Trust Building collaboration through respect, recognition and reward.
Expertise Fueling R&D with unmatched science and technology both in-house and together with our partners.
Courage Propelling scientific advancement through innovative thinking and a pioneering spirit.
Action Harnessing our talent to urgently deliver first-in-class therapies.
Our Mission
Bringing hope and relief to patients with undertreated rare diseases through the development and commercialization of curative first-in-class gene therapies.