CRISPR Therapeutics is a gene editing company focused on the development of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) based therapeutics. CRISPR/Cas9 is a technology for gene editing, the process of altering specific sequences of genomic DNA. Co. is developing several gene-edited allogeneic cell therapy programs. Co.'s main product candidate, CTX001, is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from transfusion-dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce fetal hemoglobin in red blood cells.