We build our commercial models with access to drug as a priority. This means that we price responsibly, launch globally, and have developed an industry-leading UltraCare program to support patients in navigating access to treatments. We are also committed to helping the rare disease community move forward by sharing our science and expertise to advance future development.

Our team is comprised of exceptional and diverse talent with a strong passion and commitment to helping patients. We encourage generosity, curiosity, and humility so that we are constantly learning together, fostering an environment that supports profound growth and fulfillment.

Using a dynamic development model that engages patients at every stage from discovery to translational research to clinical development, we are able to learn and adapt rapidly. This model has helped us to successfully move our programs through the clinic to approval at an average pace of 5.5 years, compared to the industry average of (at least) 7.5 years.