Companies in #Hemoglobinopathies
Found 1 company operating in this sector.
CRISPR Therapeutics is a Swiss-American biopharmaceutical company pioneering CRISPR/Cas9 gene editing therapies for serious diseases. The company developed Casgevy (exagamglogene autotemcel), the first CRISPR-based gene therapy approved by the FDA and EMA for sickle cell disease and transfusion-dependent beta thalassemia. The company is advancing a broad pipeline spanning hemoglobinopathies, oncology, autoimmune diseases, and in vivo gene editing programs.