Transforming the
Lives of Patients

WITH SEVERE GENETIC AND ORPHAN DISEASES

We are developing next generation products based on the transformative potential of gene therapy to treat patients with severe genetic and orphan diseases. We have two clinical stage products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease, a preclinical oncology program in partnership with Celgene, a world-class team and a broadly applicable gene therapy platform. READ MORE >>