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