Ataxion, Inc. is a discovery-stage biopharmaceutical company developing novel small-molecule therapeutics for rare, debilitating, and underserved neurologic diseases.
The targets of our drug discovery programs are novel ion-channels within the central nervous system. Ataxion’s researchers and collaborators are world-leaders in CNS ion-channel modulators.
Our lead program is focused on a group of orphan genetic disorders termed hereditary ataxias. These diseases are characterized by dysfunction or degeneration of the cerebellum – the brain’s movement-coordination center. Patients with these conditions develop severe difficulties: walking, speaking, and performing daily activities.
Despite significant burden of disease, there are no approved therapeutics for these conditions, and it has broadly been a neglected area of therapeutic-development.
The Ataxion drugs being developed for genetic forms of ataxia may also have the potential to treat other forms ataxia, such as those associated with other degenerative neurologic disorders including Multiple Sclerosis and Huntington’s Disease. In addition, Ataxion has programs targeting dystonias, as well as other movement disorders.