Vision, Mission,
and Strategy


A world in which transformative treatments and cures are available to the millions afflicted with neurological diseases.


To create disease-modifying neurogenetic medicines by identifying validated targets, advancing multiple therapeutic modalities, and delivering to the right areas within the central nervous system.


Define and become a leader in the field of neurogenetic medicine. We intend to achieve this by:

Advancing our prioritized CNS pipeline by achieving preclinical and clinical milestones and establishing proof-of-biology and proof-of-concept.

Fueling our future by initiating and advancing early research programs in gene therapy and other therapeutic approaches to neurogenetic medicine.
Maximizing value for all stakeholders by advancing our wholly-owned and partnered assets intended to transform the treatment of neurological disease.