Exagamglogene autotemcel

Sickle cell disease (SCD) is a genetic disorder characterized by the production of abnormal sickle-shaped red blood cells (called hemoglobin S, HbS) that initiate a pathophysiology resulting in severe pain, progressive multi-organ damage, and premature death.^A262691 Treatment options for SCD are largely focused on preventing the production and circulation of HbS and inducing the production of normal fetal hemoglobin (HbF),A262696,A262701 including hydroxyurea - a mainstay of treatment since the 1990s - as well as newer agents like crizanlizumab and voxelotor.

Exagamglogene autotemcel is an autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells which has been investigated in clinical trials for the treatment of severe SCD and severe beta-thalassemia.^L10139 Following engraftment, it causes an increase in the production of HbF and a subsequent decrease in HbS. It was approved by the FDA in December 2023 for the treatment of patients with SCD with recurrent vaso-occlusive crises.^L49231 It is the first CRISPR-based gene editing therapy to be approved in the United States.^L49246 In January 2024, exagamglogene autotemcel received an additional FDA approval for the treatment of transfusion-dependent ?-thalassemia.^L49681