DESIGNED TO DELIVER A FUNCTIONAL CURE

Delivering transformative, one-time 
treatments for patients and families 
impacted by rare disease

Our gene addition approach is designed to address disease at the genetic level, delivering one-time treatments with the potential for durable, long-term benefit.

Genetic Medicine Is Here

Our gene addition technology targets the underlying cause of disease with a single dose

See our approach

Pipeline

Explore the clinical programs
supporting our approved therapies

Our Pipeline

Sickle Cell Disease

Learn how we’re changing what’s possible for people living with sickle cell disease

Explore What's Possible

Beta-thalassemia

See how we reduce the burden of lifelong transfusions

Learn more

CALD

Learn about treatment for early, active cerebral adrenoleukodystrophy

Discover more