Companies in #Sickle Cell Disease & Beta Thalassemia
Found 1 company operating in this sector.
Vertex Pharmaceuticals is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. Vertex is the leading developer of cystic fibrosis therapies (Trikafta/Kaftrio franchise), brought the first FDA-approved CRISPR-based therapy to market (Casgevy, with CRISPR Therapeutics), and has a late-stage pipeline in acute pain, sickle cell disease, beta thalassemia, and Duchenne muscular dystrophy.