Our Guiding Principles

Our goal is to provide novel biopharmaceutical medicines that extend, improve, or enhance the lives of people with rare diseases. We will adhere to these foundational principles:


We are driven to make an impact for patients and their caregivers—from early detection to therapeutic intervention.


We are committed to providing differentiated, high-quality solutions that address unmet medical needs for those who suffer from rare diseases.


We will never compromise our integrity and will lead with transparency, accountability, and compassion.