Vertex Pharmaceuticals is researching the use of CRISPR/Cas9 gene editing technology to develop potential new treatments aimed at the underlying genetic causes of human disease, including transfusion-dependent beta thalassemia and severe sickle cell disease. Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. Vertex collaborated with CRISPR Therapeutics to discover this investigational therapy.