The company's lead program, targeting the blood diseases β-thalassemia and sickle cell disease, has entered clinical testing, and its work in immuno-oncology, focused on CRISPR/Cas9-edited allogeneic CAR-T cell therapies, could be transformative.

CRISPR has a collaboration with Vertex Pharmaceuticals in Duchenne muscular dystrophy and myotonic dystrophy Type 1 which, if successful, could earn the company more than $1 billion.