CRISPR Therapeutics develops gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform, a gene editing technology that can make precise changes to DNA. The company focuses on various disease areas such as hemoglobinopathies, oncology, regenerative medicine, and rare diseases, with a portfolio of therapeutic programs. Its lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy for transfusion-dependent beta-thalassemia or severe sickle cell disease.