Mission & Vision
A fearlessly human mission
We are on a mission to transform the lives of patients with serious and rare diseases.
Developing innovative therapies requires that we think creatively and collaborate thoughtfully. As we strive to achieve our mission, we seek to create even greater possibilities.
Our inclusive spirit brings all points of view together. And our culture embodies authenticity, intent, and purpose—allowing us to be unapologetically, fearlessly human.
One Mission
Working together to achieve our mission.
We’re working to reimagine medicine—and change the world
Our vision is to be a globally recognized leading biotech company that empowers great people to deliver, with a profound sense of urgency and compassion, life-altering therapies to small patient populations experiencing big health problems. We seek to reimagine medicine. To transform lives. And to change the world for the better.
Our Values
Our five core values set the tone for our company’s culture and guide the actions we take each day:
Collaboration
Collaboration
We check our egos at the door and share ideas openly and candidly. When we disagree, we do so with respect and a willingness to listen.
Accountability
Accountability
We are each responsible for ensuring that our actions are aligned with our values and produce results that help achieve our mission.
Passion
Passion
We are driven to expect more than others think is possible and deliver excellence to our patients, fellow employees, and stakeholders.
Respect
Respect
We value each other as parts of a whole by embracing our differences, recognizing our contributions, and creating a culture of empowerment and trust.
Integrity
Integrity
We are committed to acting in an ethical, honest, and transparent manner in everything we do.
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