Ex vivo mechanisms competing with exa-cel and beti-cel

Vertex and CRISPR’s recent standout data and bluebird’s likely FDA approval raise standards for the pipeline of ex vivo gene modification therapies in development to treat sickle cell and β-thalassemia, and their range of biological mechanisms.

Phase I/II data presented by  Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) and CRISPR Therapeutics AG (NASDAQ:CRSP) during the European Hematology Association’s (EHA) virtual meeting June 12 suggested exagamglogene autotemcel (exa-cel, CTX001) could constitute a one-time functional cure for sickle cell disease and β-thalassemia. The CRISPR-edited, fetal hemoglobin-secreting hematopoietic stem cells (HSCs) are now in Phase III testing for both indications...