Vertex, in collaboration with CRISPR Therapeutics to investigate the use of a gene-editing technology, known as CRISPR/Cas9, to discover and develop a potential one-time treatment for sickle cell disease and beta thalassemia. Exagamglogene autotemcel (exa-cel), is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy which aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin (HbF; hemoglobin F). HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The aim is to use the body’s own machinery to switch back to producing fetal hemoglobin.
Michelle OFlahertyQualified Person ATMP