Our difference is that we make a difference.
We focus on rare genetic disorders with high unmet medical need. Whether it be providing communities with their first treatment option, or improving upon the current standard of care, we focus on conditions where we believe we can make a meaningful impact.
Our scientific curiosity and pioneering spirit make it possible to take on the biggest challenges in rare disease.Learn More Our Science ⌃
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition we hope to address.Learn More about our pipline ⌃