.

FRIEDREICH’S ATAXIA

Friedreich’s ataxia (FA) is a rare, severe, inherited neurological disease caused by mutations in the frataxin (FXN) gene leading to decreased expression of FXN, which results in severe sensory impairment, progressive loss of the ability to walk, generalized weakness and loss of sensation, as well as severe and potentially fatal cardiomyopathy.

In partnership with Voyager Therapeutics as part of a strategic collaboration announced in January 2019, Neurocrine Biosciences is developing VY-FXN01, a gene therapy for the treatment of Friedreich’s ataxia. Additional preclinical studies are underway at Voyager including steps to identify a lead clinical candidate for the treatment of FA during 2019.